ND0612 Receives Complete Letter Response, Otsuka Terminates AVP-786, TSHA-102 Performs in Phase 1/2 REVEAL Studies

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

According to a recent announcement, the FDA has issued a complete response letter (CRL) to NeuroDerm for its submission of ND0612, a 24-hour/day subcutaneous infusion of liquid levodopa/carbidopa (LD/CD), as a potential treatment for ON time in Parkinson disease (PD). Mitisubishi Tanabe Pharma and its wholly owned subsidiary, NeuroDerm, noted that they are reviewing the CRL and will work closely with the FDA to address its comments to consider the future direction. ND0612’s application was supported by data from the phase 3 BouNDless trial (NCT04006210), a double-blind, double-dummy, active-controlled trial that featured 259 patients with PD who experienced at least 2.5 h/day of OFF time. In the study, treatment with the investigational agent resulted in an additional 1.72 h (95% CI, 1.08-2.36) of ON time without troublesome dyskinesia compared with oral LD/CD.

According to a recent announcement, Otsuka Pharmaceutical announced the termination of the development of its novel compound AVP-786, a CYP2D6 inhibitor, for the treatment of patients with Alzheimer disease (AD) agitation. The company noted that it plans to continue its research and development efforts to meet the unmet needs of patients who experience agitation associated with AD. The termination of the development of this drug candidate was decided by the company after a detailed analysis of results from the completed phase 3 clinical trial (NCT03393520) assessing AVP-786 in patients with AD agitation. In February 2024, top-line results of the trial showed that the treatment failed to distinguish itself from placebo in this patient population, which consisted of 550 participants across 90 centers worldwide.

Taysha Gene Therapies has announced positive data from its pivotal phase 1/2 REVEAL studies assessing TSHA-102, an adeno-associated (AAV)-based gene therapy for patients with Rett syndrome. All told, treatment with the therapy resulted in durable improvements across clinical domains in a small group of both adult and pediatric patients, including motor skills, communication/socialization, autonomic function, and seizures, in addition to demonstrating a safe profile. Designed as a one-time lumbar intrathecal treatment, TSHA-102 aims to address the genetic root cause of Rett by delivering a functional form of MECP2 to cells in the central nervous system. Following an independent data monitoring committee review of safety data from the first high dose patient in the adolescent and adult trial, dosing for a high-dose cohort is expected to commence in Q3 2024.

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