Trial of Ptau Therapy ACI-35.030 Launched, Positive Data on AMT-130 in Huntington Disease, CHMP Recommends EU Approval of Omeveloxolone

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

AC Immune SA recently announced the launch of its phase 2b trial, dubbed ReTain, to assess the efficacy and safety of ACI-35.030, an investigational anti-phosphorylated tau (pTau) immunotherapy in development for patients with Alzheimer disease (AD). The registration-enabling trial is designed to test the hypothesis that ACI-35.030 has a disease-modifying effect that can delay or prevent the onset of cognitive impairment or other clinical symptoms in individuals with preclinical AD. The phase 2b ReTain trial, a multicenter, double-blind, placebo-controlled study, is expected to include 500 patients with preclinical AD who are randomly assigned 1:1 to a single dose of ACI-35.030 or placebo for a maximum of 4 years. Change in Preclinical AD Cognitive Composite 5 (PACC-5) score, which combines tests of episodic memory, timed executive function, and global cognition, as the primary end point.

New 30-month interim data from the ongoing U.S. and European multi-center phase 1/2 clinical trials assessing AMT-130 (uniQure), an investigational gene therapy for Huntington disease (HD), showed evidence of potential dose-dependent clinical benefit relative to the nonconcurrent criteria-matched natural history. The combined data presented from these trials has a cut-off date of September 30, 2023, and does not include efficacy and biomarker data from the control patients who crossed over to treatment. Among 39 patients in the both trials, treatment with AMT-130 resulted in a 0.39-point difference on composite Unified Huntington’s Disease Rating Scale (cUHDRS) at 30 months and a 1.24-point difference at 18 months for the low- and high-dose, respectively.

According to an announcement from Biogen, the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has given a positive recommendation for omaveloxolone to be the first approved agent to treat patients with Friedreich ataxia (FA) in the EU. The therapy, which has already received FDA-approval, will continue to be marketed as Skyclarys if approved by the EMA. The European Commission (EC) will review omaveloxolone with a final decision expected in the first quarter of 2024. If approved, the therapy will be indicated for adults and adolescents with FA aged 16 years and older.

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