EX-99.2 3 tmb-20240630xex99d2.htm EX-99.2

Exhibit 99.2

ProQR Announces Second Quarter 2024 Operating and Financial Results

AX-0810 program targeting NTCP for cholestatic diseases and AX-1412 program targeting B4GALT1 for cardiovascular diseases advancing to the clinic in late 2024/early 2025 with translational data and trial design details to be announced in H2 2024
$4.5 million in milestones achieved in H1 related to progress in the Lilly partnership
€96.2 million cash position as of end of Q2 providing runway into mid-2026, plus additional potential milestones from Lilly partnership

LEIDEN, Netherlands & CAMBRIDGE, Mass., August 8, 2024 – ProQR Therapeutics NV. (Nasdaq: PRQR) (ProQR), a company dedicated to changing lives through transformative RNA therapies based on its proprietary Axiomer RNA editing technology platform, today reported its financial and operating results for the second quarter ended June 30, 2024, and provided a business update.

“In the second quarter, we took important steps toward advancing our first editing oligonucleotide programs to clinical stage, with AX-0810 targeting NTCP for cholestatic diseases and AX-1412 targeting B4GALT1 for cardiovascular diseases on track to enter the clinic in late 2024/early 2025,” said Daniel A. de Boer, Founder and CEO of ProQR. “We were excited to share data at ASGCT in May, demonstrating in vivo proof of target engagement with meaningful changes in disease-relevant biomarkers in non-human primates for our NTCP program. As we progress our CTA/IND-enabling studies, we plan to announce translational data and more about our clinical plans for AX-0810 in the second half of the year, as well as preclinical proof of concept and translational data for AX-1412.”

de Boer continued, “Beyond our own pipeline programs, we are pleased with the progress we are making in our partnership with Lilly and highlight the achievement of multiple milestones in the first half of the year. With the benefits of a productive partnership, a differentiated pipeline strategy rooted in human genetics, our leading IP portfolio, and a strong cash balance with runway into mid-2026, we look forward to continued execution as we advance our science and pipeline programs.”

Recent Progress and Anticipated Upcoming Events

In June, ProQR presented at the RNA Editing Summit in Boston, highlighting its Axiomer technology platform and AX-0810, the Company’s program targeting NTCP for cholestatic diseases.
In May at the American Society of Cell and Gene Therapy (ASGCT) Annual Meeting, ProQR presented preclinical proof of concept in vitro and in vivo data for AX-0810 for Cholestatic

Diseases targeting NTCP (poster P-705). ProQR scientists showed for the first time in the ADAR RNA editing field in vivo proof of target engagement (RNA editing) with meaningful changes in biomarkers in NHPs using Axiomer RNA Editing Oligonucleotides. At ASGCT, ProQR and partner Eli Lilly presented preclinical data related to Axiomer in poster P-726 titled “Complex Metabolism and Prolonged PK/PD of a GalNAc-Conjugated Editing Oligonucleotide (EON) in Mice”.
Anticipated Upcoming Events:
AX-0810 targeting NTCP for cholestatic diseases: clinical development candidate translational data to be reported, and design for the clinical trial to be shared in H2 2024 with program to advance to the clinic in late 2024/early 2025.
AX-1412 targeting B4GALT1 for cardiovascular diseases: preclinical proof of concept data and translational data to be reported in H2 2024 with program to advance to the clinic in late 2024/early 2025.
Potential additional new pipeline target announcements in 2024.
Continue to execute on partnership with Eli Lilly and Company (Lilly), with potential additional data updates to come in 2024, along with potential additional milestone income from existing partnership, and potential option to exercise for expansion of deal to 15 targets, which would result in a $50 million opt-in payment to ProQR.
ProQR may selectively form new partnerships, which could include multi-target discovery alliances, or product alliances on specific programs.

Financial Highlights

At June 30, 2024, ProQR held cash and cash equivalents and short term financial assets of €96.2 million, compared to €118.9 million cash and cash equivalents at December 31, 2023. Net cash used in operating activities during the three-month period ended June 30, 2024 was €6.3 million, compared to €10.3 million used for the same period last year. During the first half of 2024, the Company achieved certain milestones in the collaboration agreement with Eli Lilly amounting to $4.5 million (€4.2 million). Milestone income achieved in Q2 was received in July.

Research and development (R&D) costs were €7.1 million for the quarter ended June 30, 2024 compared to €5.9 million for the same period last year.

General and administrative costs were €3.0 million for the quarter ended June 30, 2024 compared to €4.1 million for the same period last year.

Net loss for the three-month period ended June 30, 2024 was €2.7 million, or €0.03 per diluted share, compared to €8.0 million, or €0.10 per diluted share, for the same period last year. For further financial information for the period ended June 30, 2024, please refer to the Q2 financial report filing.


About Axiomer™

ProQR is pioneering a next-generation RNA base editing technology called Axiomer, which could potentially yield a new class of medicines for diverse types of diseases. Axiomer “Editing Oligonucleotides”, or EONs, mediate single nucleotide changes to RNA in a highly specific and targeted way using molecular machinery that is present in human cells called ADAR (Adenosine Deaminase Acting on RNA). Axiomer EONs are designed to recruit and direct endogenously expressed ADARs to change an Adenosine (A) to an Inosine (I) in the RNA – an Inosine is translated as a Guanosine (G) – correcting an RNA with a disease-causing mutation back to a normal (wild type) RNA, modulating protein expression, or altering a protein so that it will have a new function that helps prevent or treat disease.

About Biliary Atresia (BA) and Primary Sclerosing Cholangitis (PSC)

Cholestatic disorders refer to a group of diseases presenting excessive and toxic buildup of bile acids in the liver due to bile ducts dysfunction. This leads to liver damage and a range of debilitating symptoms. Without treatment, liver damage can progress through various stages, ultimately leading to liver failure and elevated risk of liver malignancy, affecting life expectancy. Cholestatic diseases remain leading causes of liver transplantation. There are no approved therapies for primary sclerosing cholangitis (PSC) for adults and biliary atresia (BA) for pediatrics It is estimated that 80,000 and 20,000 individuals have PSC and BA, respectively, in North America and in Europe.

About AX-0810 targeting NTCP

The majority of the bile acids present in the liver cells originate from the enterohepatic reuptake cycle. The key transporter responsible for hepatic uptake of bile acids from portal circulation is the sodium (Na+)-taurocholate cotransporting polypeptide (NTCP, SLC10A1 gene) expressed in the liver. AX-0810 is designed to introduce a loss of function variant in SLC10A1 RNA that has been found in human genetics to prevent re-uptake of bile acids in liver via NTCP. Based on its mechanism of action, AX-0810 has the potential to become a disease modifying treatment for PSC and BA primarily among other cholestatic diseases.

About Cardiovascular Diseases

Cardiovascular diseases (CVDs) are a group of health conditions that affect the heart and blood vessels, such as atherosclerosis which can lead to severe problems like heart attacks, heart failure, and stroke. CVDs represent the leading cause of disability and death in the world. Approximately 18 million people die every year from CVDs representing one third of all the global deaths. Despite available lipid lowering therapies and hypertension medications, the risk of CVDs is still projected to increase rapidly over the coming years.


About AX-1412 targeting B4GALT1

Gene–based analysis of rare beta-1,4-galactosyltransferase 1 (B4GALT1) missense variant (p.Asn352Ser) is known to lead to B4GALT1 protein loss of function and showed an association with decreased coronary artery disease. These beneficial effects are mediated by hypo-galactosylation of the apolipoprotein B100 and fibrinogen, known – independent – drivers of increased risk of CVDs. AX-1412 introduces a protective variant into B4GALT1 RNA to address the remaining residual risk of developing cardiovascular diseases. ProQR intends to advance AX-1412 targeting B4GALT1 to early clinical proof of concept stage, then would seek to partner this program.

About ProQR

ProQR Therapeutics is dedicated to changing lives through the creation of transformative RNA therapies. ProQR is pioneering a next-generation RNA technology called Axiomer, which uses a cell’s own editing machinery called ADAR to make specific single nucleotide edits in RNA to reverse a mutation or modulate protein expression and could potentially yield a new class of medicines for both rare and prevalent diseases with unmet need. Based on our unique proprietary RNA repair platform technologies we are growing our pipeline with patients and loved ones in mind.

Learn more about ProQR at www.proqr.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 “continue,” "anticipate," "believe," "could," "estimate," "expect," "goal," "intend," "look forward to", "may," "plan," "potential," "predict," "project," "should," "will," "would" and similar expressions. Such forward-looking statements include, but are not limited to, statements regarding our business, technology, strategy, preclinical model data, our initial pipeline targets and the upcoming strategic priorities and milestones related thereto, our Axiomer platform, including the continued development and advancement of our Axiomer platform, the therapeutic potential of our Axiomer RNA editing oligonucleotides and our ability to expand preclinical in vivo and in vitro data, the timing, progress and results of our preclinical studies and other development activities, including the release of data related thereto, our patent estate, including our anticipated strength and our continued investment in it, as well as the timing of our clinical development, the potential of our technologies and product candidates, the collaboration with Lilly and the intended and potential benefits thereof, including the receipt of milestone and royalty payments from commercial product sales, if any, from the products covered by the collaboration, our ability to selectively form new partnerships and enter into future collaborations, and our financial position and cash-runway. 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. Our actual results could differ materially from those expressed or implied by these forward-looking statements for many reasons, including, without limitation, the risks, uncertainties and other factors in our filings


made with the Securities and Exchange Commission, including certain sections of our annual report filed on Form 20-F. These risks and uncertainties include, among others, the cost, timing and results of preclinical studies and clinical trials and other development activities by us and our collaborative partners whose operations and activities may be slowed or halted shortage and pressure on supply and logistics on the global market; the likelihood of our preclinical and clinical programs being initiated and executed on timelines provided and reliance on our contract research organizations and predictability of timely enrollment of subjects and patients to advance our clinical trials and maintain their own operations; our reliance on contract manufacturers to supply materials for research and development and the risk of supply interruption from a contract manufacturer; the potential for future data to alter initial and preliminary results of early-stage clinical trials; the unpredictability of the duration and results of the regulatory review of applications or clearances that are necessary to initiate and continue to advance and progress our clinical programs; the ability to secure, maintain and realize the intended benefits of collaborations with partners, including the collaboration with Lilly; the possible impairment of, inability to obtain, and costs to obtain intellectual property rights; possible safety or efficacy concerns that could emerge as new data are generated in research and development; general business, operational, financial and accounting risks, and risks related to litigation and disputes with third parties; and risks related to macroeconomic conditions and market volatility resulting from global economic developments, geopolitical instability and conflicts. 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, except as required by law.


ProQR Therapeutics N.V.

Investor contact:
Sarah Kiely
ProQR Therapeutics N.V.
T: +1 617 599 6228
skiely@proqr.com
or

Peter Kelleher
LifeSci Advisors
T: +1 617 430 7579
pkelleher@lifesciadvisors.com

Media contact:
Robert Stanislaro

FTI Consulting

T: +1 212 850 5657
robert.stanislaro@fticonsulting.com


Financial Tables

PROQR THERAPEUTICS N.V.
Unaudited Condensed Consolidated Statement of Financial Position

June 30, 

December 31, 

2024

2023

€1,000

€1,000

Assets

  

  

Current assets

  

  

Cash and cash equivalents

78,970

118,925

Financial asset - current

17,183

Prepayments and other receivables

5,116

1,538

Other taxes

497

523

Total current assets

101,766

120,986

Property, plant and equipment

16,285

16,897

Investments in financial assets

Total assets

118,051

137,883

Equity and liabilities

  

  

Equity

  

Equity attributable to owners of the Company

32,854

41,390

Total equity

32,854

41,390

Current liabilities

  

  

Borrowings

4,437

Lease liabilities

1,675

1,614

Derivative financial instruments

184

311

Trade payables

33

1,541

Social securities and other taxes

1,448

1,659

Deferred income

23,827

20,569

Other current liabilities

5,675

8,509

Total current liabilities

37,279

34,203

Borrowings

4,292

Lease liabilities

13,078

13,828

Deferred income

34,840

44,170

Total liabilities

85,197

96,493

Total equity and liabilities

118,051

137,883


PROQR THERAPEUTICS N.V.
Unaudited Condensed Consolidated Statement of Profit or Loss and OCI

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

Three month period

Six month period

ended June 30, 

 

ended June 30, 

    

2024

2023

 

2024

2023

€1,000

€1,000

€1,000

€1,000

Revenue

6,305

1,205

10,755

1,860

Other income

156

38

366

80

Research and development costs

(7,048)

(5,909)

(16,331)

(11,969)

General and administrative costs

(3,013)

(4,145)

(6,465)

(8,171)

Total operating costs

(10,061)

(10,054)

(22,796)

(20,140)

  

  

  

  

Operating result

(3,600)

(8,811)

(11,675)

(18,200)

Finance income and expense

513

470

1,001

(74)

Results related to associates and financial assets

Results related to financial liabilities measured at fair value through profit or loss

195

221

127

891

Results on derecognition of financial liabilities

101

509

  

  

  

  

Result before corporate income taxes

(2,892)

(8,019)

(10,547)

(16,874)

Income taxes

200

42

197

42

  

  

  

  

Result for the period

(2,692)

(7,977)

(10,350)

(16,832)

Other comprehensive income (foreign exchange differences on foreign operation)

85

7

276

(212)

  

  

  

  

Total comprehensive income

(2,607)

(7,970)

(10,074)

(17,044)

Result attributable to

  

  

  

  

Owners of the Company

(2,692)

(7,993)

(10,350)

(16,926)

Non-controlling interests

16

94

(2,692)

(7,977)

(10,350)

(16,832)

Total comprehensive income attributable to

Owners of the Company

(2,607)

(7,986)

(10,074)

(17,138)

Non-controlling interests

16

94

(2,607)

(7,970)

(10,074)

(17,044)

  

  

  

  

Share information

  

  

  

  

Weighted average number of shares outstanding1

81,665,565

80,939,392

81,618,038

80,913,751

Earnings per share attributable to owners of the Company (Euro per share)

Basic loss per share1

(0.03)

(0.10)

(0.13)

(0.21)

Diluted loss per share1

(0.03)

(0.10)

(0.13)

(0.21)

1.For these periods the potential exercise of share options is not included in the diluted earnings per share as the Company was loss-making. Due to the anti-dilutive nature of the outstanding options, basic and diluted earnings per share are equal.


PROQR THERAPEUTICS N.V.
Unaudited Condensed Consolidated Statement of Changes in Equity

Attributable to owners of the Company

  

Number
of shares

  

Share
Capital

  

Share
Premium

  

Equity settled
Employee
Benefit
Reserve

  

Translation
Reserve

  

Accumulated
Deficit

  

Total

  

Non-
controlling
interests

  

Total
Equity

 

  

€1,000

€1,000

€1,000

€1,000

€1,000

€1,000

€1,000

€1,000

Balance at January 1, 2023

 

84,246,967

3,370

412,540

29,052

1,212

(379,110)

67,064

(384)

66,680

Result for the period

 

(16,926)

(16,926)

94

(16,832)

Other comprehensive income

 

(212)

(212)

(212)

Recognition of share-based payments

 

1,860

1,860

1,860

Treasury shares transferred

(122,584)

Share options lapsed

(3,873)

3,873

Share options exercised

122,584

4

(231)

231

4

4

Balance at June 30, 2023

 

84,246,967

3,370

412,544

26,808

1,000

(391,932)

51,790

(290)

51,500

Balance at January 1, 2024

 

84,248,384

3,370

412,894

25,159

817

(400,850)

41,390

41,390

Result for the period

 

(10,350)

(10,350)

(10,350)

Other comprehensive income

 

276

276

276

Recognition of share-based payments

 

1,364

1,364

1,364

Treasury shares transferred

(326,455)

Share options lapsed

(359)

359

Share options exercised / RSUs vested

326,455

174

(288)

288

174

174

 

  

  

  

  

  

  

  

  

  

Balance at June 30, 2024

 

84,248,384

3,370

413,068

25,876

1,093

(410,553)

32,854

32,854


PROQR THERAPEUTICS N.V.
Unaudited Condensed Consolidated Statement of Cash Flows

Three month period 

Six month period 

ended June 30, 

 

ended June 30, 

    

2024

2023

2024

2023

€1,000

€1,000

€1,000

€1,000

Cash flows from operating activities

  

  

  

  

Net result

(2,692)

(7,977)

(10,350)

(16,832)

Adjustments for:

— Depreciation

711

594

1,402

1,143

— Share-based compensation

628

765

1,364

1,860

— Financial income and expenses

(513)

(470)

(1,001)

74

— Results related to associates and financial assets

— Results related to financial liabilities measured at fair value through profit or loss

(195)

(221)

(127)

(891)

— Results on derecognition of financial liabilities

(101)

(509)

— Income tax expenses

(200)

(197)

Changes in working capital

(4,614)

(3,622)

(13,838)

48,668

Cash (used in)/generated from operations

(6,875)

(11,032)

(22,747)

33,513

  

  

  

  

Corporate income tax received/(paid)

199

196

Interest received

610

685

1,542

865

Interest paid

(190)

(379)

  

  

  

  

Net cash (used in)/generated from operating activities

(6,256)

(10,347)

(21,388)

34,378

  

  

  

  

Cash flow from investing activities

Increase in financial asset - current

(17,000)

Purchases of property, plant and equipment

(267)

(294)

(999)

(430)

Sales of property, plant and equipment

47

  

  

  

  

Net cash used in investing activities

(267)

(294)

(17,999)

(383)

  

  

  

  

Cash flow from financing activities

  

  

  

  

Proceeds from exercise of share options

12

4

174

4

Repayment of lease liability

(294)

(647)

(875)

(906)

  

  

  

  

Net cash used in financing activities

(282)

(643)

(701)

(902)

  

  

  

  

Net (decrease)/increase in cash and cash equivalents

(6,805)

(11,284)

(40,088)

33,093

  

  

  

  

Currency effect cash and cash equivalents

62

860

133

694

Cash and cash equivalents, at beginning of the period

85,713

138,986

118,925

94,775

  

  

  

  

Cash and cash equivalents at the end of the period

78,970

128,562

78,970

128,562