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Ultragenyx Initiates Cyprus2+, a Pivotal Clinical Trial Evaluating UX701 Gene Therapy for the Treatment of Wilson Disease
NOVATO, Calif., Oct. 18, 2021 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and
About this update from Ultragenyx Pharmaceutical Inc.
[{"type":"text","content":"NOVATO, Calif., Oct. 18, 2021 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel products for rare and ultra-rare genetic diseases, today announced that it has successfully screened and enrolled multiple patients with Wilson disease into the baseline monitoring period prior to dosing in its pivotal, seamless Phase 1/2/3 study of UX701, the Cyprus2+ study. 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 establishing normal trafficking of copper in patients with Wilson disease. “Through collaboration with the FDA and other regulatory agencies we are able to bring forward an innovative and seamless phase 1/2/3 study design, that required agreement on clinical endpoints and manufacturing requirements up-front, allowing us to progress through the phases of development more efficiently,” said Eric Crombez, M.D., Chief Medical Officer of the Ultragenyx Gene Therapy development unit. “We are fortunate to have one of the most experienced and skilled teams in gene therapy and a proprietary and commercial-scale manufacturing platform that enable us to conduct this type of innovative and dynamic clinical program.” The study will enroll patients receiving ongoing standard of care medication for the treatment of Wilson disease (copper chelators and/or zinc) for at least 12 months, with no medication or dose changes for at least 6 months prior to enrollment. After initial screening that includes testing for pre-existing antibodies to the AAV9 capsid, patients will be evaluated to ensure stable measures of disease during a 4-to 12-week baseline monitoring period (including values for 24-hour urinary copper concentration, complete blood count, and liver function tests). Seamless Phase 1/2/3 study designThis study evaluating UX701 for the potential treatment of Wilson disease is designed with 3 seamless 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, 27 patients will be randomized into three cohorts in a 2:1 ratio per cohort to receive UX701 at th...