PrimeC Performs Well in High-Risk ALS, Patient Death Reported in DAYLIGHT Trial, Donanemab AdComm Hearing Date Announced

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

Newly announced analyses from the phase 2b PARADIGM trial showed that treatment with PrimeC (NeuroSense), an investigational agent, resulted in statistically significant slowing of disease progression in individuals with high-risk ALS after 6 months of treatment. NeuroSense plans to utilize the new subgroup findings to help inform the design of its upcoming trial and increase the probability of success and cost-effectiveness. In this multinational, randomized, double-blind, placebo-controlled trial, 68 patients with ALS were included to assess efficacy and safety of PrimeC. At 6 months, high-risk patients in the pre-specified per protocol (PP) population, defined by the European Network for the Cure of ALS (ENCALS) Risk Factor as those with a higher risk for rapid disease progression, demonstrated a 43% difference compared with placebo on ALS Functional Rating Scale-Revised (ALSFRS-R), the primary end point. Overall, this translated to a 5.04-point difference in favor of PrimeC.

A patient in the phase 2 DALIGHT trial (NCT05429372) assessing Pfizer’s investigational gene therapy fordadistrogene movaparvovec (PF-06939926) in boys with Duchenne muscular dystrophy (DMD) has suddenly passed away, the company wrote in a statement. Pfizer is currently gathering information and are actively working with the trial site investigator to understand what happened. DAYLIGHT is a multicenter, single-arm study assessing the safety and tolerability of the gene therapy in a small cohort of 10 boys aged between 2 and 4 years old with DMD. The boy, whose age was not disclosed, had been on the therapy since early 2023. Following the patient death, Pfizer has also decided to pause dosing in the phase 3 CIFFREO crossover trial, and will work with regulators and the independent external Data Monitoring Committee as it learns more about the event.

Months after the original announcement, Eli Lilly has announced that the FDA’s Peripheral and Central Nervous System Drugs Advisory Committee (PCNS) will meet on June 10, 2024, to discuss the new drug application of donanemab, an investigational antiamyloid therapy in development for early-stage Alzheimer disease (AD). The committee will review data from the supportive phase 3 TRAILBLAZER-ALZ 2 trial (NCT04437511) to determine whether evidence is sufficient enough to warrant recommendation for approval. TRAILBLAZER-ALZ 2, a double-blind, placebo-controlled trial led by Daniel M. Skovronsky, MD, PhD, executive vice president of science and technology at Eli Lilly, enrolled 1736 participants across 8 countries to assess the efficacy and safety of donanemab in participants age 60-85 with early symptomatic AD.

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