Donanemab Shows Decline in Alzheimer, Pfizer DMD Study Launched, REM Not Correlated With Seizures


Neurology News Network for the week ending January 16, 2021.

This week Neurology News Network detailed the phase 2 TRAILBLAZER-ALZ study of donanemab which showed slower decline in cognition for Alzheimer disease, the launch of the phase 3 CIFFREO study of Pfizer's PF-06939926 in Duchenne muscular dystrophy, and a study evaluating the correlation between rapid eye movement sleep and onset of seizures.

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.

Eli Lilly has announced results from its randomized, place-controlled, double-blind, multi-center phase 2 TRAILBLAZER-ALZ study investigating donanemab for slowing the progression of Alzheimer disease. When compared to placebo, patients treated with donanemab showed a significantly slower decline in cognition and daily function.Patients treated with donanemab declined 32% slower compared to placebo as assessed by the Integrated Alzheimer's Disease Rating Scale (iADRS) from baseline to 76 weeks. Donanemab also showed consistent improvements in secondary end points measuring cognition and function as compared to placebo, but not all of these endpoints reached statistical significance. Lilly added that the full results of the trial will be presented at a future medical congress and published in a peer-reviewed clinical journal.

The phase 3 CIFFREO study, which will evaluate the efficacy and safety of investigational gene therapy candidate PF-06939926 in boys with Duchenne muscular dystrophy, has just launched, according to an announcement from Pfizer, the agent’s manufacturer. On December 29, 2020, the first patient was dosed in the double-blind, placebo-controlled study, which is expected to enroll 99 ambulatory male patients, ages 4 through 7, across 55 clinical trial sites in 15 countries. Researchers will use change from baseline in the North Star Ambulatory Assessment (NSAA) at 1 year as the primary end point of the study.In May 2017, the investigational agent received orphan drug and rare pediatric disease designations. Three years later, in October 2020, PF-06939926 received fast track designation from the FDA.

Data from a small sample study suggested that there is no correlation between rapid eye movement (REM) sleep onset seizures and the incidence of sudden unexpected death in epilepsy (SUDEP).Among a cohort of 26 patients who died of SUDEP, the frequency of seizures arising from REM sleep was 1.9%, comparable to the 1% frequency previously reported. The investigators noted that the lack of association may be in part because of the cohort was too small to find such association. The results were presented at the American Epilepsy Society (AES) Virtual Meeting. Though the percentage of total seizures arising from REM was low, there were 2 patients (7.7%) who recorded REM-onset seizures. Each of the 2 patients who recorded REM-onset seizures had a single seizure arising from REM sleep and both had additional seizures arising from other states. The study did have notable limitations, including the impact that inpatient hospitalization and antiepileptic medication taper on the natural sleep pattern, as well as the lack of control patients in this sample.

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