Neurogene to Present Interim Clinical Data from Phase 1/2 Trial of NGN-401 Gene Therapy for Rett Syndrome

Rhea-AI Summary

Neurogene Inc. (Nasdaq: NGNE) announced it will present interim efficacy data from the low-dose cohort of its ongoing Phase 1/2 clinical trial of NGN-401 gene therapy for pediatric patients with Rett syndrome. The company will host a webcast on November 11, 2024 at 4:30 p.m. ET to review the data, which will include safety information from both low- and high-dose cohorts.

Additionally, these results will be presented in a late-breaking poster during the 53rd Annual Child Neurology Society (CNS) Meeting on November 12 at 12:30 p.m. PT in San Diego, CA. The poster, titled 'NGN-401, a Novel Regulated Gene Therapy for Rett Syndrome: Preliminary Results from the First-in-Human Study,' will be presented by Dr. Bernhard Suter from Texas Children's Hospital and Baylor College of Medicine.

Positive

• Interim efficacy data from the Phase 1/2 trial of NGN-401 for Rett syndrome to be presented
• Safety data from both low- and high-dose cohorts will be shared
• Late-breaking poster presentation at a major neurology conference (CNS Annual Meeting)

Negative

• None.

Insights

Medical Research Analyst

The upcoming presentation of interim clinical data for NGN-401, Neurogene's gene therapy for Rett syndrome, is a significant development. This Phase 1/2 trial data could provide important insights into the therapy's efficacy and safety profile. Rett syndrome, a rare genetic neurological disorder, currently has no cure, making this potential treatment highly anticipated.

Key points to consider:

• The presentation will include efficacy data from the low-dose cohort and safety data from both low- and high-dose cohorts.
• The involvement of Dr. Bernhard Suter, a respected expert in Rett syndrome, adds credibility to the study.
• As a first-in-human study, these results could be pivotal for Neurogene's pipeline and valuation.

However, investors should approach this news cautiously. Early-stage clinical data can be volatile and the full impact on Neurogene's prospects won't be clear until the complete dataset is analyzed. The market's reaction to this data could significantly affect Neurogene's stock price in the short term.

Company to host webcast to review data on November 11 at 4:30 p.m. ET

Late-breaker poster to be presented during Child Neurology Society Meeting on November 12

NEW YORK--(BUSINESS WIRE)-- Neurogene Inc. (Nasdaq: NGNE), a clinical-stage company founded to bring life-changing genetic medicines to patients and families affected by rare neurological diseases, today announced that it will host a webcast to present interim efficacy data from the low-dose cohort of its ongoing Phase 1/2 clinical trial of NGN-401 gene therapy for pediatric patients with Rett syndrome on November 11, 2024 at 4:30 p.m. ET. Safety data from the low- and high-dose cohorts will also be shared.

These data will also be presented in a late-breaking poster presentation during the 53rd Annual Child Neurology Society (CNS) Annual Meeting on November 12 at 12:30 p.m. PT in San Diego, CA.

Neurogene’s Analyst and Investor Webcast/Conference Call

The live webcast and conference call information will be accessible from the Investor Relations section of Neurogene’s website under Events, where the webcast replay will also be available for a limited time.

Neurogene’s CNS Meeting Presentation

• Poster Title: NGN-401, a Novel Regulated Gene Therapy for Rett Syndrome: Preliminary Results from the First-in-Human Study
• Presenter: Bernhard Suter, M.D., Medical Director of the Blue Bird Circle Rett Center at Texas Children’s Hospital, Associate Professor of Pediatrics and Neurology at Baylor College of Medicine, and principal investigator in the NGN-401 clinical trial

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 Houston, Texas. CGMP production of NGN-401 was conducted in this facility and will support pivotal clinical development activities. For more information, visit www.neurogene.com.

View source version on businesswire.com: https://www.businesswire.com/news/home/20241018666990/en/

Company:

Cara Mayfield

Vice President, Corporate Affairs

cara.mayfield@neurogene.com



Investor:

Melissa Forst

Argot Partners

Neurogene@argotpartners.com

Source: Neurogene

FAQ

What is Neurogene's stock symbol?

Neurogene's stock symbol is NGNE, traded on the Nasdaq.

When will Neurogene present interim data for NGN-401 in Rett syndrome?

Neurogene will present interim data for NGN-401 in Rett syndrome on November 11, 2024, during a webcast at 4:30 p.m. ET.

What type of data will be presented for NGN-401 in the Rett syndrome trial?

Neurogene will present interim efficacy data from the low-dose cohort and safety data from both low- and high-dose cohorts of the Phase 1/2 trial of NGN-401 for Rett syndrome.

Where else will Neurogene present the NGN-401 Rett syndrome trial data?

Neurogene will also present the NGN-401 Rett syndrome trial data in a late-breaking poster at the 53rd Annual Child Neurology Society Meeting on November 12, 2024, at 12:30 p.m. PT in San Diego, CA.