Acorda faces new trouble with trial halt, safety problems

November 15, 2017 - Lisa LaMotta

Dive Brief:

• Acorda Therapeutics Inc. disclosed Wednesday morning it would halt new enrollment into two long-term safety studies of its Parkinson's disease drug tozadenant after serious safety signals emerged.
• The company will also increase monitoring of patients' blood cell counts to once a week in an ongoing Phase 3 trial after cases of agranulocytosis, or low white blood cell count, were reported.
• Share of Acorda plummeted on the news, dropping 38% to trade near $17.50 Wednesday morning. The stock has been fluctuating a lot over the past 52 weeks, recording a high of $33 per share and a low of $13.60

  Dive Insight:

  Tozadenant is part of a new class of drugs called A2a receptor antagonists that are meant to treat motor dysfunctions in patients with Parkinson’s disease.

  Including a Phase 2b trial, Acorda said 890 people have been exposed to tozadenant so far in clinical development. Of those patients, there have been seven cases of sepsis — all of which were taking tozadenant as opposed to placebo. Five of those cases resulted in death and four were associated with agranulocytosis.

  "Contingent on further input from the Data Safety Monitoring Board and FDA, we continue to expect to report efficacy and safety results of the double-blind Phase 3 study in the first quarter of 2018," said Acorda CEO Ron Cohen.

  Management noted in a Wednesday conference call with analysts that there has been no pattern of the safety problems, and didn't present a hypothesis to explain why this is happening.

  Jefferies equity analyst Michael Yee wrote in a Nov. 15 note to clients that he pegs probability of the Phase 3 trial reading out positively at about 50%. Even then, the clinical benefit would have to be substantial for the Food and Drug Administration to look beyond this safety problem.

  "Even if approved, it would likely have major warnings or restrictions (weekly monitoring, etc.) on the label which could hamper commercial uptake anyways," wrote Yee.

  This is a huge setback for Acorda, which is currently in dire need of a win. The company recently had patents for its key revenue-driver Ampyra (dalfampridine) knocked down by the courts, putting nearly 95% of the company’s revenues at risk.

  The court decision led Acorda to reduce its headcount by 20% and spurred an activist shareholder to push for a sale of the company. In September, the biotech used a "poison pill" to keep Scopia Capital Management LP from acquiring a greater stake in the company.

  Elsewhere, Acorda hit a setback with its other Parkinson’s disease drug Inbrija (levodopa inhalation powder), when the FDA refused to accept the company’s New Drug Application in August.

  https://www.biopharmadive.com/news/acorda-faces-new-trouble-with-trial-halt-safety-problems/510992/