HNSA-5487 Significantly Reduces IgG Levels, FDA Removes Hold on AOC 1001, DMD Gene Therapy Shows No Impact on Cardiac Outcomes

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

Recently announced results from a 12-month follow-up analysis of the NICE-1 trial, a first-in-human study of HNSA-5487 (Hansa Biopharma), showed that treatment with the investigational agent resulted in highly robust reduction of immunoglobulin G (IgG) levels by more than 95% within a few hours post treatment. All told, these positive data open the possibilities for the company to advance the treatment in chronic autoimmune diseases like myelin glycoprotein antibody disease (MOGAD), neuromyelitis optica (NMO), and myasthenia gravis (MG). NICE-1 was a double-blind, placebo-controlled trial that included 36 healthy adult participants to test the safety, tolerability, pharmacokinetics, and pharmacodynamics of HNSA-5487, a next-generation IgG-cleaving enzyme. In the study, treatment with single ascending doses of the investigational agent were considered safe and well tolerated, with no serious adverse events (AEs) observed. In addition, the therapy demonstrated lower pre-treatment anti-drug antibody (ADA) levels and significant reduced ADA responses when compared with imlifidase, Hansa’s first-generation IgG-cleaving enzyme.

According to a recent announcement, the FDA has removed a partial clinical hold on Avidity Biosciences’ investigational agent AOC 1001 in development for patients with myotonic dystrophy type 1 (DM1). The hold, originally placed in September 2022, was related to a serious adverse event reported in a single patient treated in the 40-mg arm of the phase 1/2 MARINA trial (NCT05027269).At the time of the partial hold, nearly 40 of the anticipated 44 participants had been enrolled in MARINA and its open-label extension (OLE); however, all participants, whether on AOC 1001 or placebo, were instructed to continue dosing as planned. The company also noted that the clinical hold was not going to impact those who choose to enter the OLE.

New findings from Part 1 of the phase 3 EMBARK study (NCT05096221) revealed that delandistrogene moxeparvovec (Elevdys; Sarepta), an FDA-approved gene therapy for Duchenne muscular dystrophy (DMD), did not impact cardiac measures relative to placebo over a 52-week treatment period. After 1 year of treatment, investigators observed no adverse cardiac effects with the therapy, supporting its previously demonstrated manageable safety profile. Presented at World Muscle Society 2024, a week 52 analysis of cMRI left ventricular parameters, including ejection fraction, end diastolic volume, end systolic volume and mass, revealed no relevant differences between those on the gene therapy vs placebo in the sub-study population. An analysis of the global circumferential strain of the left ventricle also revealed no relevant differences between the active (mean change, –0.37) and placebo (mean change, 0.21) groups at that time point.

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