GenSight Biologics announced that the independent Data Safety Monitoring Board (DSMB) completed its first safety review of the ongoing PIONEER phase 1/2 clinical trial of GS030, combining gene therapy and optogenetics for the treatment of retinitis pigmentosa. The DSMB confirmed the absence of any safety issues for the first cohort of three subjects who received a single intravitreal injection of 5e10 vg combined with a wearable optronic visual stimulation device. The DSMB recommended moving forward as planned without any modification in the protocol and recruiting the second cohort of three subjects receiving an escalating dose of 1.5e11 vg.
“We are pleased to be able to move forward GS030, our second clinical stage program. We look forward to confirm the safety of GS030 at higher doses and to demonstrate efficacy in restoring useful visual functions in RP patients,” Bernard Gilly, Co-founder and Chief Executive Officer of GenSight, said in a company news release.
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, making them responsive to light and bypassing disease-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) levels of visual acuity. The extension cohort will include 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. An extension cohort will receive the highest tolerated dose. The 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.
GS030 was granted orphan drug designation in the United States and Europe. PIONEER is being conducted in three centers across the United Kingdom, France, and the United States.
GenSight expects to complete enrollment in the first half of 2020. Early findings may be available and released in the second half of 2019, and preliminary results are expected in the fourth quarter of 2020.