PHOENIX Study Meets Enrollment, Atogepant and Ubrogepant Safe, ABATE Trial Highlights Targeted Response of Alzheimer Vaccine

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

Months after AMX0035 (Relyvrio; Amylyx Pharmaceuticals) gained FDA approval as a treatment for patients with amyotrophic lateral sclerosis (ALS), the company has announced it has successfully completed enrollment in its pivotal phase 3 PHOENIX study (NCT05021536), a 48-week trial assessing the agent in 664 individuals with ALS.1 Topline results are expected in 2024. PHOENIX, a randomized, placebo-controlled trial, is expected to span across approximately 65 sites in the US and Europe, assessing the safety and efficacy of AMX0035, a coformulation of sodium phenylbutyrate-taurursodiol. In the study, patients will be randomly assigned 3:2 to AMX0035 or placebo for a 48-week treatment period, and will be assessed on change in the primary outcome of ALS Functional Rating Scale-Revised (ALSFRS-R). Other primary outcome measures include safety, as described by number of adverse events (AEs), and number of participants able to remain on study drug until planned discontinuation.

Recently published findings from a phase 1b, multicenter, open-label, fixed-sequence study showed that the combination approach of atogepant (Qulipta; AbbVie) and ubrogepant (Ubrelvy; AbbVie) at the highest FDA-approved dose strengths, was safe for patients with migraine, with no new concerns identified. Atogepant, an oral calcitonin gene-related peptide (CGRP) receptor antagonist, gained approval as a preventive treatment of episodic migraine in September 2021, while ubrogepant, another CGRP receptor antagonist was approved for the acute treatment of migrane in December 2019. Led by Andrew Blumenfeld, MD, director of the Headache Center of Southern California, the trial’s primary objective was to assess pharmacokinetic interactions of the highest approved dose of each therapy, with ubrogepant administered on a fixed-dose schedule every 3 days.

Initial interim results announced from the phase 1b/2 ABATE clinical trial showed that the low dose group of ACI-24.060 (AC Immune), an antiamyloid vaccine for patients with Alzheimer disease (AD), elicited a targeted response and was generally well tolerated with no safety concerns observed. As a result, dosing in ABATE’s second, higher dose AD cohort has now begun. In the multicenter, adaptive, placebo-controlled study, findings from the first cohort of patients with prodromal AD showed that low-dose ACI-24.060 could elicit an anti-amyloid-ß response as soon as week 6, or 2 weeks after the second injection. Following these results, the company will also begin to screen individuals with Down syndrome for part 2 of the adaptive study.

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