0001171843-14-003898.txt : 20140812 0001171843-14-003898.hdr.sgml : 20140812 20140812071450 ACCESSION NUMBER: 0001171843-14-003898 CONFORMED SUBMISSION TYPE: 6-K PUBLIC DOCUMENT COUNT: 5 CONFORMED PERIOD OF REPORT: 20140812 FILED AS OF DATE: 20140812 DATE AS OF CHANGE: 20140812 FILER: COMPANY DATA: COMPANY CONFORMED NAME: Prosensa Holding N.V. CENTRAL INDEX KEY: 0001574111 STANDARD INDUSTRIAL CLASSIFICATION: PHARMACEUTICAL PREPARATIONS [2834] IRS NUMBER: 000000000 STATE OF INCORPORATION: P7 FISCAL YEAR END: 1231 FILING VALUES: FORM TYPE: 6-K SEC ACT: 1934 Act SEC FILE NUMBER: 001-35990 FILM NUMBER: 141032410 BUSINESS ADDRESS: STREET 1: J.H. OORTWEG 21 CITY: 2133 CH LEIDEN STATE: P7 ZIP: 00000 BUSINESS PHONE: 31 0 713320100 MAIL ADDRESS: STREET 1: J.H. OORTWEG 21 CITY: 2133 CH LEIDEN STATE: P7 ZIP: 00000 FORMER COMPANY: FORMER CONFORMED NAME: Prosensa Holding B.V. DATE OF NAME CHANGE: 20130410 6-K 1 f6k_081214.htm FORM 6-K f6k_081214.htm
 
 
 
UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
Washington, D.C. 20549
__________________
 
FORM 6-K
__________________

Report of Foreign Private Issuer
Pursuant to Rule 13a-16 or 15d-16 of
the Securities Exchange Act of 1934

August 12, 2014

Commission File Number: 001-35990
__________________________________

Prosensa Holding N.V.
__________________________________

J.H. Oortweg 21
2333 CH Leiden
The Netherlands
(Address of principal executive offices)
__________________________________

Indicate by check mark whether the registrant files or will file annual reports under cover of Form 20-F or Form 40-F.
 
Form 20-F ☒                          Form 40-F ☐
 
Indicate by check mark if the registrant is submitting the Form 6-K in paper as permitted by Regulation S-T Rule 101(b)(1):
 
Indicate by check mark if the registrant is submitting the Form 6-K in paper as permitted by Regulation S-T Rule 101(b)(7):
 
 
 
 
1

 
INCORPORATION BY REFERENCE

This report on Form 6-K shall be deemed to be incorporated by reference into the registration statements on Form F-3 (Registration Number 333-197240) and Form S-8 (Registration Number 333-194650) of Prosensa Holding N.V. and to be a part thereof from the date on which this report is filed, to the extent not superseded by documents or reports subsequently filed or furnished.
 
 
 

 
SIGNATURES

Pursuant to the requirements of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned, thereunto duly authorized, in Leiden, The Netherlands on August 12, 2014.
 
 
 
PROSENSA HOLDING N.V.
 
  By:
/s/Hans G.C.P. Schikan
   
Name:
Hans G.C.P. Schikan
   
Title:
Chief Executive Officer
       
       
  By:
/s/Berndt A.E. Modig
   
Name:
Berndt A.E. Modig
   
Title:
Chief Financial Officer
 
 
 

 
EXHIBIT INDEX
 

Exhibit
Description of Exhibit
1
Prosensa Holding N.V. Unaudited Condensed Consolidated Interim Financial Statements as of June 30, 2014
2
Prosensa Holding N.V. Management’s Discussion and Analysis of Financial Condition and Results of Operations
3
 
Convertible Note Purchase Agreement dated on August 11, 2014, by and between Prosensa Holding N.V. and Pro200, LLC
Confidential treatment requested as to portions of the exhibit. Confidential material omitted and filed separately with the Securities and Exchange Commission.
 

EX-1 2 exh_1.htm EXHIBIT 1 exh_1.htm
Exhibit 1
 
 
INDEX TO UNAUDITED CONDENSED CONSOLIDATED FINANCIAL STATEMENTS
______________________________

 
 
 

 
PROSENSA HOLDING N.V.
UNAUDITED CONDENSED CONSOLIDATED FINANCIAL STATEMENTS
 
 
CONDENSED CONSOLIDATED STATEMENT OF COMPREHENSIVE INCOME (UNAUDITED)
 
 

   
Note
   
2014
   
Three months
ended June 30,
2013
   
2014
   
Six months
ended June 30,
2013
 
         
€ (‘000 except per share data)
 
License revenue                                                       
    16       -       1,286       14,695       2,693  
Collaboration revenue                                                       
            -       698       60       1,691  
Total revenue                                                       
            -       1,984       14,755       4,384  
Other income                                                       
    17       214       33       452       34  
Research and development expense
    18       (5,539 )     (4,549 )     (10,796 )     (8,609 )
General and administrative expense
    19       (2,696 )     (2,074 )     (5,151 )     (3,869 )
Other gains - net                                                       
            72       7       96       8  
Operating loss                                                       
            (7,949 )     (4,599 )     (644 )     (8,052 )
Finance income                                                       
            229       100       456       292  
Finance costs                                                       
            (242 )     (211 )     (483 )     (399 )
Finance cost – net                                                       
            (13 )     (111 )     (27 )     (107 )
Net loss                                                       
            (7,962 )     (4,710 )     (671 )     (8,159 )
Other comprehensive income                                                       
            -       -       -       -  
Total comprehensive loss*                                                       
            (7,962 )     (4,710 )     (671 )     (8,159 )
Loss per share from operations attributable to the equity holders of the company during the period (in € per share)
                                       
Basic and diluted loss per share
    21       (0.22 )     (0.16 )     (0.02 )     (0.28 )
 
* Total comprehensive loss is fully attributable to equity holders of the company
 
 
The notes are an integral part of these condensed consolidated financial statements.
 
2

 
PROSENSA HOLDING N.V.
UNAUDITED CONDENSED CONSOLIDATED FINANCIAL STATEMENTS
 
CONDENSED CONSOLIDATED BALANCE SHEET (UNAUDITED)
 
 
 
€ (‘000)  
Note
   
As of June 30,
2014
   
As of December 31
2013
 
Assets
                 
Non-current assets
                 
Leasehold improvements and equipment                                                                                        
    7       2,027       2,177  
Intangible assets                                                                                        
    8       1,036       758  
Other financial assets                                                                                        
    9       89       289  
Total non-current assets                                                                                        
            3,152       3,224  
Current assets
                       
Trade and other receivables                                                                                        
    10       3,453       4,403  
Prepayments                                                                                        
    11       998       931  
Cash and cash equivalents                                                                                        
    12       69,467       82,232  
Total current assets                                                                                        
            73,918       87,566  
Total assets                                                                                        
            77,070       90,790  
                         
Equity and liabilities
                       
Equity attributable to owners of the parent
                       
Share capital                                                                                        
            361       359  
Share premium                                                                                        
            119,442       119,222  
Other reserves                                                                                        
            3,050       2,123  
Accumulated deficit                                                                                        
            (58,494 )     (41,890 )
Unappropriated earnings                                                                                        
            (671 )     (16,604 )
Total equity                                                                                        
    13       63,688       63,210  
Liabilities
                       
Non-current liabilities
                       
Borrowings – non-current portion                                                                                        
    15       7,874       7,630  
Derivative financial instruments                                                                                        
            -       22  
Deferred revenue / income                                                                                        
    16       87       10,852  
Total non-current liabilities                                                                                        
            7,961       18,504  
Current liabilities
                       
Borrowings – current portion                                                                                        
    15       30       191  
Derivative financial instruments                                                                                        
            -       8  
Trade and other payables                                                                                        
    14       5,233       5,150  
Deferred revenue / income                                                                                        
    16       158       3,727  
Total current liabilities                                                                                        
            5,421       9,076  
Total liabilities                                                                                        
            13,382       27,580  
Total equity and liabilities                                                                                        
            77,070       90,790  
 
 
The notes are an integral part of these condensed consolidated financial statements.
 
3

 
PROSENSA HOLDING N.V.
UNAUDITED CONDENSED CONSOLIDATED FINANCIAL STATEMENTS
 
 
CONDENSED CONSOLIDATED STATEMENT OF CHANGES IN EQUITY (UNAUDITED)
 
 
 
 
€ (‘000)
 
Common
Share capital
   
Class O Share
capital
   
Class A Share
capital
   
Class B Share
capital
   
Total Share
capital
   
Share premium
   
Other reserves
   
Retained earnings
   
Unappropriated earnings
   
Total
equity
 
Balance at January 1, 2014
    359       -       -       -       359       119,222       2,123       (41,890 )     (16,604 )     63,210  
Net loss
    -       -       -       -       -       -       -       -       (671 )     (671 )
Appropriation of result
    -       -       -       -       -       -       -       (16,604 )     16,604       -  
Share-based payments
    -       -       -       -       -       -       927       -       -       927  
Proceeds from shares issued
    2       -       -       -       2       220       -       -       -       222  
Balance at June 30, 2014
    361       -       -       -       361       119,442       3,050       (58,494 )     (671 )     63,688  
                                                                                 
Balance at January 1, 2013
    35       7       74       174       290       56,118       1,056       (31,998 )     (9,892 )     15,574  
Net loss
    -       -       -       -       -       -       -       -       (8,159 )     (8,159 )
Appropriation of result
    -       -       -       -       -       -       -       (9,892 )     9,892       -  
Share-based payments
    -       -       -       -       -       -       289       -       -       289  
Proceeds from shares issued
    -       -       -       -       -       -       -       -       -       -  
Balance at June 30, 2013
    35       7       74       174       290       56,118       1,345       (41,890 )     (8,159 )     7,704  
 
 
The notes are an integral part of these condensed consolidated financial statements.
 
4

 
PROSENSA HOLDING N.V.
UNAUDITED CONDENSED CONSOLIDATED FINANCIAL STATEMENTS
 
 
CONDENSED CONSOLIDATED STATEMENT OF CASH FLOWS (UNAUDITED)
 
 
 
€ (‘000)
       
 
Six months ended June 30,
 
   
Note
   
2014
   
2013
 
Cash flows from operating activities
                 
Net loss                                                                                        
          (671 )     (8,159 )
Adjustments for:                                                                                        
                     
- Amortization/depreciation                                                                                        
    7,8       617       608  
- Costs employee share option plan                                                                                        
    20       927       289  
- Reversal finance income, net                                                                                        
            (71 )     107  
- Changes in the fair value of derivatives                                                                                        
            (30 )     (11 )
- Changes in trade and other receivables                                                                                        
    10       884       (2,301 )
- Changes in prepayments                                                                                        
    11       (67 )     (1,331 )
- Changes in trade and other payables                                                                                        
    14       (42 )     2,793  
- Currency effect (outstanding) receivables and payables
            98       (1 )
- Changes in deferred revenue                                                                                        
    16       (14,334 )     (2,693 )
              (12,689 )     (10,699 )
Interest received                                                                                        
            453       613  
Interest paid                                                                                        
            (14 )     (30 )
Cash used in operating activities                                                                                        
            (12,250 )     (10,116 )
Cash flows from investing activities
                       
Purchases of tangible fixed assets                                                                                        
    7       (338 )     (283 )
Purchases of intangible assets                                                                                        
    8       (282 )     (34 )
Decrease of other financial assets                                                                                        
            200       -  
Net cash used in investing activities                                                                                        
            (420 )     (317 )
Cash flows from financing activities
                       
Proceeds from issuance of share capital                                                                                        
    13       222       -  
Proceeds from borrowings                                                                                        
    15       100       650  
Redemption financial lease                                                                                        
    15       (61 )     (125 )
Repayments of borrowings                                                                                        
    15       (400 )     (50 )
Net cash (used in)/generated from financing activities
            (139 )     475  
Net decrease in cash and cash equivalents                                                                                        
            (12,809 )     (9,958 )
Currency effect cash and cash equivalents                                                                                        
            44       (23 )
Cash and cash equivalents at beginning of the period                                                                                        
            82,232       40,738  
Cash and cash equivalents at end of the period                                                                                        
    12       69,467       30,757  
Restricted cash                                                                                        
    9       -       500  
 
 
The notes are an integral part of these condensed consolidated financial statements.
 
5

 
NOTES TO THE CONDENSED CONSOLIDATED FINANCIAL STATEMENTS
 
1. General information
 
The activities of Prosensa Holding N.V. and its subsidiaries (together “the Company”) primarily consist of developing innovative, RNA-based therapeutics for the treatment of genetic disorders.
 
Since July 3, 2013, the Company’s ordinary shares have been listed under the ticker symbol “RNA” in the United States on the NASDAQ Global Select Market.
 
Effective January 12, 2014, GlaxoSmithKline (GSK) and the Company mutually agreed to terminate the Research and Development Collaboration and License agreement (the research and collaboration agreement) entered into on October 6, 2009. As of the effective date, the Company regained all rights for the development and commercialization of drisapersen, PRO044 and other applicable compounds in the DMD portfolio.
 
The Company is incorporated and domiciled in the Netherlands. The address of its registered office is J.H. Oortweg 21, Leiden. Prosensa Holding N.V. is the ultimate parent of the following group of entities:
 
1.
Prosensa Therapeutics B.V. (100%);
2.
Prosensa Technologies B.V. (100%);
3.
Polybiotics B.V. (100%); and
4.
Prosensa Inc. (100%)
 
The shares of Prosensa Holding N.V. are held by multiple shareholders, none of them having a share in the Company in excess of 25%.
 
The Management Board approved these condensed consolidated financial statements for issuance on August 12, 2014.
 
2. Summary of significant accounting policies
 
2.1 Basis of preparation
 
The condensed consolidated financial statements of the Company were prepared in accordance with International Financial Reporting Standards (IFRS) for interim financial information (IAS 34). Certain information and disclosures normally included in consolidated financial statements prepared in accordance with IFRS have been condensed or omitted. Accordingly, these condensed consolidated financial statements should be read in conjunction with the Company’s annual consolidated financial statements for the year ended December 31, 2013 and accompanying notes included in the Form 20-F filed with the Securities & Exchange Commission (the Company’s annual consolidated financial statements or financial statements), which have been prepared in accordance with IFRS as issued by the International Accounting Standards Board (IASB).
 
The principal accounting policies applied in the preparation of these condensed consolidated financial statements have been consistently applied to all the periods presented, unless otherwise stated and are consistent with those of the Company annual consolidated financial statement.
 
The preparation of financial statements in conformity with IFRS requires the use of certain critical accounting estimates. It also requires management to exercise its judgment in the process of applying the Company’s accounting policies. The areas involving a higher degree of judgment or complexity, or areas where assumptions and estimates are significant to these condensed consolidated financials are disclosed in note 4.
 
2.2 Changes in accounting policy and disclosures
 
The accounting policies adopted are consistent with those of the previous year, except as described below:
 
(a) New and amended standards adopted by the Company
 
The following standards and amendments to standards became effective for annual periods on January 1, 2014, and have been adopted by the Company in the preparation of the consolidated financial statements:
 
 
·
Amendment to IAS 36 Impairment of Assets
·
Amendment to IAS 39 Financial Instruments
·
IFRIC 21 Levies
 
 
6

 
The adoption of these new standards and amendments to standards had an immaterial effect to the Company’s financial position and results of operations in the periods presented.
 
(b) New standards and interpretations not yet adopted by the Company
 
IFRS 15 “Revenue from contracts with customers” is effective as from January 1st, 2017 with a restrospective effect and could have a significant effect on the consolidated financial statements of the Company. The Company has not early adopted IFRS 15 and has yet to assess IFRS 15’s full impact. There are no other standards which are currently available for early adoption which are expected to have a significant effect on the condensed consolidated financial statements of the Company.
 
3. Financial risk management
 
3.1 Financial risk factors
 
The Company’s activities expose it to a variety of financial risks: market risk (including currency risk, fair value interest rate risk, cash flow interest rate risk and price risk), credit risk and liquidity risk.
 
The condensed consolidated financial statements do not include all financial risk management information and disclosures required in the annual consolidated financial statements and should be read in conjunction with the Company’s annual consolidated financial statements for the period ended December 31, 2013.
 
There have been no changes in the financial management team that is responsible for financial risk management or in the Company’s financial risk management policies since December 31, 2013.
 
Liquidity risk
 
The table below sets forth the Company’s financial liabilities based on the remaining period at the balance sheet date to the contractual maturity date. Derivative financial liabilities are included in the analysis if their contractual maturities are essential for an understanding of the timing of the cash flows. The amounts disclosed in the table are the contractual undiscounted cash flows.
 
€ (‘000)
 
Less than
1 year
   
Between
1 and 2 years
   
Between
2 and 5 years
   
Over 5 years
   
Undefined
 
At June 30, 2014
                             
Borrowings (excl. finance lease liabilities)
    -       -       -       -       7,906  
Finance lease liabilities
    30       -       -       -       -  
Derivative financial instruments
(interest rate swap)
    -       -       -       -       -  
Trade and other payables
    5,233       -       -       -       -  
Total
    5,263       -       -       -       7,906  
                                         
At December 31, 2013
                                       
Borrowings (excl. finance lease liabilities)
    100       100       200       -       7,792  
Finance lease liabilities
    91       -       -       -       -  
Derivative financial instruments
(interest rate swap)
    8       8       14       -       -  
Trade and other payables
    5,150       -       -       -       -  
Total
    5,349       108       214       -       7,792  

3.2 Fair value estimation
 
The Company had entered into a floating-to-fixed interest rate swap to reduce the impact of volatility in changes to interest rates. The determined fair value of the interest rate swap was the impact between a fixed interest rate of 4.15% and the estimated interest rate at measurement date for the remaining period of the instrument discounted over time. The estimated interest rate and discount rates were level two fair value hierarchy inputs. During the six month period ended June 30, 2014, the interest rate swap was settled. As of June 30, 2014 and 2013, the change in fair value of the interest rate swap which was recorded through the condensed consolidated statement of comprehensive income amounted to €3 thousand gain and €6 thousand gain, respectively. Until its settlement the interest rate swap was recorded as both a non-current and current liability in the condensed consolidated balance sheet.
 
 
7

 
The carrying amount of the Company’s financial assets and financial liabilities is a reasonable approximation of their fair value.
 
4. Critical accounting estimates and judgments
 
Estimates and judgments are continually evaluated and are based on historical experience and other factors, including expectations of future events that are believed to be reasonable under the circumstances.
 
The preparation of financial statements in conformity with IFRS requires the Company to make estimates and assumptions that affect the reported amounts and classifications of assets and liabilities, revenues and expenses in the condensed consolidated financial statements. Actual results could differ materially from those estimates and assumptions.
 
The preparation of financial statements in conformity with IFRS also requires the Company to exercise judgment in applying accounting policies. Critical judgments in the application of the Company’s accounting policies and the key sources of estimation of uncertainty were the same as those applied to the consolidated financial statements for the year ended December 31, 2013.
 
The condensed consolidated financial statements do not include all disclosures for critical accounting estimates and judgments that are required in the annual consolidated financial statements and should be read in conjunction with the Company’s annual consolidated financial statements for the period ended December 31, 2013.
 
On September 20, 2013, GSK and the Company announced that the drisapersen Phase III study (DEMAND III, or DMD114044) did not meet its primary endpoint. Effective January 12, 2014, the Company and GSK terminated the research and collaboration agreement for the development of drisapersen and the Company’s other DMD product candidates. On January 16, 2014, the Company reported preliminary data on the overall clinical program for drisapersen. On March 17, 2014, the Company reported preliminary results of the DEMAND V study (DMD114876), a Phase II placebo controlled exploratory study.

On June 3, 2014, the Company announced that following positive feedback from the the U.S. Food and Drug Administration (FDA), the Company will pursue a New Drug Application (NDA) filing for drisapersen with the FDA, under an accelerated approval pathway based on existing data. The Company plans to submit an NDA later this year in which it anticipates that it would commit to the initiation of two confirmatory post-approval studies. In addition, the Company has been interacting with the European Medicines Agency (EMA) and based on these interactions the Company intends to file for conditional approval in Europe.

The outcome of these filings may have a material impact on the further development of drisapersen and other DMD compounds by the Company. A negative outcome of the regulatory approval process could alter the Company’s development plans and costs, and potentially impact the following accounts in the Company’s consolidated financial statements.

Borrowings
 
Certain loans from patient organizations have no fixed redemption schemes, and repayment is due when certain predetermined milestones are met. As of June 30, 2014, the Company recorded €7.9 million of such loans with no fixed redemption schemes. As of June 30, 2014, the maturity dates of the borrowings have been assessed, and changes in redemption dates resulted in an immaterial change to the Company’s borrowing balance.
 
Intangible assets
 
As of June 30, 2014, the Company recorded patents and licenses with a net book value of €475 thousand. As of June 30, 2014, there were no changes to management’s assumptions used to determine the patent and licenses’ recoverable amount, which would exceed the carrying value of €475 thousand even if the outcome of the evaluation is negative, and therefore no impairment is required.
 
Deferred revenue & License revenue
 
Upfront license fee payments received under the research and collaboration agreement with GSK were initially deferred and recognized based on the percentage of completion method, which required the Company to estimate the work performed to date as a proportion of the total work expected to be performed.
 
As a result of the termination of the research and collaboration agreement the Company was released from any performance obligations and recorded €14.7 million license revenue in the first quarter of 2014. As of June 30, 2014, the Company’s deferred revenue balance totals €0.2 million of grants deferred (reference is made to footnote 17).

 
8

 
5. Seasonality of Operations
 
The Company’s financial results have varied substantially and are expected to continue to vary from quarter to quarter. The Company therefore believes that period to period comparisons should not be relied upon as indicative of future financial results.
 
6. Segment information
 
The Company operates in one reportable segment, which comprises the discovery and development of innovative, RNA-based therapeutics. The Management Board is identified as the chief operating decision maker. The Management Board reviews the consolidated operating results regularly to make decisions about resources and to assess overall performance.
 
The Company derived its revenues from a single party, GSK, under the research and collaboration agreement, an exclusive worldwide collaboration for the development and commercialization of RNA-based therapeutics for DMD. The agreement was terminated effective January 12, 2014.
 
7. Leasehold improvements and equipment
 
 
€ (‘000)  
Leasehold improvements
   
Laboratory equipment
   
Office
equipment
   
Construction in progress
   
Total
 
Period ended June 30, 2014
                             
Opening net book amount
    262       1,486       224       205       2,177  
Additions
    -       370       167       (199 )     338  
Depreciation charge (note 18, 19)
    (18 )     (396 )     (74 )     -       (488 )
Closing net book amount
    244       1,460       317       6       2,027  
                                         
At June 30, 2014
                                       
Cost
    353       4,842       957       6       6,158  
Accumulated depreciation
    (109 )     (3,382 )     (640 )     -       (4,131 )
Net book amount
    244       1,460       317       6       2,027  
 
Depreciation expense of €413 thousand for the six months ended June 30, 2014 (six months ended June 30, 2013: €396 thousand) has been charged to research and development expense. Depreciation expense of €75 thousand for the six months ended June 30, 2014 (six months ended June 30, 2013: €95 thousand) has been charged to general and administrative expense.
 
Construction in progress mainly comprises laboratory and computer equipment not ready for use as of June 30, 2014.

8. Intangible assets
 
€ (‘000)
 
Patents
and licenses
   
Software
   
Total
 
Period ended June 30, 2014
                 
Opening net book amount
    522       236       758  
Additions
    -       407       407  
Amortization charge
    (47 )     (82 )     (129 )
Closing net book amount
    475       561       1,036  
                         
At June 30, 2014
                       
Cost
    939       1,140       2,079  
Accumulated amortization and impairment
    (464 )     (579 )     (1,043 )
Net book amount
    475       561       1,036  
 
Additions include €139 thousand of software which was under development as of June 30, 2014.
 
 
9

 
Amortization expense of €91 thousand for the six months ended June 30, 2014 (six months ended June 30, 2013: €81 thousand) has been charged to research and development expense. Amortization expense of €38 thousand for the six months ended June 30, 2014 (six months ended June 30, 2013: €36 thousand) has been charged to general and administrative expense.
 
As of June 30, 2014, acquired software for an amount of €125 thousand was not yet paid and accordingly not reflected in the consolidated statement of cash flows.
 
9. Other financial assets
 
 
€ (‘000)  
June 30,
2014
   
December 31,
2013
 
Deposit for rental obligations
    89       89  
Restricted cash
    -       200  
Total
    89       289  
 
The restricted cash balance secured a bank loan until its repayment in full during the six month period ended June 30, 2014. Refer to note 12 of the condensed consolidated financial statements for further detail.
 
10. Trade and other receivables
 
€ (‘000)  
June 30,
2014
   
December 31,
2013
 
Trade accounts receivable
    2,422       1,298  
Amounts to be invoiced to partners
    -       2,380  
Trade receivables
    2,422       3,678  
Value-added tax
    421       351  
Government and other grants to be received
    171       30  
Advances to personnel
    92       -  
Interest receivables on bank accounts
    279       344  
Other receivables
    68       -  
Total
    3,453       4,403  
 
As of June 30, 2014, trade receivables include an allowance related to the final settlement of the termination agreement with GSK. No other receivables were impaired or not performing. The carrying amount of the Company’s trade receivables are fully denominated in British pounds, while other receivables are fully denominated in Euros.
 
The maximum exposure to credit risk at the reporting date is the carrying value of each class of receivable mentioned above. The Company does not hold any collateral as security.
 
On June 27, 2014, the Company was awarded a $200 thousand (€146 thousand) research grant from Parent Project Muscular Dystrophy (PPMD), a not-for-profit organization founded by parents of children with Duchenne and Becker muscular dystrophy. As of June 30, 2014, the PPMD grant proceeds are included in government and other grants to be received.
 
11. Prepayments
 
As of June 30, 2014, the Company has made prepayments to suppliers for (pre)clinical studies and drug substance of €542 thousand and prepayments of €64 thousand on insurance fees.
 
12. Cash and cash equivalents
 
€ (‘000)  
June 30,
2014
   
December 31,
2013
 
Cash at bank and on hand
    9,242       9,119  
Short-term bank deposits
    60,225       73,113  
Total
    69,467       82,232  

 
10

 
In 2006, the Company received a bank loan of €900 thousand from ABN Amro N.V. The loan was fully repaid as of June 30, 2014. Repayment of the loan has been included in the repayment of borrowings line in the cash flows from financing activities. Upon repayment of the loan, €200 thousand of cash that secured the loan and accordingly had been considered restricted, and all cash and cash equivalents as of June 30, 2014 are at free disposal of the Company. As of December 31, 2013, the €200 thousand that secured the bank loan was considered restricted cash and recorded as a component of other financial assets. The remaining balance of cash and cash equivalents was at the free disposal of the Company.
 
13. Equity
 
Class of shares and stated value
 
June 30,
2014
   
December 31, 2013
 
Common shares of EUR 0.01
    36,095,972       35,932,792  
 
The par value as of June 30, 2014, is €0.01 per share (as of December 31, 2013: €0.01 per share). All issued shares are fully paid. Besides the minimum amount of share capital to be held under Dutch law, there are no distribution restrictions applicable to equity of the Company.
 
In the six month period ended June 30, 2014, 163,180 shares were issued as a result of the exercise of vested options granted under the Company’s share-based compensation plans (refer to the Company’s annual consolidated financial statements for the period ended December 31, 2013 for details of the plans). The related weighted average price at the time of exercise was $9.66 per share.
 
14. Trade and other payables
 
 
€ (‘000)  
June 30,
2014
   
December 31,
2013
 
Trade payables
    1,493       1,910  
Holiday payments and holiday rights
    410       457  
Social security and wage tax
    672       246  
Other liabilities
    2,658       2,537  
Total
    5,233       5,150  
 
Other liabilities
 
Other liabilities mainly consist of accruals for not yet billed services provided by vendors and miscellaneous liabilities.
 
15. Borrowings
 
€ (‘000)
 
June 30,
2014
   
December 31,
2013
 
Non-current
           
Bank borrowings
    -       300  
Other loans
    7,874       7,330  
Total non-current
    7,874       7,630  
Current
               
Bank borrowings
    -       100  
Finance lease liabilities
    30       91  
Total current
    30       191  
Total
    7,904       7,821  
 
Borrowings
 
In 2006, the Company received a bank loan of €900 thousand from ABN Amro N.V. which matures in 2017. The loan bears interest equal to Euribor plus 1.75% per year. The Company fully repaid the outstanding amount in the six month period ended June 30, 2014.
 
 
11

 
In the six months ended June 30, 2014, the Company received loan installments amounting to €100 thousand from Agentschap NL as part of the innovation credit facility (Innovatiekrediet) of the Dutch Ministry of Economic Affairs. During 2013 the Company received a loan installment of €99 thousand from Agentschap NL, €500 thousand from the Duchenne Children’s Trust as installment of a €1.5 million funding agreement for research and development at an interest rate that approximates the market interest rate, €250 thousand from Association Française contre les Myopathies as installment of a €3.0 million funding agreement and €202 thousand from Everest International Pte Ltd as installment of a €1.0 million funding agreement for research and development at below market interest rates.
 
The condensed consolidated financial statements do not include all disclosures for borrowings that are required in the annual consolidated financial statements and should be read in conjunction with the Company’s annual consolidated financial statements for the period ended December 31, 2013.
 
16. Revenue and deferred revenue
 
From October 2009 to January 2014, the Company operated under an exclusive worldwide collaboration with GSK for the development and commercialization of RNA-based therapeutics for DMD, with GSK exclusively licensing worldwide rights to develop and commercialize drisapersen and obtaining an option to exclusively license PRO044 and other specified assets in the Company’s DMD portfolio. Under the research and collaboration agreement, GSK paid the Company a total of £41.5 million (€47.4 million) in upfront and milestone payments. Under the research and collaboration agreement, GSK was responsible for all costs of clinical development of drisapersen.
 
On January 12, 2014, the research and collaboration agreement was mutually terminated pursuant to a termination agreement, which terminated all intellectual property license grants as well as any rights arising under the research and collaboration agreement (other than rights to payments that accrued prior to termination of the collaboration). In addition, the termination agreement required GSK to transfer to the Company certain data and know-how, inventory, regulatory filings, clinical trial sponsorships, clinical study reports and material agreements relating to the development of the Company’s products.
 
Going forward, the Company will be solely responsible for the cost of developing and commercializing drisapersen and its other product candidates, which may have significant financial and operational implications.
 
The agreement to terminate the research and collaboration agreement released the Company from any performance obligations under the upfront payments already received from GSK. As a result, in the six month period ended June 30, 2014, the Company recognized €14.5 million deferred license income. The release from any performance obligations also resulted in recognition of €0.2 million revenue related to other services delivered under the research and collaboration agreement with GSK. In the six month period ended June 30, 2014, collaboration revenue was minimal due to the termination of the research and collaboration agreement.
 
In the six month period ended June 30, 2013, an amount of €1,718 thousand of the initial upfront payment under the research and collaboration agreement with GSK was recognized as license revenue in the consolidated condensed income statement. In the six month period ended June 30, 2013, the Company recognized revenue of €975 thousand related to other upfront payments under the research and collaboration agreement.
 
The condensed consolidated financial statements do not include all disclosures for revenue and deferred revenue that are required in the annual consolidated financial statements and should be read in conjunction with the Company’s annual consolidated financial statements for the period ended December 31, 2013.
 
17. Other income
 
The Company is part of two pan-European consortia, each of which has been awarded a Framework Programme 7 (FP7) research grant of €6 million from the European Commission to support the ongoing clinical study of PRO045 and the development of imaging biomarkers for Duchenne muscular dystrophy (DMD), respectively. The Company also received a research grant from a private non-profit organization and governmental research grants. Grant proceeds are deferred and recognized in other income based on the percentage of completion method in the amount of €446 thousand in the six months ended June 30, 2014 (for the six months ended June 30, 2013: €34 thousand).

The Company obtained certain loans made to support research and development that generally bear interest at a rate below the market interest rate, considered by the Company to be 12% over the last four years. The difference between fair value and the notional amount at inception is treated as a grant received for certain research performed by the Company and recognized in other income over the periods during which expenses are incurred.

 
12

 
18. Research and development expense
 
Research and development expenses increased from €8.6 million to €10.8 million in the six months ended June 30, 2013 and 2014, respectively. The increase is mainly due to the expansion of our development and regulatory activities, directly impacted by the termination of the research and collaboration agreement with GSK, as well as the costs of preparing for the regulatory filing for drisapersen, and the progressing of clinical phase I/II studies of PRO045 and PRO053.
 
 
19. General and administrative expense
 
General and administrative expense increased from €3.9 million to €5.2 million in the six months ended June 30, 2013 and 2014, respectively. The increase is primarily due to share-based compensation and costs associated with operating as a public company in the period ended June 30, 2014 offset by expenses related to our initial public offering (IPO) in the same period in 2013.
 
On July 3, 2014 the Company filed a shelf registration statement (Form F-3) that provides the flexibility to raise up to $150 million in a primary offering if the Company chooses to do so. Costs incurred related to the Form F-3 in the period ended June 30, 2014 were recorded in the consolidated statement of comprehensive income in an amount of €168 thousand.
 
20. Share-based Payments
 
Share-based compensation expenses of €927 thousand were recognized during the six month period ended June 30, 2014 (for the six month period ended June 30, 2013: €289 thousand). A total of 469,000 options were granted in the six month period ended June 30, 2014. The exercise price of the options is the quoted share price at the time of grant. The Company used similar valuation assumptions, such as volatility, as used for previously granted options during the second half of 2013.
 
The condensed consolidated financial statements do not include all disclosures for share-based payments that are required in the annual consolidated financial statements and should be read in conjunction with the Company’s annual consolidated financial statements for the period ended December 31, 2013.
 
21. Loss per share
 
Basic
 
Basic loss per share is calculated by dividing the profit/(loss) attributable to equity holders of the Company by the weighted average number of ordinary and preferred shares in issue during the year.
 
   
Three months ended June 30,
   
Six months ended June 30,
 
   
2014
   
2013
   
2014
   
2013
 
Loss attributable to equity holders of the company in EUR (‘000)
    (7,962 )     (4,710 )     (671 )     (8,159 )
Weighted average number of Common and Preference shares in issue
    35,985,467       29,002,298       35,959,275       29,002,298  
 
Diluted
 
Diluted earnings/(loss) per share is calculated by adjusting the weighted average number of ordinary shares outstanding to assume conversion of all dilutive potential ordinary shares. Due to the fact that the Company is loss making, all potential ordinary shares had an antidilutive effect if converted, and thus have been excluded from the computation of loss per share.
 
22. Income tax expense
 
No tax charge or income has been recognized in the six month period ended June 30, 2014, or the corresponding period in 2013. The Company has a history of tax losses and expects to record a loss for the year ended December 31, 2014. Management’s judgment is that sufficient evidence is currently not available that future taxable profit will be available against which the unused tax losses or unused tax credits can be utilized by the fiscal unity, therefore a deferred tax asset is not recognized.

 
13

 
23. Related-party transactions
 
In the period ended June 30, 2014 and 2013, the Management Board was paid regular salaries and contributions to post-employment schemes. Additionally, in the period ended June 30, 2013 selected members of the Supervisory Board received compensation for their services in the form of cash compensation. In the period ended June 30, 2014, members of the Boards also received share-based compensation. No loans, advances or guarantees were made to the Management Board or Supervisory Board members as of June 30, 2014 and 2013.
 
The condensed consolidated financial statements do not include all disclosures for related-party transactions that are required in the annual consolidated financial statements and should be read in conjunction with the Company’s annual consolidated financial statements for the period ended December 31, 2013.
 
24. Events after the balance sheet date
 
On July 3, 2014, the Company filed a shelf registration statement (Form F-3) that provides the flexibility to raise up to $150 million in a primary offering. The filing of the registration statement is intended to enable the Company the flexibility to raise up to $150 million in one or more primary offerings while the shelf registration statement is effective, if the Company chooses to do so. The specific terms of any securities that the Company may offer, if it chooses to do so, will be determined at the time of such offering and will be described in a separately filed prospectus supplement at the time of such offering. The shelf registration statement also includes the shares of the Company’s three largest shareholders. As of the date of issue of these financial statements, the Company has not offered or sold securities
 
On August 11, 2014, the Company entered into an agreement to sell unsecured convertible notes in the amount of up to €5 million to an affiliate of CureDuchenne, a non-profit organization. An amount of €0.5 million will be issued as an initial note and the remaining notes will be issued if and when specified milestones are met. The notes bear interest at a below-market interest rate that accumulates to the principal amount of the note. The notes must be repaid on the earliest to occur of a change of control, twelve months from when the Company obtains regulatory approval of its first product candidate and June 30, 2019. CureDuchenne has the option, under specified conditions, to convert the notes into the Company’s ordinary shares prior to maturity at a conversion price based on the Company’s share price at the time of conversion.
 
In July 2014, the Company and certain of its managing directors and supervisory directors were named as defendants in Singh v. Schikan et al., a purported class action lawsuit filed in the U.S. District Court for the Southern District of New York. The complaint asserts claims under the federal securities laws on behalf of a professed class consisting of all those who purchased the Company’s ordinary shares pursuant and/or traceable to the registration statement used in connection with the Company’s IPO. The complaint alleges that the Company omitted and/or misstated certain facts in the registration statement concerning the Phase III trial of drisapersen that, as announced on September 20, 2013, did not meet its primary endpoint. The litigation is in its earliest stages, and the Company and the individual defendants intend to defend the action vigorously. The Company is not able at present to reasonably estimate potential losses, if any, in connection with the litigation, but an adverse resolution could have a material adverse effect on the Company’s financial position, results of operations and cash flows.
 
14

 
EX-2 3 exh_2.htm EXHIBIT 2 exh_2.htm
Exhibit 2
 
Management’s Discussion and Analysis of Financial Condition and Results of Operations
 
This section should be read in conjunction with our unaudited condensed consolidated financial statements and related notes included in Exhibit 1 of this Quarterly Report and the section contained in our Annual Report on Form 20-F (our “Annual Report”) — Operating And Financial Review And Prospects”. This discussion contains certain forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. Many of the forward-looking statements contained in this Quarterly Report can be identified by the use of forward-looking words such as “anticipate,” “believe,” “could,” “expect,” “should,” “plan,” “intend,” “will,” “estimate” and “potential,” among others. Forward-looking statements appear in a number of places in this report and include, but are not limited to, statements regarding our intent, belief or current expectations. Forward-looking statements are based on our management’s beliefs and assumptions and on information currently available to our management. Such statements are subject to risks and uncertainties, and actual results may differ materially from those expressed or implied in the forward-looking statements due to various factors, including, but not limited to, those identified under the section “Item 3. Key Information-D. Risk factors” of our Annual Report.

Forward-looking statements speak only as of the date they are made, and we do not undertake any obligation to update them in light of new information or future developments or to release publicly any revisions to these statements in order to reflect later events or circumstances or to reflect the occurrence of unanticipated events.
 
These risks and uncertainties include factors relating to:

 
·
the timing or likelihood of regulatory filings and approvals;
 
 
·
our expectations regarding regulators’ acceptance of accelerated approval pathways for drisapersen and our follow-on product candidates by the FDA and the EMA;
 
 
·
our expectation on the timing and success of confirmatory studies for drisapersen;
 
 
·
the evaluation of the benefit-to-risk profile of drisapersen treatment across all studies and potential impact on the development and commercial pathway of all our product candidates;
 
 
·
the timing and conduct of our trials of drisapersen and our other product candidates, including statements regarding the timing of initiation and completion of the trials and when results of the trials will be made public;
 
 
·
our plans to pursue research and development of our product candidates for DMD and product candidates for other indications;
 
 
·
the potential advantages of our RNA modulation therapies, in particular drisapersen and our other product candidates for DMD;
 
 
·
the clinical utility of drisapersen and our other product candidates;
 
 
·
our estimates regarding the market opportunity for drisapersen and our other product candidates;
 
 
·
our ability to establish sales, marketing and distribution capabilities;
 
 
·
our ability to establish and maintain manufacturing arrangements for our product candidates;
 
 
·
our intellectual property position, including the outcome of interference proceedings relating to our product candidates, including both drisapersen (PRO051) and PRO053;
 
 
·
our estimates regarding expenses, future revenues, capital requirements and the need for additional financing;
 
 
·
the impact of government laws and regulations;
 
 
·
our competitive position;
 
 
·
the outcome of litigation in which we are involved; and
 
 
·
other risk factors discussed under “Risk Factors” included in our Annual Report on Form 20-F.
 
 
 

 
From October 2009 to January 2014, we operated under an exclusive worldwide collaboration with GSK for the development and commercialization of RNA-based therapeutics for DMD, with GSK exclusively licensing worldwide rights to develop and commercialize drisapersen and obtaining an option to exclusively license PRO044 and other specified assets in our DMD portfolio. Under the research and collaboration agreement, GSK paid us a total of £41.5 million (€47.4 million) in upfront and milestone payments and GSK was responsible for all costs of clinical development of drisapersen.
 
On January 12, 2014, we and GSK mutually terminated the research and collaboration agreement pursuant to a termination agreement, which terminated all intellectual property license grants as well as any rights arising under the collaboration agreement (other than rights to payments that accrued prior to termination of the collaboration). In addition, the termination agreement required GSK to transfer to us certain data and know-how, inventory, regulatory filings, clinical trial sponsorships, clinical study reports and material agreements relating to the development of our products.
 
On January 16, 2014, we reported preliminary data on the overall clinical program for drisapersen. On March 17, 2014 we reported 48-week data from our U.S.-based, Phase II placebo-controlled study (DMD114876 or DEMAND V) of our lead compound, drisapersen. The results of this study suggest that, compared to placebo, boys in the higher-dose drisapersen group (6 mg/kg once weekly) experienced stabilization and even improvements in their muscle function and physical activity as measured by the six-minute walk test (6MWT) for the 24-week treatment phase and maintained this improvement during the 24-week follow-up period. Additionally, when evaluating the percent-predicted six-minute walk distance (6MWD), a clinically meaningful treatment difference was observed at week 24 and at week 48.

On June 3, 2014 we announced that following positive feedback from the meeting that took place on May 14, 2014 with the United States Food and Drug Administration (FDA) we will pursue a New Drug Application (NDA) filing for drisapersen with the FDA, under an accelerated approval pathway based on existing data. We plan to submit an NDA later this year in which we anticipate that we would commit to the initiation of two confirmatory post-approval studies. In addition, we have been interacting with the European Medicines Agency (EMA) and based on these interactions we also intend to file for conditional approval in Europe.

In July 2014, the Patent Trial and Appeal Board (the “PTAB”) of the United States Patent and Trademark Office (the “USPTO”) declared patent interferences between certain allowed patent applications exclusively licensed to Prosensa and patents held by Sarepta Therapeutics, Inc. (“Sarepta”) related to exon 51 and exon 53 skipping product candidates designed to treat DMD.  A patent interference is an administrative proceeding conducted by the USPTO with respect to patents filed before the enactment of the America Invents Act and is used to determine which party first invented a technology and is therefore entitled to a patent covering that technology. The PTAB declared Interference No. 106,008 (relating to exon 51 skipping products) that identifies Sarepta’s U.S. Patent Nos. 7,807,816 and 7,960,541 as interfering with Prosensa’s allowed U.S. Application No. 13/550,210. The PTAB also declared Interference No. 106,007 (relating to exon 53 skipping product products) that identifies Sarepta’s U.S. Patent No. 8,455,636 as interfering with Prosensa’s allowed U.S. Application No. 11/233,495. In both cases Prosensa’s patent applications have earlier filing dates than the applicable Sarepta patents.
 
In July 2014, the Company and certain of its managing directors and supervisory directors were named as defendants in Singh v. Schikan et al., a purported class action lawsuit filed in the U.S. District Court for the Southern District of New York. The complaint asserts claims under the federal securities laws on behalf of a professed class consisting of all those who purchased the Company’s ordinary shares pursuant and/or traceable to the registration statement used in connection with the Company’s IPO. The complaint alleges that the Company omitted and/or misstated certain facts in the registration statement concerning the Phase III trial of drisapersen that, as announced on September 20, 2013, did not meet its primary endpoint. The litigation is in its earliest stages, and the Company and the individual defendants intend to defend the action vigorously. The Company is not able at present to reasonably estimate potential losses, if any, in connection with the litigation, but an adverse resolution could have a material adverse effect on the Company’s financial position, results of operations and cash flows.
 
 
2

 
Results of Operations - Comparison of the Three Months Ended June 30, 2014 and 2013
 
   
Three months ended June 30,
 
   
2014
   
2013
   
Change
 
   
(€ in '000)
   
%
 
License revenue                                                                                        
          1,286       (100.0 )
Collaboration revenue                                                                                        
          698       (100.0 )
Total revenue                                                                                        
          1,984       (100.0 )
Other income                                                                                        
    214       33       548.5  
Research and development expense                                                                                        
    (5,539 )     (4,549 )     21.8  
General and administrative expense                                                                                        
    (2,696 )     (2,074 )     30.0  
Other gains - net                                                                                        
    72       7       928.6  
Operating loss                                                                                        
    (7,949 )     (4,599 )     72.8  
Finance income                                                                                        
    229       100       129.0  
Finance costs                                                                                        
    (242 )     (211 )     14.7  
Finance cost –  net                                                                                        
    (13 )     (111 )     (88.3 )
Net loss                                                                                        
    (7,962 )     (4,710 )     69.0  
 
Total revenue
 
License revenue was nil in the three month period ended June 30, 2014, compared to the corresponding period in 2013 due to the termination of the research and collaboration agreement with GSK effective January 12, 2014.
 
The decrease in collaboration revenue to nil in the three month period ended June 30, 2014 from €0.7 million in the three month period ended June 30, 2013 is due to the termination of the research and collaboration agreement with GSK.
 
The timing of our operating cash flows may vary significantly from the recognition of the related revenue, as revenue from some upfront or initiation payments is deferred and recognized as revenue when earned, while other revenue is earned when received, such as milestone payments or service fees. Our revenue has varied, and varies substantially from quarter to quarter and year to year, depending upon, among other things, the number of milestones achieved and the level of revenues earned for ongoing development efforts. Pursuant to the termination of our collaboration with GSK, we do not expect any future license or collaboration revenue under the collaboration. Any new collaboration arrangements we may enter into and the terms we are able to negotiate may impact our revenue for future periods. We therefore believe that period to period comparisons should not be relied upon as indicative of our future revenues.
 
Other income
 
Other income for the three months ended June 30, 2014, amounts to €0.2 million (three months ended June 30, 2013:€0.03 million). We are part of two pan-European consortia, each of which has been awarded Framework Programme 7 (FP7) research grants from the European Commission, and we have also received governmental research grants. Grant proceeds are deferred, and other income is recognized based on the percentage of completion method.
 
We obtained certain loans made to support research and development that generally bear interest at a rate below the market interest rate, considered by us to be 12% over the last four years. The difference between fair value and the notional amount at inception is treated as a grant received for certain research performed by us and is deferred and will be recognized in other income over the periods when expenses are incurred.

 
3

 
Research and Development Expense for the three months ended June 30, 2014 and 2013
 
Project expenses by project
 
Three months ended June 30,
 
   
2014
   
2013
   
Change
 
   
(€ in '000)
   
%
 
DMD Projects                                                                                        
    3,617       2,367       52.8  
PRO044                                                                                       
    103       316       (67.4 )
PRO045 and PRO053                                                                                       
    1,399       860       62.7  
Other DMD projects                                                                                       
    2,115       1,191       77.6  
Non-DMD projects                                                                                        
    207       272       (23.9 )
Infrastructure costs                                                                                        
    1,715       1,910       (10.2 )
Total                                                                                        
    5,539       4,549       21.8  
 
Research and development expense increased from €4.5 million in the three months ended June 30, 2013 to €5.5 million in the three months ended June 30, 2014. Our research and development expense is highly dependent on the development phases of our projects and therefore fluctuates highly from period to period.
 
The variance in our research and development expense during the three months ended June 30, 2014, and the corresponding period in 2013 is primarily related to the following projects:
 
 
·
DMD projects. The DMD project expenses mainly consist of salaries, costs for production of the compounds, costs paid to contract research organizations and costs relating to preparing our regulatory filings for drisapersen. During the three month period ended June 30, 2014, we mostly incurred expenses related to the expansion of our development and regulatory capabilities (directly impacted by the termination of the research and collaboration agreement with GSK) and costs for progressing clinical Phase I/II studies of both PRO045 and PRO053 (€1.4 million). An extension study for PRO044 is planned to start the second half of this year, resulting in lower expenses in the three month period ended June 30, 2014 compared to the corresponding period in 2013. In the three month period ended June 30, 2014, we also incurred expenses for drisapersen (€1.4 million) and for our other DMD projects, such as the Natural History study, PROSPECT and PRO052.
 
 
·
Non-DMD projects. The expenses for our non-DMD projects DM1 and HD mainly consist of outsourced studies.
 
 
·
Infrastructure cost: we incur a significant amount of costs associated with our research and development that are less dependent on individual ongoing programs so they are not allocated to specific projects.
 
General and Administrative Expense
 
General and administrative expense increased from €2.1 million to €2.7 million in the three months ended June 30, 2013 and 2014, respectively. The increase is primarily due to share-based compensation and costs associated with operating as a public company in the period ended June 30, 2014, offset by expenses related to our IPO in the same period in 2013.
 
On July 3, 2014, we filed a shelf registration statement (Form F-3) that provides the flexibility to raise up to $150 million in a primary offering if we choose to do so. Cost incurred expenses related to the Form F-3 in the period ended June 30, 2014 were recorded in the consolidated statement of comprehensive income for an amount of €168 thousand.
 
Other Gains-net
 
Other gains mainly related to currency effects on outstanding receivables in the three months ended June 30, 2014, and were insignificant in the three months ended June 30, 2013.
 
Finance Income
 
Finance income increased €0.1 million in the three months ended June 30, 2014, compared to the three months ended June 30, 2013 due to higher cash balances offset by lower interest rates.
 
 
4

 
Finance Cost
 
Finance cost increased €31 thousand in the three months ended June 30, 2014, compared to the three months ended June 30, 2013. Higher finance costs were mainly due to higher outstanding borrowing balances in the three month period ended June 30, 2014.

 
 
Results of Operations - Comparison of the Six Months Ended June 30, 2014 and 2013
 
   
Six months ended June 30,
 
   
2014
   
2013
   
Change
 
   
(€ in '000)
   
%
 
License revenue                                                                                        
    14,695       2,693       445.7  
Collaboration revenue                                                                                        
    60       1,691       (96.5 )
Total revenue                                                                                        
    14,755       4,384       236.6  
Other income                                                                                        
    452       34       1,229.4  
Research and development expense                                                                                        
    (10,796 )     (8,609 )     25.4  
General and administrative expense                                                                                        
    (5,151 )     (3,869 )     33.1  
Other gains - net                                                                                        
    96       8       1,100.0  
Operating loss                                                                                        
    (644 )     (8,052 )     (92.0 )
Finance income                                                                                        
    456       292       56.2  
Finance costs                                                                                        
    (483 )     (399 )     21.1  
Finance cost –  net                                                                                        
    (27 )     (107 )     (74.8 )
Net loss                                                                                        
    (671 )     (8,159 )     (91.8 )
 
 
Total Revenue
 
License revenue increased €12.0 million in the six month period ended June 30, 2014, compared to the corresponding period in 2013 due to the termination of the research and collaboration agreement with GSK and the related release of deferred revenue balances.
 
Collaboration revenue is revenue from contracts, typically for research and development activities related to the services provided under the research and collaboration agreement. The decrease in collaboration revenue to €0.1 million in the six month period ended June 30, 2014 from €1.7 million in the six month period ended June 30, 2013 is due to the termination of the research and collaboration agreement effective January 12, 2014.
 
The timing of our operating cash flows may vary significantly from the recognition of the related revenue, as the revenue from some upfront or initiation payments is deferred and recognized as revenue when earned, while other revenue is earned when received, such as milestone payments or service fees. Our revenue has varied, and varies substantially from quarter to quarter and year to year, depending upon, among other things, the number of milestones achieved and the level of revenues earned for ongoing development efforts. Pursuant to the termination of our research and collaboration agreement, we do not expect any future license or collaboration revenue under the collaboration. Any new collaboration arrangements we may enter into and the terms we are able to negotiate may impact our revenue for future periods. We therefore believe that period to period comparisons should not be relied upon as indicative of our future revenues.
 
Other income
 
Other income for the six month period ended June 30, 2014, amounts to €0.5 million (six month period ended June 30, 2013: €0.03 million). We are part of two pan-European consortia, each of which has been awarded Framework Programme 7 (“FP7”) research grants from the European Commission, and we have also received governmental research grants. Grant proceeds are deferred, and other income is recognized based on the percentage of completion method.
 
We obtained certain loans made to support research and development that generally bear interest at a rate below the market interest rate, considered by us to be 12% over the last four years. The difference between fair value and the notional amount at inception is treated as a grant received for certain research performed by us and is deferred and will be recognized in other income over the periods when expenses are incurred.

 
5

 
Research and Development Expense for the six months ended June 30, 2014 and 2013
 
Project expenses by project
 
Six months ended June 30,
 
   
2014
   
2013
   
Change
 
   
(€ in '000)
   
%
 
DMD Projects                                                                                        
    6,941       4,874       42.4  
PRO044                                                                                       
    223       671       (66.8 )
PRO045 and PRO053                                                                                       
    2,810       1,830       53.6  
Other DMD projects                                                                                       
    3,908       2,373       64.7  
Non-DMD projects                                                                                        
    478       527       (9.3 )
Infrastructure costs                                                                                        
    3,377       3,208       5.3  
Total                                                                                        
    10,796       8,609       25.4  
 
Research and development expense increased from €8.6 million in the six month period ended June 30, 2013 to €10.8 million in the six month period ended June 30, 2014. Our research and development expense is highly dependent on the development phases of our projects and therefore fluctuates highly from period to period.
 
The variance in our research and development expense during the six month period ended June 30, 2014, and the corresponding period in 2013 is primarily related to the following projects:
 
 
·
DMD projects. The DMD project expenses mainly consist of salaries, costs for production of the compounds, costs paid to contract research organizations and costs relating to preparing our regulatory filings for drisapersen. During the six month period ended June 30, 2014, we mostly incurred expenses related to the expansion of our development and regulatory capabilities (directly impacted by the termination of the research and collaboration agreement with GSK) and costs for progressing clinical Phase I/II studies of both PRO045 and PRO053 (€2.8 million). An extension study for PRO044 is planned to start the second half of this year, resulting in lower expenses in the six month period ended June 30, 2014 compared to the corresponding period in 2013. In the six month period ended June 30, 2014, we also incurred research and development expenses for drisapersen (€1.9 million) and for our other DMD projects, such as the Natural History study, PROSPECT and PRO052.
 
 
·
Non-DMD projects. The expenses for our non-DMD projects DM1 and HD mainly consist of outsourced studies.
 
 
·
Infrastructure cost: we incur a significant amount of costs associated with our research and development that are less dependent on individual ongoing programs so they are not allocated to specific projects.
 
General and Administrative Expense
 
General and administrative expense increased from €3.9 million to €5.2 million in the six month period ended June 30, 2013 and 2014, respectively. The increase is primarily due to share-based compensation expense and costs associated with operating as a public company in the period ended June 30, 2014 offset by expenses related to our IPO in the same period in 2013.
 
On July 3, 2014 we filed a shelf registration statement (Form F-3) that provides the flexibility to raise up to $150 million in a primary offering if we choose to do so. Cost incurred expenses related to the Form F-3 in the period ended June 30, 2014 were recorded in the consolidated statement of comprehensive income for an amount of €168 thousand.
 
Other Gains-net
 
Other gains mainly related to currency effects on outstanding receivables in the six month period ended June 30, 2014, and were insignificant in the six month period ended June 30, 2013.
 
Finance Income
 
Finance income increased €0.2 million in the six month period ended June 30, 2014, compared to the six month period ended June 30, 2013 mainly due to higher cash balances.
 
 
6

 
Finance Cost
 
Finance cost increased €0.1 million in the six month period ended June 30, 2014, compared to the six month period ended June 30, 2013. Higher finance costs were mainly due to higher outstanding borrowing balances in six month period ended June 30, 2014.
 
 
 
Liquidity and Capital Resources
 
To date, we have financed our operations through private placements of our equity securities and our IPO, upfront, milestone and expense reimbursement payments received from GSK, as well as funding from patient organizations, governmental bodies and bank loans.
 
Cash Flows
 
Our cash and cash equivalents as of June 30, 2014, were €69.5 million. The table below summarizes our consolidated unaudited statement of cash flows for each of the six month period ended June 30, 2014 and 2013:
 
   
Six months ended June 30,
 
 (€ in '000)
 
2014
   
2013
 
Net cash used in operating activities                                                                                                        
    (12,250 )     (10,116 )
Net cash used in investing activities                                                                                                        
    (420 )     (317 )
Net cash (used in)/generated from financing activities                                                                                                        
    (139 )     475  
Net decrease in cash and cash equivalents                                                                                                        
    (12,809 )     (9,958 )
Currency effect cash and cash equivalents                                                                                                        
    44       (23 )
Cash, cash equivalents and bank overdrafts at the beginning of the period
    82,232       40,738  
Cash, cash equivalents and bank overdrafts at the end of the period
    69,467       30,757  
 
The net cash used in operating activities of €12.3 million in the six month period ended June 30, 2014, increased from net cash used in operating activities of €10.1 million in 2013 mainly due to a higher operating loss excluding license income and share based compensation expenses (both non-cash items) for a net amount of €3.5 million, less interest received in the amount of €0.2 million and increased cash generated from changes in working capital for an amount of €1.6 million. For an explanation of the operating loss, please see “Results of Operations”.
 
The net cash used in investing activities increased to €0.4 million in the six month period ended June 30, 2014 from €0.3 million in the six month period ended June 30, 2013 due to higher investments in fixed assets offset by the decrease in restricted cash.
 
The decrease in net cash generated from financing activities to net cash used of €0.1 million in the six month period ended June 30, 2014 from net cash generated of €0.5 million in the six month period ended June 30, 2013 is due to a decrease in received borrowings and increase in repayments of €0.8 million in total, offset by proceeds received from issuance of share capital of €0.2 million for options exercised under our share-based compensation plans in the six months period ended June 30, 2014 compared to the same period in 2013.
 
Funding Requirements
 
Our funding requirements may vary substantially from the periods presented in this report. Under our historical collaboration with GSK, GSK reimbursed us for the cost of the clinical trials of drisapersen as well as a substantial portion of the costs of the clinical trials of PRO044 and our natural history study of DMD, and paid us milestone payments upon successful development. Following the mutual termination of the collaboration in January 2014, we will bear the full cost of any additional clinical trials of drisapersen and our other DMD product candidates and will receive no future payments under the collaboration other than those accrued on the date of the termination.
 
Pursuant to the guidance received from the US FDA on June 2, 2014, we anticipate initiating two confirmatory studies prior to the potential accelerated approval of drisapersen , during the first half of 2015. We are financially responsible for conducting these studies, and as a result of the feedback from the FDA and ongoing communication with
 
 
7

 
the EMA, we expect that we will expend significant additional financial resources on clinical development of drisapersen and the rest of our DMD portfolio.
 
On August 11, 2014, the Company entered into an agreement to sell unsecured convertible notes in the amount of up to €5 million to an affiliate of CureDuchenne, a non-profit organization. An amount of €0.5 million will be issued as an initial note and the remaining notes will be issued if and when specified milestones are met. The notes bear interest at a below-market interest rate that accumulates to the principal amount of the note. The notes must be repaid on the earliest to occur of a change of control, twelve months from when the Company obtains regulatory approval of its first product candidate and June 30, 2019. CureDuchenne has the option, under specified conditions, to convert the notes into the Company’s ordinary shares prior to maturity at a conversion price based on the Company’s share price at the time of conversion.
 
We believe that our existing cash and cash equivalents and research funding that we expect to receive will be sufficient to fund our operating expenses, debt service obligations and capital expenditure requirements for at least the next 12 months. We have based this estimate on assumptions that may prove to be wrong, and we could use our capital resources sooner than we currently expect.
 
Our present and future funding requirements will depend on many factors, including, among other things:
 
 
the time and costs involved in obtaining regulatory approval for drisapersen as well our other compounds and any delays we may encounter as a result of evolving regulatory requirements or adverse results with respect to any of these compounds;
 
 
the progress, timing and completion of preclinical testing and clinical trials for any current or future compounds, including our DMD compounds;
 
 
the number of potential new compounds we identify and decide to develop;
 
 
the costs involved in filing patent applications and maintaining and enforcing patents or defending against claims or infringements raised by third parties;
 
 
the marketing activities undertaken in connection with the anticipated commercialization of our DMD compounds and any other current or future compounds and costs involved in the creation of an effective sales and marketing organization;
 
 
the amount of revenues, if any, we may derive either directly or in the form of royalty payments from future sales of our products; and
 
 
 •
the outcome of litigation and other proceedings in which we are involved.
 
Capital Expenditures
 
The following table sets forth our capital expenditures for the six month period ended June 30, 2014 and 2013.
 
   
Six months ended June 30,
 
   
2014
   
2013
 
Investments in tangible fixed assets                                                                                                        
    338       283  
Investments in intangible assets                                                                                                        
    282       34  
Total                                                                                                        
    620       317  

We plan to make investments during the remainder of 2014 to enhance our research and development capacity. The total investments will depend on a possible path forward of drisapersen. For the six month period ended June 30, 2014, we invested €0.6 million in tangible and intangible fixed assets.
 
 
8

 
JOBS Act exemptions
 
On April 5, 2012, the JOBS Act was signed into law. The JOBS Act contains provisions that, among other things, reduce certain reporting requirements for an “emerging growth company.” As an emerging growth company, we are electing to take advantage of the following exemptions:
 
 
·
not providing an auditor attestation report on our system of internal controls over financial reporting;
 
 
·
not providing all of the compensation disclosure that may be required of non-emerging growth public companies under the U.S. Dodd-Frank Wall Street Reform and Consumer Protection Act; and
 
 
·
not disclosing certain executive compensation-related items such as the correlation between executive compensation and performance and comparisons of the Chief Executive Officer’s compensation to median employee compensation.
 
These exemptions will apply for a period of five years following the completion of our IPO or until we no longer meet the requirements of being an “emerging growth company,” whichever is earlier. We would cease to be an emerging growth company if we have more than $1.0 billion in annual revenue, have more than $700 million in market value of our ordinary shares held by non-affiliates or issue more than $1.0 billion of non-convertible debt over a three-year period.
 
9

 
EX-3 4 exh_3.htm EXHIBIT 3 exh_3.htm
Exhibit 3

CONFIDENTIAL TREATMENT REQUESTED UNDER RULE 24b-2 UNDER THE SECURITIES EXCHANGE ACT OF 1934, AS AMENDED.

[*****] INDICATES OMITTED MATERIAL THAT IS THE SUBJECT OF A CONFIDENTIAL TREATMENT REQUEST FILED SEPARATELY WITH THE COMMISSION. THE OMITTED MATERIAL HAS BEEN FILED SEPARATELY WITH THE COMMISSION.

 
PROSENSA HOLDING NV.
 
______________
 
CONVERTIBLE NOTE PURCHASE AGREEMENT
 
 
August 11, 2014
 
 
 

 
TABLE OF CONTENTS
__________________________
 
   
Page
     
Section 1. DEFINITIONS
  3
   
Section 2. ISSUANCE AND SALE OF THE SECURITIES
  4
2.1
The Initial Note
4
2.2
The Second Tranche Note, the Third Tranche Note, the Fourth Tranche Note and the Fifth Tranche Note
4
     
Section 3. THE CLOSINGS
 4
3.1
Initial Closing
4
3.2
Second Closing
4
3.3
Third Closing
4
3.4
Fourth Closing
4
3.5
Fifth Closing
4
3.6
Order of Closings
5
3.7
Deliveries by the Company
5
3.8
Deliveries by the Investor
5
     
Section 4. REPRESENTATIONS, WARRANTIES AND COVENANTS
  6
4.1
Representations and Warranties of the Company
6
4.2
Representations and Warranties of the Investor
7
4.3
Covenant of the Company
8
     
Section 5. CONDITIONS TO CLOSING
  8
5.1
Conditions to Closing by the Investor
8
5.2
Conditions to Closing by the Company
8
     
Section 6. MISCELLANEOUS
  9
6.1
Waivers and Amendments
9
6.2
Costs and Expenses
9
6.3
Remedies Cumulative
9
6.4
Remedies Not Waived
9
6.5
Entire Agreement
9
6.6
Specific Performance
9
6.7
Governing Law
10
6.8
Notices
10
6.9
Counterparts
10
6.1
Successors and Assigns
11
6.11
Third Parties
11
6.12
Schedules and Exhibits
11
6.13
Headings
11
6.14
Publicity
11
 
 
 

 
CONVERTIBLE NOTE PURCHASE AGREEMENT
 
THIS CONVERTIBLE NOTE PURCHASE AGREEMENT (this “Agreement”), dated as of August 11, 2014, is entered into by and between Prosensa Holding N.V., a public company with limited liability (naamloze vennootschap) organized under the laws of The Netherlands (the “Company”), and PRO200, LLC, a Delaware limited liability company (the “Investor”).
 
RECITALS
 
Whereas, the Company desires to implement a project (as described in Exhibit B hereto, the “DMD Project”) to develop certain experimental pharmaceutical products for the treatment of Duchenne muscular dystrophy;
 
Whereas, the Investor is affiliated with CureDuchenne, a 501(c)(3) registered tax-exempt nonprofit organization organized under the laws of California, and Investor, in furtherance of the charitable mission of its affiliated nonprofit, desires to provide financial support to assist the Company in the development of pharmaceutical products for the treatment of Duchenne muscular dystrophy;
 
Whereas, the parties have agreed that the financial support for the DMD Project will be structured as a loan from the Investor where such loan may convert into ordinary shares of the Company;
 
Whereas, the Company has authorized the sale and issuance of unsecured convertible promissory notes in the form attached hereto as Exhibit A in the aggregate principal amount of up to €5,000,000 (the “Notes”) (the securities issuable on conversion of the Notes, the “Conversion Shares” and, along with the Notes, the “Securities”) for an aggregate purchase price of up to €5,000,000, pursuant to the terms of this Agreement;
 
Whereas, the Investor desires to purchase the Securities on the terms and conditions set forth herein; and
 
Whereas, the Company desires to issue and sell the Securities to the Investor on the terms and conditions set forth herein.
 
AGREEMENT
 
Now, Therefore, in consideration of the foregoing recitals and the mutual promises, representations, warranties, and covenants hereinafter set forth and for other good and valuable consideration, the receipt and sufficiency of which are hereby acknowledged, the Company and the Investor agree as follows:
 
SECTION 1. DEFINITIONS
 
The following terms when used in this Agreement shall have the following respective meanings:
 
Closing Date” means the Initial Closing Date, the Second Closing Date, the Third Closing Date, the Fourth Closing Date or the Fifth Closing Date as applicable.
 
Exchange Act” means the Securities Exchange Act of 1934, as amended.
 
Governmental Authority” means the United States, any state, county or municipality, the government of any foreign country, any subdivision of any of the foregoing or any authority, department, commission, board, bureau, agency, court or instrumentality of any of the foregoing.
 
Lien” means any mortgage, lien, pledge, security interest, easement, conditional sale or other title retention agreement or other encumbrance of any kind except for liens relating to taxes that have accrued but are not yet payable that could not be reasonably expected to have a Material Adverse Effect.
 
Material Adverse Effect” means a material adverse effect on the business, properties, management, financial position, stockholders’ equity or results of operations of the Company and its subsidiaries taken as a whole or on the performance by the Company of its obligations under this Agreement.
 
 
 

 
Person” means an individual, corporation, partnership, joint venture, trust, unincorporated organization, or Governmental Authority.
 
SEC” means the U.S. Securities and Exchange Commission.
 
Securities Act” means the Securities Act of 1933, as amended.
 
SECTION 2. ISSUANCE AND SALE OF THE SECURITIES
 
2.1 The Initial Note
 
The Company shall issue and sell to the Investor, and the Investor shall purchase for an aggregate purchase price of €500,000 (the “Initial Note Purchase Price”), from the Company, a Note in the principal amount of €500,000 (the “Initial Note”).
 
2.2 The Second Tranche Note, the Third Tranche Note, the Fourth Tranche Note and the Fifth Tranche Note
 
The Company and the Investor agree that up to four further closings, the “Second Closing,” the “Third Closing,” the “Fourth Closing,” and the “Fifth Closing,” respectively, may be completed as provided for in, and subject to, Sections 3.2, 3.3, 3.4 and 3.5, respectively. At the Second Closing, the Company shall issue and sell to the Investor, and the Investor shall purchase for an aggregate purchase price of €2.0 million (the “Second Note Purchase Price”) from the Company, a Note in the principal amount of  €2.0 million (the “Second Tranche Note”). At the Third Closing, the Company shall issue and sell to the Investor, and the Investor shall purchase for an aggregate purchase price of €1.5 million (the “Third Note Purchase Price”) from the Company, a Note in the principal amount of €1.5 million (the “Third Tranche Note”). At the Fourth Closing, the Company shall issue and sell to the Investor, and the Investor shall purchase for an aggregate purchase price of €500,000 (the “Fourth Note Purchase Price”) from the Company, a Note in the principal amount of €500,000 (the “Fourth Tranche Note”). At the Fifth Closing, the Company shall issue and sell to the Investor, and the Investor shall purchase for an aggregate purchase price of €500,000 (the “Fifth Note Purchase Price”) from the Company, a Note in the principal amount of €500,000 (the “Fifth Tranche Note”).
 
SECTION 3. THE CLOSINGS
 
3.1 Initial Closing
 
The closing of the issuance and sale of the Initial Note (the “Initial Closing,” each of the Initial Closing, Second Closing and Third Closing, a “Closing”) shall take place on September 22, 2014, at the offices of Davis Polk & Wardwell LLP, 450 Lexington Avenue, New York, New York 10017, or at such other time or place as the Company and the Investor shall mutually agree (the actual date being referred to herein as the “Initial Closing Date”). Each Closing may take place via electronic transmission of signature pages.
 
3.2 Second Closing
 
The obligation of the Investor to effect the Second Closing is subject to the dosing of the first patient in the CLIN-02 extension study of PRO044 no later than June 30, 2015 (the “Second Closing Pre-Condition”).  The Company will use its commercially reasonable best efforts to satisfy the Second Closing Pre-Condition on or before January 1, 2015.  Within thirty (30) days of receipt from the Company of notice of the satisfaction of the Second Closing Pre-Condition (which shall be provided within three business days of satisfaction of the condition and which shall include evidence reasonably satisfactory to Investor), the closing of the issuance and sale of the Second Tranche Note shall take place at the offices of Davis Polk & Wardwell LLP, 450 Lexington Avenue, New York, New York 10017, or at such other time or place as the Company and the Investor shall mutually agree (the actual date being referred to herein as the “Second Closing Date”).
 
3.3 Third Closing
 
The obligation of the Investor to effect the Third Closing is subject to the dosing of the first patient in the United States in a Company-sponsored Phase II clinical trial of PRO044 no later than December 31, 2015 (the “Third Closing Pre-Condition”).  The Company will use its commercially reasonable best efforts to satisfy the
 
 
 

 
Third Closing Pre-Condition on or before June 1, 2015.  Within thirty (30) days of receipt from the Company of notice of the satisfaction of the Third Closing Pre-Condition (which shall be provided within three business days of satisfaction of the condition and which shall include evidence reasonably satisfactory to Investor), the closing of the issuance and sale of the Third Tranche Note shall take place at the offices of Davis Polk & Wardwell LLP, 450 Lexington Avenue, New York, New York 10017, or at such other time or place as the Parties shall mutually agree (the actual date being referred to herein as the “Third Closing Date”).
 
3.4 Fourth Closing
 
The obligation of the Investor to effect the Fourth Closing is subject to drisapersen being made available to U.S. trial participants no later than December 31, 2015 (the “Fourth Closing Pre-Condition”).  The Company will use its commercially reasonable best efforts to satisfy the Fourth Closing Pre-Condition on or before June 30, 2015.  Within thirty (30) days of receipt from the Company of notice of the satisfaction of the Fourth Closing Pre-Condition (which shall be provided within three business days of satisfaction of the condition and which shall include evidence reasonably satisfactory to Investor), the closing of the issuance and sale of the Fourth Tranche Note shall take place at the offices of Davis Polk & Wardwell LLP, 450 Lexington Avenue, New York, New York 10017, or at such other time or place as the Parties shall mutually agree (the actual date being referred to herein as the “Fourth Closing Date”).
 
3.5 Fifth Closing
 
The obligation of the Investor to effect the Fifth Closing is subject to the satisfaction of the first to occur of (a) the dosing of the first patient in a Company-sponsored treatment-phase clinical trial of PRO045 and (b) the dosing of the first patient in a Company-sponsored treatment-phase clinical trial of PRO053, no later than December 31, 2015 (the “Fifth Closing Pre-Condition”).  The Company will use its commercially reasonable best efforts to satisfy the Fifth Closing Pre-Condition on or before June 30, 2015.  Within thirty (30) days of receipt from the Company of notice of the satisfaction of the Fifth Closing Pre-Condition (which shall be provided within three business days of satisfaction of the condition and which shall include evidence reasonably satisfactory to Investor), the closing of the issuance and sale of the Fifth Tranche Note shall take place at the offices of Davis Polk & Wardwell LLP, 450 Lexington Avenue, New York, New York 10017, or at such other time or place as the Parties shall mutually agree (the actual date being referred to herein as the “Fifth Closing Date”).
 
3.6 Order of Closings
 
(a) The Third Closing may be consummated prior to the Second Closing should the Third Closing Pre-Condition be satisfied prior to the Second Closing Pre-Condition. If the Fourth Closing Pre-Condition or the Fifth Closing Pre-Condition occurs prior to the Third Closing Pre-Condition, the Fourth Closing or the Fifth Closing, respectively, shall be postponed and shall take place, if at all, on the Third Closing Date. The Fifth Closing may be consummated prior to the Fourth Closing should the Fifth Closing Pre-Condition be satisfied prior to the Fourth Closing Pre-Condition.
 
3.7 Deliveries by the Company
 
(a) At or prior to the Initial Closing, the Company shall deliver or cause to be delivered to the Investor the following items:
 
(i) The Initial Note purchased by the Investor hereunder, registered in the name of the Investor and subject to the legends and other restrictions set forth herein;
 
(ii) a counterpart of this Agreement duly executed by the Company.
 
(b) At or prior to the Second Closing, the Company shall deliver or cause to be delivered to the Investor the Second Tranche Note, registered in the name of the Investor and subject to the legends and other restrictions set forth herein.
 
(c) At or prior to the Third Closing, the Company shall deliver or cause to be delivered to the Investor the Third Tranche Note, registered in the name of the Investor and subject to the legends and other restrictions set forth herein.
 
 
 

 
(d) At or prior to the Fourth Closing, the Company shall deliver or cause to be delivered to the Investor the Fourth Tranche Note, registered in the name of the Investor and subject to the legends and other restrictions set forth herein.
 
(e) At or prior to the Fifth Closing, the Company shall deliver or cause to be delivered to the Investor the Fifth Tranche Note, registered in the name of the Investor and subject to the legends and other restrictions set forth herein.
 
3.8 Deliveries by the Investor
 
(a) At or prior to the Initial Closing, the Investor shall deliver or cause to be delivered to the Company the following items:
 
(i) payment of the Initial Note Purchase Price in immediately available funds by wire transfer to an account designated in writing by the Company prior to the Initial Closing Date; and
 
(ii) a counterpart of this Agreement duly executed by the Investor.
 
(b) At or prior to the Second Closing, the Investor shall deliver or cause to be delivered to the Company payment of the Second Note Purchase Price in immediately available funds by wire transfer to an account designated in writing by the Company prior to the Second Closing Date.
 
(c) At or prior to the Third Closing, the Investor shall deliver or cause to be delivered to the Company payment of the Third Note Purchase Price in immediately available funds by wire transfer to an account designated in writing by the Company prior to the Third Closing Date.
 
(d) At or prior to the Fourth Closing, the Investor shall deliver or cause to be delivered to the Company payment of the Fourth Note Purchase Price in immediately available funds by wire transfer to an account designated in writing by the Company prior to the Fourth Closing Date.
 
(e) At or prior to the Fifth Closing, the Investor shall deliver or cause to be delivered to the Company payment of the Fifth Note Purchase Price in immediately available funds by wire transfer to an account designated in writing by the Company prior to the Fifth Closing Date.
 
SECTION 4. REPRESENTATIONS, WARRANTIES AND COVENANTS
 
4.1 Representations and Warranties of the Company
 
In order to induce the Investor to purchase the Securities it is purchasing hereunder, the Company represents and warrants to the Investor as of the date hereof that:
 
(a) Organization and Standing. The Company has been duly organized and is validly existing under the laws of The Netherlands and has all requisite corporate power and authority to own or lease its properties and assets and to conduct its business as it is presently being conducted.
 
(b) Capitalization. The outstanding ordinary shares of the Company have been duly and validly authorized and issued and are fully paid and nonassessable.
 
(c) Capacity of the Company; Consents; Execution of Agreements. The Company has full right, power and authority to execute and deliver this Agreement and to perform its obligations hereunder; and all action required to be taken for the due and proper authorization, execution and delivery by it of this Agreement and the consummation by it of the transactions contemplated hereby has been duly and validly taken. This Agreement has been duly executed and delivered by the Company and constitutes a valid and legally binding agreement of the Company, enforceable in accordance with its terms, except as enforcement thereof may be limited by bankruptcy, insolvency, reorganization, moratorium or other similar laws, both state and federal, affecting the enforcement of creditors’ rights or remedies in general from time to time in effect and the exercise by courts of equity powers or their application of principles of public policy.
 
 
 

 
(d) Status of the Conversion Shares; Reservation of Share Capital. Upon issuance and delivery of the Notes in accordance with this Agreement, the Notes will be convertible into Conversion Shares in accordance the terms of the Notes; the Conversion Shares reserved for issuance upon conversion of the Notes have been duly authorized and reserved and, when issued upon conversion of the Notes in accordance with the terms of the Notes, will be validly issued, fully paid and non assessable, and the issuance of the Conversion Shares will not be subject to any preemptive or similar rights.
 
(e) Conflicts; Defaults. The execution and delivery of this Agreement by the Company and the performance by the Company of the transactions and obligations contemplated hereby to be performed by it will not (i) violate or conflict with any of the provisions of the Company’s Articles of Association, (ii) violate or conflict with, or constitute a default under, or result in the acceleration of any obligation under, any material contract, note, debt instrument, security agreement, or other instrument to which the Company is a party or by which the Company, or any of its assets is bound (collectively, the “Material Agreements”); (iii) result in the creation or imposition of any Liens or claims upon the Company’s assets or upon the Company’s outstanding share capital; (iv) assuming the accuracy of the Investor’s representations in Section 4.2, constitute a material violation of any law, statute, judgment, decree, order, rule, or regulation of a Governmental Authority applicable to the Company; or (v) constitute an event which, after notice or lapse of time or both, would result in any of the foregoing; except in the case of clauses (ii) through (v), as would not individually or in the aggregate, be reasonably expected to have a Material Adverse Effect.
 
(f) Disclosure. None of the reports filed by the Company with the SEC, at the time filed (or if amended or superseded by a filing prior to the date of this Agreement, then on the date of such filing), contained any untrue statement of a material fact or omitted to state a material fact required to be stated therein or necessary in order to make the statements therein, in light of the circumstances under which they were made, not misleading.
 
4.2 Representations and Warranties of the Investor
 
The Investor hereby represents and warrants to the Company that as of the date hereof:
 
(a) Investment Intent. The Securities to be purchased by the Investor hereunder are being purchased for its own account and not with the view to, or for resale in connection with, any distribution or public offering thereof within the meaning of the Securities Act. The Investor understands that the Securities have not been registered under the Securities Act by reason of their issuance in transactions exempt from the registration and prospectus delivery requirements of the Securities Act pursuant to Section 4(a)(2) thereof, the availability of which depends upon, inter alia, the bona fide nature of the Investor’s investment intent and the accuracy of the Investor’s representations in this Agreement. The Investor further understands that the certificates representing the Conversion Shares that may be issued pursuant to the conversion of the Notes will bear the following legend and the Investor agrees that it will hold such shares subject thereto:
 
THIS CONVERTIBLE PROMISSORY NOTE AND THE SECURITIES ISSUABLE UPON THE CONVERSION HEREOF HAVE NOT BEEN REGISTERED UNDER THE SECURITIES ACT OF 1933, AS AMENDED, AND HAVE BEEN ACQUIRED FOR INVESTMENT AND NOT WITH A VIEW TO, OR IN CONNECTION WITH, THE SALE OR DISTRIBUTION THEREOF. THIS CONVERTIBLE PROMISSORY NOTE AND ANY SECURITIES ISSUABLE UPON THE CONVERSION HEREOF MAY NOT BE OFFERED, SOLD, ASSIGNED, PLEDGED OR OTHERWISE TRANSFERRED EXCEPT WITH THE COMPANY’S PRIOR WRITTEN CONSENT, WHICH MAY BE GRANTED OR WITHHELD IN THE COMPANY’S SOLE DISCRETION.  ANY PURPORTED TRANSFER IN VIOLATION OF THIS LEGEND SHALL BE NULL AND VOID AB INITIO. BY ITS ACQUISITION HEREOF, THE PURCHASER OF THIS CONVERTIBLE PROMISSORY NOTE AGREES TO COMPLY WITH THE RESTRICTIONS SET FORTH IN THIS LEGEND.
 
The Company need not register a transfer of legended Conversion Shares, and may also instruct its transfer agent not to register the transfer of the Conversion Shares, unless the conditions specified in the legends is satisfied.
 
(b) Capacity of the Investor; Execution of Agreement. The Investor has all requisite power, authority and capacity to enter into this Agreement, deliver the Purchase Price, and to perform the transactions and
 
 
 

 
obligations to be performed by it hereunder. This Agreement has been duly authorized, executed and delivered by them and constitutes its valid and legally binding obligation, enforceable in accordance with its terms, except as enforcement thereof may be limited by bankruptcy, insolvency, reorganization, moratorium or other similar laws, both state and federal, affecting the enforcement of creditors’ rights or remedies in general from time to time in effect and the exercise by courts of equity powers or their application of principles of public policy.
 
(c) Accredited Investor. The Investor is an “accredited investor” as defined in Rule 501(a) of Regulation D promulgated under the Securities Act (“Regulation D”).  Specifically, the Investor is an organization all of the beneficial owners of which are “accredited investors.”
 
(d) Suitability and Sophistication. (i)The Investor has such knowledge and experience in financial and business matters that it is capable of independently evaluating the risks and merits of purchasing the Securities; (ii) the Investor has independently evaluated the risks and merits of purchasing the Securities and has independently determined that the Securities are a suitable investment for it; and (iii) the Investor has sufficient financial resources to bear the loss of its entire investment in the Securities.
 
(e) Receipt of Information. The Investor believes, after due inquiry and investigation, that it has received all of the information that it considers necessary or appropriate for deciding whether to purchase the Securities. The Investor further represents that it has had an opportunity to ask questions and receive answers from the Company regarding the terms and conditions of the offering of the Securities and the business, properties, prospects and financial condition of the Company and to obtain additional information (to the extent the Company possessed such information or could acquire it without unreasonable effort or expense) necessary to verify the accuracy of any information furnished to the Investor.
 
(f) Address for Blue Sky. The Investor’s address set forth in Section 6.8 of this Agreement is the office of the Investor’s principal place of business, upon which the Company may rely for the purpose of complying with applicable “Blue Sky” laws.
 
(g) Rule 144. The Investor acknowledges that the Securities must be held indefinitely unless subsequently registered under the Securities Act or unless an exemption from such registration is available.  It is aware of the provisions of Rule 144 (“Rule 144”) promulgated under the Securities Act which permit limited resale of securities purchased in a private placement subject to the satisfaction of certain conditions.
 
(h) No Public Market. The Investor understands that no public market now exists for the Notes and that the Company has made no assurances that a public market will ever exist for the Notes. The Company does not intend to register the Notes or the Conversion Shares.
 
(i) Investor Counsel; Tax Advice.  The Investor acknowledges that it has had the opportunity to review this Agreement, the Notes and the exhibits and schedules hereto and thereto and the transactions contemplated by this Agreement with its own legal counsel. The Investor has reviewed with its own tax advisors the tax consequences of the transactions contemplated by this Agreement.  It relies solely on such advisors and not on any statements or representations of the Company or any of the Company’s agents regarding such tax consequences.  It understands that it, and not the Company, shall be responsible for its own tax liability that may arise as a result of the transactions contemplated by this Agreement.
 
4.3 Covenants of the Company
 
(a) Use of Proceeds.  The Company shall use the proceeds from the sale of the Notes to perform the development activities set forth on Exhibit B.  The Company shall use commercially reasonable efforts to complete the activities set forth in such plan in accordance with the timelines set forth therein.
 
(b) Rule 144 Sales; Cooperation By The Company; Listing. If the Investor shall transfer any Conversion Shares pursuant to Rule 144, the Company shall cooperate, to the extent commercially reasonable, with the Investor by: (i) making and keeping available public information, as those terms are contemplated by Rule 144; (ii) timely filing with the SEC all reports and other documents required to be filed under the Exchange Act; and (iii) furnishing to the Investor upon request a written statement by the Company as to its compliance with the reporting requirements of the Exchange Act, a copy of the most recent annual or quarterly report of the Company, and such other information as the Investor may reasonably request in order to avail itself of any rule or regulation of the SEC allowing the Investor to sell any Conversion Shares without registration under the Securities Act.  For so
 
 
 

 
long as the Investor owns Notes or Conversion Shares, the Company shall use its best efforts to maintain the listing of its ordinary shares on the Nasdaq Stock Market.
 
SECTION 5. CONDITIONS TO CLOSING
 
5.1 Conditions to Closing by the Investor
 
The obligations of the Investor to consummate the purchase of the Notes pursuant to Section 2 hereof are subject to the satisfaction on or prior to each Closing Date of the following conditions, any of which may be waived in whole or in part in writing by the Investor:
 
(a) all representations and warranties of the Company contained in this Agreement shall be true and correct as of the date of this Agreement and as of the Closing Date as though made anew as of such date (unless another date is specified);
 
(b) the Company shall have delivered to the Investor the applicable items required by Section 3.5 of this Agreement prior to or as of the applicable Closing Date;
 
(c) the Company shall have performed and complied with all agreements and conditions required by this Agreement to be performed and complied with by it prior to or as of the applicable Closing Date; and
 
(d) all pre-issuance registrations, qualifications, permits and approvals required, if any, under applicable state securities laws or stock exchange listing rules for the lawful execution and delivery of this Agreement and the offer, sale, issuance and delivery of the Securities shall have been obtained.
 
5.2 Conditions to Closing by the Company
 
The obligations of the Company to consummate the issuance and sale of the Securities pursuant to Section 2 hereof are subject to the satisfaction on or prior to each Closing Date of the following conditions, any of which may be waived in whole or in part in writing by the Company:
 
(a) all representations and warranties of the Investor contained in this Agreement shall be true and correct as of the date of this Agreement and as of the Closing Date as though made anew as of such date;
 
(b) the Investor shall have delivered to the Company the applicable items required by Section 3.6 of this Agreement prior to or as of the applicable Closing Date;
 
(c) the Investor shall have performed and complied with all agreements and conditions required by this Agreement to be performed and complied with by it prior to or as of the applicable Closing Date; and
 
(d) all pre-issuance registrations, qualifications, permits and approvals required, if any, under applicable state securities laws or stock exchange listing rules for the lawful execution and delivery of this Agreement and the offer, sale, issuance and delivery of the Securities shall have been obtained.
 
SECTION 6. MISCELLANEOUS
 
6.1 Waivers and Amendments
 
This Agreement may be amended or modified in whole or in part only by a writing which makes reference to this Agreement that is executed by the Investor and the Company. The obligations of any Party hereunder may be waived (either generally or in a particular instance and either retroactively or prospectively) only with the written consent of the party claimed to have given the waiver; provided, however, that any waiver by any party of any violation of, breach of, or default under any provision of this Agreement or any other agreement provided for herein shall not be construed as, or constitute, a continuing waiver of such provision, or waiver of any other violation of, breach of or default under any other provision of this Agreement or any other agreement provided for herein.
 
6.2 Costs and Expenses
 
 
 

 
Each party agrees to pay its own costs and expenses in connection with the preparation, execution and delivery of this Agreement and other instruments and documents to be delivered hereunder.
 
6.3 Remedies Cumulative
 
No specific right, power, or remedy conferred by this Agreement shall be exclusive, and each such right, power, or remedy shall be cumulative and in addition to every other right, power, or remedy, whether conferred hereby or by any security of the Company or now or hereafter available, at law or in equity, by statute or otherwise.
 
6.4 Remedies Not Waived
 
No course of dealing between the Company and the Investor, and no delay in exercising any right, power, or remedy conferred hereby or by any security issued by the Company, or now or hereafter available at law or in equity, by statute or otherwise, shall operate as a waiver of or otherwise prejudice any such right, power, or remedy.
 
6.5 Entire Agreement
 
This Agreement and the other agreements and instruments expressly provided for herein together set forth the entire understanding of the parties hereto and supersede in their entirety all prior contracts, agreements, arrangements, communications, discussions, representations and warranties, whether oral or written, among the parties with respect to the subject matter hereof.
 
6.6 Specific Performance
 
The Company and the Investor acknowledge and agree that irreparable damage would occur in the event that any of the provisions of this Agreement were not performed in accordance with the specific terms hereof or were otherwise breached. It is accordingly agreed that, to the fullest extent permitted by law or equity, each of the parties shall be entitled to an injunction or injunctions to prevent or cure breaches of the provisions of this Agreement and to enforce specifically the terms and provisions hereof, this being in addition to any other remedy to which the parties may be entitled by law or equity.
 
6.7 Governing Law
 
This Agreement shall in all respects be governed by and construed in accordance with the laws of the State of New York without giving effect to the principles of conflicts of law thereof.  The Company and the Investor irrevocably submit to the non-exclusive jurisdiction of any New York State or United States Federal court sitting in The City of New York over any suit, action or proceeding involving any of the parties hereto that arises out of or relates to this Agreement or the transactions contemplated hereby
 
6.8 Notices
 
Any notice, request or other communication required or permitted hereunder shall be in writing and be deemed to have been duly given (a) when personally delivered or sent by email or facsimile transmission, (b) one business day after being sent by a nationally recognized overnight courier service or (c) three business days after being sent by registered or certified mail, return receipt requested, postage prepaid, to the parties at their respective addresses set forth below.
 
If to the Company:
 
Prosensa Holding N.V.
J.H. Oortweg 21
2333 CH Leiden, The Netherlands,
Fax: +31 (0)71 3322088)
Email: b.modig@prosensa.nl
Attention: Berndt Modig, Chief Financial Officer

With a copy, which shall not constitute notice, to:
Davis Polk & Wardwell LLP
450 Lexington Avenue
 
 
 

 
New York, NY 10017
Fax (212) 701-5800
Email: sophia.hudson@davispolk.com
Attention: Sophia Hudson

If to the Investor:
 
PRO200, LLC
c/o CureDuchenne
1400 Quail Street, Suite 110
Newport Beach, CA 92660
Email: debra@cureduchenne.org
Attention: Debra Miller, President
 
With a copy, which shall not constitute notice, to:
 
Hutchison, PLLC
3110  Edwards Mill Road, Suite 300
Raleigh, NC  27612
Fax: (866) 479-7550
Email: bwofford@hutchlaw.com
Attention: Bill Wofford

Any party by written notice to the others may change the address or the persons to whom notices or copies thereof shall be directed.
 
6.9 Counterparts
 
This Agreement may be executed in counterparts, each of which shall be deemed to be an original, and all of which together shall constitute one and the same instrument.
 
6.10 Successors and Assigns
 
This Agreement shall be binding upon and shall inure to the benefit of the parties hereto and their respective successors and assigns.
 
6.11 Third Parties
 
Nothing expressed or implied in this Agreement is intended, or shall be construed, to confer upon or give any Person other than the parties hereto any rights or remedies under or by reason of this Agreement.
 
6.12 Schedules and Exhibits
 
The schedules and exhibits attached to this Agreement are incorporated herein and shall be part of this Agreement for all purposes.
 
6.13 Headings
 
The headings in this Agreement are solely for convenience of reference and shall not be given any effect in the construction or interpretation of this Agreement.
 
6.14 Publicity
 
Upon consummation of the Initial Closing, the Company and the Investor shall issue a joint press release in the form of Exhibit C hereto.
 
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IN WITNESS WHEREOF, the parties have duly executed, or have caused their duly authorized officer or representative to execute, this Convertible Note Purchase Agreement as of the date first above written.
 
 
 
 
PROSENSA HOLDING N.V.
 
  By: /s/ Hans Schikan
    Name: Hans Schikan
    Title: Chief Executive Officer
     
 
 
 
  By: /s/ Berndt Modig
    Name: Bernt Modig
    Title: Chief Financial Officer
     
   

 
 

 
IN WITNESS WHEREOF, the parties have duly executed, or have caused their duly authorized officer or representative to execute, this Convertible Note Purchase Agreement as of the date first above written.
 

 
PRO200, LLC
 
By: /s/ Debra Miller_______________
Name: Debra Miller
Title: Manager
 
 
 

 
Exhibit A
 
FORM OF NOTE
 
 
 
 

 
THIS CONVERTIBLE PROMISSORY NOTE AND THE SECURITIES ISSUABLE UPON THE CONVERSION HEREOF HAVE NOT BEEN REGISTERED UNDER THE SECURITIES ACT OF 1933, AS AMENDED, AND HAVE BEEN ACQUIRED FOR INVESTMENT AND NOT WITH A VIEW TO, OR IN CONNECTION WITH, THE SALE OR DISTRIBUTION THEREOF. THIS CONVERTIBLE PROMISSORY NOTE AND ANY SECURITIES ISSUABLE UPON THE CONVERSION HEREOF MAY NOT BE OFFERED, SOLD, ASSIGNED, PLEDGED OR OTHERWISE TRANSFERRED EXCEPT WITH THE COMPANY’S PRIOR WRITTEN CONSENT, WHICH MAY BE GRANTED OR WITHHELD IN THE COMPANY’S SOLE DISCRETION.  ANY PURPORTED TRANSFER IN VIOLATION OF THIS LEGEND SHALL BE NULL AND VOID AB INITIO. BY ITS ACQUISITION HEREOF, THE PURCHASER OF THIS CONVERTIBLE PROMISSORY NOTE AGREES TO COMPLY WITH THE RESTRICTIONS SET FORTH IN THIS LEGEND.
 
CONVERTIBLE PROMISSORY NOTE
 
€[________]1     [_____], 2014
            
 
For value received, Prosensa Holding N.V., a public company with limited liability (naamloze vennootschap) organized under the laws of The Netherlands (the “Company”), promises to pay to PRO200, LLC, a Delaware limited liability company (the “Holder”), the principal sum set forth above. Interest shall accrue from the date of this Convertible Promissory Note (this “Note”) on the unpaid principal amount at a rate equal to (x) at all times other than during the continuance of an Event of Default, five percent (5%) per annum, and (y) during the continuance of an Event of Default, to the extent permitted by applicable law, fifteen percent (15%) per annum, it being understood that accrued interest shall not compound or be payable prior to the Maturity Date, and shall be calculated on the basis of a 360 day year comprised of 30 day months (or in the case of a partial month, the actual number of days elapsed therein).  “Event of Default” means (i) any failure by the Company to pay the entire unpaid principal sum of this Note (unless earlier converted), together with accrued and unpaid interest thereon, when required pursuant to Section 1(a) hereof, (ii) any failure of the Company to deliver the full number of Ordinary Shares (as defined below) issuable upon conversion of this Note pursuant to Section 2 hereof that continues for ten business days, or (iii) a Bankruptcy Event (as defined below).  This Note is subject to the following terms and conditions:
 
1. Maturity.
 
(a) Scheduled Maturity. Unless converted pursuant to Section 2, the entire unpaid principal sum of this Note, together with accrued and unpaid interest thereon, will be payable on the Maturity Date. Subject to Section 3, interest shall accrue on this Note but shall not be due and payable until the written demand of the Holder on or after the Maturity Date.  The “Maturity Date” shall occur on the earliest of:
 
(i) the consummation of a Change of Control (as defined below);
 
(ii) the date twelve months following the first marketing approval of a new drug application submitted by or on behalf of the Company from either the European Medicines Agency or the U.S. Food and Drug Administration of a product targeting the treatment of patients with Duchenne muscular dystrophy; and
 
(iii) June 30, 2019.
 
As used herein, “Change of Control” means:
 
(A) any “person” or “group” within the meaning of Section 13(d) of the Securities Exchange Act of 1934, as amended (the “Exchange Act”), other than the Company, its wholly owned subsidiaries or its or their employee benefit plans, has filed a Schedule 13D disclosing that such person or group has become the direct or indirect “beneficial owner” (as defined in Rule 13d-3 under the Exchange Act) of more than 50% of the voting power of the Company’s common equity; or
_________________________
1 To be €500,000 for first tranche, €2m for the second tranche, €1.5 million for the third tranche, €500,000 each for the fourth and fifth tranches.
 
 
 

 
(B) (i) the consummation of any consolidation or merger of the Company, or a similar transaction, as determined by the Company in good faith, as a result of which the Company’s ordinary share capital would be converted into, or exchanged for, other securities or property, unless the holders of all classes of the Company’s common equity immediately prior to the relevant transaction(s) and/or event(s) own, directly or indirectly, more than 50% of all classes of common equity of the continuing or surviving entity or the parent thereof immediately after such transaction(s) and/or event(s) or (ii) the sale of all or substantially all of the Company’s consolidated assets to any entity other than one of the Company’s subsidiaries.
 
(b) Acceleration.  Notwithstanding the foregoing, the entire unpaid principal sum of this Note, together with accrued and unpaid interest thereon, shall become immediately due and payable upon the commission of any act of bankruptcy by the Company, the execution by the Company of a general assignment for the benefit of creditors, the filing by or against the Company of a petition in bankruptcy, suspension of payment or similar insolvency proceedings or any other petition for relief under the U.S. federal bankruptcy code or the Dutch Bankruptcy Act (Faillissementswet), or the appointment of a receiver, trustee or similar officer to take possession of the property or assets of the Company (any of the foregoing, a “Bankruptcy Event”); provided that no Bankruptcy Event will occur in respect of any proceedings or presentation of a petition or application by a third party creditor being contested by the Company in good faith and where such proceedings are dismissed, stayed or discharged within ninety (90) days of commencement.
 
2. Conversion.
 
(a) Optional Conversion. At any time prior to repayment of this Note, this Note may, at the option of the Holder, be voluntarily converted, in whole but not in part, into ordinary shares, nominal value €0.01 per share (the “Ordinary Shares”) of the Company or such other securities or property for which this Note may become convertible as a result of any adjustment described in Section 2(b). The number of Ordinary Shares to be issued upon such conversion shall be equal to (i) the principal amount of the Note plus accrued interest divided by (ii) the Conversion Price (as defined below), rounded down to the nearest whole number of shares. The “Conversion Price” shall be equal to the Euro equivalent, as determined by the Company in good faith on the conversion date, of:  (A) if the consummation of any issuance of Ordinary Shares by the Company in which the Company raises gross cash proceeds of at least $40 million (any such financing transaction, a “Qualified Financing”) has occurred prior to the relevant conversion, ninety-five (95%) of the price per Ordinary Share paid to the Company in such Qualified Financing and (B) otherwise, the greater of (1) US $20.00 per share and (2) ninety percent (90%) of the Share Price. The Conversion Price shall in any case at least be equal to the nominal value of the shares delivered.  “Share Price” means (x) the closing price per Ordinary Share  on the NASDAQ Global Select Market (or such other market on which such shares are traded) or (y) if the Ordinary Shares are not then listed on the NASDAQ Global Select Market or otherwise traded on a market, the fair market value per Ordinary Share, in each case on the relevant conversion date, as determined by the Company in good faith. The Company shall deliver to the Holder the consideration due upon conversion on the third business day following the conversion date.  For purposes hereof, this Note shall be considered to have been “converted,” and the “conversion date” to have occurred, on the date that the Note and the Conversion Notice is delivered to the Company pursuant to clause (c) below.
 
(b) Adjustment.
 
(i) In the event of changes in the outstanding share capital of the Company by reason of stock dividends, stock splits, recapitalizations, reclassifications, combinations or exchanges of shares, reorganizations, liquidations, consolidation, merger or acquisition of the Company, or a similar transaction or event, as determined by the Company in good faith (any such transaction or event, a “Share Exchange Event”), with respect to any conversion that is settled on or after the effective date therefor, (A) in lieu of each Ordinary Share  that would otherwise have been deliverable hereunder by the Company upon conversion, the Company shall deliver the types and amounts of securities or property deliverable pursuant to such Share Exchange Event to a holder of one Ordinary Share (or, if holders of Ordinary Shares can elect the types of consideration they receive in such event, the weighted average of the types and amounts of securities, cash or other property deliverable to such holders on a per share basis) (the amount of such securities or property deliverable with respect to one Ordinary Share, a “Unit of Reference Property”) and (B) the Share Price shall be equal to the fair market value of a Unit of Reference Property, as determined by the Company in good faith. If requested by the Company, the Holder shall promptly
 
 
 

 
execute an amendment to this Note to evidence such changes to the terms of the Note; provided that no such amendment shall be necessary for such changes to be effective.
 
(ii) Upon the occurrence of each adjustment pursuant to this Section 2(b), the Company at its expense will, at the written request of the Holder, promptly notify the Holder of the composition of a Unit of Reference Property.
 
(c) Mechanics and Effect of Conversion. Upon conversion of this Note pursuant to this Section 2, the Holder shall surrender this Note at the principal offices of the Company, along with a duly executed Conversion Notice in the form attached as Appendix A hereto. At its expense, the Company will, as soon as practicable thereafter, cause to be issued to such Holder the number of Ordinary Shares to which such Holder is entitled upon such conversion or, in the case of a conversion following the first anniversary of the issuance date of this Note, unless the Company reasonably determines that inclusion of a restrictive legend is necessary or advisable to ensure compliance with applicable securities laws, beneficial interests representing such number of Ordinary Shares to be held through The Depositary Trust Company. No additional consideration will be due upon conversion of this Note into Ordinary Shares.  Upon conversion of this Note, the Company will be forever released from all of its obligations and liabilities under this Note, including without limitation the obligations to pay the principal amount thereof and accrued interest thereon. The Holder acknowledges and agrees that if this Note is converted prior to the date that is one year following the date of issuance of this Note, or the Company otherwise reasonably determines that inclusion of such a legend is necessary or advisable, any certificate representing Ordinary Shares issued upon conversion will bear a restrictive legend designed to ensure compliance with applicable securities laws.
 
3. Payment Terms. All payments shall be made in Euros by wire transfer to such account as notified in writing by the Holder at least fifteen calendar days prior to the relevant payment date. Payment shall be credited first to the accrued interest then due and payable and the remainder applied to principal. Payment of this Note may be made only upon the written consent of the Holder, which consent shall not be unreasonably withheld or delayed in the case of a payment in connection with a Change of Control; provided, however, that the Company shall provide at least fifteen (15) calendar days’ prior written notice of any requested payment in connection with a Change of Control; provided, further, that the Company shall not be required to provide written notice of any requested payment in connection with a Change of Control prior to the public announcement of such Change of Control.  “Euro” or “” means the lawful currency of the member states of the European Union that have adopted the single currency in accordance with the Treaty establishing the European Community, as amended from time to time.
 
4. Transfer; Successors and Assigns . The terms and conditions of this Note shall inure to the benefit of and be binding upon the respective successors and assigns of the parties. Notwithstanding the foregoing, the Holder may not offer, sell, assign, pledge, or otherwise transfer this Note without the prior written consent of the Company. Subject to the preceding sentence, this Note may be transferred only upon surrender of the original Note for registration of transfer, duly endorsed, or accompanied by a duly executed written instrument of transfer in form satisfactory to the Company. Thereupon, a new convertible promissory note for the same principal amount and interest will be issued to, and registered in the name of, the transferee. Interest and principal are payable only to the registered holder of this Note.  Upon any consolidation or merger of the Company with or into, or sale of all or substantially all of the assets of the Company to, any other entity, or any transaction similar thereto (as determined by the Company in good faith), such other entity shall automatically succeed to and be substituted for the Company, with the same effect as if named herein as the “Company,” and the entity named herein as the “Company” may be dissolved, wound up or liquidated and shall be released from its liabilities as obligor and maker of this Note. Upon any such succession, if the Company (or such successor) so requests, the Holder shall promptly execute an amendment to this Note to evidence such succession; provided that no such amendment shall be necessary for such succession to be effective.
 
5. Governing Law. This Note and all acts and transactions pursuant hereto and the rights and obligations of the parties hereto shall be governed, construed and interpreted in accordance with the laws of the State of New York, without giving effect to principles of conflicts of law to the extent inconsistent with such choice of New York law.
 
6. Notices. Any notice required or permitted by this Note shall be in writing and shall be deemed sufficient upon receipt, when delivered personally or by courier, overnight delivery service or confirmed facsimile or e-mail, or forty-eight (48) hours after being deposited in the U.S. mail as certified or registered mail with postage
 
 
 

 
prepaid, if such notice is addressed to the party to be notified at such party’s address, facsimile number or e-mail as set forth on the signature page hereto or as subsequently modified by written notice.
 
7. Amendments and Waivers . Any term of this Note may be amended or waived only with the written consent of the Company and the Holder. Any amendment or waiver effected in accordance with this Section 7 shall be binding upon the Company, the Holder and their successors and assigns.
 
8. Shareholders and Directors Not Liable. In no event shall any shareholder, supervisory director or managing director of the Company be liable for any amounts due or payable pursuant to this Note.
 
9. Counterparts. This Note may be executed in two or more counterparts, all of which together shall constitute one and the same instrument. This Note may also be executed and delivered by facsimile or other electronic delivery of signature.
 
10. Titles and Subtitles. The titles and subtitles used in this Note are used for convenience only and are not to be considered in construing or interpreting this Note.
 
[Signature Page Follows]
 
 
 

 
This Note is executed and delivered as of the date first set forth above.
 
 
COMPANY:
 
 
PROSENSA HOLDING N.V.
 
 
 
By:
 
   
Name:
 
   
Title:
 
       
 
By:
 
   
Name:
 
   
Title:
 
     
   
Address for notices:
   
Berndt Modig
   
Tel: + 31 61 47 90 201
   
J.H. Oortweg 21
   
2333 CH Leiden, the Netherlands
 
 
 
AGREED TO AND ACCEPTED:
 
PRO200, LLC
 
 
By:
   
   
Name:
 
   
Title:
 
    Address for notices:  
       
 
PRO200, LLC
 
  1400 Quail Street, Suite 110  
  Newport Beach, CA 92660  
  Attention: Debra Miller  

 
 

 
Appendix A
 
Conversion Notice
 
The undersigned hereby elects to convert the herein-enclosed Convertible Promissory Note of Prosensa Holding N.V., dated [____], 2014, with principal amount equal to €[________].
 
 
PRO200, LLC
 
 
 
By:
 
   
Name:
 
   
Title:
 
   
Date:
 
   
 
Deliver this Notice with the original Note to the offices of the Company in accordance with Section 2(c) of the Note.
 
 
 

 
Exhibit B
 
DMD Project
 
*****
 
 
 
 

 
Exhibit C
 
Press Release
 
 
 
 

 
 
Prosensa and CureDuchenne Strengthen Long-term Collaboration with an Additional €5M to Advance DMD Pipeline

LEIDEN, The Netherlands August 11, 2014 – Prosensa Holding N.V. (NASDAQ: RNA), the biopharmaceutical company focusing on RNA-modulating therapeutics for rare diseases with high unmet need, today announced that an affiliate of CureDuchenne, a US national nonprofit organization dedicated to finding a cure for Duchenne Muscular Dystrophy (DMD), will provide Prosensa with up to €5 million by means of convertible promissory notes to support the company and accelerate the development and patient access of much needed DMD therapies. The closing of €4.5 million of the notes is contingent upon specified milestones in the advancement of drisapersen and the Company’s other exon skipping candidates in Prosensa’s DMD portfolio.
“The ability for industry and patient organizations to work collaboratively is crucial to developing much needed treatment options for rare diseases such as DMD,” said Hans Schikan, CEO of Prosensa. “CureDuchenne has been a dedicated supporter of Prosensa since the company’s inception, and we are very appreciative of the additional funding for our extensive DMD program.”

The funding arrangement will assist the Company in a number of efforts that are core to Prosensa’s mission of developing innovative, RNA-based therapeutics to address unmet medical needs for patients with rare genetic disorders including:
 
 
·
Progressing the second exon skipping candidate for the treatment of DMD, PRO044, by initiating a Phase II clinical extension study in Europe by the end of 2014 and a placebo-controlled trial in the US in the first half of 2015, which may serve as one of two confirmatory studies to support a potential accelerated approval for drisapersen.

 
 
·
Supporting re-dosing efforts for drisapersen clinical trial participants in North America and Europe and facilitate the drug’s New Drug Application (NDA) filing in the US in 2014.

 
 
·
Supporting the development of other Prosensa’s other exon skipping compounds, PRO045 and PRO053.
 
Prosensa and CureDuchenne have been collaborating since 2004 when CureDuchenne provided valuable financial support to help advance the company’s extensive pipeline of DMD products.

“We are very encouraged by the pioneering progress that Prosensa has made in Duchenne and are committed to the long-standing collaboration we have with Prosensa to support the development of treatments for boys diagnosed with this rare and devastating genetic disease,” said Debra Miller, CEO & Founder of CureDuchenne. “This partnership underscores the important role that patient groups play in accelerating the research and development for Duchenne and other diseases,” she added.

In June Prosensa received positive feedback from the United States Food and Drug Administration (FDA) enabling it to pursue an NDA filing for its lead DMD therapy, drisapersen, under an accelerated approval pathway based on existing data. The FDA’s guidance also requested that the company commence a confirmatory randomized, placebo controlled study in a follow-on exon skipping drug with a similar mechanism of action prior to a potential approval for drisapersen. The placebo controlled trial with PRO044 anticipated to start in the first half of 2015 may serve this purpose.

 
 

 
About Prosensa Holding N.V.
Prosensa (NASDAQ: RNA) is a Dutch biotechnology company engaged in the discovery and development of RNA-modulating therapeutics for the treatment of genetic disorders. Its primary focus is on rare neuromuscular and neurodegenerative disorders with a large unmet medical need, including Duchenne muscular dystrophy (DMD), myotonic dystrophy and Huntington’s disease.

Prosensa’s current portfolio includes six compounds for the treatment of DMD, all of which have received orphan drug status in the United States and the European Union. The compounds use an innovative technique called exon-skipping to provide a personalized medicine approach to treat different populations of DMD patients. www.prosensa.com

About CureDuchenne
CureDuchenne is a US national nonprofit organization located in Newport Beach, California, dedicated to finding a cure for Duchenne, the most common and most lethal form of muscular dystrophy. As the leading genetic killer of young boys, Duchenne affects more than 300,000 boys worldwide.
CureDuchenne has garnered international attention for its efforts to raise funds and awareness for Duchenne through venture philanthropy. With the help of CureDuchenne’s distinguished international panel of Scientific Advisors, funds raised by CureDuchenne support the most promising research aimed at treating and curing Duchenne. To date, seven CureDuchenne research projects have made their way into human clinical trials – a unique accomplishment as few health-related nonprofits have been as successful in being a catalyst for human clinical trials. www.cureduchenne.org

About DMD
Duchenne muscular dystrophy (DMD) is a severely debilitating childhood neuromuscular disease that affects up to 1 in 3,500 live male births. This rare disease is caused by mutations in the dystrophin gene, resulting in the absence or defect of the dystrophin protein. As a result, patients suffer from progressive loss of muscle strength, often rendering them wheelchair-bound before the age of 12. Respiratory and cardiac muscle can also be affected by the disease and most patients die in early adulthood due to respiratory and cardiac failure.

About exon skipping
The dystrophin gene is the largest gene in the body, consisting of 79 exons. Exons are small segments of genetic code which, via an intermediate step involving RNA, lead to the assembly of sections of protein. In DMD, when certain exons are mutated/deleted, the RNA cannot be processed past the fault. This prevents the remainder of the exons from being read, resulting in a non-functional dystrophin protein and the severe symptoms of DMD. RNA-based therapeutics, specifically antisense oligonucleotides inducing exon skipping, are currently in development for DMD. These antisense oligonucleotides skip an exon next to, or containing, the fault and thereby correct the RNA processing, enabling the production of a novel, largely functional dystrophin protein. Prosensa’s exon skipping technology was licensed from Leiden University Medical Center.

About PRO044
PRO044 induces exon 44 skipping in the dystrophin gene and is intended for up to approximately 6% of all DMD patients, including those with deletions of exon 43, exon 45, exons 38-43, exons 40-43, exons 42-43, and exons 45-54. PRO044 has been granted orphan drug status in the European Union and the United States.

PRO044 has completed a Phase I/II dose-escalation study in Europe. Data from this study were presented at the World Muscle Society Congress in October 2013. PRO044 was generally well-tolerated up to dose levels of 12mg/kg for five weeks by subcutaneous or intravenous administration. Safety findings of the study are consistent with the known class safety profile and no drug related serious adverse events were reported.

About the notes
A total of €5.0 million of notes may be issued under a note purchase agreement executed on August 11, 2014.  A €0.5 million note will be issued initially, and the remaining €4.5 million of notes will be issued if and when specified milestones are met. The notes bear interest at a below-market interest rate that accumulates to the principal amount of the note. The notes must be repaid on fixed terms on the earliest to occur of a change of control, twelve months from when the Company obtains regulatory approval of its first product candidate and June 30, 2019. CureDuchenne has the option, under specified conditions, to convert the notes into the Company’s ordinary shares prior to maturity at a conversion price based on the Company’s share price at the time of conversion.

 
 

 
Forward Looking Statement
This press release contains certain forward-looking statements.  All statements, other than statements of historical facts, contained in this press release, including statements regarding our strategy, future operations, future financial position, future revenues, projected costs, prospects, plans and objectives of management, are forward-looking statements. The words “anticipate,” “believe,” “estimate,” “expect,” “intend,” “may,” “plan,” “predict,” “project,” “target,” “potential,” “will,” “would,” “could,” “should,” “continue,” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Forward-looking statements in this press release include statements around our exon skipping programs, drisapersen, PRO044, PRO045, PRO053, funding around these programs and the regulatory review of our product candidates. Actual results may differ materially from those projected or implied in such forward-looking statements.  Such forward-looking information involves risks and uncertainties that could significantly affect expected results.  These risks and uncertainties are discussed in the Company’s SEC filings, including, but not limited to, the Company’s Form 6-K’s and the Company’s Annual Report on Form 20-F. In addition, any forward-looking statements represent our views only as of today and should not be relied upon as representing our views as of any subsequent date. While we may elect to update these forward-looking statements at some point in the future, we specifically disclaim any obligation to do so, even if our views change.

Contact:
Prosensa Holding N.V.
Celia Economides, Senior Director IR & Corporate Communications
Phone: +1 917 941 9059
Email: c.economides@prosensa.nl
 

 
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