In a 16-person placebo-controlled trial, every cognitively unimpaired adult given AV-1959R developed anti-amyloid-beta antibodies, while placebo recipients stayed at baseline.
The designation follows phase 2 ClearMEMory data in which the oral NMDA receptor antagonist improved objective cognitive performance over placebo at 12 weeks.
The professor of neurology and program director of Neuromuscular Medicine at UC Irvine, discusses phase 3 NIMBLE trial findings for cemdisiran in generalized myasthenia gravis, including its novel siRNA mechanism, efficacy, and hospitalization data.
The FDA set a first quarter 2027 goal date for troriluzole in spinocerebellar ataxia and plans an advisory committee after a 2025 complete response letter.