Sumifilam Improves Alzheimer Biomarkers, Fenfluramine Phase 3 Results, Ozanimod Lowers ARR


Neurology News Network for the week ending September 26, 2020.

This week Neurology News Network covered sumifilam's ability to improve multiple validated Alzheimer disease biomarkers, the third phase 3 results of fenfluramine, and the interim analysis of ozanimod in the DAYBREAK study.

Welcome to this special edition of Neurology News Network. I’m Marco Meglio. Please excuse our appearance this week as a majority of the US workforce, including the NeurologyLive team, moves to working remote as we come together to help reduce the spread of the novel coronavirus.

Cassava Sciences announced that its lead drug candidate, sumifilam, significantly improved a panel of validated Alzheimer disease biomarkers in patients with a clinical diagnosis of AD in a phase 2b study, something that no drug has shown the ability to do to date. Patients with AD administered sumifilam in the randomized, placebo-controlled, double-blind, multicenter clinical study showed improvements in tests of episodic and spatial working memory compared with patients on placebo. Additionally, improvements in cognition correlated most strongly with decreases in P-tau181, of which an 8% to 11% reduction was recorded versus placebo.Jeffrey Cummings, MD, ScD, founding director, Cleveland Clinic Lou Ruvo Center for Brain Health, and Chambers Professor of Brain Science, University of Nevada–Las Vegas, said in a statement, “I am pleased to see early evidence of disease-modifying effects in patients with this investigational drug. The data appear to represent a step forward toward urgently needed treatments for Alzheimer disease.”

Zogenix has announced that the top-line results of its third phase 3 study—aptly named Study 3—of fenfluramine in patients with Dravet syndrome are positive, corroborating the findings of the 2 prior phase 3 trials, Study 1 and Study 2. Ultimately, the international, randomized, double-blind trial included 143 children and young adults and revealed that those treated with 0.7 mg/kg per day fenfluramine experienced a 64.8% greater reduction in mean monthly convulsive seizures compared to the placebo group. The median reduction in the monthly frequency of convulsive seizures was 73.7% for the fenfluramine group. Comparatively, the placebo group experienced a 7.6% monthly reduction. The study also expanded the evaluation of fenfluramine to more locales, this time including patients from Japan, in line with the company’s plans to submit a new drug application (J-NDA) there for the oral agent in 2021. The agent was approved in the US for the treatment of Dravet syndrome in June 2020.

Interim analysis from an open-label extension of patients with relapsing forms of multiple sclerosis enrolled in the DAYBREAK study showed an association between ozanimod and low annualized relapse rate and low counts of new or enlarging T2 and gadolinium-enhancing lesions over time. At Months 24 and 36, 79% and 75% of participants, respectively, were relapse free. A cohort of 2492 participants with a mean ozanimod exposure of 35.4 months revealed an adjusted ARR of 0.112. Regardless of parent-trial treatment group mean number of new/enlarging T2 lesions per scan at 24 hours was similar, as well as the mean number of GdE lesions at month 24 (range, 0.2–0.4). Confirmed disability progression was observed in 10.8% and 8.6% of participants at 3- and 6-months, respectively.

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