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

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

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Ascendis Pharma A/S Announces Presentations for its Endocrinology Rare Disease Clinical Programs at Upcoming Medical Conferences

COPENHAGEN, Denmark, Oct. 21, 2020 (GLOBE NEWSWIRE) — Ascendis Pharma A/S (Nasdaq: ASND), a biopharmaceutical company that utilizes its innovative TransCon™ technologies to address unmet medical needs, today announced five presentations featuring the company’s endocrinology rare disease programs at two upcoming medical conferences: European Calcified Tissue Society (ECTS) 2020, taking place online October 22–24, 2020, and the Pediatric Endocrinology Nursing Society (PENS) national conference, taking place online November 2–5, 2020.

During ECTS, results from the phase 2 PaTH Forward Trial of TransCon PTH in adult hypoparathyroidism (HP) will be presented. During PENS, data from the phase 3 fliGHt Trial of TransCon hGH (lonapegsomatropin) in pediatric growth hormone deficiency (GHD) and from two clinical trials of the auto-injector for lonapegsomatropin. Additionally, the company will present posters highlighting the impact of achondroplasia (ACH) on the quality of life in children and their parents, which will help inform the TransCon CNP program.

“Ascendis Pharma is committed to supporting the endocrinology community, and we are excited to present data across all three of our endocrinology rare disease programs,” said Aimee Shu, M.D., Senior Medical Director, Clinical Development at Ascendis Pharma. “The data being presented at this year’s ECTS and PENS meetings highlight our portfolio of investigational product candidates and demonstrate important clinical and quality of life outcomes for patients and caregivers, including our first presentation of clinical data on our proprietary auto-injector for lonapegsomatropin.”

Presentation Details

ECTS

Poster Presentation

Title

 

Date/Time

 

Design and Topline Results of TransCon PTH, a Long-acting Parathyroid Hormone (PTH), Phase 2 Trial in Patients with Hypoparathyroidism

 

Presented on Saturday, October 24, 2020 at 4:55–5:00 p.m. (CST) with live Q&A to follow.

 

PENS 2020

Poster Presentations

Title

 

Date/Time

 

Introduction of a Novel GH Auto-Injector for Once-weekly Administration of TransCon hGH (lonapegsomatropin)

 

Posters are available during PENS with live Q&A on Thursday, November 5, 2020 from 2:15–2:45 p.m. (ET).

 

Treatment Experience of Children with Growth Hormone Deficiency in the Phase 3 fliGHt Trial: Switching from Daily Growth Hormone to Once-weekly TransCon hGH (lonapegsomatropin)

 

 

Pediatric Achondroplasia: Impacts on Children’s Functioning and Well-being

 

 

Experiences of Parents of Children with Achondroplasia: Impacts on Quality of Life

 

 

The posters will be available on the Ascendis website under Selected Publications in the Pipeline section: https://ascendispharma.com/product-pipeline/publications/.   If you are a healthcare provider who would like more information, please contact: [email protected]

About TransCon™ Technology Platform

TransCon refers to “transient conjugation.” The proprietary TransCon platform is an innovative technology to create new therapies that are designed to potentially optimize therapeutic effect, including efficacy, safety and dosing frequency. TransCon molecules have three components: an unmodified parent drug, an inert carrier that protects it, and a linker that temporarily binds the two. When bound, the carrier inactivates and shields the parent drug from clearance. When injected into the body, physiologic conditions (e.g., pH and temperature) initiate the release of the active, unmodified parent drug in a predictable manner. Because the parent drug is unmodified, its original mode of action is expected to be maintained. TransCon technologies can be

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