Positive Phase 1 Study of Monepantel, CRL Given to GA Depot, FDA Rejects Tasimelteon sNDA

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Welcome to this special edition of Neurology News Network. I’m Marco Meglio.

Newly announced findings from the phase 1 MEND study (NCT04894240) showed that monepantel (PharmAust), a veterinary drug, met its primary safety and tolerability end points, coupled with slowing in disease progression, in patients with motor neuron disease (MND)/amyotrophic lateral sclerosis (ALS). The trial featured 12 patients with MND/ALS who were split into either cohort 1 (2 and 6 mg/kg dose levels) or cohort 2 (4 and 10 mg/kg dose levels) of monepantel for a 24-hour single-dose pharmacokinetic study, followed by a 4-week repeated escalating dose study. For all 12 patients, the estimated rate of decline was –0.74 in ALS Functional Rating Scale-Revised (ALSFRS-R) points per month or a 39% slowing in ALSFRS-R decline when compared with the PRO-ACT database, an external control cohort. All patients completed the study, with no dose limiting toxicities experienced.

According to an announcement, the FDA has issued a complete response letter (CRL) for Viatris and Mapi Pharma’s investigational agent GA Depot, a long-acting, once-monthly glatiramer acetate (GA Depot) solution as a potential treatment for patients with relapsing forms of multiple sclerosis (MS). The companies are reviewing the content of the CRL and will be determining the next steps to advance this treatment for patients with MS. The agency originally accepted the new drug application (NDA), which comprised of data from a phase 3, multinational, double-blind, placebo-controlled study (NCT04121221) of GA Depot in November 2023. In the trial, treatment with GA Depot led to statistically significant reduction of annualized relapse rate (ARR) relative to placebo (P = .0066) over a 52-week period. Findings from the study were strengthened by MRI end points as well.

According to a recent announcement, the FDA rejected Vanda Pharmaceuticals' supplemental new drug application (sNDA) for tasimelteon (Hetlioz) in the treatment of insomnia. In July 2023, the FDA assigned a PDUF date of March 4, 2024, to make a decision on tasimelteon for insomnia, a condition characterized by difficulties with sleep initiation. A month prior, on February 4, 2024, the FDA identified deficiencies regarding labeling and post-marketing requirements. As a result of these deficiencies, the FDA issued a Complete Response Letter (CRL) stating the submission of tasimelteon could not be approved in the treatment's current form.

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