ProQR Announces Virtual Presentations at American Academy of Ophthalmology (AAO)

Rhea-AI Summary

ProQR Therapeutics (Nasdaq: PRQR) announced virtual presentations at the American Academy of Ophthalmology Annual Meeting from November 13-15, 2020. Findings will include Phase 1b/2 trial results of sepofarsen for Leber congenital amaurosis 10 (LCA10), presented by Stephen R. Russell, MD. Another presentation by Allen Ho, MD, will focus on patient responses assessed via full-field stimulus testing. Sepofarsen is a pivotal RNA therapy targeting the p.Cys998X mutation in the CEP290 gene, designed to restore normal protein function and has received several designations from health authorities.

Positive

• Sepofarsen is a first-in-class RNA therapy addressing the underlying cause of LCA10.
• The drug has received orphan drug designation in the U.S. and EU, fast-track status from the FDA, and PRIME access by the EMA.
• The presentations at AAO may enhance visibility and support for sepofarsen's clinical development.

Negative

• None.

LEIDEN, Netherlands & CAMBRIDGE, Mass., Nov. 02, 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 virtual poster presentations at the Annual Meeting of the American Academy of Ophthalmology (AAO) held November 13-15, 2020.

ProQR’s presentations

Presentation title: Phase 1b/2 trial results of intravitreal sepofarsen RNA therapy in Leber congenital amaurosis 10 (LCA10)

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

Date: The poster presentation will be available on the AAO meeting portal starting November 11, 2020

Presentation title: Full-field stimulus testing (FST) to assess sepofarsen patient response in Leber congenital amaurosis type 10 (LCA10)

Presenter: Allen Ho, MD, Professor of Ophthalmology, Wills Eye Hospital, Thomas Jefferson University

Date: The poster presentation will be available on the AAO meeting portal starting November 11, 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 presentations at AAO. 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
skiely@proqr.com
or
Hans Vitzthum
LifeSci Advisors
T: +1 617 535 7743
hans@lifesciadvisors.com  

Media Contact:
Alison Chen
LifeSci Communications
T: +1 646 876 4932
achen@lifescicomms.com

FAQ

What are the key findings from ProQR's presentations at the AAO meeting?

ProQR's presentations will showcase Phase 1b/2 trial results of sepofarsen for LCA10 and its patient response assessment methods.

When will ProQR's poster presentations be available?

The poster presentations will be available on the AAO meeting portal starting November 11, 2020.

What is sepofarsen and its significance?

Sepofarsen is an investigational RNA therapy aimed at treating Leber congenital amaurosis 10 by correcting the underlying genetic mutation.

What designations has sepofarsen received from regulatory authorities?

Sepofarsen has received orphan drug designation in the U.S. and EU, fast-track designation from the FDA, and access to the PRIME scheme by the EMA.