Intrathecal Zolgensma Meets Primary End Point, Deramiocel BLA Submitted, Imlifidase Improves Function in Guillain-Barré Syndrome

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

In newly announced data from the phase 3 STEER study (NCT05089656), results revealed that OAV101IT (Novartis), an intrathecal (IT) investigational formulation of an approved gene therapy for spinal muscular atrophy (SMA), met its primary end point among treatment-naïve patients with the disease. Novartis plans to share these results with regulatory agencies with the goal of bringing this IT formulation to market.In STEER, more than 100 patients with SMA Type 2 were randomly assigned to receive OAV101 by IT injection or to receive a sham procedure for a 52-week period, followed by a crossover phase. All told, treatment with the agent met its primary end point, demonstrating enhancements in total Hammersmith Functional Motor Scale-Expanded (HFMSE) scores, considered a gold standard for SMA-specific assessment of motor ability and disease progression. OAV101 IT demonstrated a favorable safety profile, with similar rates of adverse and serious adverse events between arms, and the most common events being upper respiratory tract infection, pyrexia, and vomiting.

Capricor Therapeutics has recently completed the submission of its biologics license application (BLA), seeking full approval for its investigational agent deramiocel as treatment for patients with Duchenne muscular dystrophy (DMD) cardiomyopathy, along with a request for priority review. Completed in late December 2024, the full submission of the rolling BLA was supported by data from the company’s phase 2 HOPE-2 (NCT03406780) and HOPE-2 open label extension (OLE) trials which compared natural history data from a large cohort of patients.HOPE-2, which enrolled originally 26 patients from 2018 to 2020, had original data published in The Lancet in 2022. In total, 8 patients were randomly assigned to deramiocel and 12 patients to placebo, with 6 patients not randomized because of screening failure. Overall, among those who had a post-treatment PUL1.2 assessment, the mean 12-month change from baseline in mid-level elbow PUL1.2 favored deramiocel over placebo.

Newly announced data from 15-HMedldeS-09, a single-arm phase 2 study (NCT03943589) of imlifidase (Hansa Biopharma), showed that treatment with the immunoglobulin (IgG)-cleaving enzyme resulted in improved functional status among patients with Guillain-Barré syndrome (GBS). The company plans to publish data from the study, as well as from an indirect comparison analysis using sample data from the International Guillain-Barré Syndrome Outcome Study (IGOS).In GBS, it is known that reducing IgG levels can deplete pathological antibodies, potentially halting disease progression, accelerating recovery, and lessening severity. In the study, patients with severe GBS treated with a single dose of imlifidase at 0.25 mg/kg with added intravenous immunoglobulin (IVIg) demonstrated rapid growth in recovery of muscle strength, fast return to independently walking, and a median time to independently walk—defined by Guillain-Barré Syndrome Disability Scale (GBS DS) scores of 2 or less—by 16 days of treatment.

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