Phase 2 Trial of Alzheimer Psychosis Initiated, FDA Declines to Lift Hold on ENTR-601-44, Levetiracetam and Clobazam With Safety Warnings


Neurology News Network for the week ending December 1, 2023. [WATCH TIME: 4 minutes]

WATCH TIME: 4 minutes

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

In recent news, Acadia Pharmaceuticals announced a phase 2 trial program assessing ACP-204, an inverse agonist, as a potential treatment for patients with Alzheimer disease (AD) psychosis, a condition for which there is no FDA-approved medication. The initial phase 2 study is part of a larger phase 2/3 program that includes 2 other phase 3 studies of identical design. Randomized, double-blinded, and placebo-controlled in design, the phase 2 study will enroll approximately 318 patients with AD psychosis and follow them over a 6-week treatment period. Patients will receive either 30 or 60 mg of ACP-204 or placebo, with change on the Scale for the Assessment of Positive Symptoms-Hallucinations and Delusions (SAPS-H+D) subscales total score as the primary end point.

In recent news, the FDA declined to lift the clinical hold on Entrada Therapeutics' investigational new drug (IND) application for the phase 1 clinical trial of ENTR-601-44 in Duchenne muscular dystrophy despite the company providing additional information to the agency.1 The company noted that the information submitted to the FDA at least supported the initiation of a United Kingdom-based phase 1 clinical trial of ENTR-601-44 among healthy volunteers, which was announced months ago. ENTR-601-44, an exon 44 skipping oligonucleotide developed with Entrada’s Endosomal Escape Vehicle (EEV) platform, aims to target the underlying genetic cause of DMD to allow muscle cells to produce functional dystrophin. For context, roughly 7.5% of the DMD population are considered exon 44 skipping amenable.

The FDA has issued a warning for the use of antiseizure medicines levetiracetam (Keppra, Keppra XR, Elepsia XR, Spritam) and clobazam (Onfi, Sympazan), which can cause drug reaction with eosinophilia and systemic symptoms (DRESS), a rare but serious adverse effect. The reaction may start as a rash but can quickly progress, resulting in injury to internal organs, the need for hospitalization, and even death. As a result, the FDA is requiring new warnings about this risk to be added to the prescribing information and patient medication guides for these medicines. In the cumulative review of these therapies, the FDA observed several reports of serious cases of DRESS in pediatric patients and adults globally.

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