Question · Q4 2025
Yigal Dov Nochomovitz requested more details on the DTX-301 (OTC) study, its prominence in success expectations, and what would be needed for the study to hit. He also asked if the UX111 IRL requests, which would typically occur during an inspection, mean the inspection would be limited or different.
Answer
Emil Kakkis, Chief Executive Officer and President, explained that the DTX-301 Phase III trial (37 patients) is looking at change in ammonia between treated and control groups as the primary endpoint, with a secondary focus on patients getting off standard of care. He noted it's an important program but not their top priority. Regarding the UX111 IRL, he expects the FDA will still inspect, but providing comprehensive documentation upfront aims to give them greater confidence that all requests have been addressed.
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