NEW YORK - Neurogene Inc. (NASDAQ: NGNE), a biotechnology firm focused on developing genetic therapies for rare neurological diseases, announced today that it has expanded its ongoing Phase 1/2 clinical trial for its gene therapy product, NGN-401, for pediatric patients with Rett syndrome. The trial will now include additional patients and a new dose-escalation cohort.
The trial's protocol has been updated to include eight patients in Cohort 1, which will receive a total dose of 1×10^15 total vector genomes delivered via intracerebroventricular administration. Notably, the staggered dosing approach previously employed in Cohort 1 has been eliminated, allowing for parallel dosing of the remaining patients. This change is expected to accelerate the completion of Cohort 1 dosing, now anticipated in the second half of 2024.
A new Cohort 2 has also been introduced, aiming to enroll a total of eight patients who will receive a higher dose of 3×10^15 total vector genomes. The initial three patients of this cohort will be dosed staggered, with the first patient expected to be dosed in the second quarter of 2024. Following a safety review by the Data and Safety Monitoring Board, the remaining patients will also be dosed in parallel.
Additionally, the trial has implemented a targeted immunosuppression regimen for Cohort 2 to mitigate potential immune responses related to the adeno-associated virus vector used in the therapy. This regimen includes rituximab, sirolimus, and a shortened course of corticosteroids, while Cohort 1 will continue with corticosteroids alone.
The company has reported that NGN-401 has been well-tolerated with no serious adverse events or signs of toxicity observed in any patient to date. Interim clinical data from Cohort 1 is expected to be reported in the fourth quarter of 2024, with additional data from Cohort 2 anticipated in the second half of 2025.
NGN-401 utilizes Neurogene's proprietary EXACT gene regulation technology designed to deliver controlled levels of MECP2 gene expression, which is crucial for treating Rett syndrome without causing toxic effects associated with overexpression.
This clinical trial expansion aligns with Neurogene's strategic approach to develop more comprehensive data to support future regulatory discussions for NGN-401. The information provided here is based on a press release statement from Neurogene Inc.
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