Neurogene update on 3rd high dose patient
On 11 November 2024, during an investor call reporting positive efficacy data, Neurogene shared that they had become aware of an emerging treatment-related serious adverse event in the third high dose participant on their clinical trial of Rett syndrome gene therapy, NGN-401.
On 18 November 2024, Neurogene shared an update about this patient who is now in critical condition. The third high-dose patient on the NGN-401 paediatric trial was dosed on November 5th. She subsequently experienced a rare immune response that has been previously reported in other patients receiving high doses of AAV gene therapy. The situation is continuing to evolve.
Rachel McMinn, Ph.D., Founder and Chief Executive Officer of Neurogene said:
“We are deeply saddened for the family. While no words could possibly provide comfort to her family, we ask the Rett syndrome community to join us in sending heartfelt thoughts to her family, friends and the dedicated clinicians who are caring for her. The safety of the participants in our clinical trial is and remains our foremost priority as we work to find solutions for this devastating disease.”
Rachael Stevenson, CEO of Reverse Rett said: ‘All of us at Reverse Rett have this patient and her family at the forefront of our minds during this difficult time. Families taking part in these clinical trials of pioneering treatments for Rett are making a tremendous contribution to the Rett community, both for patients living now and for future generations. Never has the weight of their contribution been as evident as it is right now. Our thoughts are with this courageous family and everyone involved in her treatment.’
Key takeaways from today’s announcement
- The third patient on high dose NGN-401 remains in critical condition.
- There were no previous serious adverse events in the first two patients on high-dose NGN-401 or in the five patients in the low-dose cohort.
- The FDA completed a review of the safety data for NGN-401 and allowed Neurogene to proceed with the Phase 1/2 trial using the low-dose NGN-401.
- No further patients will be dosed in Q4 2024 but dosing will resume in the new year.
- Neurogene paused further use of high-dose NGN-401 on initial notification of the SAE and does not plan to enroll any further participants at that dose level.
- The planned enrolment of 3 adult patients which was announced on November 11 will go ahead with low dose NGN-401.
Read the full press release here
Read Neurogene’s letter to the community here
At Reverse Rett, we have worked since our inception to accelerate treatments for Rett syndrome, including contributing to the academic work that led to the Neurogene gene therapy program. We have worked consistently with Neurogene throughout the development of their program including the initiation of UK clinical trials.
Today’s news is obviously difficult for the Rett community and at Reverse Rett, we echo Neurogene’s request that Rett families worldwide hold this patient and her family in their thoughts at this difficult time.