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Neurogene Reports Positive Interim Efficacy Data from First Four Low-Dose Pediatric Participants in NGN-401 Gene Therapy Clinical Trial for Rett Syndrome
All participants experienced a 2-point improvement in the clinician-rated Clinical Global Impression-Improvement (CGI-I) scale from baseline All participants
About this update from Neurogene Inc.
All participants experienced a 2-point improvement in the clinician-rated Clinical Global Impression-Improvement (CGI-I) scale from baseline All participants improved in the caregiver-completed Rett Syndrome Behavior Questionnaire (RSBQ), ranging from 28 to 52 percent improvement from baseline All participants with disruptions in sleep, constipation, and dysphagia at baseline demonstrated objective improvements Gains in skill and developmental milestones were consistent, durable, deepened over time and demonstrated improvements not expected based on natural history data Low-dose NGN-401 well-tolerated with favorable safety profile Company plans to provide an update of registrational trial design in the first half of 2025 Company to host investor/analyst webcast today, November 11, 2024 , at 4:30 p.m. ET NEW YORK --(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 positive interim clinical data in the first four participants in the low-dose cohort of its ongoing Phase 1/2 open-label trial designed to evaluate NGN-401 gene therapy for the treatment of female pediatric patients with Rett syndrome. Low-dose NGN-401 has demonstrated a favorable safety profile. “Today marks an important day for Neurogene and the Rett syndrome community as we share positive interim data for NGN-401 from our low-dose cohort that shows the first four participants demonstrated meaningful gains of skills and developmental milestones in core clinical domains of Rett syndrome, which are not expected to occur when compared to and contextualized against the natural history of Rett syndrome. Data were also concordant across multiple scales and show consistency of effect across patients, despite their unique clinical presentations at baseline,” said Rachel McMinn , Ph.D., Founder and Chief Executive Officer of Neurogene. “We are incredibly thankful to the participants, caregivers and Rett syndrome trial sites who are participating in our study.” “Rett syndrome is a devastating neurodevelopmental disease that is incredibly challenging for patients and their caregivers given there are no treatment options available to address the underlying cause of the disease,” said Aleksandra Jacobs , M.D., Ph.D., Professor of Pediatric Neurology, Albert Einstein College of Medicine and Director of the Center for Rett Syndrome in the Children’s Hospital at Montefiore Medical Center . “The totality of the outcomes shared today with NGN-401 gene therapy have never been seen before in the treatment of Rett syndrome. Notably, these initial participants acquired developmental skills post-treatment during the period in which the natural history of Rett syndrome indicates girls would not. I look forward to the continued progress in this program and additional data to come.” Interim Clinical Data as of Data Cut-Off Date of October 17, 2024 Interim Safety Data (N=7)* Low-dose (1E15 vg) and high-dose (3E15 vg) NGN-401 have been well-tolerated with a favorable safety profile in the first seven pediatric participants (N=5 low-dose; N=2 high-dose): No treatment-related serious adverse events (SAEs) No signs or symptoms indicative of MeCP2 overexpression toxicity Most treatment-related adverse events (AEs) are known potential risks of adeno-associated virus (AAV), have been responsive to steroids, and are resolved or are resolving No intracerebroventricular (ICV)-related AEs No seizures for any participants following NGN-401 treatment *Today, Neurogene became aware of an emerging treatment-related SAE consistent with known risks of AAV gene therapy in the third high-dose participant who was recently dosed. Low-Dose Interim Efficacy Data (N=4) The first four participants (age range 4-7 years old, efficacy assessments at 15, 12, 9, and 3 months post-dosing) in low-dose Cohort 1 showed consistent, concordant and durable improvements across key Rett syndrome assessments: All participants achieved a rating of “much improved,” or a score of 2, on the clinician-rated Clinical Global Impression Scale of Improvement (CGI-I) from baseline; a score of