Acorda dumps $363M Parkinson’s drug after patient deaths

Acorda Therapeutics
Acorda Therapeutics' offices.

Acorda Therapeutics is scrapping its Parkinson’s disease drug tozadenant following patient deaths in its phase 3 trial. The action marks the end for an oral adenosine A2a receptor antagonist Acorda paid $363 million in cash for last year. 

Ardsley, New York-based Acorda flagged up problems with the phase 3 trial last week when the deaths of five patients from sepsis forced it to suspend enrollment in the study. Now, Acorda has stopped dosing of patients already enrolled in the trial following a review of new information obtained from the phase 3 program.

Acoda is yet to provide full details of that information, saying only that it ties to “agranulocytosis and associated serious adverse events” and left it unsure whether “weekly white blood cell count screening would sufficiently ensure patient safety.”  

Those doubts were enough to prompt Acorda to call time on the trial. 

“Patient safety is our top priority,” Acorda CEO Ron Cohen, M.D., said in a statement. “While we are deeply disappointed by this outcome, we remain committed to the Parkinson’s community.”

Acorda reaffirmed its commitment to Parkinson’s disease early last year with the $363 million all-cash takeover of Biotie. That deal gave Acorda the rights to tozadenant, which had already delivered clinical proof-of-concept data hinting at its potential to reduce “off” times in Parkinson’s patients taking the combination of carbidopa and levodopa. 

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Back then, Acorda saw tozadenant racking up sales of $400 million. That made tozadenant a key part of a clutch of drugs tipped to generate $1 billion a year for Acorda. The other key component of that anticipated franchise, CVT-301, has also since run into difficulties that raise doubts about its ability to pull in the $500 million a year once forecast by Acorda. 

The FDA knocked back a filing for approval of CVT-301 following a preliminary review earlier this year after concluding Acorda had failed to submit all the required information.

These pipeline misfires have taken place against the backdrop of a countdown to the loss of patent protection for Ampyra, the drug on which Acorda relies. Tozadenant and CVT-301 were supposed to get to market in time to offset the hit from generic competition for Ampyra. The other drug in Acorda’s $1 billion revenue plan, Plumiaz, also ran aground last year.