FDA Approves Vamorolone for DMD, Tau Agent BIIB080 Shows Promising Results, Subcutaneous Lecanemab More Effective in Amyloid Removal

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

According to an announcement, the FDA has approved Santhera Pharmaceuticals' investigational agent vamorolone (now marketed as Agamree) oral suspension 40 mg/mL for the treatment of patients with Duchenne muscular dystrophy (DMD) aged 2 years and older. The therapy is a first-in-class dissociative steroid, that aims to retain the anti-inflammatory activity of corticosteroids while decreasing the deleterious adverse events (AEs). Data from phase 2b data from the VISION-DMD study was the basis for the approval, in which the agent met its primary end point of superiority of change in time to stand test (TTSTAND) velocity relative to placebo. The primary end point, superiority in change of TTSTAND velocity, was represented by a difference of 0.06 (95% CI, 0.02-0.10) rises per second from baseline in the treated group.

New data from the phase 1b trial assessing BIIB080 (Biogen), an investigational antisense oligonucleotide (ASO) therapy targeting tau, showed numerical differences favoring the therapy in high-dose groups on multiple cognitive and functional scales at completion of the multiple ascending dose (MAD) and long-term extension (LTE) in patients with mild Alzheimer disease (AD). These findings support further investigation of BIIB080’s potential clinical efficacy and safety in patients with mild cognitive impairment (MCI) because of AD or mild AD in the ongoing CELIA phase 2 trial. The data showed differences in baseline clinical scores between treated groups, low-dose groups and high-dose groups, compared with the pooled placebo. At week 37, end of the MAD, favorable differences were reported in the high-dose groups on the Mini-Mental State Exam (MMSE) cognitive scales and Functional Activities Questionnaire (FAQ), and Repeatable Battery for the Assessment of Neuropsychological Status (RBANS) scales compared with the placebo. Notably, in the low-dose groups, unfavorable group averages were observed relative to the placebo.

In a late breaking symposium presented at the 2023 Clinical Trials on Alzheimer’s Disease (CTAD) conference held October 24-27, in Boston, Massachusetts, data from an open-label extension (OLE) of the pivotal phase 3 Clarity AD study (NCT03887455) showed that a new novel subcutaneous form of lecanemab (Leqembi; Eisai) resulted in greater amyloid plaque removal than biweekly intravenous (IV) administration. The company plans to submit a biologics license application for the subcutaneous formulation to the FDA by March 31, 2024. In a preliminary 6-month analysis of the OLE, data from a subgroup of patients showed a 14% increased amyloid plaque removal with the subcutaneous method than IV administration, the administration for which it was FDA-approved under earlier this year. Presented by Reisa Sperling, MD, a neurologist at Brigham and Women’s Hospital, Harvard Medical School, the data comprised of 72 patients with early Alzheimer disease (AD) who received lecanemab for the first time in a subcutaneous way and 322 patients who received IV lecanemab in the Clarity AD core study followed by subcutaneous administration in the substudy.

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