What's Happening?
Encoded Therapeutics has announced positive interim results from its POLARIS clinical development program, which is evaluating ETX101, a gene therapy for children with SCN1A+ Dravet syndrome. The trials,
conducted in the US, UK, and Australia, have shown a median seizure reduction of 78% over seven months at the third dose level. The therapy has also demonstrated significant neurodevelopmental improvements, particularly in cognitive skills, when administered before the age of two. ETX101 has been well-tolerated across all dose levels, with no treatment-related serious adverse events reported. The company plans to present further efficacy data and initiate a pivotal study in 2026.
Why It's Important?
The interim results of ETX101 are significant as they offer hope for a disease-modifying treatment for Dravet syndrome, a severe neurological disorder with no current approved therapies. The observed reduction in seizures and neurodevelopmental improvements could greatly enhance the quality of life for affected children and their families. The success of ETX101 could pave the way for similar gene therapies targeting other genetic disorders, potentially transforming the landscape of treatment for rare neurological conditions.
What's Next?
Encoded Therapeutics plans to present additional efficacy data from the fourth dose level and advance ETX101 towards a pivotal clinical trial in 2026. The continuation of these trials will be crucial in determining the long-term efficacy and safety of ETX101. The results could influence regulatory decisions and the future availability of this therapy for Dravet syndrome patients.
