Polyunsaturated Fats and ALS Disease Progression, CAP-1002 Improves Motor Function in DMD, Lecanemab Granted Traditional Approval

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

Using participants from the EMPOWER clinical trial, recently published findings in Neurology showed that higher levels of alpha-linolenic acid (ALA) were associated with longer survival and slower functional decline in individuals with ALS. Prior to the study, limited research had suggested that higher dietary intake and plasma levels of polyunsaturated fatty acids (PUFAs), in particular ALA, are associated with a lower risk of ALS. In the most recent analysis, after adjusting for age, sex, and baseline ALS Functional Rating Scale (ALSFRS-R) score, the HR for death comparing the highest and lowest quartile of ALA was 0.50. The study had several strengths, including the large number of patients with ALS with similar disease duration and progression. Investigators also had access to comprehensive data on demographic characteristics and clinical variables relevant for the diagnosis, prognosis, and progression of ALS.

Newly announced data from the open-label extension (OLE) period of the phase 2 HOPE-2 trial (NCT03406780) showed continued improvement of patients’ left ventricular ejection fraction (LVEF) after 2 years of treatment with CAP-1002 (Capricor), a cell therapy in development for Duchenne muscular dystrophy (DMD). Following the double-blind, placebo-controlled period, participants entered the OLE where they received CAP-1002 at 150 million cells per infusion every 3 months over the course of 24 months. In addition to showing improvement in LVEF, which was indicative of cardiac function, treated patients showed statistically significant benefits in Performance of the Upper Limb (PUL v2.0) scale after 2 years in comparison with the original rate of decline from the placebo group at 1 year. The therapy was well-tolerated with a safety profile that was consistent to previous observations.

According to an announcement, the FDA has granted traditional approval to Eisai’s Alzheimer disease (AD) medication lecanemab (Leqembi), a major step in enabling broader access and coverage of the therapy to the aging population. It originally received FDA approval under the accelerated approval pathway in January. The decision was expected after a recent meeting between the FDA’s Peripheral and Central Nervous System Drugs Advisory Committee in which the panel voted unanimously in favor of the agent. The committee was asked to vote on whether the results of the phase 3 Clarity AD trial, the confirmatory study, verified the clinical benefit of lecanemab for the treatment of AD. Lecanemab is only the second early AD treatment that has received FDA approval in the past 20 years following the contentious approval of aducanumab (Aduhelm; Eisai/Biogen) in 2021, which remains under conditional approval.

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