Acorda axes Parkinson’s drug after trial deaths
Acorda Therapeutics has abandoned development of its Parkinson’s drug, tozadenant, just days after putting trials on hold following a series of patient deaths.
Based in New York state, Acorda said it made the decision after finding new information from phase 3 studies, related to the previously disclosed safety issues with agranulocytosis – a lack of white blood cells in the blood.
Acorda’s phase 3 development programme included an ongoing pivotal efficacy and safety study and two long-term safety studies.
The company revealed last week that seven patients developed sepsis and five of the cases proved fatal.
Four of the sepsis cases were associated with agranulocytosis, two had no white blood cell counts available at the time of the event, and one had a high white blood cell count.
The biotech said it could not be confident that weekly white blood cell count screening could ensure patient safety, and has informed regulators that it will close ongoing studies.
More than 90% of the participants in the placebo-controlled phase 3 efficacy and safety study, CL-05, have completed the study.
Acorda will present data from them in the first quarter of 2018 at an appropriate medical or scientific conference.
Shares in the company ticked up slightly following the news – but last week’s announcement saw them nosedive by almost 40%.
President and CEO Ron Cohen said in a statement that patient safety was the company’s top priority. He added: “While we are deeply disappointed by this outcome, we remain committed to the Parkinson’s community, which is in great need of new therapeutic options. We are grateful to the patients who volunteered for the tozadenant studies and to their care partners, clinical investigators, and the internal and external study teams.”
Tozadenant was the second most developed drug in Acorda’s clinical pipeline – closest to market is a self-administered inhaled formulation of levodopa, for treating ‘off’ periods in people already taking oral carbidopa/levodopa therapy.
The company announced phase 3 results earlier this year, but in August the FDA rejected Acorda’s filing, saying it was not ready.
Acorda said the FDA served a “refusal to file” letter, saying it could not review the data without details of when a manufacturing site will be ready for inspection, and further technical information about production.
Its subsidiary Biotie Therapies has a candidate for Parkinson’s disease-related dementia in phase 2 development, and also a phase 2 drug for primary sclerosing cholangitis, and potentially other inflammatory and fibrotic diseases.
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