Ultragenyx Reports Positive Interim Phase 1/2 Data With GTX-102 - Quick Facts

(RTTNews) - Ultragenyx Pharmaceutical Inc. (RARE) announced data from the Phase 1/2 study of GTX-102 for the treatment of Angelman syndrome. The company said patients in Expansion Cohorts A & B treated with a set dose and regimen of GTX-102 showed rapid and clinically meaningful improvement across multiple domains consistent with or exceeding Dose-escalation Cohorts 4-7 data at Day 170. Treatment of the dose-escalation Cohorts 4-7 showed long-term increasing and sustained clinical benefit far exceeding Natural History data at Day 758.

Eric Crombez, chief medical officer at Ultragenyx, said: "Our next step is an end of Phase 2 meeting with the FDA and interactions with other health authorities to enable timely initiation of a Phase 3 pivotal study."

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