The heat is on Acorda Therapeutics once again. Months after Acorda lost a key group of patents, the FDA has declined to review, at least for now, an experimental Parkinson’s disease drug that is critical to the company’s future.
Acorda (NASDAQ: ACOR) said this morning that the FDA has sent the Ardsley, NY, company a “refusal to file” letter for the Parkinson’s drug it hopes to sell as Inbrija. The FDA sends out those letters when it refuses to review a drug application it deems insufficient for one reason or another. In Acorda’s case, the FDA requested more information about Inbrija, an inhalable form of the long-used Parkinson’s treatment levodopa, such as when the drug’s manufacturing site would be ready for an inspection. Acorda filed for approval of Inbrija in June.
Acorda stressed that the FDA’s concerns are “addressable” and that the agency hasn’t asked the company to run any new clinical trials. It will seek “immediate guidance” from the agency, including a meeting, to respond.
“We will work with the FDA as quickly as possible to address the open issues and to clarify the path to successfully re-file our application,” said Acorda president and CEO Ron Cohen. “We remain confident in Inbrija’s data package and its promise as an important new therapy for people with Parkinson’s disease.”
Nonetheless, the news blindsided analysts and investors. Leerink analyst Paul Matteis called the FDA’s letter “surprising, puzzling, [and] disconcerting” in a research note given the agency’s questions aren’t about the drug’s data but two “vague” requests about manufacturing. It is a “somewhat unvettable situation until we get more color,” Matteis wrote. Acorda shares fell 29 percent in pre-market trading Tuesday morning.
Indeed, the news represents a delay for Acorda at a time that the pressure is on for its Parkinson’s drugs to come through and quickly produce 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.
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, through a buyout of Civitas Therapeutics, and another late-stage Parkinson’s drug, tozadenant, it got via a deal for Biotie Therapies. Acorda’s future now rests with those drugs, starting with Inbrija, which succeeded in late-stage testing earlier this year. Defying pressure from activist investor Scopia Capital Management in August—which called for the company to sell itself—Acorda said it would do better if it stayed the course and won approval of the two Parkinson’s drugs. That remains the plan, and investors had recently showed their support, sending shares up about $10 apiece from their lows this year as Inbrija progressed. Today’s setback, however, raises new questions.
“The [letter] is mysterious to us, and we expect the stock to be under pressure until opacity is lifted,” Matteis wrote.