Yet Another Biogen's Retinal Disease Gene Therapy Fails In Late-Stage Study

While basking in the glow of the approval for its Alzheimer’s treatment, Biogen Inc BIIB announced that a gene therapy candidate for eye disease failed a late-stage clinical trial, yet again.
The company announced topline results from Phase 3 STAR study of timrepigene emparvovec (BIIB111/AAV2-REP1) for choroideremia, a rare inherited retinal disease that results in progressive vision loss.
The treatment failed the primary endpoint, the proportion of participants with more than 15 letter improvement from baseline in best-corrected visual acuity (BCVA) at Month 12 — as well as all key secondaries.
The safety results were consistent with previous studies. Phase 3 involved 169 men with a confirmed diagnosis of choroideremia.
Biogen will evaluate the full dataset from timrepigene’s Star study as they consider the next steps for the program. Full results will be presented at a future scientific forum.
Just a month back, cotoretigene toliparvovec gene therapy in X-linked retinitis pigmentosa failed on the primary endpoint but did seem to improve vision in low light conditions.
Both gene therapies came out of Biogen’s $877 million deal for Nightstar Therapeutics.
Price Action: BIIB shares closed at $406.14 on Monday.