A drug developed by Biohaven Pharmaceuticals treats disease by modulating neurotransmitters important for normal brain function Failure A pivotal study in a rare neuromuscular disease marks the third clinical trial failure in many indications in the past two years.
The latest disappointing data comes from testing for spinocerebellar ataxia, a genetic disorder that causes problems with muscle coordination, balance, movement and speech. Symptoms of neurodegenerative disease begin in adulthood and lead to a progressive deterioration in muscle control, eventually leading to the patient’s need for a wheelchair.
There are three types of spinocerebellar ataxia, none of which have any FDA-approved treatments. In all types of spinocerebellar ataxia, preliminary data showed that the drug troluzole did not beat a placebo on a scale used to assess muscle control and movement, Biohaven said Monday. The New Haven, Connecticut-based biotech company attributed the results to lower-than-expected disease progression during the study.
Despite the Phase 3 failure, Biohaven said an analysis of the unblinded data showed that the treatment was effective in patients with spinocerebellar ataxia type 3, the most common form of the disease. In the study of 213 patients, this group comprised 41 percent of the participants. In this group, Biohaven said the scores showed numerical improvements compared to placebo. Given the unmet patient need for the disease, Biohaven said it plans to submit these results to the FDA to see if there is a way forward for the drug,
In a research note sent to investors Monday, William Blair analyst Tim Lugo wrote that while the FDA may offer some flexibility for rare diseases such as spinocerebellar ataxia, the company will wait for more , before assigning any value to the item. He added that investors didn’t expect much of troluzole and its recent failure would be seen as a “liquidation event,” meaning it cleared the way for the company to focus its energies elsewhere.
Troriluzole is designed to lower levels of glutamate, a neurotransmitter. Dysfunction of this brain chemical has been linked to a range of diseases, including amyotrophic lateral sclerosis, Alzheimer’s disease and obsessive-compulsive disorder. Biohaven’s drug is designed to increase the activity of an amino acid that clears glutamate from synapses.
So far, traluzole has not been successful in clinical trials. In 2020, Biohaven reported that the drug Failed Phase 3 Study in Generalized Anxiety Disorder. Last year, the company reported Alzheimer’s disease stage 2/3 failure. The glutamate-clearing drug has one more shot. Phase 3 clinical trials for OCD are still ongoing.In its Report For its financial results for the first quarter of 2022, the company said registration for the study is expected to be completed in the second half of the year.
Biohaven is being acquired by Pfizer for $11.6 billionThe deal focuses on commercializing the migraine drug Nurtec ODT, as well as other pain compounds targeting the same protein. Pfizer doesn’t get the rest of Biohaven’s neuroscience drug pipeline, which will be spun off as a separate public company that will retain the Biohaven name. Troriluzole is one of the projects to be spun out as part of the new Biohaven.
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