Valtoco Gains Expanded Indication, FDA Approves Digital Therapeutic for Migraine, Phase 1 Study of AMX0114 in ALS Begins

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

The FDA has approved an expanded indication for diazepam nasal spray (Valtoco; Neurelis), now allowing its use in children ages 2 to 5 for the treatment of seizure clusters, also known as acute repetitive seizures. Previously approved for patients aged 6 and older, this update broadens access to an important rescue therapy for younger children experiencing recurrent seizures. Valtoco, a nasal spray formulation of diazepam, was originally approved in 2020 under the 505(b)(2) regulatory pathway as the first intranasal rescue formulation for patients with seizure clusters aged 6 and older.2 Through the intranasal route of administration, it allows the treatment to be rapidly absorbed through the highly vascularized nasal mucosa, as well as avoidance of first-pass metabolism. For years, diazepam nasal spray has been considered a faster and easier option in emergency seizure situations, especially in community or home settings.

According to a new announcement, the FDA has made a major decision, approving Click Therapeutics’ CT-132 as the first prescription digital therapeutic for the preventive treatment of episodic migraine in adults. Approved through the De Novo pathway, the therapy is indicated for patients aged 18 years and older, intended for adjunctive use alongside acute and/or other preventive treatments for migraine. The medication’s approval was primarily based on data from 2, double-blind, decentralized randomized trials: the phase 3 ReMMi-D trial (NCT05853900) and the ReMMiD-C bridging study (NCT06004388). In its label, CT-132 carries no contraindications, and is not intended to be used as a standalone therapy. CT-132 does not replace or substitute other migraine treatments, and patients who opt for this digital therapeutic should continue their current treatment as directed.

Dosing has begun for a new phase 1, placebo-controlled study (NCT06665165) testing Amylyx Pharmaceuticals’ AMX0114, investigational antisense oligonucleotide (ASO) targeting calpain-2 (CAPN2), in patients with amyotrophic lateral sclerosis (ALS). Dubbed LUMINA, the randomized study is expected to have early cohort data expected this year.The trial, expected to enroll 48 patients with ALS across North America, randomly assigns participants 3:1 to receive AMX0114 or placebo by intrathecal administration once every 4 weeks for a total of up to 4 doses. Primarily designed to assess safety, tolerability, pharmacokinetics, and pharmacodynamics of the agent, LUMINA also includes other secondary outcomes like change in calpain-2 levels, neurofilament light (NfL) levels, and other pharmacodynamic biomarkers of ALS.

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