Loading...
Loading...
- 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.
© 2024 Benzinga.com. Benzinga does not provide investment advice. All rights reserved.
Comments
Loading...
Date | ticker | name | Actual EPS | EPS Surprise | Actual Rev | Rev Surprise |
---|
Benzinga simplifies the market for smarter investing
Trade confidently with insights and alerts from analyst ratings, free reports and breaking news that affects the stocks you care about.
Join Now: Free!
Already a member?Sign in