LEIDEN, Netherlands & CAMBRIDGE, Mass., April 26, 2022 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (Nasdaq: PRQR) (the “Company”), a company dedicated to changing lives through the creation of transformative RNA therapies, today announced several presentations at the 7th Annual Retinal Cell and Gene Therapy Innovation Summit being held Friday, April 29, 2022 and the Annual Meeting of the Association for Research in Vision and Ophthalmology (ARVO) being held May 1-4, 2022, both in Denver, CO, U.S.
Presentations at the Retinal Cell and Gene Therapy Innovation Summit
Presentation title: AON treatment for CEP290-LCA: Efficacy, safety, and durability
Presenter: Dr. Artur Cideciyan, University of Pennsylvania
Presentation type: Pre-recorded talk
Session: Session 4: Antisense Oligonucleotide Therapy
Date: April 29, 2022 at 4:00pm MDT
Presentation title: Efficacy and safety of sepofarsen, an intravitreal RNA antisense oligonucleotide, for the treatment of CEP290-associated inherited retinal disease called Leber congenital amaurosis (LCA10): A randomized, double-masked, sham-controlled, phase III study (ILLUMINATE)
Presenter: Dr. Bart Leroy, Ghent University Hospital
Presentation type: Oral presentation
Session: Session 4: Antisense Oligonucleotide Therapy
Date: April 29, 2022 at 4:15pm MDT
Presentations at ARVO
Presentation title: Long-term safety and efficacy of sepofarsen in a Ph1b/2 INSIGHT extension trial in CEP290-associated Leber congenital amaurosis (LCA10)
Presenter: Dr. Stephen R. Russell, Iowa University
Presentation type: Oral presentation
Date: May 1, 2022 at 3:36pm MDT
Presentation title: QR-1011 corrects splicing in the Stargardt disease type 1-causing variant ABCA4 c.5461-10T>C
Presenter: Melita Kaltak, ProQR Therapeutics
Presentation type: Oral presentation
Date: May 4, 2022 at 10:34am MDT
Presentation title: Efficacy and safety of sepofarsen, an intravitreal RNA antisense oligonucleotide, for the treatment of CEP290-associated Leber congenital amaurosis (LCA10): a randomized, double-masked, sham-controlled, Phase 3 study (ILLUMINATE)
Presenter: Dr. Bart Leroy, Ghent University Hospital
Presentation type: Poster presentation
Date: May 4, 2022 at 3:00pm MDT
“CEP290-mediated Leber congenital amaurosis 10, or LCA10, is a difficult to treat ultra-rare inherited retinal disease for which there is currently no treatment,” said Dr. Bart Leroy, Head of the Ophthalmology Department and Professor of Ophthalmology and Ophthalmic Genetics at Ghent University in Belgium and Attending Physician at The Children's Hospital of Philadelphia. “After missing the primary endpoint in the Illuminate Phase 2/3 clinical trial, sepofarsen showed an encouraging efficacy signal across multiple endpoints in post-hoc analyses comparing sepofarsen treated and sham treated eyes to their corresponding untreated contralateral eyes. I look forward to continuing to work with ProQR on this investigational treatment.”
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 c.2991+1655A>G (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 an investigational RNA therapy designed to restore vision in Leber congenital amaurosis 10 due to the c.2991+1655A>G mutation (p.Cys998X) in the CEP290 gene. The 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. ProQR is pioneering a next-generation RNA technology called Axiomer®, which uses a cell’s own editing machinery called ADAR (adenosine deaminase acting on RNA) 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 genetic diseases. 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 "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 participation in these conferences as well as sepofarsen (QR-110) and the clinical development and the therapeutic potential thereof, and our planned interactions with regulatory authorities relating to our programs. 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 later data to alter initial and preliminary results of early-stage clinical trials, including as a result of differences in the trial designs and protocols across different 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 outcomes of our planned interactions with regulatory authorities; 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; our ability to maintain and service our loan facility with Pontifax and Kreos; general business, operational, financial and accounting risks; and risks related to litigation and disputes with third parties. 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
Hans Vitzthum
LifeSci Advisors
T: +1 617 430 7578
hans@lifesciadvisors.com
Media Contact:
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FTI Consulting
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