Neurogene Inc., a clinical-stage company focused on genetic medicines for rare neurological disorders, announced that its NGN-401 gene therapy for Rett syndrome has been chosen to participate in the FDA's Support for Clinical Trials Advancing Rare Disease Therapeutics (START) Pilot Program. This selection will enable Neurogene to benefit from enhanced communication with the FDA, facilitating the development of NGN-401. The START Program will provide frequent guidance and consultations on various aspects of product development, such as clinical study design, control group selection, and patient population criteria.

Neurogene, stated, "We are honored that NGN-401 gene therapy for Rett syndrome has been chosen as one of only three CBER programs for the FDA’s START Pilot Program. We are grateful for the FDA’s commitment to accelerating the development of NGN-401. We look forward to working closely with the FDA to advance NGN-401 toward a potential registrational study for patients and families affected by this devastating disease."

The Phase 1/2 clinical trial of NGN-401 is investigating two dose levels in female pediatric patients with Rett syndrome. The trial focuses on the safety, tolerability, and preliminary efficacy of a one-time intracerebroventricular (ICV) administration. Recently, Neurogene presented favorable safety data from the first three patients dosed, with interim efficacy data expected in the fourth quarter of 2024.

The START Program aims to accelerate the development of new therapies for rare diseases by facilitating more frequent and rapid interactions with the FDA. To be considered for the program, applications needed to include detailed clinical, CMC (chemistry, manufacturing, and controls), and non-clinical development plans, as well as updates on current development status. The FDA selected participants based on factors such as potential clinical benefits, alignment of development plans, and the ability to advance towards a marketing application. Program participants will receive ongoing advice from FDA staff to ensure the generation of high-quality data to support future marketing applications.

NGN-401 is an investigational AAV9 gene therapy designed as a one-time treatment for Rett syndrome. It is the first clinical candidate to deliver the full-length human MECP2 gene using Neurogene’s EXACT technology. This technology enables targeted levels of MECP2 transgene expression, avoiding the toxic effects associated with overexpression in conventional gene therapy.

NGN-401's selection for the START Pilot Program follows several previous recognitions, including orphan drug designation, Fast Track designation, and rare pediatric designation from the FDA. Additionally, NGN-401 received orphan designation and advanced therapy medicinal product designation from the EMA, and the Innovative Licensing and Application Pathway (ILAP) designation from the UK MHRA.

 

Source: businesswire.com

Report Abuse