STK-001 Shows Disease-Modifying Impacts in Dravet, FDA Approves Ravulizumab, Satralizumab Falls Short in Myasthenia Gravis

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

New data from 2 open-label phase 1/2 studies and their open-label extensions showed that treatment with STK-001 (Stoke Therapeutics), an investigational antisense oligonucleotide (ASO), resulted in substantial and durable reductions in convulsive seizure frequency among patients with Dravet syndrome (DS). These improvements, coupled with benefits in measures of cognition and behavior, further demonstrate the potential disease-modifying impacts of this agent in DS. Stoke reported on data from MONARCH (NCT04740476) and ADMIIRAL (NCT04442295), 2 open-label studies of children and adolescents ages 2 to 18 with DS, confirmed by evidence of a genetic mutation in the SCN1A gene. The new analysis focused on 19 patients treated in the highest dose group (70 mg), with results showing a 43% (n = 8) median reduction in convulsive seizure frequency at 3 months after last dose with 1 dose of STK-001 and 85% (n = 10) reduction in 70 mg patients who received 2 or 3 doses.

The FDA has approved ravulizumab-cwvz (Ultomiris; Alexion), a terminal compliment C5 inhibitor, for the treatment of patients with anti-aquaporin (AQP4) antibody-positive neuromyelitis optica spectrum disorder (NMOSD). With the decision, ravulizumab-cwvz becomes the fourth approved therapy for this patient population, following behind eculizumab (Soliris; Alexion), inebilizumab (Uplizna; Horizon Therapeutics), and satralizumab (Enspryng; Genentech). In September 2023, the FDA issued a complete response letter to the company for the supplemental biologics license application (sBLA) of ravulizumab-cwvz for NMOSD. In its response, the agency requested modifications to enhance the Risk Evaluation and Mitigation Strategy program and did not need any additional analysis or reanalysis of the phase 3 CHAMPION-NMOSD study (NCT04201262), the supporting trial for ravulizumab-cwvz’s sBLA.

According to an update from Chugai Pharmaceutical, the company’s genetical recombination of satralizumab (Enspryng) demonstrated statistically significant data on the primary end point in the phase 3 LUMINESCE study (NCT04963270) of patients with myasthenia gravis (MG); however, the results did not reach the expected degree of clinical benefit. Detailed results are expected to be presented as an oral Emerging Science abstract at the upcoming American Academy of Neurology (AAN) Annual Meeting, held 13-18, in Denver, Colorado. The multicenter, global trial randomly assigned 188 adults and adolescents aged 12 years and older with MG to either satralizumab or placebo for 24 weeks. Change in total Myasthenia Gravis Activities of Daily Living (MG-ADL) score among those who were anti-acetylcholine receptor (AChR)-seropositive served as the primary end point. While the therapy did not meet the investigators anticipated benefit, it did show a safety profile that was consistent with its previous indication, neuromyelitis optica spectrum disorder.

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