Neurogene Announces NGN-401 Gene Therapy for Rett Syndrome Selected by FDA for START Pilot Program
Neurogene (NASDAQ: NGNE) announced that its NGN-401 gene therapy for Rett syndrome has been chosen by the FDA for the START Pilot Program, aimed at accelerating the development of rare disease therapies. NGN-401 is one of only three programs selected by the CBER division, due to its potential clinical benefits and readiness for clinical development.
This selection allows Neurogene enhanced communication with the FDA to resolve product-specific development issues promptly. Currently, NGN-401 is being evaluated in a Phase 1/2 clinical trial, focusing on safety, tolerability, and preliminary efficacy in female pediatric patients. Interim efficacy data is expected in Q4 2024.
Neurogene recently reported favorable safety data from the first three patients dosed. The START Program aims to provide frequent FDA advice to facilitate the program's development and generate high-quality data for potential future marketing applications.
- NGN-401 selected for FDA's START Pilot Program, one of only three programs chosen.
- Enhanced communication with FDA could accelerate NGN-401's development.
- Favorable safety data reported from the first three patients dosed.
- Interim efficacy data expected in Q4 2024.
- Potential for NGN-401 to move towards a registrational study for Rett syndrome.
- Current status only at Phase 1/2 clinical trial, implying significant time before potential market availability.
- No efficacy data reported yet; only interim results expected by Q4 2024.
- Selection in the START Program does not guarantee successful development or FDA approval.
Insights
The selection of Neurogene’s NGN-401 gene therapy for the FDA’s START Pilot Program is significant. This program accelerates the development of therapies for rare diseases through enhanced interactions with the FDA. For a gene therapy targeting Rett syndrome, a debilitating neurological disorder primarily affecting females, this means faster transitions from clinical trials to potential approval. The presence of two dose levels in the Phase 1/2 trial evaluates safety, tolerability and initial efficacy, important for understanding how well the therapy might work in real-world scenarios. The program's milestone-driven nature and frequent advice can help in refining clinical studies, ensuring a robust submission for future marketing applications. The readiness and potential clinical benefits cited are promising, but investors should remain cautious until more substantial efficacy data, expected by late 2024, is available.
Neurogene's inclusion in the START Pilot Program not only boosts the timeline for NGN-401 but also signals strong FDA confidence in its potential. For investors, this represents a potential de-risking event, given the FDA's commitment to allocate resources to this program. The favorable safety data from the initial set of patients is encouraging, yet the true financial impact hinges on the forthcoming efficacy data. While there might be short-term stock appreciation due to this news, the long-term valuation will depend on the final trial results and the subsequent steps toward commercialization. Investors should be aware of the timelines and prepare for possible volatility as upcoming data releases approach.
START Program provides sponsors enhanced communications with FDA to accelerate development of rare disease therapies
“We are honored that
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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
About Neurogene
The mission of Neurogene is to treat devastating neurological diseases to improve the lives of patients and families impacted by these rare diseases. Neurogene is developing novel approaches and treatments to address the limitations of conventional gene therapy in central nervous system disorders. This includes selecting a delivery approach to maximize distribution to target tissues and designing products to maximize potency and purity for an optimized efficacy and safety profile. The Company’s novel and proprietary EXACT transgene regulation platform technology allows for the delivery of therapeutic levels while limiting transgene toxicity associated with conventional gene therapy. Neurogene has constructed a state-of-the-art gene therapy manufacturing facility in
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Company Contact:
Cara Mayfield
Vice President, Corporate Affairs
cara.mayfield@neurogene.com
Investor Contact:
Melissa Forst
Argot Partners
Neurogene@argotpartners.com
Source: Neurogene Inc.
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