GenSight Biologics

Key Opinion Leaders Highlight GS010 Efficacy and Patient Benefits in Discussion of Findings from REVERSE Phase III Clinical Trial - Seite 3

• Subjects who entered study with better vision (on-chart eyes) tended to have better clinical outcomes. At week 48, in on-chart best-seeing eyes, GS010-treated eyes gained on average +12 ETDRS letters (-0.236 LogMAR) compared to +4 ETDRS letters (-0.075 LogMAR) in sham-treated eyes.
• Subjects whose vision loss was less than 9 months tended to have better clinical outcomes. 75% of GS010-treated eyes that showed a trend in visual acuity improvement at week 48 had vision loss for less than 9 months at time of treatment administration.
• Subjects who were younger (< 21 years) at enrollment tended to have better clinical outcomes.
• The proportion of subjects who achieved a large improvement in visual acuity (0.5 logMar, 25 ETDRS letters) was higher than in sham-treated eyes.

“In my opinion, LHON is a genetic emergency,” Dr. Sergott concluded.

In the panel discussion that followed, the medical experts were asked to interpret what the two-line visual acuity improvement observed in both eyes meant to patients. Dr. Newman cautioned against overemphasizing quantitative measures that may understate the actual improvement in patients’ quality of life. “There is good work being done on more meaningful measurement of the functional impact of improved central acuity for LHON patients, but these measures have yet to be discussed and agreed with authorities,” said Dr. Newman. “Ultimately, it’s up to the patients to decide what a two-line improvement means; we should listen to them.” Ms. Poincenot echoed this assessment: “For patients, slight differences mean a lot in day-to-day life. A little improvement can translate into making life easier.”

Bernard Gilly, co-founder and CEO of GenSight, reaffirmed GenSight’s push to bring GS010 to market. “We are committed to working with the relevant authorities to bring this product to the approval stage as soon as possible,” he said. Aiming to maintain its plan to submit dossiers to the FDA and EMA in Q2 2019, the company is scheduling consultations with those agencies to discuss REVERSE results and trends. These discussions will help frame the results from RESCUE, which will be available in October this year, and inform potential refinements to the design of REFLECT, which has begun patient recruitment.