Biogen's Retinal Disease Gene Therapy Flunks Mid-Stage Study

Biogen Inc BIIB has announced topline results from the Phase 2/3 XIRIUS study evaluating cotoretigene toliparvovec (BIIB112), a gene therapy being investigated as a one-time therapy for patients with X-linked retinitis pigmentosa (XLRP).
XLRP is a rare, inherited retinal disease associated with progressive vision loss as the retina's light-sensing cells gradually deteriorate.
The XIRIUS study did not meet its primary endpoint of demonstrating a statistically significant improvement in the proportion of treated study eyes with more than 7 dB improvement from baseline.
This assessment was performed at 12 months and compared to the study eye of patients randomized to the untreated control group.
The company says that positive trends were observed across several clinically relevant prespecified secondary endpoints.
Most adverse events were ocular in nature, mild-to-moderate in severity, and resolved.
Complete analysis of the XIRIUS study is ongoing, and detailed results will be shared in a future scientific forum.
Cotoretigene toliparvovec is designed to provide full-length functioning retinitis pigmentosa GTPase regulator (RPGR) protein in XLRP patients caused by mutations in the RPGR gene.
By replacing the gene, cotoretigene toliparvovec leads to increased levels of the RPGR protein, which may potentially slow, stop or prevent further degeneration of photoreceptors in patients with RPGR-associated XLRP.
Price Action: BIIB shares are trading 0.55% lower at $280.16 during the premarket session on the last check Friday.

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