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Welcome to this special edition of Neurology News Network. Im Marco Meglio.

According to an announcement, Amylyx Pharmaceuticals will voluntarily discontinue AMX0035 (Relyvrio) and remove it from the market in the United States and Canada based onnegative topline datafrom its phase 3 PHOENIX trial (NCT05021536) that showed AMX0035 did not meet its primary end point of change in ALS Functional Rating Scale-Revised (ALSFRS-R).1,2AlthoughAMX0035will no longer be available for new patients with amyotrophic lateral sclerosis (ALS), those currently on therapy in the US and Canada who wish to stay on treatment and consult with their cliniciancan be transitioned to a free drug program. In PHOENIX, results showed no significant difference on ALSFRS-R between AMX0035-treated and placebo-treated patients over a 48-week treatment period (P = .667).

In a newly published first-in-human trial (NCT02362438), treatment with scAAV9/JeT-GAN, an investigational gene therapy administered directly into the spinal fluid, was well tolerated and showed signs of therapeutic benefit among children with giant axonal neuropathy (GAN). Published in theNew England Journal of Medicine,the investigators observed various rates of slowed motor function decline, with nearly half of the small cohort regaining sensory nerve response. scAAV9/JeT-GAN is a self-complementary adeno-associated viral (AAV) serotype 9 vector that carries a codon-optimized humanGANtransgene with expression controlled by the minimal synthetic recombinant JeT promoter consisting of 5 elements.

According to an announcement from Eisai and Biogen, the companies have officially submitted a supplemental biologics license application (sBLA) for a new monthly intravenous (IV) maintenance dosing for lecanemab-irmb (Leqembi), its FDA-approved therapy for early-stage Alzheimer disease (AD). Those whove already completed the biweekly IV initiation phase are now eligible to receive a monthly IV does that maintains effective drug concentration to sustain the clearance of highly toxic protofibrils. Lecanemab, a humanized immunoglobulin gamma 1 monoclonal antibody directed against aggregated soluble and insoluble forms of amyloid-, was approved in July 2023 in 100 mg/mL injections for IV use. In the newly submitted sBLA, the companies included data from Study 201 (NCT01767311), a phase 2 trial, and its open-label extension (OLE), as well as the phase 3 Clarity AD trial (NCT03887455), the study that lecanemab was traditionally approved on, and its OLE.

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Amylyx Pulls ALS Drug AMX0035, Gene Therapy Shows Promise in Giant Axonal Neuropathy, Eisai Submits sBLA for ... - Neurology Live