GenSight begins Phase III retinal disease trials
pharmafile | February 29, 2016 | News story | Research and Development | GenSight, Leber’s Hereditary Optic Neuropathy, retinal disease
French biotech GenSight has begun enrolling patients for two parallel pivotal Phase III trials with its lead candidate GS010, a drug used to treat the retinal disease Leber’s Hereditary Optic Neuropathy (LHON) – a condition which can lead to severe vision loss.
Bernard Gilly, chairman and chief executive of GenSight, comments: “With the first patient recruited, we are now entering the last mile of GS010’s development, which we hope will demonstrate GS010’s ability to durably stop, if not restore, the brutal vision loss caused by LHON. GenSight continues to deliver on its strategy to develop novel approaches against blinding diseases.”
The trials aim to determine whether GS010 can halt or reverse vision loss from LHON due to the NADH dehydrogenase 4 (ND4) mutation, or if it can be as effective as prophylaxis for vision loss in an eye not yet affected. The trials will also seek to identify the therapeutic window of opportunity for treatment after onset of disease – early intervention is considered a potential major factor in maximising therapeutic success.
For this reason, the two clinical trials – RESCUE and REVERSE – will focus on treating patients who have manifested visual decline for up to one year. RESCUE is expected to enroll 36 patients with an onset of vision loss up to six months, and REVERSE the same amount of patients who have experienced vision loss onset for between seven and 12 12 months. The first patient has already been injected with GS010.
GS010 will be administered as a single intravitreal injection to one eye of each subject, while the other eye will receive a sham or placebo procedure. After the initial 48-week study period, a minimal patients will be monitored for three years to determine the sustainability of efficacy outcomes and long-term safety of treatment.
The trials will be conducted in parallel in seven centres across the US, UK, France, Germany and Italy. European sites will open in the coming weeks upon final approval of regulatory agencies, GenSight says, and topline results at 48 weeks are expected by the end of 2017.
Joel Levy
