Acorda Therapeutics said Monday that it will stop work on an experimental treatment for Parkinson’s disease, spelling the end for a drug the Ardsley, NY, company added to its pipeline last year in a $363 million deal.
The announcement follows the recent emergence of safety problems for the Acorda (NASDAQ: ACOR) drug, tozadenant. Acorda said last week that cases of arangulocytosis, dangerously low levels of infection-fighting white blood cells, were found in patients during the Phase 3 clinical trial. Some of those cases led to sepsis and death. No such cases were reported in patients given a placebo.
Acorda had said it planned to address the problem by testing white blood cell counts more frequently. The study is close to complete and Acorda was set to report the full results early next year. But in a statement Monday, Acorda said analysis of the clinical trial data led the company to conclude that even more frequent white blood cell testing wouldn’t be enough to keep patients safe. Acorda will still disclose the trial results at a medical meeting next year, but it will no longer develop tozadenant. Patients already enrolled in the study will stop receiving doses of the Acorda drug.
Acorda will now put all of its resources behind Inbrija, an inhalable version of the decades-old Parkinson’s drug levodopa that the company has submitted to the FDA for approval. Acorda acquired that drug in 2014 from its $525 million acquisition of Chelsea, MA-based Civitas Therapeutics. Inbrija succeeded in late-stage testing earlier this year, but in August the FDA sent the company a “refusal to file” letter,” meaning it wanted more information about the drug before it would consider approving the therapy. Acorda said it plans to resubmit its approval application by the end of the year.
In a note to investors, Leerink Partners analyst Paul Matteis called the decision to dump tozadenant, save cash, and put it towards Inbrija a “good strategic step” given the likely limited commercial potential of tozadenant even if it were approved. Acorda also recently grabbed $53 million in cash by selling some royalty streams to Healthcare Royalty Partners and Lundbeck. Acorda can now launch Inbrija without selling more shares of stock, Matteis wrote. Shares climbed 6.5 percent in early trading Monday morning.
Acorda had turned to acquisitions in its effort to expand its scope beyond its multiple sclerosis drug dalfampridine—a quest that has become more urgent now that the last of dalfampridine’s patents will expire next year. Following the Civitas deal, Acorda last year bought Finland-based Biotie Therapies for $363 million in cash, which gave the company tozadenant and a second experimental Parkinson’s drug, SYN120, currently in Phase 2 clinical testing. The trial, which has some financial support from the Michael J. Fox Foundation, is expected to be completed in the first half of 2018.