Neurogene Inc. announced that its NGN-401 gene therapy for Rett syndrome has been selected to participate in the U.S. Food and Drug Administration (FDA) Support for Clinical Trials Advancing Rare Disease Therapeutics (START) Pilot Program. As part of the START Program, Neurogene will have opportunities for enhanced communications with the FDA, with the aim to further accelerate the pace of NGN-401?s development. These opportunities are designed to provide frequent advice and regular ad-hoc conversations to address product-specific development issues, including, but not limited to, clinical study design, choice of control group and fine-tuning the choice of patient population.

Two dose levels of NGN-401 are being evaluated in a Phase 1/2 clinical trial. The trial is assessing safety, tolerability, and preliminary efficacy of a one-time intracerebroventricular (ICV) administration of NGN-401 in female pediatric patients with Rett syndrome. Neurogene recently presented favorable safety data from the first three patients dosed with NGN-401, and the Company remains on track to report interim efficacy data from the trial in the fourth quarter of 2024.

The milestone-driven START Program was launched to help further accelerate development of novel drug and biological product candidates intended to address rare diseases through more frequent communication and rapid interactions with the FDA. START Program applications required clinical, CMC (chemistry, manufacturing and controls), and non-clinical development plans as well as an update on the current status of development. The FDA reported that it would be considering multiple factors in selecting participants, including potential clinical benefits of the product candidate, the alignment of clinical development and CMC plans, and the ability to move the program forward toward a marketing application.

Selected sponsors are expected to receive more frequent advice from FDA staff with the goal of facilitating program development and an expectation of generating high quality and reliable data to support a potential future marketing application. About NGN-401: NGN-401 is an investigational AAV9 gene therapy being developed as a one-time treatment for Rett syndrome. It is the first clinical candidate to deliver the full-length human MECP2 gene under the control of Neurogene?s EXACT technology.

The EXACT technology utilized in NGN-401 is an important advancement in gene therapy for Rett syndrome, specifically because the disorder requires a treatment approach that enables targeted levels of MECP2 transgene expression without causing overexpression-related toxic effects associated with conventional gene therapy. NGN-401 was one of three Center for Biologics Evaluation and Research (CBER) programs selected by the U.S. Food and Drug Administration (FDA) for its START Pilot Program. NGN-401 previously received orphan drug designation, Fast Track designation and rare pediatric designation from the FDA.

Neurogene was previously granted an INTERACT meeting with the FDA regarding the EXACT technology. NGN-401 also received orphan designation and advanced therapy medicinal product designation from the European Medicines Agency (EMA) and the Innovative Licensing and Application Pathway (ILAP) designation from the United Kingdom (UK) Medicines and Healthcare products Regulatory Agency (MHRA).