Biogen Phase 2/3 gene therapy trial misses primary endpoint

 

• While the Phase 2/3 XIRIUS study did not meet its primary endpoint, data indicated positive trends in several prespecified secondary endpoints, such as a clinically relevant measure of visual acuity
  

• Biogen will communicate next steps for the program after analyzing the complete data set

• Currently there are no approved treatments for X-linked retinitis pigmentosa (XLRP), a rare inherited retinal disease that ultimately leads to blindness in most people with the condition
  

 Biogen Inc. (Nasdaq: BIIB) today announced topline results from the Phase 2/3 XIRIUS study of 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 that is associated with progressive vision loss as the light-sensing cells of the retina gradually deteriorate. Initial symptoms are difficulty seeing at night, followed by restriction of the field of vision and eventually blindness in most people by the age of 40. Patients living with XLRP currently have no approved treatments.

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.

“Although the Phase 2/3 XIRIUS study of cotoretigene toliparvovec did not meet its primary endpoint, we are encouraged by positive trends in other pre-specified clinically relevant endpoints, such as a measure of visual acuity under low light conditions,” said Katherine Dawson, M.D., head of the therapeutics development unit at Biogen. “XLRP is a serious, early-onset form of retinitis pigmentosa, and people living with it face almost certain blindness by the end of the fourth decade, commonly leading to loss of independence, depression and unemployment. We are working to further evaluate the data from the XIRIUS study before communicating potential next steps for the cotoretigene toliparvovec clinical development program.”

https://finance.yahoo.com/news/biogen-announces-topline-results-phase-113000443.html