More bad news has come down for Biohaven’s small molecule troriluzole. After the FDA decided to convene an advisory committee to consider the asset’s potential approval in a rare disease, pushing the PDUFA date to the fourth quarter, troriluzole has now failed a phase 3 trial in obsessive-compulsive disorder (OCD).
As a result, the Connecticut-based biotech is ending troriluzole’s development in OCD, according to Biohaven’s second-quarter earnings release, to instead direct resources to other programs. The company plans to share the results of the trial at an upcoming conference.
Troriluzole is a prodrug of riluzole, which was approved in 1995 as the first drug for the treatment of amyotrophic lateral sclerosis.
Troriluzole also failed to meet the primary outcome of a prior phase 2/3 OCD trial in 2020 and has amassed a string of other clinical collapses since then. The molecule followed up with a 2021 failure in a phase 2/3 trial in Alzheimer’s disease and even failed its first phase 3 trial in spinocerebellar ataxia, the indication it is now being considered for approval in by the FDA.
After refusing to review troriluzole in 2023, the FDA decided to give the asset another chance after a second phase 3 test showed more promising signs that the molecule could significantly slow the progression of the rare disease. In May, Biohaven revealed that the FDA had decided to assemble an adcomm for the asset, with the agency’s decision now expected in the fourth quarter of 2025.