Being studied for GAD, OCD, AD and spinocerebellar ataxia, Biohaven’s troriluzole has now struck out in 3 late-stage studies.
Troriluzole is proving itself against Alzheimer’s disease.
Alzheimer’s disease has found itself in the top three most expensive diseases. Billions of dollars are poured into research each year, yet the push for effective treatment and hope for a cure is dashed time and again. This week another biopharma announced the disappointing news that their trial too had failed to make an impact on the devastating progression of Alzheimer’s.
Connecticut-based Biohaven Pharmaceuticals was looking pretty hopeful with its third-generation prodrug to modulate glutamate, the most abundant excitatory neurotransmitter in the human body. The pharma company hoped to find success for troriluzole in multiple indications and had studies evaluating efficacy in generalized anxiety disorder (GAD), obsessive compulsive disorder, spinocerebellar ataxia and Alzheimer’s disease (AD).
Unfortunately, Biohaven’s troriluzole did not show a statistical improvement for patients when compared to those on the placebo at the 48-week marker. There were some indications that the treatment could be somewhat effective on patients with early-stage Alzheimer’s. The company may pursue further study into patients in that category.
Vlad Coric, M.D., Biohaven CEO said, “Alzheimer’s is a devastating disease and we must continue to advance the science to improve treatment outcomes for the many patients who are in need. Our goal was to efficiently assess whether troriluzole could benefit patients relatively late in the disease process with mild-to-moderate AD. This study was well-conducted but unfortunately it is clear from this preliminary analysis that troriluzole is not efficacious as a symptomatic treatment in a mixed population of patients with mild and moderate AD.”
The past 12 months have been one disappointment after another for troriluzole. Last February came the announcement that the prodrug did no better than the placebo in treating GAD. In June the company released results for the Phase II/III program for obsessive-compulsive disorder. Although there was consistent numerical improvement over placebo, it did not meet the primary outcome measures at week 12. Nonetheless, the company plans to advance the drug to a full Phase III program with a larger sample size and a higher-dose arm.
The trial found that troriluzole was well tolerated at the 280 mg dose. It’s a prodrug formulation of riluzole, which inhibits synaptic levels of glutamate. Glutamate dysfunction is a feature of AD. The prodrug formulation makes it easier to take than the drug riluzole. Riluzole is taken twice a day on an empty stomach, while troliluzole is only needed once daily and is unaffected by food. As a prodrug, it also causes a lower burden on the liver to process.
Irfan Qureshi, M.D., Vice President of Neurology at Biohaven, stated “We are extremely grateful to the community of patients and researchers who participated in this program and sincerely appreciate all that they have done to see this study through to completion. The topline data we released today only represent the early analyses that are available and multiple analyses including important biomarker data are expected in the near term. Additional planned analysis of the clinical outcome scales and biomarkers will help to inform our long-term development plans for troriluzole in AD.”
