GenSight to Pursue Approval for LHON Gene Therapy Candidate, Despite Phase III Setback

June 15, 2018: By Jon Swedien

GenSight Biologics will continue to pursue US and European regulatory approvals for its gene therapy candidate for Leber hereditary optic neuropathy (LHON) despite missing a primary endpoint in a Phase III trial, CEO Bernard Gilly said June 12.

The candidate, GS010 (rAAV2/2-ND4), improved subjects’ vision during the REVERSE trial but missed the primary endpoint because it failed to outperform the sham.

Paris-based GenSight said it is encouraged by the results, and it has other clinical trials for GS010 in progress.

“We are now going to discuss the full results with regulatory authorities so that we are aligned on how best to bring GS010 to market with our existing Phase III program within our defined timelines,” Gilly said.

REVERSE evaluated a single intravitreal injection of GS010 in 37 subjects who lost vision due to LHON between six and 12 months prior to the beginning of the study.

Subjects received GS010 in one randomly selected eye and sham treatment (pressure to the eye using the blunt end of a syringe) in the other.

Treated eyes improved +11 ETDRS letters at 48 weeks compared with baseline, while untreated eyes improved +10 ETDRS letters, the company said.

The data from REVERSE suggest that GS010 may provide meaningful bilateral improvement of vision, which GenSight had not expected, Chief Medical Officer Barrett Katz said.

Katz said GS010-treated eyes were significantly more likely to achieve vision of 20/200 or better when compared with eyes treated with the sham.

He said there could be a potentially larger benefit for subjects at earlier stages of the disease.

GenSight said the gene therapy met some secondary endpoints, specifically the change in ganglion cell layer macular volume measured from baseline to week 48 and change in thickness of the temporal quadrant of the retinal nerve fiber layer from baseline to week 48.

These results demonstrated a direct biologic and physiological impact of GS010 on the anatomy relevant to LHON, GenSight said.

Also, contrast sensitivity, as determined by Pelli-Robson low‑vision testing, almost doubled in the GS010‑treated eyes compared to sham‑treated eyes.

GenSight noted that subjects who were age 21 and younger at enrollment tended to have better clinical outcomes.

The company said all 37 subjects will be evaluated again at 96 weeks. That data will be reported in Q1-2019.

GS010 is also being investigated in two additional ongoing Phase III trials—RESCUE and REFLECT.

 

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