Welcome to this special edition of Neurology News Network. I'm Marco Meglio.
A clinical readout from the phase 3 REVITALYZ trial (NCT06525077) testing Avadel’s once-nightly sodium oxybate (Lumryz) treatment in patients with idiopathic hypersomnia (IH) is expected in Q2 2026, as the company recently announced completed enrollment for the study and its final patients. REVITALYZ, which began dosing in August 2024, is a double-blind, placebo-controlled, randomized withdrawal study of approximately 150 patients with IH who receive either once-nightly sodium oxybate or placebo for 42 weeks. The study’s primary end point, change in Epworth Sleepiness Scale (ESS) total score, will be assessed at 14 weeks. To date, there is only 1 FDA-approved option for IH: Jazz Pharmaceuticals’ Xywav, a combination of calcium, magnesium, potassium, and sodium oxybates, which gained greenlight in 2021.
In recent news, Continuem’s PIPE-307, an M1 receptor antagonist developed for patients with relapsing-remitting multiple sclerosis (RRMS), failed to meet its primary end point in the phase 2 VISTA trial (NCT06083753), a placebo-controlled trial testing the drug’s effect on remyelination. Although the study failed to achieve its prespecified end points, the company is still planning to take a more detailed look at the exploratory end points, as well as present and publish the findings at a later time in a peer-reviewed journal. VISTA, a double-blind, proof-of-concept trial, comprised 168 patients with RRMS testing 2 doses of PIPE-307 against placebo for a 30-week treatment period. The trial’s primary end point, change in binocular 2.5% low contrast letter acuity (LCLA), was ultimately not successful for the investigational agent.
Recently presented findings from the initial cohort of the phase 1 LUMINA trial (NCT06665165) showed that investigational AMX0114 (Amylyx Pharmaceuticals), was safe and led to no treatment-related serious adverse events (SAEs) in patients with amyotrophic lateral sclerosis (ALS). Based on these data, the company is expected to begin enrollment for the second cohort of participants in Canada later this month and in the U.S. in January. LUMINA, a double-blind, placebo-controlled, multiple-ascending dose study, tests the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) of AMX0114, an investigational antisense oligonucleotide (ASO) targeting calpain-2 (CAPN2). In recent years, Calpain-2 has emerged as a promising therapeutic target in ALS because its dysregulated activation contributes to motor neuron injury, axonal degeneration, and neuroinflammation that drive disease progression.
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