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Ultragenyx Announces Initiation of Dosing in Second Cohort of Pivotal Phase 1/2/3 Cyprus2+ Trial Evaluating UX701 Gene Therapy for the Treatment of Wilson Disease
Initial data expected in first half of 2024 NOVATO, Calif., July 31, 2023 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today announced it
About this update from Ultragenyx Pharmaceutical Inc.
[{"type":"text","content":"Initial data expected in first half of 2024\nNOVATO, Calif., July 31, 2023 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today announced it has begun dosing the second dose-escalation cohort in its pivotal Phase 1/2/3 Cyprus2+ study following completion of dosing and safety review in the first cohort. The company's investigational AAV9 gene therapy is designed to deliver stable expression of the ATP7B copper transporter following a single intravenous infusion, with the goal of normalizing copper metabolism in patients with Wilson disease. In the first dose cohort, UX701 has been well tolerated with no unexpected related treatment emergent adverse events observed as of July 11, 2023. The Data Safety Monitoring Board recommended that the company proceed with dosing patients at the higher dose of 1.0 x 10^13 GC/kg. Investigators have dosed the first patient and have identified and screened the remaining four patients for dosing in Cohort 2. The company is on track to complete enrollment in Stage 1 of the Phase 1/2/3 trial this year and expects to share initial data in the first half of 2024. “We are encouraged by the safety data and early signals of the establishment of normal trafficking of copper observed in Cohort 1 and with acceleration of enrollment following improvements in study design and entry criteria,” said Eric Crombez, M.D., chief medical officer at Ultragenyx. “The shift to open label design and broadening of inclusion criteria allows us to enroll subjects who may not be well managed on current standard of care with chelators and/or zinc.” U.S. residents can learn more by visiting ultraclinicaltrials.com. Cyprus2+ Phase 1/2/3 study design This study evaluating UX701 for the potential treatment of Wilson disease is designed with three stages. During the first stage, the safety and efficacy of up to three dose levels of UX701 will be evaluated over the course of 52 weeks and a dose will be selected for further evaluation in Stage 2. In this first stage, 15 patients will be enrolled into three sequential dosing cohorts to evaluate doses of 5.0 x 10^12 GC/kg, 1.0 x 10^13 GC/kg, and 2.0 x 10^13 GC/kg. In Stage 2, a new cohort of patients will be randomized 2:1 to receive the selected dose of UX701 or placebo. The primary safety and efficacy analyses will be conducted at Week 52 of Stage 2. The primary e...