FDA Approves Intrathecal Version of Zolgensma, Ozempic Falls Short in EVOKE Trials, Asundexian Meets End Point in Stroke Prevention Trial

WATCH TIME: 4 minutes

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

According to a new announcement, the FDA has approved an intrathecal formulation of Novartis’ onasemnogene abeparvovec-brve, a gene therapy indicated for children, teens, and adults with spinal muscular atrophy (SMA). Marketed as Itvisma, the medication becomes the first intrathecally administered treatment for SMA, giving patients a new optional route of administration. The gene therapy, approved for a broad SMA population, is designed to address the genetic root cause of SMA by providing a functional copy of the human SMN1 gene to improve motor function through sustained survival motor neuron (SMN) protein expression. An adeno-associated, virus vector-based medication, onasemnogene abeparvovec was originally approved in 2019 as a single intravenous infusion for a broad range of SMA.

Topline results from the randomized, double-blinded phase 3 EVOKE (NCT04777396) and EVOKE+ (NCT04777409) studies showed that semaglutide (Ozempic; Novo Nordisk), a glucagon-like peptide-1 (GLP-1) receptor agonist, did not demonstrate superiority over placebo in reducing disease progression in patients with early-stage symptomatic Alzheimer disease (AD), despite improvements observed in AD-related biomarkers. The 2 trials randomized a total of 3808 adults aged 55 to 85 years with mild cognitive impairment or mild dementia because of AD to investigate the efficacy and safety of oral semaglutide compared with placebo plus standard of care. Findings, to be presented at the upcoming 2025 Clinical Trials on Alzheimer’s Disease (CTAD) Conference, held December 1-4, in San Diego, California, showed that semaglutide was well tolerated and consistent with prior safety data. Based on efficacy results observed in the overall study population, Novo Nordisk noted in its announcement that the 1-year extension period in the trials will be discontinued.

According to a new announcement, Bayer’s asundexian, an investigational factor XIa inhibitor, met its primary end point in the phase 3 OCEANIC-STROKE trial, further supporting its efficacy and safety as a treatment for secondary stroke prevention. Using these notable data, the company expects to engage with regulatory authorities in preparation for a submission of asundexian in the near future. OCEANIC-STROKE was a multicenter, international, randomized, placebo-controlled double-blind, event-driven phase 3 trial testing whether adding asundexian to standard anticoagulant therapy reduces the risk of recurrent stroke in patients who recently had a non-cardioembolic ischemic stroke or high-risk transient ischemic attack (TIA). Overall, the trial featured more than 12,300 patients, with main study results expected to be presented at an upcoming scientific congress.