Biohaven Ltd. recently announced positive results from a pivotal study, BHV4157-206-RWE, on the efficacy of troriluzole, for spinocerebellar ataxia (SCA). SCA is a progressive, inherited disorder characterized by the loss of motor control and increasing disability over time, with no currently approved treatments available.
Troriluzole is designed as a prodrug of riluzole, targeting the neurotransmitter glutamate. It increases the action of amino acid transporters that clear excess glutamate from nerve cells, helping to protect neurons and ease symptoms.
Troriluzole was administered orally at a dose of 200 mg once daily over the three-year study period.
The study focused on the change in patients’ modified functional Scale for the Assessment and Rating of Ataxia (f-SARA). This scale, used as the primary endpoint, measures patients’ motor function and coordination. Secondary endpoints included analyses of disease progression based on a worsening of two or more points on the f-SARA.
Troriluzole was tested against three external control groups: the US Clinical Research Consortium for the Study of Cerebellar Ataxia (CRC-SCA) as the US natural history control, the European SCA natural history study (EUROSCA) as the European Union (EU) natural history control and a combined US and EU control.
Results showed a statistically significant improvement in patients treated with troriluzole compared to all control groups. At the one, two and three-year marks, troriluzole demonstrated a 50 to 70 percent slower disease progression, equating to a 1.5- to 2.2-year delay in motor function decline. In the pooled analysis using both CRC-SCA and EUROSCA data, patients treated with troriluzole were 4.1 times more likely to avoid significant disease progression compared to those in the untreated control arms.
The trial used a real-world evidence approach, leveraging Phase III data from matched, untreated patients with SCA to create a comparison group. Propensity score matching ensured the external control groups were well-aligned with the troriluzole treatment group, enhancing the study’s validity.
Biohaven plans to submit a New Drug Application (NDA) to the US Food and Drug Administration (FDA) in the fourth quarter of 2024, leveraging the Orphan Drug and Fast-Track designations previously granted. This status may enable a priority review process, potentially making troriluzole available to patients in 2025 if approved.
Alongside troriluzole, the company is advancing other pipeline programs, including investigational treatments such as the Kv7 activator BHV-7000 for epilepsy and taldefgrobep alfa for spinal muscular atrophy, both in Phase III trials.
More recently, Biohaven announced a public offering of $250 million to support its ongoing corporate and research activities.
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