Neurogene said it has completed dosing in its EMBOLDEN registrational trial of NGN-401 for Rett syndrome, enrolling 25 participants, above the original target of 20.
The company had built its statistical plan around an intent-to-treat population of up to 24 participants, but it chose to overenroll and dose all eligible patients already in screening, bringing the total to 25.
At the 1e15 vg dose, NGN-401 has now been administered to 35 participants across the phase 1/2 and EMBOLDEN studies, and Neurogene said the treatment continues to be generally well tolerated. The company said there have been no cases of hemophagocytic lymphohistiocytosis, or HLH, at that dose in either trial.
EMBOLDEN’s primary success threshold is a minimum response rate of 33%, or 8 of 24 participants. The primary analysis tied to the planned biologics license application is expected after the first 24 participants complete 12 months of follow-up.
Neurogene said topline data from EMBOLDEN is now expected in the second half of 2027, while updated interim phase 1/2 data are slated for mid-2026 and will include at least 12 months of follow-up on all 10 participants. The market has reacted to these announcements by moving the company's shares 0.04% to a price of $26.50. For the full picture, make sure to review Neurogene's 8-K report.
