EX-99.1 2 a14-24729_1ex99d1.htm PRESS RELEASE OF UNIQURE N.V. DATED DECEMBER 1, 2014, ANNOUNCING THE COMPANY'S RESULTS

Exhibit 99.1

 

 

FOR IMMEDIATE RELEASE

 

uniQure Announces Results for the Third Quarter of 2014

 

Amsterdam, the Netherlands, December 01, 2014 — uniQure N.V. (NASDAQ: QURE), a leader in human gene therapy, today announced results for the third quarter of 2014.

 

Corporate Highlights and Pipeline Program Updates

 

·                  Hemophilia B Program - uniQure is finalizing all regulatory, trial site and administrative preparations for the initiation of its Phase 1/2 clinical trial in hemophilia B. The Company expects to complete these efforts as planned by the end of 2014 and anticipates the dosing of the first patient to be announced in early 2015. The Company still expects to announce initial results in mid-2015.

·                  Glybera® (alipogen tiparvovec) - uniQure’s commercialization partner Chiesi confirmed that the first commercial treatment of a LPLD patient with Glybera will occur in the last quarter of 2014/first quarter 2015.

·                  Collaborator-sponsored Pipeline Programs - In October, top-line results from the Phase 1 clinical trial in acute intermittent porphyria (AIP) confirmed the safety and successful transduction of patients’ liver cells with the porphobilinogen deaminase gene (PBGD) using uniQure’s proprietary AAV5 viral vector, corroborating the interim analysis presented in the second quarter of 2014. These data were presented by the AIPGENE Consortium, a pan-European collaboration supported by the EU Seventh Framework Programme, at the European Society of Gene and Cell Therapy (ESGCT) and Netherlands Society of Gene and Cell Therapy (NVGCT) Collaborative Congress. In the ongoing Parkinson’s disease clinical trial conducted by uniQure’s partners at UCSF and NIH, the sixth and final patient in the first dose cohort has been treated.

·                  Lexington, Massachusetts Manufacturing Facility - Approximately 40 employees have initiated preparations for production of the first GMP batch expected in H1 2015.

·                  InoCard Acquisition - uniQure has completed the integration of the German cardiology gene therapy company Inocard, acquired in July 2014, which is now called uniQure Germany GmbH.

 

Jörn Aldag, uniQure Chief Executive Officer, commented: “uniQure continues to direct key resources toward advancing the Company’s core programs, most prominently hemophilia B, where we expect to announce preliminary safety and efficacy data in mid-2015, and our earlier-staged hemophilia A and congestive heart failure preclinical development programs.”

 

Financial Highlights

 

As of September 30, 2014, the Company held cash and cash equivalents of €62.8 million. Total revenues (consisting of licensing and collaboration revenues) for the three months ended September 30, 2014 were €1.0 million, compared with €1.3 million in the same period of 2013. For the nine months ended September 30, 2014, total revenues were €3.2 million, compared to €2.1 million in the first nine months of 2013. Collaboration revenues represent development activities that are reimbursable by Chiesi under the Company’s co-development agreement for hemophilia B. License revenues represent the monthly amortization of the upfront payments received under the Chiesi agreements related to Glybera® entered into in June 2013.

 

Research and development expenses were €9.5 million for the three months ended September 30, 2014, compared to €3.2 million for the same period in 2013. Research and development expenses for the nine months ended September 30, 2014 were €23.7 million, compared to €9.6 million for the same period in 2013. The increase in research and development expenses reflect additional development and clinical activities required to support the planned commercial launch of Glybera®, in addition to the continued progression of uniQure’s other programs through late stage research and early clinical development.

 

Net loss for the three months ended September 30, 2014 was €9.1 million or €0.51 per share, compared to €7.0 million or €0.61 per share for the same period in 2013. Net loss for the nine months ended September 30, 2014 was €25.9

 



 

million or €1.54 per share, compared to €20.0 million or €1.94 per share for the same period in 2013. Net loss for both the three and nine months ended September 30, 2014 was affected by a favourable €3.6 million related to foreign exchange results.

 

For further financial information for the period ending September 30, 2014, please refer to the financial statements appearing at the end of this release.

 

Webcast

 

uniQure will host an Investor Day in New York City and simultaneous webcast on December 1, 2014, at 8:30 am ET to provide a detailed update on the Company’s programs as well as a discussion on the future of gene therapy with distinguished guest speakers. To access the webcast, log on to http://www.media-server.com/m/p/tfx7rb2k or go to the “Events” section on the “Media” page of uniQure’s website, at http://www.uniqure.com/news/calendar-of-events. A replay of the webcast will be available on uniQure’s website for at least 30 days following the event.

 

About uniQure

 

uniQure is delivering on the promise of gene therapy through single treatments with potentially curative results. We have developed a modular platform to rapidly bring new disease-modifying therapies to patients with severe disorders. We are engaged in multiple partnerships and have obtained regulatory approval of our lead product, Glybera, in the European Union for a subset of patients with LPLD. www.uniQure.com

 

FORWARD-LOOKING STATEMENTS

 

This press release contains forward-looking statements. All statements other than statements of historical fact are forward-looking statements, which are often indicated by terms such as “anticipate,” “believe,” “could,” “estimate,” “expect,” “goal,” “intend,” “look forward to”, “may,” “plan,” “potential,” “predict,” “project,” “should,” “will,” “would” and similar expressions. Forward-looking statements are based on management’s beliefs and assumptions and on information available to management only as of the date of this press release. These forward-looking statements include, but are not limited to, statements regarding the timeframe and anticipated results of our research and development programs and those of our collaborators, as well as the commercial success of Glybera. Our actual results could differ materially from those anticipated in these forward-looking statements for many reasons, including, without limitation, risks associated with our clinical development activities, including the risk of cessation or delay of any of our ongoing or planned clinical studies and/or development of our product candidates; the risk of delay or failure to successfully commercialize or obtain or maintain regulatory approval of our products; risks related to our manufacturing processes and facilities, including our compliance with applicable regulatory requirements; the risk that our collaborations or our other collaboration partners will not continue or will not be successful; risks related to regulatory oversight, including our compliance with post-approval requirements; risks related to product commercialization; risks related to intellectual property claims; and the risks, uncertainties and other factors described under the heading “Risk Factors” in uniQure’s Form 20-F filed with the Securities and Exchange Commission dated April 25, 2014. Given these risks, uncertainties and other factors, you should not place undue reliance on these forward-looking statements, and we assume no obligation to update these forward-looking statements, even if new information becomes available in the future.

 

uniQure:

Aicha Diba

Investor Relations

Phone: +31 20 240 6100

a.diba@uniQure.com

 

Media inquiries:

Gretchen Schweitzer

MacDougall Biomedical Communications

Direct: +49 172 861 8540

Main: +49 89 2424 3494 or +1 781 235 3060

gschweitzer@macbiocom.com

 



 

UNIQURE N.V.

 

Unaudited Condensed Consolidated Balance Sheets

(€ in thousands)

 

 

 

DECEMBER 31,

 

SEPTEMBER, 30

 

 

 

2013

 

2014

 

Assets

 

 

 

 

 

Non-current assets

 

 

 

 

 

Goodwill

 

 

1,342

 

Other Intangible assets, net

 

7,775

 

15,311

 

Property, plant and equipment

 

2,614

 

17,055

 

Other non-current assets

 

923

 

978

 

Total non-current assets

 

11,312

 

34,686

 

Current assets

 

 

 

 

 

Receivables from related parties

 

1,425

 

1,138

 

Trade and Other Receivables

 

1,557

 

2,188

 

Inventories

 

865

 

539

 

Cash and cash equivalents

 

23,810

 

62,782

 

Total current assets

 

27,657

 

66,647

 

Total assets

 

38,969

 

101,333

 

Equity

 

 

 

 

 

Share capital

 

610

 

892

 

Share premium

 

142,459

 

205,811

 

Other reserves

 

6,536

 

14,422

 

Accumulated deficit

 

(144,041

)

(169,920

)

Total equity

 

5,564

 

51,205

 

Liabilities

 

 

 

 

 

Non-current liabilities

 

 

 

 

 

Borrowings

 

6,292

 

15,684

 

Financial lease liabilities

 

302

 

177

 

Deferred rent

 

680

 

5,525

 

Deferred revenue

 

15,679

 

15,017

 

Deferred Tax Liabilities

 

 

1,379

 

Contingent Considerations

 

 

1,301

 

Total non-current liabilities

 

22,953

 

39,083

 

Current liabilities

 

 

 

 

 

Trade and other payables

 

7,601

 

8,924

 

Debt to related party - derivative

 

722

 

272

 

Borrowings

 

633

 

 

Borrowings - derivative

 

217

 

93

 

Deferred rent

 

 

451

 

Deferred revenue

 

1,279

 

1,305

 

Total Current Liabilities

 

10,452

 

11,045

 

Total liabilities

 

33,405

 

50,128

 

Total equity and liabilities

 

38,969

 

101,333

 

 



 

UNIQURE N.V.

 

Unaudited Condensed Consolidated Statements of Comprehensive Loss

(€ in thousands, except share and per share data)

 

 

 

THREE MONTHS ENDED

 

NINE MONTHS ENDED

 

 

 

SEPTEMBER 30,

 

SEPTEMBER 30,

 

 

 

as restated

 

 

 

as restated

 

 

 

 

 

2013

 

2014

 

2013

 

2014

 

 

 

(€
in thousands)

 

 

 

 

 

 

 

License revenues

 

220

 

221

 

220

 

662

 

Collaboration revenues

 

1,073

 

780

 

1,831

 

2,551

 

Total revenues

 

1,293

 

1,001

 

2,051

 

3,213

 

Cost of goods sold

 

 

 

(800

)

 

Other income

 

171

 

208

 

562

 

598

 

Research and development expenses

 

(3,152

)

(9,514

)

(9,573

)

(23,740

)

Selling, general and administrative expenses

 

(3,394

)

(3,218

)

(7,551

)

(8,035

)

Other gains / losses, net

 

(304

)

3,630

 

(269

)

3,694

 

Total Operating Costs

 

(6,679

)

(8,894

)

(16,831

)

(27,483

)

Operating result

 

(5,386

)

(7,893

)

(15,580

)

(24,270

)

Finance income

 

4

 

54

 

48

 

125

 

Finance expense

 

(1,656

)

(1,220

)

(4,470

)

(1,734

)

Finance income/(expense)—net

 

(1,652

)

(1,166

)

(4,422

)

(1,609

)

Result before corporate income tax

 

(7,038

)

(9,059

)

(20,002

)

(25,879

)

Corporate income taxes

 

 

 

 

 

Net Loss

 

(7,038

)

(9,059

)

(20,002

)

(25,879

)

Items that may be subsequently reclassified to profit or loss

 

6

 

703

 

6

 

693

 

Other comprehensive income

 

6

 

703

 

6

 

693

 

Total comprehensive loss

 

(7,032

)

(8,356

)

(19,996

)

(25,186

)

Loss per share attributable to the equity holders of the Company during the year

 

 

 

 

 

 

 

 

 

Basic and diluted loss per share

 

(0.61

)

(0.51

)

(1.94

)

(1.54

)

 



 

UNIQURE N.V.

 

Unaudited Condensed Consolidated Statement of Changes in Equity/Deficit

(€ in thousands)

 

 

 

Total

 

 

 

 

 

 

 

 

 

 

 

Share

 

Share

 

Other

 

Accumulated

 

Total

 

 

 

Capital

 

Premium

 

Reserves

 

Deficit

 

Equity/Deficit

 

Balance at January 1, 2013

 

483

 

114,795

 

1,508

 

(117,234

)

(448

)

Result for the period

 

 

 

 

(20,002

)

(20,002

)

Other Comprehensive Income

 

 

 

 

 

 

6

 

6

 

Capital contributions

 

127

 

27,649

 

 

 

27,776

 

Result on conversion of the loan

 

 

 

3,005

 

 

3,005

 

Share based payment/expense

 

 

 

1,411

 

 

 

1,411

 

Balance at September 30, 2013 (as restated)

 

610

 

142,444

 

5,924

 

(137,230

)

11,748

 

Result for the period

 

 

 

 

(6,818

)

(6,818

)

Other Comprehensive Income

 

 

 

 

7

 

7

 

Capital contributions

 

 

15

 

 

 

15

 

Result on conversion of the loan

 

 

 

 

 

 

Share based payment/expense

 

 

 

612

 

 

612

 

Balance at December 31, 2013

 

610

 

142,459

 

6,536

 

(144,041

)

5,564

 

Result for the period

 

 

 

 

(25,879

)

(25,879

)

Other Comprehensive Income

 

 

 

693

 

 

693

 

Proceeds from shares issued

 

282

 

64,020

 

 

 

64,302

 

Share issuance costs

 

 

(668

)

 

 

(668

)

Share based payment/expense

 

 

 

7,193

 

 

7,193

 

Balance at: September 30, 2014

 

892

 

205,811

 

14,422

 

(169,920

)

51,205

 

 



 

UNIQURE N.V.

 

Unaudited Condensed Consolidated Statement of Cash Flows

(€ in thousands)

 

 

 

NINE MONTHS ENDED

 

 

 

SEPTEMBER 30,

 

 

 

as restated

 

 

 

 

 

2013

 

2014

 

Cash flow from operating activities

 

 

 

 

 

Net loss

 

(20,002

)

(25,879

)

Adjustments for:

 

 

 

 

 

Depreciation

 

398

 

859

 

Lease incentive

 

 

4,854

 

Derivative result

 

2,339

 

(574

)

Derivative result arising on early conversion of loan

 

1,333

 

 

Exchange result

 

(4

)

(2,545

)

Other non-cash items

 

 

(1

)

Share-based expenses

 

1,411

 

7,193

 

Changes in other non-current assets

 

(917

)

 

Changes in trade and other receivables

 

(1,937

)

(142

)

Movement in inventories

 

(427

)

326

 

Changes in trade and other payables

 

(282

)

(1,812

)

Changes in deferred revenue and provisions

 

17,102

 

(635

)

Movement in other liabilities

 

1,701

 

1,209

 

Interest (income) / expense

 

956

 

1,035

 

Cash used in operations

 

1,671

 

(16,112

)

Interest paid

 

(17

)

(807

)

Net cash used in operating activities

 

1,654

 

(16,919

)

Cash flow from investing activities

 

 

 

 

 

Purchases of property, plant and equipment

 

(536

)

(13,365

)

Purchases of intangible assets

 

(3,623

)

(2,129

)

Interest received

 

 

95

 

Acquisition of businesses

 

 

(1,463

)

Net cash used in investing activities

 

(4,159

)

(16,862

)

Cash flow from financing activities

 

 

 

 

 

Capital contribution from shareholders

 

14,278

 

 

Proceeds from shares issued

 

 

62,786

 

Share issuance cost

 

 

(668

)

Convertible loans drawn down

 

11,999

 

 

Proceeds from borrowings

 

7,492

 

7,184

 

Redemption of financial lease

 

(106

)

(116

)

Net cash generated from financing activities

 

33,663

 

69,186

 

Net increase in cash, cash equivalents and bank overdrafts

 

31,158

 

35,405

 

Currency effect cash and cash equivalents

 

6

 

3,567

 

Cash, cash equivalents and bank overdrafts at beginning of the period

 

263

 

23,810

 

Cash, cash equivalents and bank overdrafts at end of the period

 

31,427

 

62,782