PRQR ProQR Therapeutics N.V.

4.98
-0.26  -5%
Previous Close 5.24
Open 5.24
52 Week Low 4.46
52 Week High 10.98
Market Cap $248,833,892
Shares 49,966,645
Float 40,247,056
Enterprise Value $166,691,778
Volume 164,735
Av. Daily Volume 145,360
Stock charts supplied by TradingView

Upcoming Catalysts

Drug Stage Catalyst Date
QR-1123
autosomal dominant retinitis pigmentosa (adRP)
Phase 1/2
Phase 1/2
Premium membership is required to view catalyst dates, analyst ratings, earnings dates and cash burn data. Click here to unlock and sign up to a 14-day FREE TRIAL.

Drug Pipeline

Drug Stage Notes
Sepofarsen (QR-110 )
Leber's Congenital Amaurosis (LCA) - Genetic blindness
Phase 2/3
Phase 2/3
Phase 2/3 trial delayed due to COVID-19.
QR-421a - STELLAR
Usher Syndrome Type 2
Phase 1/2
Phase 1/2
Phase 1/2 interim data March 31, 2020. 2/8 patients demonstrated benefit across multiple concordant outcome measures.
QR-010
Cystic Fibrosis
Phase 1b
Phase 1b
Phase 1b top-line data released September 25, 2017. FEV levels not significant across four doses. One of four doses showed significance in subgroup.
QR-313
Epidermolysis bullosa
Phase 1/2
Phase 1/2
Phase 1/2 interim analysis announced March 26, 2019. Future development to be conducted by Wings Therapeutics.

Latest News

  1. LEIDEN, Netherlands & CAMBRIDGE, Mass., June 12, 2020 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (NASDAQ:PRQR), a company dedicated to changing lives through the creation of transformative RNA therapies for severe genetic rare diseases, today announced that the Company will launch a series of Expert Perspectives calls to provide an in depth review of topics related to the Company's pipeline and platform.

    The inaugural call in the series will be hosted on June 22, 2020 at 12pm EDT and will focus on Usher syndrome and retinitis pigmentosa, The call will feature a fireside chat between Aniz Girach, MD, Chief Medical Officer of ProQR Therapeutics, and Robert Koenekoop, MD, PhD of McGill University/Montreal Children's Hospital. Drs. Girach and…

    LEIDEN, Netherlands & CAMBRIDGE, Mass., June 12, 2020 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (NASDAQ:PRQR), a company dedicated to changing lives through the creation of transformative RNA therapies for severe genetic rare diseases, today announced that the Company will launch a series of Expert Perspectives calls to provide an in depth review of topics related to the Company's pipeline and platform.

    The inaugural call in the series will be hosted on June 22, 2020 at 12pm EDT and will focus on Usher syndrome and retinitis pigmentosa, The call will feature a fireside chat between Aniz Girach, MD, Chief Medical Officer of ProQR Therapeutics, and Robert Koenekoop, MD, PhD of McGill University/Montreal Children's Hospital. Drs. Girach and Koenekoop will also discuss QR-421a, ProQR's investigational therapy for patients with Usher syndrome type 2A and retinitis pigmentosa. They will discuss the role of genetic testing and outcome measures related to visual field and visual acuity, including OCT EZ area, DAC, FST, and BCVA. Additionally, they will discuss relative significance of slowing disease progression, stabilization, and visual improvement. An Usher syndrome patient will be participating in the call to discuss her experience with the disease.

    Future topics for the Expert Perspectives call series are planned, including autosomal dominant retinitis pigmentosa and ProQR's RNA oligonucleotide platform and Axiomer RNA editing approaches.

    Event Details

    Date/Time: June 22, 2020, 12-1pm EDT

    Topic: Usher syndrome and retinitis pigmentosa

    To register: https://lifescipartners.zoom.us/webinar/register/WN_h3OpQ_jURDuZKBe6URtBOg

    Following the discussion, a portion of the call will be dedicated to Q&A. The archived presentation will be available on the Company's website for approximately 30 days following the presentation date.

    Dr. Koenekoop is a Professor of Pediatric Surgery, Human Genetics and Ophthalmology at McGill University. He is also the Director of the Laboratory for Retinal Genetics and Therapeutics. Additionally, he is Chief of Pediatric Ophthalmology and has been involved in numerous global clinical trials for patients with inherited retinal diseases (IRDs). As a clinician-scientist, he focuses on finding genetic causes and new treatments for childhood blindness and has participated in international collaborations leading to the discoveries of multiple new IRD genes.

    He is currently the PI on seven different human clinical trials in Montreal, testing the safety and efficacy of therapies in adults and children with severe visual loss due to Choroideremia, RPGR related x-linked retinitis pigmentosa, Usher syndrome type 2, Leber congenital amaurosis due to CEP290 mutations and Usher syndrome type 1c patients. He has published over 150 refereed publications, lectured around the world extensively, and holds grants from NIH, CIHR, Fighting Blindness Canada and Reseau du Quebec.

    About ProQR

    ProQR Therapeutics is dedicated to changing lives through the creation of transformative RNA therapies for the treatment of severe genetic rare diseases such as Leber congenital amaurosis 10, Usher syndrome and retinitis pigmentosa. Based on our unique proprietary RNA repair platform technologies we are growing our pipeline with patients and loved ones in mind.

    *Since 2012*

    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. Such statements include those relating to our pipeline and platform. 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 anticipated in 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. 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



    or

    Hans Vitzthum

    LifeSci Advisors

    T: +1 617 535 7743

     

    Media Contact:

    Sara Zelkovic

    LifeSci Public Relations

    T: +1 646 876 4933

    Primary Logo

    View Full Article Hide Full Article
  2. LEIDEN, Netherlands & CAMBRIDGE, Mass., June 08, 2020 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (NASDAQ:PRQR), a company dedicated to changing lives through the creation of transformative RNA therapies for severe genetic rare diseases, today announced a virtual presentation of data from the Company's Phase 1/2 trial of sepofarsen. The data will be shared via a video presentation through the Association for Research in Vision and Ophthalmology (ARVO). It is the first time this data, previously announced in a press release and conference call in October 2019, will be presented in association with an ophthalmology medical congress.

    Due to COVID-19, the ARVO 2020 Annual Meeting was cancelled and instead video-recorded presentations will be…

    LEIDEN, Netherlands & CAMBRIDGE, Mass., June 08, 2020 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (NASDAQ:PRQR), a company dedicated to changing lives through the creation of transformative RNA therapies for severe genetic rare diseases, today announced a virtual presentation of data from the Company's Phase 1/2 trial of sepofarsen. The data will be shared via a video presentation through the Association for Research in Vision and Ophthalmology (ARVO). It is the first time this data, previously announced in a press release and conference call in October 2019, will be presented in association with an ophthalmology medical congress.

    Due to COVID-19, the ARVO 2020 Annual Meeting was cancelled and instead video-recorded presentations will be available on ARVOLearn, ARVO's online learning platform.

    Details of ProQR's presentation are as follows:

    Presenter: Stephen R. Russell, MD, Professor and Director of Vitreoretinal Diseases and Surgery Service, Department of Ophthalmology and Visual Sciences, University of Iowa

    Presentation Title: Results of a phase 1b/2 trial of intravitreal (IVT) sepofarsen (QR-110) antisense oligonucleotide in Leber congenital amaurosis 10 (LCA10) due to p.Cys998X mutation in the CEP290 gene

    Date: The video presentation will be available on ARVOLearn starting June 15, 2020.

    About Sepofarsen

    Sepofarsen (QR-110) is being evaluated in the pivotal Phase 2/3 Illuminate trial and is a first-in-class investigational RNA therapy designed to address the underlying cause of Leber congenital amaurosis 10 due to the p.Cys998X mutation (also known as the c.2991+1655A>G mutation) in the CEP290 gene. The p.Cys998X mutation leads to aberrant splicing of the mRNA and non-functional CEP290 protein. Sepofarsen is designed to enable normal splicing, resulting in restoration of normal (wild type) CEP290 mRNA and subsequent production of functional CEP290 protein. Sepofarsen is intended to be administered through intravitreal injections in the eye and has been granted orphan drug designation in the United States and the European Union and received fast-track designation and rare pediatric disease designation from the FDA as well as access to the PRIME scheme by the EMA.

    About ProQR

    ProQR Therapeutics is dedicated to changing lives through the creation of transformative RNA therapies for the treatment of severe genetic rare diseases such as Leber congenital amaurosis 10, Usher syndrome and autosomal dominant retinitis pigmentosa. Based on our unique proprietary RNA repair platform technologies we are growing our pipeline with patients and loved ones in mind.

    *Since 2012*

    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. Such statements include those relating to our presentation of data through ARVO. 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 anticipated in 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. 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



    or

    Hans Vitzthum

    LifeSci Advisors

    T: +1 617 535 7743

     

    Media Contact:

    Sara Zelkovic

    LifeSci Public Relations

    T: +1 646 876 4933

    Primary Logo

    View Full Article Hide Full Article
  3. Leiden, Netherlands & Cambridge, Mass., May 25, 2020 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (NASDAQ:PRQR) (the "Company"), a company dedicated to changing lives through the creation of transformative RNA therapies for severe genetic rare diseases, today announced that the Annual General Meeting of Shareholders will take place on Tuesday, June 23, 2020 at 15:00 CET, via videoconference.

    All relevant documents and information for the meeting, including the notice and agenda, are or will be made available in the "Investors" section of ProQR's website (www.proqr.com) under "Financial Information". The documents will also be made available on the SEC's website at www.sec.gov.  Shareholders that wish to attend the videoconference should register…

    Leiden, Netherlands & Cambridge, Mass., May 25, 2020 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (NASDAQ:PRQR) (the "Company"), a company dedicated to changing lives through the creation of transformative RNA therapies for severe genetic rare diseases, today announced that the Annual General Meeting of Shareholders will take place on Tuesday, June 23, 2020 at 15:00 CET, via videoconference.

    All relevant documents and information for the meeting, including the notice and agenda, are or will be made available in the "Investors" section of ProQR's website (www.proqr.com) under "Financial Information". The documents will also be made available on the SEC's website at www.sec.gov.  Shareholders that wish to attend the videoconference should register for attendance as described in the notice and agenda, after which they will receive login details for the videoconference.

    About ProQR

    ProQR Therapeutics is dedicated to changing lives through the creation of transformative RNA therapies for the treatment of severe genetic rare diseases such as Leber congenital amaurosis 10, Usher syndrome and autosomal dominant retinitis pigmentosa. Based on our unique proprietary RNA repair platform technologies we are growing our pipeline with patients and loved ones in mind.
    *Since 2012*

    ProQR Therapeutics N.V.

    Investor Contact:
    Sarah Kiely
    ProQR Therapeutics N.V.
    T: +1 617 599 6228

    or
    Hans Vitzthum
    LifeSci Advisors
    T: +1 617 535 7743
     

    Media Contact:
    Sara Zelkovic
    LifeSci Public Relations
    T: +1 646 876 4933

    Primary Logo

    View Full Article Hide Full Article
    • Reported positive interim analysis findings from Phase 1/2 Stellar trial of QR-421a for Usher syndrome and non-syndromic retinitis pigmentosa – study ongoing with dose expansion and escalation planned;
    • Updated data from the Phase 1/2 InSight extension study of sepofarsen, including data from contralateral eye treatment, on track to be reported in H2 2020;
    • Three clinical stage RNA therapies in development for inherited retinal diseases, with fourth ophthalmic program slated to enter mechanistic proof-of-concept studies;
    • ProQR anticipates its cash runway will fund operations into H2 2022

    LEIDEN, Netherlands & CAMBRIDGE, Mass., May 07, 2020 (GLOBE NEWSWIRE) --  ProQR Therapeutics N.V. (NASDAQ:PRQR) (the "Company"), a company dedicated to changing…

    • Reported positive interim analysis findings from Phase 1/2 Stellar trial of QR-421a for Usher syndrome and non-syndromic retinitis pigmentosa – study ongoing with dose expansion and escalation planned;
    • Updated data from the Phase 1/2 InSight extension study of sepofarsen, including data from contralateral eye treatment, on track to be reported in H2 2020;
    • Three clinical stage RNA therapies in development for inherited retinal diseases, with fourth ophthalmic program slated to enter mechanistic proof-of-concept studies;
    • ProQR anticipates its cash runway will fund operations into H2 2022

    LEIDEN, Netherlands & CAMBRIDGE, Mass., May 07, 2020 (GLOBE NEWSWIRE) --  ProQR Therapeutics N.V. (NASDAQ:PRQR) (the "Company"), a company dedicated to changing lives through the creation of transformative RNA therapies for severe genetic rare diseases, today reported its financial results for the first quarter ended March 31, 2020 and provided a business update.

    "At the end of the first quarter we shared positive findings from the interim analysis of the Phase 1/2 Stellar trial of QR-421a for Usher's syndrome, which adds to the growing body of evidence further validating the potential of our platform," said Daniel A. de Boer, Chief Executive Officer of ProQR. "Based on these findings, we are continuing the trial as designed, with dose expansion and escalation cohorts planned. We are working closely with our clinical trial sites to monitor the evolving COVID-19 situation and preparing to rapidly ramp up enrollment once it is deemed safe to do so. In the second half of 2020 we look forward to sharing updated data from the Phase 1/2 InSight extension study of sepofarsen for LCA10, including data from the contralateral eye treatment. We are confident in our fundamentals – we have a productive platform, a deep pipeline, and are well capitalized – as we continue our work to bring novel RNA therapies to patients."

    Business Operations and Program Updates

    Ongoing clinical studies of sepofarsen for LCA10, QR-421a for Usher syndrome and nsRP, and QR-1123 for adRP are all currently active, but the effects of the COVID-19 pandemic are resulting in disruptions to patient enrollment across these programs. In consultation with clinical trial sites, ProQR is implementing mitigation procedures that support a rapid ramp up in enrollment as soon as the disruption allows, including ongoing patient identification activities, pre-screening, and documentation for additional site activations. Additionally, ProQR is continuing to monitor previously enrolled trial subjects. The impact of COVID-19 continues to be a dynamic and evolving situation.

    Sepofarsen, lead clinical candidate for Leber congenital amaurosis 10 (LCA10):

    • Based on COVID-19-related disruptions at clinical trial sites, the enrollment timeline for the pivotal Phase 2/3 Illuminate trial is delayed. Additional site activation and patient pre-screening activities are ongoing, which are designed to enable rapid ramp up in enrollment once the trial sites are able to dose patients.
    • Updated data from the Phase 1/2 InSight extension study of sepofarsen for LCA10, including data from contralateral eye treatment, are on track to be reported in H2 2020.

     QR-421a for Usher's syndrome and non-syndromic retinitis pigmentosa: 

    • In March, the Company reported interim analysis findings from the Phase 1/2 Stellar trial of QR-421a in patients with Usher syndrome and non-syndromic retinitis pigmentosa, or nsRP. The data demonstrated that thus far, QR-421a is generally well tolerated with no serious adverse events noted. There were early signals of target engagement and clinical activity supported by concordant benefit observed across multiple outcome measures for 25% (2 of 8) of patients in the trial, which support continuing the trial as designed, with both cohort expansion and dose escalation planned. 

    QR-1123 for autosomal dominant retinitis pigmentosa (adRP):

    • The Phase 1/2 Aurora trial is ongoing with initial data on track for 2021.

     QR-504a for Fuchs Endothelial Corneal Dystrophy (FECD):

    • QR-504a is expected to be the Company's next pipeline candidate to enter clinical development for patients with FECD type 3 who are scheduled for corneal transplant.

    My Retina Tracker Program:

    • In February, ProQR announced its participation in the Foundation Fighting Blindness "My Retina Tracker Program", a collaborative, open access program in the United States providing no-cost genetic testing and genetic counseling for individuals with a clinical diagnosis of an inherited retinal disease, such as LCA and Usher syndrome.

    Financial Highlights

    At March 31, 2020, ProQR held cash and cash equivalents of €98.1 million, compared to €112.0 million at December 31, 2019. Net cash used in operating activities during the three-month period ended March 31, 2020 was €15.0 million, compared to €12.4 million for the same period last year.

    Research and development (R&D) costs were €12.8 million for the quarter ended March 31, 2020 compared to €12.0 million for the same period last year and were comprised of allocated employee costs including share-based payments, the costs of materials and laboratory consumables, outsourced activities, license and intellectual property costs, and other allocated costs.

    General and administrative costs were €3.9 million for the quarter ended March 31, 2020 compared to €3.2 million for the same period last year.

    Net loss for the three-month period ended March 31, 2020 was €16.1 million, or €0.32 per diluted share, compared to €14.2 million, or €0.36 per diluted share, for the same period last year. For further financial information for the period ending March 31, 2020, please refer to the financial statements appearing at the end of this release.

    About Leber Congenital Amaurosis 10 (LCA10)

    Leber congenital amaurosis (LCA) is the most common cause of blindness due to genetic disease in children. It consists of a group of diseases of which LCA10 is the most frequent and one of the most severe forms. LCA10 is caused by mutations in the CEP290 gene, of which the p.Cys998X mutation has the highest prevalence. LCA10 leads to early loss of vision causing most people to lose their sight in the first few years of life. To date, there are no treatments approved that treat the underlying cause of the disease. Approximately 2,000 people in the Western world have LCA10 because of this mutation.

    About Sepofarsen

    Sepofarsen (QR-110) is being evaluated in the pivotal Phase 2/3 Illuminate trial and is a first-in-class investigational RNA therapy designed to address the underlying cause of Leber congenital amaurosis 10 due to the p.Cys998X mutation (also known as the c.2991+1655A>G mutation) in the CEP290 gene. The p.Cys998X mutation leads to aberrant splicing of the mRNA and non-functional CEP290 protein. Sepofarsen is designed to enable normal splicing, resulting in restoration of normal (wild type) CEP290 mRNA and subsequent production of functional CEP290 protein. Sepofarsen is intended to be administered through intravitreal injections in the eye and has been granted orphan drug designation in the United States and the European Union and received fast-track designation and rare pediatric disease designation from the FDA as well as access to the PRIME scheme by the EMA.

    About Usher Syndrome Type 2 and Non-Syndromic Retinitis Pigmentosa

    Usher syndrome is the leading cause of combined deafness and blindness. People with Usher syndrome type 2 are usually born with hearing loss and start to have progressive vision loss during adulthood. The vision loss can also occur without hearing loss in a disease called non-syndromic retinitis pigmentosa. Usher syndrome type 2 and non-syndromic retinitis pigmentosa can be caused by mutations in the USH2A gene. To date, there are no pharmaceutical treatments approved or in clinical development that treat the vision loss associated with mutations in USH2A.

    About QR-421a

    QR-421a is being evaluated in the Phase 1/2 Stellar trial and is a first-in-class investigational RNA therapy designed to address the underlying cause of vision loss in Usher syndrome type 2 and non-syndromic retinitis pigmentosa (RP) due to mutations in exon 13 of the USH2A gene. QR-421a is designed to restore functional usherin protein by using an exon skipping approach with the aim to stop or reverse vision loss in patients. QR-421a is intended to be administered through intravitreal injections in the eye and has been granted orphan drug designation in the US and the European Union and received fast-track and rare pediatric disease designations from the FDA.

    About Autosomal Dominant Retinitis Pigmentosa (adRP)

    Autosomal dominant retinitis pigmentosa, or adRP, is a severe and rare genetic disease that causes progressive problems in night vision during childhood, leading to visual field loss and frequently resulting in blindness in mid adulthood. In the United States, the most prevalent mutation associated with adRP is the P23H point mutation (also known as the c.68C>A mutation) in the rhodopsin (RHO) gene and affects approximately 2,500 people. This mutation causes misfolding of the rhodopsin protein that becomes toxic to the photoreceptor cells and at the same time diminishes the function of the wild type allele. Over time this results in cell death and progressive vision loss. There are currently no therapies approved or in clinical development for P23H adRP. A natural history study in patients with P23H adRP has been conducted.

    About QR-1123

    QR-1123 is being evaluated in the Phase 1/2 Aurora trial and is a first-in-class investigational RNA therapy designed to treat adRP due to the P23H mutation in the RHO gene. QR-1123 was discovered and developed by Ionis Pharmaceuticals using Ionis' proprietary antisense technology. The therapy aims to inhibit the formation of the mutated toxic version of the rhodopsin protein by specifically binding the mutated RHO mRNA. Binding of QR-1123 causes allele specific knockdown of the mutant mRNA by a mechanism called RNase H mediated cleavage without affecting the normal RHO mRNA. QR-1123 is intended to be administered through intravitreal injections in the eye. QR-1123 was in-licensed from Ionis Pharmaceuticals in 2018. QR-1123 has been granted Orphan Drug designation in the United States and received Fast Track designation from the FDA.

    About Fuchs Endothelial Corneal Dystrophy (FECD)

    Fuchs endothelial corneal dystrophy (FECD) is a common inherited condition characterized by the dysfunction and degeneration of the corneal endothelium, a single cell layer of cells on the inside of the cornea. FECD is a common disorder; it is estimated that FECD affects more than 4% of individuals over the age of 40 in the U.S., and similar prevalence is noted for other global regions. There are different types of this disease and we focus on age-related FECD (FECD3). Some patients with age-related FECD develop advanced disease with corneal edema and corneal clouding. These symptoms can lead to complete vision loss and the need for surgery and a corneal transplant.

    About QR-504a

    We are developing QR-504a as an RNA therapy for the treatment of FECD3. We plan to advance the QR-504a program into a first clinical trial in late-stage disease patients in 2020. QR-504a is designed to target the intronic TNRs in the TCF4 RNA. The aim is to reduce aggregation and the formation of RNA foci in order to normalize the RNA splicing patterns, and prevent or halt corneal degeneration in patients with FECD3.

    About ProQR

    ProQR Therapeutics is dedicated to changing lives through the creation of transformative RNA therapies for the treatment of severe genetic rare diseases such as Leber's congenital amaurosis 10, Usher syndrome and autosomal dominant retinitis pigmentosa. Based on our unique proprietary RNA repair platform technologies we are growing our pipeline with patients and loved ones in mind.

    *Since 2012*

    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. Such forward-looking statements include, but are not limited to, statements regarding sepofarsen (QR-110) and the clinical development and the therapeutic potential thereof, statements regarding our pipeline of programs targeting inherited retinal dystrophies, statements regarding QR-421a, and the clinical development and the therapeutic potential thereof,  statements regarding QR-1123 and the clinical development and therapeutic potential thereof, our other programs and business operations, including timing of commencing clinical trials and enrollment of patients therein, the expected impact of the COVID-19 on our business operations, including our research and development plans and timelines and the supply chain for our clinical and development programs, 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 anticipated in 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 by the COVID-19 pandemic; the likelihood of our clinical programs being 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; 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; and general business, financial and accounting risks and litigation. 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.

    Cautionary Note on Future Updates

    The statements contained in this press release reflect our current views with respect to future events, which may change significantly as the global consequences of the COVID-19 pandemic rapidly develop. Accordingly, we do not undertake and specifically disclaim any obligation to update any forward-looking statements.

    ProQR Therapeutics N.V.

    Investor Contact:
    Sarah Kiely
    ProQR Therapeutics N.V.
    T: +1 617 599 6228

    or
    Hans Vitzthum
    LifeSci Advisors
    T: +1 617 535 7743
     

    Media Contact:
    Sara Zelkovic
    LifeSci Public Relations
    T: +1 646 876 4933


     

          

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

             
             
        March 31,    December 31, 
        2020   2019
        € 1,000   € 1,000
    Assets        
    Current assets        
    Cash and cash equivalents    98,063    111,950
    Prepayments and other receivables    1,987    1,866
    Social securities and other taxes    998    850
             
    Total current assets    101,048    114,666
             
    Property, plant and equipment    2,291    2,440
    Investments in associates    295    429
             
    Total assets    103,634    117,535
             
    Equity and liabilities        
    Equity        
    Equity attributable to owners of the Company    81,869    94,329
    Non-controlling interests    (519)    (496)
    Total equity    81,350    93,833
             
    Current liabilities        
    Borrowings    519    343
    Lease liabilities    306    508
    Trade payables    578    445
    Current income tax liability    65    64
    Social securities and other taxes    18    108
    Pension premiums    21    2
    Deferred income    557    711
    Other current liabilities    7,089    8,812
             
    Total current liabilities    9,153    10,993
             
    Borrowings    13,131    12,709
             
    Total liabilities    22,284    23,702
             
    Total equity and liabilities    103,634    117,535


     

    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
        ended March 31, 
             
        2020   2019
        € 1,000   € 1,000
    Other income    263    416
             
    Research and development costs    (12,825)    (11,963)
    General and administrative costs    (3,918)    (3,191)
             
    Total operating costs    (16,743)    (15,154)
             
    Operating result    (16,480)    (14,738)
    Finance income and expense    536    494
    Results related to associates    (134)    —
             
    Result before corporate income taxes    (16,078)    (14,244)
    Income taxes    —    —
             
    Result for the period    (16,078)    (14,244)
    Other comprehensive income    256    12
             
    Total comprehensive income (attributable to owners of the Company)    (15,822)    (14,232)
             
    Result attributable to        
    Owners of the Company    (16,055)    (14,157)
    Non-controlling interests    (23)    (87)
         (16,078)    (14,244)
             
    Share information        
    Weighted average number of shares outstanding1    49,906,033    38,885,428
             
    Earnings per share attributable to the equity holders of the Company (expressed in Euro per share)        
    Basic loss per share1    (0.32)    (0.36)
    Diluted loss per share1    (0.32)    (0.36)

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


     

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

                                         
                                         
        Attributable to owners of the Company        
                                         
                    Equity settled                    
                    Employee               Non-    
        Number of   Share   Share   Benefit   Translation   Accumulated       controlling   Total
        shares   Capital   Premium   Reserve   Reserve   Deficit   Total   interests   Equity
            € 1,000   € 1,000   € 1,000   € 1,000   € 1,000   € 1,000   € 1,000   € 1,000
    Balance at January 1, 2019    43,149,987    1,726    235,744    10,780    108    (155,443)    92,915    (230)    92,685
    Result for the period    —    —    —    —    —    (14,157)    (14,157)    (87)    (14,244)
    Other comprehensive income    —    —    —    —    12    —    12    —    12
    Recognition of share-based payments    —    —    —    2,288    —    —    2,288    —    2,288
    Issuance of ordinary shares    —    —    —    —    —    —    —    —    —
    Treasury shares transferred    —    —    —    —    —    —    —    —    —
    Share options lapsed    —    —    —    —    —    —    —    —    —
    Share options exercised    —    —    71    (49)    —    49    71    —    71
                                         
    Balance at March 31, 2019    43,149,987    1,726    235,815    13,019    120    (169,551)    81,129    (317)    80,812
                                         
    Balance at January 1, 2020    53,975,838    2,159    287,214    16,551    151    (211,746)    94,329    (496)    93,833
    Result for the period    —    —    —    —    —    (16,055)    (16,055)    (23)    (16,078)
    Other comprehensive income    —    —    —    —    256    —    256    —    256
    Recognition of share-based payments    —    —    —    2,870    —    —    2,870    —    2,870
    Issuance of ordinary shares    —    —    —    —    —    —    —    —    —
    Treasury shares transferred    (220,958)    —    —    —    —    —    —    —    —
    Share options lapsed    —    —    —    (2)    —    2    —    —    —
    Share options exercised    220,958    —    469    (304)    —    304    469    —    469
                                         
    Balance at March 31, 2020    53,975,838    2,159    287,683    19,115    407    (227,495)    81,869    (519)    81,350


     

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

             
             
        Three month period 
        ended March 31, 
             
        2020   2019
        € 1,000   € 1,000
    Cash flows from operating activities        
    Net result    (16,078)    (14,244)
    Adjustments for:        
    — Depreciation    522    521
    — Share-based compensation    2,870    2,288
    — Financial income and expenses    (536)    (494)
    — Results related to associates    134    —
    — Net foreign exchange gain / (loss)    256    12
             
    Changes in working capital    (2,200)    (474)
    Cash used in operations    (15,032)    (12,391)
             
    Corporate income tax paid    —    —
    Interest received    29    54
    Interest paid    (4)    (27)
             
    Net cash used in operating activities    (15,007)    (12,364)
             
    Cash flow from investing activities        
    Purchases of property, plant and equipment    (198)    (223)
             
    Net cash used in investing activities    (198)    (223)
             
    Cash flow from financing activities        
    Proceeds from issuance of shares, net of transaction costs    —    —
    Proceeds from exercise of share options    469    71
    Proceeds from borrowings    290    —
    Proceeds from convertible loans    —    690
    Repayment of lease liability    (202)    (284)
             
    Net cash (used in)/generated by financing activities    557    477
             
    Net increase/(decrease) in cash and cash equivalents    (14,648)    (12,110)
             
    Currency effect cash and cash equivalents    761    610
    Cash and cash equivalents, at beginning of the period    111,950    105,580
             
    Cash and cash equivalents at the end of the period    98,063    94,080

     

    Primary Logo

    View Full Article Hide Full Article
    • QR-421a showed early and encouraging evidence of activity, with 25% of patients showing a benefit across multiple concordant outcome measures and was well tolerated with no serious adverse events
    • QR-421a is the second ophthalmology program where clinical activity was predicted by translational models, further validating the platform
    • COVID-19 pandemic expected to impact timelines for the pipeline
    • ProQR anticipates its cash runway will fund operations into H2 2022

    LEIDEN, Netherlands & CAMBRIDGE, Mass., March 31, 2020 (GLOBE NEWSWIRE) --  March 31, 2020 -- ProQR Therapeutics N.V. (NASDAQ:PRQR) (the "Company"), a company dedicated to changing lives through the creation of transformative RNA therapies for severe genetic rare diseases, today announces…

    • QR-421a showed early and encouraging evidence of activity, with 25% of patients showing a benefit across multiple concordant outcome measures and was well tolerated with no serious adverse events
    • QR-421a is the second ophthalmology program where clinical activity was predicted by translational models, further validating the platform
    • COVID-19 pandemic expected to impact timelines for the pipeline
    • ProQR anticipates its cash runway will fund operations into H2 2022

    LEIDEN, Netherlands & CAMBRIDGE, Mass., March 31, 2020 (GLOBE NEWSWIRE) --  March 31, 2020 -- ProQR Therapeutics N.V. (NASDAQ:PRQR) (the "Company"), a company dedicated to changing lives through the creation of transformative RNA therapies for severe genetic rare diseases, today announces positive findings from a planned three-month interim analysis of its Phase 1/2 Stellar trial of QR-421a in adults with Usher syndrome and non-syndromic retinitis pigmentosa (nsRP) due to USH2A exon 13 mutations. The Company is also providing an update on business operations in relation to the COVID-19 pandemic. Management will host a conference call today at 8:15 am ET.

    "The goal of the interim analysis of this 24 month Stellar trial of QR-421a was to assess safety and early signs of efficacy for the purpose of informing next steps in development and future trial strategy," said David Rodman, M.D., Executive Vice President of Research and Development of ProQR. "We are pleased with the current safety profile and are very encouraged by early signals of target engagement and clinical activity supported by concordant benefit observed across multiple outcome measures for 25% of QR-421a-treated patients thus far in this trial. The findings support continuing the trial as planned, with both cohort expansion and dose escalation in order to identify a potential development path to registration. Importantly, these data represent the second program from our ophthalmology pipeline that is supported by preclinical predictions from human retinal organoids, providing further validation of our translational approach and platform technology."

    Phase 1/2 Three-Month Interim Analysis

    The interim analysis (IA) is based on nine and three month data from the first and second dose  cohorts, respectively, of the Stellar Phase 1/2 clinical trial of QR-421a, an investigational RNA therapy. The Stellar trial is a randomized, single ascending dose, global multicenter, longitudinal, 24-month study, involving active versus sham procedure. The first two cohorts include a total of 14 subjects (ranging from 24-65 years in age), of which eight received a single dose of QR-421a and six received a single sham procedure for masking. Six subjects were enrolled in the 50 µg cohort ("low dose"), of which four received treatment and two were randomized to sham; eight patients were enrolled in the 100 µg cohort ("mid dose") of which four received treatment and four were randomized to sham. The population varied in disease characteristics with both Usher syndrome (n=6) and nsRP (n=8) affected subjects included, genetic background with both homozygous (n= 4) and heterozygous (n=10) subjects for USH2A exon 13 mutations, and visual impairment at baseline ranging from mild to severe.

    Key initial findings include the following:

    Safety data: Across both cohorts thus far, QR-421a was observed to be generally well tolerated with no serious adverse events noted.

    Efficacy data:, In the six sham treated subjects (two followed for 9 months and four for 3 months), outcome measures demonstrated no consistent pattern of response above the "noise" level.  In contrast, two of eight QR‑421a-treated patients (one each in the 50 µg and 100 µg dose cohorts) demonstrated benefit across multiple concordant outcome measures.  

    • Responder 1: One of four treated patients in the low dose group was classified as a responder, with onset of action observed by the 3 month visit. Benefit was maintained for 6 months or longer, which is consistent with the expected half-life of QR-421a in photoreceptors. This Usher syndrome patient was homozygous for USH2A exon 13 mutations and had moderate visual impairment at baseline (peripheral vision affected). Concordant benefit was observed across multiple relevant measures appropriate to the severity of the patient's disease, including full field stimulus threshold test (FST) [deterioration by 5 dB in untreated eye, treated eye stable], dark adapted chromatic (DAC) perimetry [15 dB.steradian improvement in peripheral sensitivity in treated eye, <5 dB.steradian change in untreated eye], and optical coherence tomography (OCT) assessment of photoreceptor Ellipsoid Zone (EZ area). For FST and OCT, the contralateral, untreated eye demonstrated modest deterioration while the treated eye showed stabilization. For DAC perimetry the untreated eye was unchanged, whereas the treated eye demonstrated improvement.
    • Responder 2: One of four treated patients in the mid dose group was classified as a responder with onset of action observed by 3 months. This non-syndromic RP patient was heterozygous for USH2A exon 13 mutations and had severe visual impairment at baseline (peripheral and central vision affected) with baseline best corrected visual acuity (BCVA) of 33 and 36 letters (approximate Snellen equivalent: 20/250 and 20/200) in the treated and untreated eye, respectively. Concordant benefit was observed across multiple relevant measures appropriate for the stage of disease including FST (improvement by 12 dB in treated eye, no improvement in untreated eye), DAC (up to 10 dB.steradian improvement in treated eye, with deterioration in the untreated eye), and BCVA (7 letter improvement from baseline of 33 letters, which is more than one line on the ETDRS eye chart, compared to no change in the untreated eye).

    Next steps: Based on the safety profile and early evidence of efficacy observed to date, the Company plans to take advantage of the adaptive design, and expand the 100 µg cohort with additional subjects who are homozygous for exon 13 mutations. Dose escalation to 200 µg ("high dose") is planned to occur in parallel. An interim analysis of dose- and gene copy-dependent safety and efficacy will be planned once all additional subjects have reached at least 3 months of treatment.

    Business Update Related to COVID-19 Pandemic

    The COVID-19 pandemic is rapidly evolving and has prompted global health concerns, the duration, severity and exact impact of which are currently unknown and cannot be predicted with confidence. In consultation with the trial sites, due to the COVID-19 pandemic the Company also expects a delay in all of its ongoing and scheduled trials, including the pivotal trial of sepofarsen for Leber congenital amaurosis 10. ProQR is implementing mitigation procedures that support a rapid ramp up in enrollment as soon as the disruption resolves, including additional patient identification activities and documentation for additional site activations, while prioritizing the safety of trial participants and healthcare providers. For the trials of QR-421a and QR-1123, patients have already been identified for the next dose cohorts and the Company expects to begin dosing as soon as practical after clinical sites are ready and able to do so. This will be the same for the start of the clinical trial for QR-504a. The Company currently does not believe that its supply chain will be affected.

    Due to the COVID-19-related delays, the Company has undertaken a budget review process and now anticipates its cash runway will fund operations into the second half of 2022.

    "In these uncertain times, the health and safety of patients in our trials, their caregivers, and our employees remains our top priority. We believe we have taken appropriate measures designed to limit their risk," said Daniel A. de Boer, Chief Executive Officer of ProQR. "While we manage the challenges stemming from the COVID-19 pandemic, we remain confident in the fundamentals of our business, as demonstrated in part by the data we are sharing today from our QR-421a program. We have a productive platform, a deep pipeline of RNA therapies focused on inherited retinal diseases, and the benefit of a strong balance sheet, which positions us well to endure the current disruption and continue the important work with the communities we serve."

    Conference Call

    Management will discuss the data and next steps for development during a webcasted conference call today, March 31, 2020, at 8:15 a.m. ET. The live and archived webcast of this presentation can be accessed through the Events and Presentations page on the Investors section of the Company's website, www.ProQR.com. The dial-in details for the call are +1 631-510-7495 (US), +31 (0) 207143545 (NL), conference ID: 5986384. The archived webcasts will be available for approximately 30 days following the presentation date.

    About Usher Syndrome Type 2 and Non-Syndromic Retinitis Pigmentosa

    Usher syndrome is the leading cause of combined deafness and blindness. People with Usher syndrome type 2 are usually born with hearing loss and start to have progressive vision loss during adulthood. The vision loss can also occur without hearing loss in a disease called non-syndromic retinitis pigmentosa. Usher syndrome type 2 and non-syndromic retinitis pigmentosa can be caused by mutations in the USH2A gene. To date, there are no pharmaceutical treatments approved or in clinical development that treat the vision loss associated with mutations in USH2A.

    About QR-421a

    QR-421a is being evaluated in the Phase 1/2 Stellar trial and is a first-in-class investigational RNA therapy designed to address the underlying cause of vision loss in Usher syndrome type 2 and non-syndromic retinitis pigmentosa (RP) due to mutations in exon 13 of the USH2A gene. QR-421a is designed to restore functional Usherin protein by using an exon skipping approach with the aim to stop or reverse vision loss in patients. QR-421a is intended to be administered through intravitreal injections in the eye and has been granted orphan drug designation in the US and the European Union and received fast-track and Rare Pediatric Disease designations from the FDA.

    About the Stellar Phase 1/2 Trial of QR-421a

    Stellar, or PQ-421a-001, is a first-in-human study of QR-421a in adults who have vision loss due to mutations in exon 13 of the USH2A gene and is conducted at expert sites in North America and Europe. It is a double-masked, randomized, 24-month study exploring the safety and efficacy of a single intravitreal injection of several dose levels of QR-421a and a control sham procedure into one eye.

    About ProQR

    ProQR Therapeutics is dedicated to changing lives through the creation of transformative RNA therapies for the treatment of severe genetic rare diseases such as Leber congenital amaurosis 10, Usher syndrome and autosomal dominant retinitis pigmentosa. Based on our unique proprietary RNA repair platform technologies we are growing our pipeline with patients and loved ones in mind.
    *Since 2012*

    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. Such forward-looking statements include, but are not limited to, statements regarding QR-421a, and the clinical development and the therapeutic potential thereof, our other programs and business operations, including timing of commencing clinical trials and enrollment of patients therein, the expected impact of the COVID-19 on our business operations, including our research and development plans and timelines and the supply chain for our clinical and development programs, 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 anticipated in 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 by the COVID-19 pandemic; the likelihood of our clinical programs being 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; 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; and general business, financial and accounting risks and litigation. 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.

    Cautionary Note on Future Updates

    The statements contained in this press release reflect our current views with respect to future events, which may change significantly as the global consequences of the COVID-19 pandemic rapidly develop. Accordingly, we do not undertake and specifically disclaim any obligation to update any forward-looking statements.

    ProQR Therapeutics N.V.

    Investor Contact:
    Sarah Kiely
    ProQR Therapeutics N.V.
    T: +1 617 599 6228

    or
    Hans Vitzthum
    LifeSci Advisors
    T: +1 617 430 7578
     

    Media Contact:
    Sara Zelkovic
    LifeSci Public Relations
    T: +1 646 876 4933

    Primary Logo

    View Full Article Hide Full Article
View All ProQR Therapeutics N.V. News