Business
Neurogene Reports Fourth Quarter and Full Year 2023 Financial Results and Highlights Recent Updates
Expanded Phase 1/2 gene therapy trial for Rett syndrome to inform future registrational study design; Company remains on track to share interim clinical data
About this update from Neurogene Inc.
[{"type":"text","content":"\nExpanded Phase 1/2 gene therapy trial for Rett syndrome to inform future registrational study design; Company remains on track to share interim clinical data in 4Q:24\n\n\nStrong financial position with runway into 2H:26 following reverse merger and private financing in December 2023\n\n\n NEW YORK--(BUSINESS WIRE)--\nNeurogene 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 fourth quarter and full year 2023 financial results and highlighted recent corporate updates.\n\n\n“We started the year with strong execution in our Phase 1/2 NGN-401 gene therapy trial for female pediatric patients with Rett syndrome, meeting our goal to expand the trial and add a high-dose cohort. We believe this expanded dataset will support future regulatory discussions to align on the design of a potential registrational study,” said Rachel McMinn, Ph.D., Chief Executive Officer and Founder of Neurogene. “NGN-401 has the potential to be a best-in-class treatment option with a targeted route of administration to deliver full-length, functional copies of the MECP2 gene. We are pleased that NGN-401 has been well-tolerated in all three patients dosed, with no signs of overexpression-related toxicity, demonstrating the potential of our EXACT transgene regulation technology to constrain MECP2 levels within a narrow and therapeutically relevant range. As we look ahead, we remain on track for interim clinical data from the NGN-401 trial in the fourth quarter of this year.”\n\n\nContinued Dr. McMinn, “Our decision to execute a reverse merger, along with the concurrent private financing, provides us with cash runway into the second half of 2026, beyond key inflection points in our NGN-401 program and our NGN-101 program for CLN5 Batten disease. We look forward to sharing data from both programs later this year and continuing to advance our pipeline to enable an additional development candidate using EXACT in 2025.”\n\n\nFourth Quarter 2023 and Recent Highlights, and Anticipated Milestones\n\n\nPhase 1/2 Trial of NGN-401 Gene Therapy for Treatment of Rett Syndrome\n\n\n\nAs previously planned, amended the protocol, expanding the trial to include more patients in low-dose Cohort 1 and added a high-dose Cohort 2; these updates are expecte...