Dive Brief:
- Acorda Therapeutics Inc. said Monday it will discontinue development of its experimental drug tozadenant, five days after announcing five patients had died in a late-stage study of the Parkinson's disease medication.
- Last week, Acorda paused new enrollment into two long-term safety studies, but chose at the time to continue its pivotal Phase 3 trial while adding additional safety measures. Those precautions were subsequently determined not to be enough to keep patients safe.
- The company will now shutter all clinical development of tozadenant, including an immediate stoppage of dosing of patients in the Phase 3 study. Results from the patients who completed the study will be announced at a future medical meeting.
Dive Insight:
The termination of its late-stage asset is a huge blow for Acorda, which has been facing a string of setbacks since a court decided earlier this year that patents for its best-selling drug Ampyra (dalfampridine) were no longer valid.
That decision, which puts nearly 90% of Acorda's revenues at stake, was the first domino in a discouraging chain of events, including pressure from an activist investor to sell the company.
The recent announcement of the safety issues with tozadenant pushed the company's stock down more than 30%, but investors seem to be responding positively to the news that Acorda will discontinue the program. Acorda’s stock was up nearly 9% to trade above $19 per share in morning trading on Monday.
The patient deaths and potential safety problems with tozadenant meant that the drug was a long-shot for approval or — at the very least — that any future commercial potential would be greatly limited if Acorda managed to squeak by the Food and Drug Administration. Investors are likely happy the biotech has chosen to cut its losses and not waste any more resources on the drug's development.
But with tozadenant now out of the picture, Acorda is relying more heavily on Inbrija, also known as CVT-301, which is also a treatment for Parkinson’s disease.
The company filed the drug with the FDA earlier this year, but received a Refusal-to-File letter from the agency. The FDA said the application wasn't complete and asked Acorda to provide information regarding the date when the manufacturing site would be ready for inspection, as well as answer questions about submission of the drug master production record. The company has yet to refile for approval.