New Biohaven hits an old problem

Expectations were low for troriluzole in the movement disorder spinocerebellar ataxia given its phase 2 failure, but as this project is one of the few clinical-stage assets to be spun out into the new incarnation of Biohaven after this month’s deal with Pfizer the company surely needed a hit. Instead topline data from the phase 3 trial show a miss on the primary endpoint, change from baseline to week 48 on f-Sara, a scale that assesses gait and motor impairment. The company attributed the miss to milder than expected disease progression over the course of the study. Its attempt at a data dredge, claiming an advantage over placebo in patients with the SCA Type 3 genotype, came to nothing since this did not even meet the threshold for nominal significance. Biohaven intends to take these subgroup data to the FDA, but this seems a longer shot than ever; Mizuho analysts have removed all SCA sales from their model. Realistically the company’s value would now seem to rest on troriluzole’s other indication, obsessive compulsive disorder, as well as the remaining pipeline. The next major readout will come from verdiperstat in ALS, but according to Mizuho “expectations are currently low”.

New Biohaven's clinical pipeline
Product Mechanism  Status and indication
Troriluzole Glutamate modulator Ph3 in OCD, data poss Q2 2023 (previously failed ph2); failed ph3 in spinocerebellar ataxia May 2022 (previously failed ph2)
RG6206 (taldefgrobep
Myostatin (GDF-8) inhibitor Ph3 Resilient in spinal muscular atrophy, data poss H2 2023
Verdiperstat Myeloperoxidase inhibitor Ph2/3 Healey ALS platform trial*, topline data due H2 2022
BHV-1100 CD38-induced natural killer (iNK) cell therapy Ph1 in multiple myeloma, data due H2 2023
*Investigator-sponsored trial. OCD = obsessive compulsive disorder. ALS = amyotrophic lateral sclerosis. Source: Evaluate Pharma & company website.

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