October Neurology News: Huntington Disease Gene Therapy, Remibrutinib in MS, and AI Medical Advice

In this update episode of Mind the Meds, host Erica Marini, PharmD, MS, BCPS, opens with regulatory news.

The FDA accepted uniQure’s application for AMT-130, a gene therapy for Huntington disease, under priority review. An advisory committee meeting is tentatively planned, and a decision is possible in May 2027. AV-1959R (Nuravax, Inc), an investigational amyloid-β vaccine, received fast track designation for preventing Alzheimer disease in cognitively unimpaired individuals with biomarker evidence of amyloid pathology. FDA decisions are expected in November on asundexian (Bayer HealthCare), an oral factor XIa inhibitor for secondary stroke prevention, and cemdisiran (Regeneron), a C5-targeting small interfering RNA for generalized myasthenia gravis. Marini also notes the $5.6 billion acquisition of Option Care, a major infusion provider. The deal is expected to close in early 2027 and could affect outpatient neurology pharmacy teams.

On the clinical side, Bristol Myers Squibb reported the death of a patient with multiple sclerosis after its chimeric antigen receptor (CAR) T-cell trials were halted. In the phase 3 REMODEL-1 (NCT05147220) and REMODEL-2 (NCT05156281) trials, the Bruton tyrosine kinase inhibitor remibrutinib (Rhapsido; Novartis) reduced annualized relapse rates by approximately 35% and 31.2% vs teriflunomide (Aubagio; Genzyme Corp), with no liver safety concerns. However, its effect on 3-month confirmed disability progression was not statistically significant. Other studies found:

• a marginal, likely not clinically relevant, difference in relapse rates between ofatumumab (Kesimpta; Novartis) and ocrelizumab (Ocrevus; Genentech);
• a higher risk of inflammatory bowel disease with rituximab (Rituxan; Genentech, Biogen; number needed to harm, 131 at 5 years);
• a negative phase 2 HIMALAYA trial (NCT05237284) of SAR443820 in amyotrophic lateral sclerosis;
• a higher risk of spontaneous abortion with early pregnancy exposure to anti-CGRP monoclonal antibodies than with propranolol;
• and positive topline phase 3 results for solengepras (Cerevance), which reduced daily off time in Parkinson disease.

Marini closes with a New York Times report on a woman with epilepsy who died after following ChatGPT’s plan to stop her antiseizure medication. The chatbot also undermined her trust in her physician and dismissed her symptoms as she tapered off. That model has since been taken offline. Marini says she hopes for regulation and urges pharmacists to make themselves available so patients have a real person to turn to with medication questions.