Anti-Tau Therapy Posdinemab Gets Fast Track, Gene Therapy SGT-212 to Enter Friedreich Ataxia Trial, Zorevunersen Eyes Phase 3 Trial of Dravet

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

According to a new announcement, the FDA has assigned fast track designation to Johnson & Johnson’s investigational tau-directed monoclonal antibody posdinemab as a potential treatment for patients with early-stage Alzheimer disease (AD). Anti-tau therapies have shown promise in slowing disease progression by addressing tau pathology, a key driver of cognitive decline in later stages of the disease. Posdinemab is currently being investigated in the phase 2b AuTonomy study (NCT04619420), a double-blind, placebo-controlled, randomized, parallel-group, common-close study that it is the first to employ a plasma biomarker as a screening tool. The study, which assesses the effect of 2 doses of posdinemab (low or high dose) or placebo, every 4 weeks over a 104-week treatment period, includes patients with symptomatic AD who meet clinical and plasma phosphorylated (p-tau)217 criteria followed by intermediate levels of tau burden on tau PET.

The FDA has given clearance to Solid Biosciences’ investigational new drug application (IND) for SGT-212, an adeno-associated virus (AAV)-based gene therapy candidate for Friedreich ataxia (FA). A phase 1b, first-in-human, open-label, dose-finding trial featuring non-ambulatory and ambulatory adults with FA is expected to begin the second half of 2025. SGT-212 is designed to deliver full-length human frataxin (Fxn) via a dual route of administration: intradentate nucleus (IDN) infusion, using an MRI-guided device, followed by an intravenous (IV) infusion to increase therapeutic Fxn levels in the cerebellar dentate nuclei and in the cardiomyocytes, respectively. A unique agent, SGT-212 is geared towards treating both the neurologic and systemic clinical manifestations of FA to address the full spectrum of disease progression.

Following successful conversations and alignment with regulatory agencies, Stoke Therapeutics has announced the design of its phase 3 registrational trial, dubbed EMPEROR, that will test the effects of zorevunersen, an investigational antisense medicine, in children and adolescents with Dravet syndrome (DS). Expected to initiate in mid-2025, the study will include 150 patients with DS who have a confirmed variant in the SCN1A gene not associated with a gain of function. The global study, which spans across the United Kingdom, the United States, European Union, and Japan, lasts a total of 60 weeks, and is comprised of an 8-week baseline period followed by a 52-week treatment period. Using reductions in major motor seizure frequency as the primary end point, the trial is expected to have data read out by the end of 2027, pending enrollment and study timelines. Following the double-blind portion, patients will be eligible to continue zorevunersen as part of an open-label extension study.

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