Neurogene’s updated positive interim clinical data to improve Rett syndrome treatment

Announcement

Neurogene Inc., a clinical-level leading company built to introduce life-changing, transformative genetic medicines to families and patient groups suffering from rare neurological diseases, have proudly proved its efforts to bring updated positive interim clinical data in the pediatric group ranging from 4-10 years old from its latest ongoing Phase 1/2 trial examining NGN-401 gene therapy for Rett syndrome treatment for females. To this small yet effective step, Neurogene’s founder and chief executive officer precisely opened up to the impact of the data.

The PhD, Chief Executive Officer and Founder of Neurogene, Rachel McMinn said, “The new interim trimmed data from our Phase 1/2 trial of NGN-401 showed that every pediatric participant, despite of any baseline diseases, has successfully achieved a developmental experience or skills/success to its functionality, along with consistent skill acquisition over time and durability. This skill achievement has set a competence bar for our Embolden registrational trial and has joined with NGN-401’s tolerability profile at the 1E15 vg dose.”

Rachel added, “This builds a capability space for excellent gene therapy for Rett syndrome. It feels proud to witness these advantages of our smart strategy to embed local delivery to the brain associated with the naturally found full-length human gene as a major one to uncover valuable efficacy in this adverse disorder. For now, we’re the only company examining a gene therapy in the children's population (as young as three years) showing Rett syndrome in a single trial.”

Updated Interim Phase 1/2 clinical data

Firstly, the safety data showed that the 1E15 vg and NGN-401 dose is considered a tolerated dose with the best safety profile. Each treatment based on adverse events has been moderate or mild in severity, and others in massive numbers are recognised as capable of risk of AAV that has resolved or are under resolution. No hemophagocytic lymphohistiocytosis (HLH) was noticed in any participant.

The efficacy data showed that all pediatric participants witnessed functional gains throughout the vast space of disease severity, with around 35 developmental achievements throughout the intense clinical area of Rett syndrome. The participants with a lengthy follow-up continued to achieve developmental skills/milestones, with a registered success in motor planning and executive function for the longest follow-up participants. The additional clinical data will be out next year. The Neurogene management will stress over the Embolden™ registrational trial and results at the Stifel 2025 Healthcare Conference.