People with a kind of rare neurological disorder may have a new treatment option in the not-too-distant future, as a medicine from Connecticut-based drugmaker Biohaven just succeeded in a large clinical trial.
To date, the Food and Drug Administration has not approved anything for this group of brain disorders, which are known as “spinocerebellar ataxia” and often impair muscle movement and coordination. But Biohaven believes its medicine could be the first, and intends to submit an approval application to the agency before the end of the year.
Supporting Biohaven’s case are freshly released data from a trial where participants took the company’s drug once a day and were followed over the course of three years to see if it changed the course of their spinocerebellar ataxia. The company said it did, with the disease slowed between 50% and 70%, which translates to a delay in progression of 1.5-2.2 years over the three-year period.
The study didn’t have a placebo group but instead compared participants to “natural history” data from an external pool of untreated patients. The two groups were then evaluated across a series of tests. The newly released data cover a total of 63 participants who had completed three years of treatment.
On the study’s main goal, which used a 16-point scale to gauge a patient’s ability to walk, stand, sit and speak, Biohaven said its drug significantly outperformed the control group. The company said the same was true for eight other key measures in the experiment.
“I cannot underscore enough the impact of a potential treatment that can slow [spinocerebellar ataxia],” Jeremy Schmahmann, a neurology professor at Harvard Medical School and the founding director of the Ataxia Center at Massachusetts General Hospital, said in a statement from Biohaven.
Vlad Coric, Biohaven’s CEO, noted how the trial was designed using both input from the FDA as well as principles outlined in the agency’s guidance for using “real-world evidence.” The company also analyzed results against a separate natural history cohort of European patients with spinocerebellar ataxia, and found its drug still succeeded in the main goal at all timepoints.
“We look forward to interacting with regulatory agencies to bring [our drug] to patients,” Coric said.
Biohaven’s medicine, troriluzole, is a prodrug of a compound called riluzole, which has been shown to provide modest protection to nerve cells and is frequently given as a treatment for ALS.
As one of Biohaven’s most advanced research projects, troriluzole could play an important role as the company looks to rebuild its deck of marketed products. In the spring of 2022, Biohaven agreed to sell its migraine business, including the Nurtec ODT pill, to Pfizer for $11.6 billion.
Around that same time, troriluzole hit a major setback by flunking a different late-stage study of spinocerebellar ataxia patients. Results showed that patients declined about the same regardless of whether they received Biohaven’s drug or a placebo. Yet, Biohaven found enough silver linings in the data not to give up on the program.
The company plans to ask the FDA for approval sometime between October and December. The drug has been granted a classification that could expedite the agency’s review process, and Biohaven estimates that it will be prepared to commercialize in the U.S. next year.
Biohaven’s stock price was up 15% at market’s open Monday, to trade at its highest point since mid-April. Shares were going for just north of $45 apiece by late morning.
