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Neurogene Reports Third Quarter 2024 Financial Results and Highlights Recent Updates
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In This Article:

Announced positive interim clinical data with 1E15 vg dose of NGN-401 gene therapy trial for Rett syndrome

Neurogene to advance NGN-401 at 1E15 vg dose

Expects to provide an update on registrational trial design in first half of 2025

Strong cash position provides runway into the second half of 2027

NEW YORK, November 18, 2024--(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 third quarter 2024 financial results and highlighted recent corporate updates.

"The recently announced interim data from participants dosed with 1E15 vg in the NGN-401 clinical trial showed consistent improvements across multiple domains that define Rett syndrome, in contrast to what is expected based on the natural history of the disease," stated Rachel McMinn, Ph.D., Founder and Chief Executive Officer of Neurogene. "We have observed girls gaining complex skills rarely ever learned in this population, as well as skills that were once present and lost during the phase of developmental regression. The favorable safety and efficacy data for NGN-401 at the 1E15 vg dose demonstrate its potential to have a meaningful impact on the disease course of Rett syndrome. We will continue to engage with the FDA on the planning for a future registrational clinical trial and anticipate providing an update on the trial design in the first half of 2025."

Third Quarter 2024 and Recent Highlights, and Anticipated Milestones

Phase 1/2 Trial of NGN-401 Gene Therapy for Treatment of Rett Syndrome

NGN-401 is currently being evaluated in an ongoing, open-label Phase 1/2 trial. Key updates include:

  • Announced today an update to the NGN-401 clinical trial

    • Following a treatment-related serious adverse event (SAE) in the third participant dosed with 3E15 vg, Neurogene paused further use of the 3E15 vg dose and does not plan to enroll any further participants at the 3E15 vg dose

    • The U.S. Food and Drug Administration (FDA) has completed a review of the safety data for NGN-401 and has allowed Neurogene to proceed with the Phase 1/2 trial using the 1E15 vg dose

    • Neurogene’s prior guidance of completing enrollment this quarter in the 1E15 vg dose cohort was updated as a result of the need to revise the protocol to remove the 3E15 vg dose; Neurogene expects to resume dosing once the revisions to the protocol are completed

  • Previously announced positive interim clinical data from the participants dosed with 1E15 vg:

    • The first four participants showed consistent, concordant improvements across key Rett syndrome scales; all achieved clinically meaningful rating of "much improved" on the Clinical Global Impression-Improvement (CGI-I) scale and improved by 28 to 52 percent on the Rett Syndrome Behavioral Questionnaire (RSBQ)

    • Participants achieved meaningful gains of function and developmental milestones in the core clinical domains of Rett syndrome – hand function/fine motor, communication/language, and ambulation/gross motor; these improvements were achieved despite heterogeneous clinical presentation at baseline, and not expected based on the natural history of Rett syndrome

    • NGN-401 was well-tolerated with a favorable safety profile in the first five participants who received the 1E15 vg dose; all treatment-related adverse events (AEs) in these participants were Grade 1 (mild)

  • Gained alignment with the FDA on CMC scale-up plans to support commercial launch and potency assay strategy for the program

  • Plans to provide an update on registrational trial design in the first half of 2025

  • Expects to announce additional interim Phase 1/2 clinical data in the second half of 2025

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