Phase 3 LAVENDER Trial Results Published, Protective and Risk Factors of Insomnia, Lecanemab Gets Positive FDA AdComm Review


Neurology News Network for the week ending June 17, 2023. [WATCH TIME: 3 minutes]

WATCH TIME: 3 minutes

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

Recently published in Nature Medicine, results from the pivotal phase 3 LAVENDER trial showed statistically significant differences between trofinetide (Daybue; Acadia Pharmaceuticals)and placebo on efficacy endpoints in patients with Rett syndrome between the ages of 5 to 20 years. These findings suggest trofinetide has the capability to modify the core symptoms consistent with the underlying pathophysiology of the disease. At week 12, the data demonstrated statistically significant improvement compared with placebo on both coprimary efficacy end points, as measured by the change from baseline in Rett Syndrome Behaviour Questionnaire (RSBQ) total score (P = .018) and the Clinical Global Impression-Improvement (CGI-I) scale score (P= .003). In March 2023, these results supported the FDA approval of the treatment, a novel synthetic analog of the amino‐terminal tripeptide of IGF-1 designed to potentially reduce neuroinflammation and support synaptic function.

In a recent population-based sample study, findings over the course of 5 years revealed a number of protective and risk factors, divided as predisposing or precipitating factors, that were significantly associated with insomnia in adults. As insomnia is a persistent condition with adverse health outcomes, these results provide new information that could be beneficial for preventing the onset and long-term occurrence of insomnia by targeting the specific precipitating factors and predisposing factors. In the survival analysis for discrete events, significant predisposing factors for insomnia included anxiety, depression, perceived stress, and number of negative life events. Also, perceived worse general health, pain, vulnerability to stress, and maladaptive emotional coping were significant predisposing factors for insomnia.

The FDA’s Peripheral and Central Drugs Advisory Committee has voted unanimously that the current data on lecanemab (Leqembi, Eisai), an antiamyloid therapy for early Alzheimer disease, is sufficient enough to warrant traditional approval by the agency. The therapy has already been conditionally approved under the accelerated approval pathway, with a PDUFA date of July 6, 2023, for traditional approval. Following the discussion topics, the panel was asked to vote on whether the results of the Clarity AD trial verified the clinical benefit of lecanemab for the treatment of AD. The panel voted 6–0 (6 Yes; 0 No; 0 Abstain) in favor of the agent.

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