Acorda Therapeutics’ rebuilding plan just suffered another setback, leaving the fate of one of the experimental Parkinson’s disease drugs that is closest to market unclear.
Ardsley, NY-based Acorda (NASDAQ: ACOR) said Wednesday morning that it has seen troubling safety problems in a Phase 3 trial of its experimental Parkinson’s disease drug tozadenant. Acorda reported cases of arangulocytosis, dangerously low levels of infection-fighting white blood cells, and some of them have led to sepsis and death. Acorda said some of these incidents could be related to tozadenant treatment.
According to Acorda, including a previous mid-stage trial, 890 total patients have gotten tozadenant, and 234 a placebo. Seven of the patients on Acorda’s drug have suffered sepsis. Five of those patients died, and four of the sepsis cases were associated with arangulocytosis. No sepsis or arangulocytosis cases have been reported among placebo patients.
Acorda will now test blood cell counts more frequently—every week—in its ongoing Phase 3 trial. It has also stopped enrolling patients in two long-term safety studies of the drug, pending discussions with a data safety monitoring board and the FDA. As of now, Acorda still expects to wrap up the study and report data in the first quarter of next year, but CEO Ron Cohen acknowledged in a statement that that will now depend on the input of the FDA and the monitoring board.
“We have taken these steps in the best interests of the safety of patients in the tozadenant studies, which is our top priority,” Cohen said.
Acorda shares fell almost 38 percent in pre-market trading.
The news marks another hit for Acorda in an already tumultuous year. Tozadenant, acquired via a buyout of Biotie Therapies in January 2016, is one of two experimental Parkinson’s drugs Acorda is counting on to quickly become a new revenue driver. In March, a district court judge invalidated four of the five patents covering dalfampridine (Ampyra), Acorda’s top selling drug. Acorda became profitable because of dalfampridine, a drug that helps improve the walking ability of multiple sclerosis patients. But it has had a tough time proving the drug’s worth for other diseases, and the district court’s ruling left the company with a single dalfampridine patent that expires in the middle of 2018. Dalfampridine generated $132.6 million in sales in its last quarter.
After the ruling, Acorda promptly cut 20 percent of its staff and put its resources behind two different Parkinson’s drugs it acquired through deals in 2014 and 2016—Inbrija, an inhalable form of the Parkinson’s drug levodopa that it got through a buyout of Civitas Therapeutics, and tozadenant. Acorda’s future now rests with those drugs, and it has even fought off pressure from activist investor Scopia Capital Management in August—which called for the company to sell itself—by saying it would do better if it stayed the course and got them to market.
Both of those drugs have now suffered setbacks. 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. That may ultimately just amount to a delay. Acorda had previously expected to know by September whether the FDA would approve Inbrija. Upon reporting its latest financial results in October, Acorda said it plans to resubmit its approval application by the end of the year.
Now, however, new questions have emerged regarding tozadenant at a time in which rival drugs have been recently approved to help combat some of the symptoms of Parkinson’s, which affects about 1 million Americans and 4 to 6 million people worldwide and causes tremors, confusion, and memory loss. In 2016, San Diego, CA-based Acadia Pharmaceuticals (NASDAQ: ACAD) won FDA approval of a drug, pimavanserin (Nuplazid), meant to reduce hallucinations associated with Parkinson’s. In August, the agency approved a drug from Emeryville, CA-based Adamas Pharmaceuticals (NASDAQ: ADMS), called amantadine (Gocovri), for levodopa-induced dyskinesia.
Tozadenant is also meant to combat Parkinson’s symptoms. Given the competition, Leerink analyst Paul Matteis, who called the disclosure a “major surprise,” wrote in a note that, even if approved, tozadenant’s commercial potential will be significantly compromised.
Acorda will hold a conference call this morning to discuss the news.