Ionis-Biogen Partnered Amyotrophic Lateral Sclerosis Trial Disappoints

Biogen Inc BIIB and Ionis Pharmaceuticals Inc IONS have announced topline results from the Phase 1 study of BIIB078 (IONIS-C9Rx) for C9orf72-associated amyotrophic lateral sclerosis (ALS).

• In this Phase 1 study, BIIB078 was generally well-tolerated. The adverse events (AEs) were mostly mild to moderate in severity and occurred similarly across BIIB078 and placebo groups. 
• The most common AEs were falls, procedural pain, and headache.
• BIIB078 did not meet any secondary efficacy endpoints and did not demonstrate clinical benefit. 
• In the dose cohorts up to 60 mg, there were no consistent differences between the BIIB078 and placebo groups. 
• Participants in the BIIB078 90 mg dose cohort trended toward a greater decline than those in the placebo group across secondary endpoints. 
• The companies have decided to discontinue the BIIB078 clinical development program, including its open-label extension study.
• Biogen exercised its option to license BIIB115/ION306 from Ionis for spinal muscular atrophy (SMA).
• Biogen made a one-time payment of $60 million to Ionis in Q4 FY21. 
• Price Action: IONS shares are down 0.93% at $36.33, and BIIB stock is up 0.59% at $212.21 during the market session on the last check Monday.