09.10.19

MeiraGTx To Present Clinical Data from Phase 1/2 Trial of AAV-RPE65 at Upcoming Scientific Conferences

Source: MeiraGTx Holdings

MeiraGTx Holdings announced that clinical data updates from the company’s phase 1/2 trial of AAV-RPE65 will be presented at two upcoming medical meetings.

The phase 1/2 open-label, multicenter, dose-finding trial evaluated AAV-RPE65 in patients with retinal dystrophy associated with disease-causing variants in the RPE65 gene, a condition that causes severe sight impairment beginning at birth. AAV-RPE65 is a second-generation gene therapy candidate developed specifically to treat RPE65-deficiency, and optimized for transduction efficiency, potency and stability. MeiraGTx previously reported positive topline data from this trial demonstrating safety and tolerability, as well as statistically significant improvement across several endpoints assessing clinical activity.   

Michel Michaelides, BSc, MB, BS, MD(Res), FRCOphth, FACS, trial investigator, Consultant Ophthalmologist, Moorfields Eye Hospital and Professor of Ophthalmology, University College London, will present updated data at the following meetings:

52nd Annual Scientific Meeting of The Retina Society

  • Podium Presentation: Results of a Phase 1/2 Trial of an Optimized Gene Therapy in Adults and Children with Retinal Dystrophy Associated with Bi-allelic Variants in RPE65
  • Session Date and Time: Sunday, September 15, 2019, 11:01 a.m. GMT
  • Session Title: Late Breaking Presentations
  • Room: Grand Ballroom

Retina Subspecialty Day of the American Academy of Ophthalmology (AAO) 2019 Annual Meeting

  • Podium Presentation: Results of a Phase 1/2 Trial of an Optimized Gene Therapy in Adults and Children with Retinal Dystrophy Associated with Bi-allelic Variants in RPE65
  • Session Date and Time: Saturday, October 12, 2019, 9:17 a.m. PDT
  • Session Title: RET15- Section XI: Late Breaking Developments, Part II
  • Room: West 3002

MeiraGTx will host a conference call and live webcast following Professor Michaelides’ presentation at AAO to review the data from the Phase 1/2 trial.

About AAV-RPE65

AAV-RPE65 is a novel second-generation gene therapy candidate in development for the treatment of patients with RPE65-deficiency, a condition that causes severe sight impairment beginning at birth. Delivered via subretinal injection, AAV-RPE65 is designed to deliver a normal copy of the RPE65 gene, which is essential for photoreceptor function in the eye. AAV-RPE65 has been granted orphan designation by the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) for the treatment of Leber congenital amaurosis (LCA) caused by disease-causing variants in the RPE65 gene. The FDA has also granted AAV-RPE65 rare pediatric disease designation for the treatment of inherited retinal dystrophy due to biallelic RPE65 disease-causing variants.

About RPE65-Deficiency

RPE65-deficiency is a rare, genetic disorder caused by disease-causing variants in the RPE65 gene. Due to rod photoreceptor dysfunction, RPE65-deficiency causes impaired vision from birth and results in the degeneration of the entire retina over time. Most RPE65-deficient patients experience poor vision in low-light conditions from a young age and suffer from central vision loss that progresses to complete blindness by early adulthood.

RPE65-deficiency is often characterized as a specific subtype of LCA caused by disease-causing variants in the RPE65 gene (LCA2), or a specific subtype of retinitis pigmentosa (RP) caused by disease-causing variants in the RPE65 gene (RP20).

RPE65-deficiency occurs in approximately one in 125,000 people in the U.S. There are estimated to be approximately 6,000 RPE65-deficient patients in the U.S., Japan and EU5, with almost 30% of those patients under the age of 30 years old. Approximately 50 new cases are diagnosed annually.

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