May. 13 at 5:15 PM
$DTIL
Preliminary data from participants enrolled in three dose cohorts (n=7) demonstrate that 71% of participants experienced
--> NO <-- hyperammonemic crises (HACs) following ECUR-506 treatment, as of the April 20, 2026 data cutoff date.
HACs are defined as plasma ammonia levels above 100µmol/L with neurologic status change.
Data from these cohorts also demonstrate approximately 52% reduction in the annualized rate of HACs following ECUR-506 administration.
HACs are the --> most clinically significant neurometabolic manifestation of ornithine transcarbamylase (OTC) deficiency. <--
Reducing or preventing these crises is a key treatment goal, clinically meaningful measure of patient benefit, and an important efficacy endpoint in this highly vulnerable population.
ECUR-506 was generally well tolerated across all dose cohorts to date, with --> NO <-- unexpected safety events.
