An experimental Biohaven Pharmaceutial therapy intended to treat anxiety has failed in a late-stage study, the first of four shots that the company is taking with the drug to try to address neurological disorders.
The results released Monday for Biohaven (NASDAQ: BHVN) drug troriluzole were for generalized anxiety disorder, a persistent feeling of worry or nervousness. The main goal of the Phase 3 study was to beat a placebo in a widely used rating scale that measures anxiety. The eight-week clinical trial randomly assigned 402 adults to receive the Biohaven drug or a placebo twice a day.
Under the rating scale used, the higher the score, the higher the level of anxiety. Patients treated with the Biohaven drug did show a reduction in anxiety, with an average change in score from baseline of -9.28 points. But that was not enough to beat the placebo group, where the average change was -9.35 points.
Following the announcement of the clinical trial results, Biohaven’s stock price fell nearly 12 percent to $45.19.
Troriluzole is one of three compounds in the Biohaven pipeline being developed to modulate the glutamate system. Glutamate is a neurotransmitter that nerve cells use to send signals to other cells in the central nervous system. Troriluzole was designed to increase the activity of amino acids that clear glutamate, which is necessary for the synapses to properly activate, Biohaven explains in securities filings. Clearing away the neurotransmitter also prevents damage to the neurons from excessive activation of glutamate receptors.
In addition to anxiety, Biohaven is evaluating troriluzole in other disorders where glutamate has been linked to how disease develops. Clinical trials are ongoing in obsessive compulsive disorder (OCD), Alzheimer’s disease, and spinocerebellar ataxia. Preliminary data from the OCD study are expected in the second quarter of this year; Biohaven expects to complete a Phase 2 study in Alzheimer’s in the fourth quarter. A late-stage study in spinocerebellar ataxia is currently enrolling patients.
In a research note Monday, SVB Leerink analyst Marc Goodman wrote that the results mean that Biohaven misses out on adding another neuro drug to potentially commercialize alongside its migraine pill, rimegepant. An FDA decision on that drug is expected later this quarter.
Goodman also said that while the troriluzole results are disappointing, there is still a chance that the compound becomes an approved drug in one of the other diseases for which it is currently being tested. The positive takeaway from the anxiety clinical trial data is that the rate of patients stopping treatment due to side effects was low—4 percent for the troriluzole versus 4.5 percent for the placebo group. That’s encouraging safety news for the other ongoing studies testing the drug, Goodman wrote.