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., Aug. 08, 2024 (GLOBE NEWSWIRE) --  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

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