GenSight Biologics announced that the UK Medicines and Healthcare Regulatory Agency (MHRA) has accepted the company’s clinical trial application (CTA) to initiate the PIONEER phase 1/2 study of GS030 in patients with retinitis pigmentosa (RP).
PIONEER is a first-in-man, multicenter, open label, dose-escalation study to evaluate the safety and tolerability of GS030 in subjects with retinitis pigmentosa. GS030 is the combination of a gene therapy (GS030-DP) administered via a single intravitreal injection and a wearable optronic visual stimulation device (GS030-MD).
Eligible patients will be those affected by end-stage non-syndromic retinitis pigmentosa with vision not better than “counting fingers."
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 higher dose.
The primary outcome analysis will be the safety and tolerability at 1 year post-injection. GenSight expects to treat the first patient in the United Kingdom in the first quarter of 2018.
“I am particularly excited to have GS030 moving into the clinic," Bernard Gilly, CEO and cofounder of GenSight, said in a company news release. "This represents a major milestone for the company. For the first time ever in ophthalmology, an optogenetic approach combined with a medical device will be tested in humans. If proven safe and effective, this therapy could be transferable from Retinitis Pigmentosa to dry-AMD."
GS030 was granted Orphan Drug Designation in the U.S. and Europe. GenSight is in active dialogue with global regulatory agencies and intends to file additional IND and CTA submissions in 2018.