After tracking 7 cases of sepsis — with 5 deaths — in studies of its late-stage Parkinson’s drug tozadenant, Acorda is increasing patient surveillance while pausing enrollment in its long-term safety studies to get a handle on the issue.
Acorda’s shares plunged 40% on the news.
The safety issue arose after researchers became aware of the cases of agranulocytosis, wiping out the white blood cells needed to fight off an infection. Now they say they will check those cell counts on a weekly basis as they study the potential drug problem with the FDA and the monitoring committee.
Acorda CEO Ron Cohen says they still expect to report out Phase III safety and efficacy data in Q1 coming up, but any threat to its number two development program — behind CVT-301 — is likely to rattle its backers.
Leerink’s Paul Matteis says the surprising news of a major safety issue at Acorda leaves the drug under a cloud.
The need for frequent white blood cell monitoring is not unheard of for approved neurology products: agranulocytosis risk is a known limitation for clozapine, an approved, and very effective atypical antipsychotic. But the risk/benefit for Toz, in the context of an apparent mortality signal – and what is already a crowded category – is now significantly compromised, given that the drug is not disease-modifying and will be targeted toward an already fragile elderly population.
Then, later on Wednesday, Matteis virtually wrote it off as a loser.
We are now very concerned regarding the approvability of Toz – even if it does work – given its positioning as a symptomatic therapy with a moderate level of benefit. We have significant questions regarding the commercial viability of a new symptomatic Parkinson’s therapy with such significant safety risks.
Acorda swooped in to buy Finland’s Biotie in early 2016 for $363 million in cash to bag the Phase III Parkinson’s drug and add a badly needed late-stage drug to the pipeline. Tozadenant is an oral adenosine A2a receptor antagonist that’s registered proof of concept data for reducing “off” times among Parkinson’s patients taking the standard levodopa/carbidopa remedy.
There’s little margin for error at Acorda.
With its patents on the company’s cash cow Ampyra under attack, possibly opening the road to a generic rival next summer, the biotech has had to re-file its application for CVT-301. The FDA had issued a refuse-to-file the application for the drug, delaying a therapy which will be needed to rescue the company from generic competition for Ampyra.
The biotech, meanwhile, has moved to restructure, block a potential hostile takeover attempt and deflect criticism for leaving itself vulnerable to the crisis it’s in. If its safety issue with tozadenant spins out of control, Acorda’s executive team will be under twice as much pressure to perform on CVT-301.
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