Acorda Takes a $363M Hit as Deaths Lead to Termination of Parkinson's Drug
On Nov. 15, Acorda Therapeutics reported that it had ended enrollment in its Phase III clinical trial of tozadenant for Parkinson’s disease due to several patient deaths. Today, the company announced that it was discontinuing clinical development of the drug altogether.
In both the Phase IIb study and the Phase III trial, about 890 patients received tozadenant and 234 received placebo. There were five fatalities out of seven patients with sepsis, all in the tozadenant group. Agranulocytosis is an absence of white blood cells, which are key immune cells. Four of the cases of sepsis were related to agranulocytosis, two had no white blood cell counts at the time of the event and one patient had a high white blood cell count.
The Nov. 15 announcement indicated that in addition to pausing enrollment, the company was going to discuss options with the independent Data Safety Monitoring Board (DSMB) and the U.S. Food and Drug Administration (FDA), and planned to increase the number of times it conducted blood cell counts on participants in the trial.
Now, after analyzing additional information, Acorda concluded that blood cell counts wouldn’t ensure patient safety. “Patient safety is our top priority,” said Ron Cohen, Acorda’s president and chief executive officer, in a statement. “While we are deeply disappointed by this outcome, we remain committed to the Parkinson’s community, which is in great need of new therapeutic options. We are grateful to the patients who volunteered for the tozadenant studies and to their care partners, clinical investigators, and the internal and external study teams.”
Tozadenant is an oral adenosine A2a receptor antagonist. It is in development as an adjunctive treatment to levodopa in Parkinson’s disease patients in an effort to reduce OFF time. OFF time refers to the motor fluctuations in Parkinson’s patients when the treatment, usually levodopa, is not working best and symptoms return.
The company picked up the drug in early 2016 when it acquired Finland’s BioTie for $363 million.
But the company now has a major problem. John Carroll, writing for Endpoints News, notes, “Acorda’s cash cow, Ampyra, faces the patent guillotine next summer, if the courts uphold a decision that would open the door to generic competition. Without another drug ready to take its place with the marketing team, Acorda’s revenue could start to swoon. So at a time the biotech needs to impress everyone with its ability to execute—as it did with a drug it purchased late in the clinic to reduce the chances of failure—Acorda continues to rack up snafus.”
One of those was in March, when a district court judge invalidated four out of five patents that cover Ampyra (dalfampridine), Acorda’s top-selling drug. Ampyra is prescribed to multiple sclerosis patients to improve walking ability. The company has tried to get it approved for other diseases, but hasn’t had success doing so. Meanwhile, the single patent expires in the middle of 2018. In the last quarter, Ampyra brought in $132.6 million in sales.
Acorda responded by cutting 20 percent of its staff and shifting resources to two Parkinson’s drugs, Inbrija, an inhalable version of levodopa, and tozadenant. Inbrija had some success in a late-stage trial this year, but in August the FDA hit Acorda with a “refusal to file” letter, indicating it wanted more data about the drug before it would consider approving it. It’s possible that’s just a delay, and Acorda indicated in its October financial report that it plans to resubmit the application by the end of this year.