Setback for Biogen’s ophthalmic gene therapy aspirations as XLRP program misses in Phase II/III trial

For one of two gene therapy programs Biogen acquired in its takeout of Nightstar, the future is in doubt following its failure to meet the primary endpoint in a Phase II/III trial to treat X-linked retinitis pigmentosa.

On Friday, Biogen Inc. (NASDAQ:BIIB) said cotoretigene toliparvovec (BIIB112) failed to significantly improve light sensitivity from baseline compared with untreated controls at 12 months in the XIRIUS trial. The endpoint assessed the proportion of treated study eyes with at least a 7 dB improvement from baseline at certain loci, assessed by Macular Integrity Assessment (MAIA) microperimetry...