Neurogene Announces Business Update and 2024 Outlook

Neurogene Inc. (NASDAQ: NGNE) (“Neurogene” or “the Company”), a clinical-stage company founded to bring life-changing genetic medicines to patients and families affected by rare neurological diseases, announced today a business update and provided its outlook for 2024. Key milestones for the Company’s clinical-stage gene therapy product candidates remain on track for 2024, while the Company continues to advance its discovery-stage transgene regulation portfolio.

Anticipated 2024 Key Milestones

• Rett Syndrome – NGN-401
  • Expand ongoing Phase 1/2 clinical trial for NGN-401 for Rett syndrome in 1H:24 to enroll a larger cohort of patients
  • Report interim Phase 1/2 clinical data for NGN-401 for Rett syndrome in 4Q:24
• CLN5 Batten Disease – NGN-101
  • Report interim Phase 1/2 clinical data for NGN-101 in 2H:24
  • Engage in discussions with U.S. Food and Drug Administration (FDA) regarding a streamlined registrational pathway for NGN-101 in 2H:24

“2023 was a transformational year for Neurogene, with the clearance by the FDA of our Investigational New Drug application for NGN-401 in female pediatric patients with Rett syndrome, completing the manufacturing of clinical-grade NGN-401 in-house at our GMP viral vector manufacturing facility, dosing the first two patients in our Phase 1/2 clinical trial for Rett syndrome with no treatment-emergent or procedure-related serious adverse events, or signs of overexpression-related toxicity, and completing a reverse merger and private financing to capitalize Neurogene into the second half of 2026,” said Founder and Chief Executive Officer, Rachel McMinn, Ph.D. “We remain on track to deliver interim results for our potentially best-in-class Rett syndrome gene therapy program in the fourth quarter of 2024.”

Rett Syndrome Program Update

Neurogene announced that the Data Safety Monitoring Board (“DSMB”) for the ongoing Phase 1/2 clinical trial of NGN-401 in female pediatric patients with Rett syndrome has recommended that the trial continue, which enables dosing of the third patient. This recommendation was based on safety data collected to date, following dosing of the first patient in the third quarter of 2023 and the second patient in the fourth quarter of 2023.