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

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Biogen's Retinal Disease Gene Therapy Flunks Mid-Stage Study
  • Biogen Inc (NASDAQ: BIIBhas 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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