Neurogene Inc. (NASDAQ: NGNE) has released positive interim clinical data for the pediatric cohort (ages 4-10) from its ongoing phase 1/2 trial evaluating ngn-401 gene therapy for the treatment of females with Rett syndrome. The updated data shows that all pediatric participants, regardless of baseline disease severity, have experienced developmental milestones/skills gains or functional improvements.
Safety data as of the cutoff date of October 30, 2025, indicates that ngn-401 at the 1e15 vg dose has been generally well-tolerated with a favorable safety profile across both the pediatric and adolescent/adult cohorts. All treatment-related adverse events have been mild (grade 1) or moderate (grade 2) in severity, with the majority being known potential risks of AAV and have either resolved or are resolving. Importantly, no evidence of hemophagocytic lymphohistiocytosis (HLH) has been observed in any participant.
In terms of efficacy, all pediatric participants have shown functional gains across the spectrum of disease severity, with a total of 35 developmental milestones/skills acquired across core clinical domains of Rett syndrome, including hand function/fine motor, language/communication, and ambulation/gross motor. These gains have been durable, with multidomain improvements observed in key areas. Participants with longer-term follow-up continued to gain developmental milestones/skills, particularly in executive function and motor planning. Notably, even participants recently dosed with six months of follow-up have exhibited consistent developmental gains.
The previously reported improvements in the Clinical Global Impression-Improvement (CGI-I) have been shown to be durable, and additional clinical data is expected to be reported in 2026.
This positive update has been described as exceeding the bar for the embolden registrational trial, and the company's management will be discussing these results and the embolden trial during the Stifel 2025 Healthcare Conference webcast.
Following these announcements, the company's shares moved 0.84%, and are now trading at a price of $31.13. For more information, read the company's full 8-K submission here.
