공시 • Aug 08
Neurogene Inc. Announces RMAT Designation for NGN-401 Investigational Gene Therapy for Rett Syndrome
Neurogene Inc. announced that NGN-401 received Regenerative Medicine Advanced Therapy (RMAT) designation from the U.S. Food and Drug Administration (FDA) for the treatment of Rett syndrome. The designation was based on preliminary clinical evidence from the ongoing Phase 1/2 clinical trial with NGN-401 that shows potential to address unmet medical needs in this disease. RMAT designation is granted for regenerative medicines intended to treat, modify, reverse, or cure a serious or life-threatening disease or condition, with preliminary clinical evidence that indicates that the drug has the potential to address unmet medical need. Benefits of the RMAT designation program include all the benefits of Fast Track and Breakthrough Therapy designation programs, including early and frequent communications with FDA senior managers, intensive guidance on efficient drug development and eligibility for an Accelerated Approval pathway and Priority Review. In addition, NGN-401 was previously selected by the FDA for its Support for clinical Trials Advancing Rare disease Therapeutics (START) Pilot Program based on potential for clinical benefit and clinical development program readiness. The START Program is designed to provide the Sponsor access to more frequent and ad hoc interactions with FDA staff to facilitate program development and an expectation of generating high quality and reliable data to support a potential future marketing application. NGN-401 is an investigational AAV9 gene therapy being developed as a one-time treatment for Rett syndrome. NGN-401 was selected by the U.S. Food & Drug Administration (FDA)for its START Pilot Program. In addition to Regenerative Medicine Advance Therapy (RMAT) designation, 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. These statements may discuss goals, intentions and expectations as to future plans, trends, events, results of operations or financial condition, or otherwise, based on current expectations and beliefs of the management of Neurogene, as well as assumptions made by, and information currently available to, management of Neurogene, including, but not limited to, statements regarding: the therapeutic potential and utility, efficacy and clinical benefits of NGN-401; the safety and tolerability profile of NGN-401; trial designs, clinical development plans and timing of the presentation of clinical trial data for NGN-401, and the anticipated benefits of the FDA's RMAT designation as well as participation in the FDA's START program. Such risks, uncertainties and assumptions include, among other things: risks related to the potential for negative impacts to patients dosed in the ongoing Phase 1/2clinical trial for NGN-401, including patients in Cohort 2 receiving a high dose of NGN-401; The risk that the Company may not be able to report its data on the predicted timeline; risks related to Neurogene's ability to effectively use the RMAT designation or theSTART program to accelerate development of NGN-401 or its ability to obtain regulatory approval for, and ultimately commercialize, NGN-401 at all; and other risks and uncertainties identified under the heading "Risk Factors" included in the Company's Annual Report on Form 10-K for the year ended December 31, 2023, filed with the Securities and Exchange Commission ("SEC") ("SEC") ("SEC") (SEC") ("SEC") in the Annual Report on Form10-K for the year endedDecember 31, 2023, filed With the Securities and Exchange Commission (" SEC") and the Company's Annual Report on form 10-K for the year ending December 31, 2023, filing with the Securities and Exchange Commission ('SEC") for the year ended December 31.