Neurogene Receives RMAT for Rett Syndrome Gene Therapy NGN-401

Neurogene Inc. (NASDAQ: NGNE), a clinical-stage company dedicated to developing genetic medicines for rare neurological diseases, has announced that their investigational therapy NGN-401 has been granted Regenerative Medicine Advanced Therapy (RMAT) designation by the U.S. Food and Drug Administration (FDA) for treating Rett syndrome. This designation highlights the potential of NGN-401 to address unmet medical needs, based on preliminary clinical evidence from the ongoing Phase 1/2 clinical trial.

Rachel McMinn, Ph.D., the Founder and CEO of Neurogene, expressed gratitude for the FDA's efforts to expedite the development of NGN-401. The RMAT designation comes in addition to NGN-401's earlier selection for the FDA's Support for clinical Trials Advancing Rare disease Therapeutics (START) Pilot Program. Both initiatives aim to fast-track the development of promising treatments for patients with significant medical needs. Neurogene plans to share interim efficacy data from the low-dose cohort in the fourth quarter of this year and additional data, including from the high-dose cohort, in the second half of 2025.

RMAT designation is granted to regenerative medicines that show potential to treat, modify, reverse, or cure serious or life-threatening conditions based on preliminary clinical evidence. This designation offers several benefits, including all the advantages of the Fast Track and Breakthrough Therapy designation programs. These benefits include early and frequent communication with FDA senior managers, intensive guidance on efficient drug development, and eligibility for an Accelerated Approval pathway and Priority Review.

The FDA's START Pilot Program also selected NGN-401 based on its potential clinical benefit and readiness for clinical development. The START Program aims to provide sponsors with more frequent and flexible interactions with FDA staff, facilitating program development and ensuring high-quality and reliable data to support future marketing applications.

NGN-401 is an investigational AAV9 gene therapy intended 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 proprietary EXACT™ transgene regulation technology. This technology represents a significant advancement in gene therapy for Rett syndrome, as it enables targeted MECP2 transgene expression levels without causing the toxic effects associated with conventional gene therapy.

Beyond the RMAT designation, NGN-401 has received multiple recognitions from the FDA, including orphan drug designation, Fast Track designation, and rare pediatric designation. Neurogene has also been granted an INTERACT meeting with the FDA to discuss the EXACT technology. Additionally, NGN-401 has 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).

Neurogene’s mission is to develop treatments for devastating neurological diseases, aiming to improve the lives of patients and their families. The company focuses on innovative approaches to overcome the limitations of conventional gene therapy for central nervous system disorders. This includes optimizing delivery methods to maximize tissue distribution and designing products to enhance potency and purity for better efficacy and safety. Neurogene has built a state-of-the-art gene therapy manufacturing facility in Houston, Texas, where CGMP production of NGN-401 has been conducted to support pivotal clinical development activities.

In summary, Neurogene Inc. is making significant strides in developing NGN-401 for Rett syndrome, with the recent RMAT and START designations from the FDA highlighting the therapeutic potential of this gene therapy. These designations, along with multiple other recognitions, reflect the company's commitment to accelerating the development of life-changing treatments for rare neurological diseases. Neurogene continues to work towards delivering effective and safe gene therapies to patients in need.