Biogen, Inc. reported that one of the ocular gene therapy candidates it acquired in its 2019 buyout of Nightstar Therapeutics plc missed its primary endpoint in a Phase II/III study, but said it viewed the result as a minor setback. BIIB112 (cotoretigene toliparvovec) failed to demonstrate statistical significance compared to an untreated control group in X-linked retinitis pigmentosa (XLRP) on a measure evaluated by Macular Integrity Assessment (MAIA) microperimetry.
Biogen said on 14 May that while the miss is disappointing, the study did show positive trends on secondary endpoints, such as visual acuity under low light conditions