Neurogene has received several questions and concerns regarding the exclusion of female children with Rett syndrome who have been exposed to DAYBUE* (trofinetide) from our first-in-human clinical trial for the investigational gene therapy, NGN-401. In response, we would like to clarify the communication from last week.

First, and most importantly, initiating trofinetide treatment will not preclude a female patient with Rett syndrome from participating in the Neurogene-sponsored NGN-401 clinical trial in the future.  We anticipate that only the first several patients in our clinical trial will be required to have no trofinetide exposure in order to independently evaluate the safety and efficacy profile of NGN-401. We are continuing to consult with medical experts to determine the best path forward to include trofinetide-experienced patients in the future.

Second, Neurogene is pleased that trofinetide is now commercially available for patients affected by Rett syndrome. We believe that any FDA approval is an important step forward in patient care, and that families, and the entire community, can only benefit from having multiple treatment options available in the future.

Neurogene is committed to the global Rett syndrome community for the long-term. We will continue to develop our plans with an open mind, with the needs of families and input of medical experts helping to guide our work.


*DAYBUE is a trademark of Acadia Pharmaceuticals Inc.