Drug Development

Acorda writes off another R&D program as Ampyra flops in stroke study

Ron Cohen

Ron Cohen

Six months after Acorda was forced to write off one of its drug candidates in the wake of a trial flop, the biotech is back with more bad news for investors. Its franchise drug Ampyra (dalfampridine) failed a pivotal study for helping stroke victims walk better. And Acorda is writing the effort off as a loser.

A little more than three years ago Acorda $ACOR was stoked by evidence of success in a small study of the same patient population. But an analysis of the data this time showed that the drug essentially lined up with a placebo when put to a major test.

Acorda’s shares were dented again by the news, dropping 13% in pre-market trading. Their shares have lost half their value over the course of the past 12 months.

Evaluating data from 368 patients in the study, 23 of 121 patients taking 10 mg dalfampridine, 17 of 121 patients taking 7.5 mg of the drug and 17 of 126 taking a placebo demonstrated at least a 20% improvement in a two-minute walk test. That may have reflected a slight drug effect, but it wasn’t nearly good enough for an expanded indication.

Back in May Acorda was also forced to finally punt the epilepsy program for Plumiaz, rejected in 2013, after the nasal spray formulation for diazepam failed a bioequivalence study. Plumiaz was one of the most advanced R&D efforts at Acorda. And while it was considered less potentially lucrative than the inhaled levodopa therapy CVT-301 ($500 million peak sales estimate by the company) and tozadenant ($400 million peak), the biotech had made it a key part of its $1 billion-plus plan to boost revenue by a blockbuster measure of success.

Acorda CEO Ron Cohen, BIO’s chair, acknowledged the most recent failure and then used it as an example of the high-risk nature of drug development — a common theme of his as the industry organization looks to steer the current debate over drug pricing toward favorable treatment for biotech innovation.

“We are disappointed by this outcome. The study indicated there was activity related to walking in people with PSWD, as suggested by the prior Phase 2 study, but overall this was not sufficiently clinically meaningful. I want to express our gratitude to the study participants, their care partners and clinicians, who gave their time and commitment to this research. This outcome underscores the risks that companies in the biopharmaceutical industry must take in order to develop innovative medicines. Over the past three years, we have successfully diversified our pipeline portfolio to account for this risk. We plan to focus R&D resources on developing our promising late-stage Parkinson’s disease therapies, CVT-301 and tozadenant, as well as advancing our earlier stage assets, CVT-427 in migraine, SYN120 in Parkinson’s disease dementia, and rHIgM22 in MS.”


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RAPS Regulatory Convergence 2017