Disappointing results from Biogen's gene therapy study for XLRP

In a disappointing outcome for its gene therapy aspirations, US biotech major Biogen (Nasdaq: BIIB) on Friday announced top-line results from the Phase II/III XIRIUS study of cotoretigene toliparvovec (BIIB112), a gene therapy being investigated as a one-time therapy for patients with X-linked retinitis pigmentosa (XLRP), that missed its goal.

The XIRIUS study did not meet its primary endpoint of demonstrating a statistically-significant improvement in the proportion of treated study eyes with ≥7 dB improvement from baseline at ≥5 of the 16 central loci of the 10-2 grid assessed by Macular Integrity Assessment (MAIA) microperimetry. This assessment was performed at 12 months and compared to the study eye of patients randomized to the untreated control group. Positive trends were observed across several clinically relevant prespecified secondary endpoints.

Drug came with Nightstar acquisition