Business
Neurogene Reports Second Quarter 2024 Financial Results and Highlights Recent Updates
NGN-401 gene therapy for Rett syndrome received RMAT designation from FDA based on preliminary clinical evidence indicating the potential to address unmet
About this update from Neurogene Inc.
[{"type":"text","content":"\nNGN-401 gene therapy for Rett syndrome received RMAT designation from FDA based on preliminary clinical evidence indicating the potential to address unmet medical needs\n\n\nNGN-401 selected for FDA START Program, also designed to accelerate development\n\n\nInterim NGN-401 efficacy data from Cohort 1 remains on track for 4Q:24\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 second quarter 2024 financial results and highlighted recent corporate updates.\n\n\n“We are pleased that NGN-401 gene therapy for Rett syndrome received RMAT designation, in addition to the recent selection by the U.S. FDA for its START Pilot Program, two programs that provide distinct opportunities for enhanced collaboration and communication with the FDA to accelerate NGN-401’s development,” said Rachel McMinn, Ph.D., Founder and Chief Executive Officer of Neurogene. “We continued to advance the NGN-401 program with the first patient dosed in Cohort 2 in May, marking an important program milestone and demonstrating an early favorable safety profile with high-dose NGN-401, and reporting in mid-June that low-dose NGN-401 remained well-tolerated by the first three patients dosed in Cohort 1. As we look ahead, we continue to expect to release interim efficacy data from the low-dose cohort in the fourth quarter of this year, with plans to share additional low-dose as well as high-dose data in the second half of next year.”\n\n\nSecond Quarter 2024 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\nNGN-401 received Regenerative Medicine Advanced Therapy (RMAT) designation from the U.S. Food and Drug Administration (FDA) for the treatment of Rett syndrome, and was selected by the FDA for the Support for clinical Trials Advancing Rare disease Therapeutics (START) Pilot Program\n\n\nRMAT designation requires preliminary clinical evidence to show the potential to address unmet medical needs for a serious or life-threatening disease or condition, and it provides opportunity for an Accelerated Approval pathway under the FDA’s guidance\n\n\n\nSTART selection criteria included potential for clinical benefit and clini...