Acorda Therapeutics to discontinue development of dalfampridine
Acorda Therapeutics announced that the MILESTONE clinical study did not show sufficient efficacy to support further development of dalfampridine to improve post-stroke walking difficulties. "We are disappointed by this outcome. The study indicated there was activity related to walking in people with PSWD, as suggested by the prior Phase 2 study, but overall this was not sufficiently clinically meaningful. I want to express our gratitude to the study participants, their care partners and clinicians, who gave their time and commitment to this research," said Ron Cohen, M.D., President and CEO of Acorda. "This outcome underscores the risks that companies in the biopharmaceutical industry must take in order to develop innovative medicines. Over the past three years, we have successfully diversified our pipeline portfolio to account for this risk. We plan to focus R&D resources on developing our promising late-stage Parkinson's disease therapies, CVT-301 and tozadenant, as well as advancing our earlier stage assets, CVT-427 in migraine, SYN120 in Parkinson's disease dementia, and rHIgM22 in MS." As part of the PSWD development program, a multi-dose pharmacokinetic study confirmed the Company has developed a potentially viable once-daily formulation of dalfampridine. The Company elected to stop enrollment and to conduct an unblinded analysis of the MILESTONE trial after reaching enrollment of 377 participants. This analysis included 368 participants who received either 10 mg or 7.5 mg of dalfampridine twice daily or placebo. The primary outcome measure of the study was the proportion of participants who showed at least a 20% improvement on the Two Minute Walk Test at Week 12 as compared to baseline. The 2MinWT measures the distance a subject can walk in 2 minutes, and is a validated scale used to assess walking capacity. The study found that 23 of 121 (19.0%) participants receiving 10 mg of dalfampridine BID and 17 of 121 (14.0%) participants receiving 7.5 mg of dalfampridine BID showed at least a 20% improvement on the 2MinWT, compared to 17 of 126 (13.5%) participants receiving placebo. In this study, dalfampridine was well tolerated in the post-stroke population. The safety profile overall was similar to that observed in multiple sclerosis clinical trials and post-marketing surveillance. The most common adverse events (greater than or equal to 5%) reported in this study were: falls (10 mg: 10.7%, 7.5 mg: 9.5%, placebo: 5.6%), urinary tract infections (10 mg: 9.0%, 7.5 mg: 6.3%, placebo: 2.4%), dizziness (10 mg: 3.3%, 7.5 mg: 7.9%, placebo: 2.4%) and fatigue (10mg: 2.5%, 7.5 mg: 3.2%, placebo: 6.3%). There were no seizures reported in the dalfampridine 10 mg group. There were 2 seizures reported in the 7.5 mg group and 3 reported in the placebo group.