Positive Results of STK-001 in Dravet, Zavegepant Meets Primary End Points, FDA Accepts NDA of Zilucoplan for Myasthenia Gravis

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

Recent news from the ongoing interim analysis of the phase 1/2a MONARCH study reported positive safety in the STK-001 data in children and adolescents with Dravet syndrome (DS). The results showed that single and multiple doses of STK-001 up to 45 mg were found to be well-tolerated for patients with DS.All told, 74% of 27 patients treated with 3 doses of STK-001 demonstrated reductions observed from baseline in convulsive seizure frequency. Specifically, investigators observed median reductions of 55% in the 45mg, 20% in the 30 mg, and 41% in the 20 mg cohorts from day 29 of their first dose to 3 months after receiving their last dose. Treatment-emergent adverse events (TEAEs) in relation to the study drug were observed in 15 out of the 55 patients (27%). The adverse events were mild to moderate in severity with the study drug and none of the TEAEs patients experienced led to withdrawal from the drug.

Recently, positive findings from the phase 2/3 trial of zavegepant nasal spray (Biohaven), a calcitonin gene-related peptide (CGRP) receptor antagonist currently under review for the acute treatment of migraine, were published in Headache. All told, zavegepant 10 and 20 mg were more effective than placebo on the coprimary end points of pain freedom at 2 hours postdose and freedom from the most bothersome symptom (MBS) at 2 hours postdose. With a PDUFA date set for Q1 2023, zavegepant aims to become the first FDA-approved mediation to target CGRP in an intranasal formulation, giving patients a new treatment option that provides ultra-rapid pain relief. Although the 10 and 20 mg doses of zavegepant proved significance for the coprimary end points, the findings for the 5 mg dose were not significant. In terms of secondary efficacy end points, 53.6% of those on placebo had pain relief at 2 hours postdose vs 60.6% and 61.2% for the zavegepant 10- and 20-mg groups, respectively.

According to an announcement, the FDA has accepted the new drug application (NDA) of UCB Pharma’s investigational, subcutaneously delivered agent zilucoplan for the treatment of adults with AChR-Ab+ generalized myasthenia gravis (gMG). The news comes shortly after the European Medicines Agency validated the Marketing Authorization Application (MAA) of zilucoplan for the same indication.Zilucoplan has its NDA supported by data from the pivotal phase 3 RAISE study, in which treatment with the agent at doses of 0.3 mg/kg daily resulted in meaningful and statistically significant improvements in key gMG-specific outcomes relative to placebo after 12 weeks of treatment. All told, zilucoplan, a self-administered complement component 5 inhibitor, met its primary end point in Myasthenia Gravis Activities of Daily Living (MG-ADL) score at week 12, with placebo-corrected mean improvements of 2.09 points. The EMA’s validation of the MAA for zilucoplan formally recognizes that it is complete and the formal review process by the EMA’s Committee for Medicinal Products for Human Use can begin. Zilucoplan was previously granted orphan drug designation by the FDA in 2019 and by the European Commission in 2022. The company did not include projected final review dates for which a decision on the agent may be made.

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