ProQR Therapeutics 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.
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.