GenSight Biologics announced that the first subject was treated in the first-in-man PIONEER phase I/II clinical trial of GS030 at the Moorfields Eye Hospital in London, United Kingdom.
“We are thrilled to see our second lead program, GS030, now entering the clinic. This is a fantastic achievement for the team, and a promising step forward for patients. Optogenetics has been studied for years in research laboratories, and it bears a great clinical promise in fighting vision impairment and other degenerative sensorial and neurological disorders,” Bernard Gilly, Co-founder and Chief Executive Officer of GenSight, said in a company news release. “GenSight continues to deliver on its strategy to develop novel approaches for treating and curing blinding diseases.”
PIONEER is a first-in-man, multicenter, open label dose-escalation study to evaluate the safety and tolerability of GS030 in 18 subjects with retinitis pigmentosa. GS030 combines a gene therapy (GS030-DP) administered via a single intravitreal injection with a wearable optronic visual stimulation device (GS030-MD).
GS030 is based on the Optogenetics technology platform developed by GenSight, which uses gene therapy to introduce a gene encoding for a light-sensitive protein into retinal ganglion cells by a single intravitreal injection, thereby making them responsive to light and bypassing destroyed photoreceptors.
Eligible patients in the first three cohorts will be those affected by end-stage non-syndromic retinitis pigmentosa with no light perception (NLP) or light perception (LP) level of visual acuity. The extension cohort will also include less severe patients, with hand motion (HM) and counting fingers (CF) levels of visual acuity.
As per protocol, three cohorts of three subjects each will be administered an increasing dose of GS030-DP via a single intravitreal injection in their worse affected eye. A fourth extension cohort will receive the highest tolerated dose. An independent Data Safety Monitoring Board (DSMB) will review safety data of all treated subjects in each cohort and make recommendations before moving to the next dose.
The primary outcome analysis will be the safety and tolerability at 1-year post-injection.
“I am particularly pleased with this milestone because it potentially paves the way for a substantial improvement in the quality of sight and life of patients with RP. It is also a recognition of the quality of the collaboration between the team at the Vision Institute and GenSight,” said Dr. José-Alain Sahel, Director of the Institut de la Vision(Sorbonne-Université/Inserm/CNRS), Paris; Chairman of the Department of Ophthalmology at Centre Hospitalier National d’Ophtalmologie des XV-XX, Paris; Professor and Chairman of the Department of Ophthalmology at University of Pittsburgh School of Medicine and UPMC (University of Pittsburgh Medical Center).
GS030 was granted Orphan Drug Designation in the U.S. and Europe. PIONEER is conducted in three centers across the United Kingdom, France and the United States.
GenSight expects to complete enrollment by the fourth quarter of 2019. Early findings for the first cohort are expected in the first half of 2019, and topline results for all subjects are expected in the fourth quarter of 2020.