Safety and initial efficacy data from Stage 1 expected in the first half of 2024

Dose selection and initiation of Stage 2 to follow in the second half of 2024

“With the support of patients, physicians and the Wilson disease community, we’ve completed Stage 1 enrollment in the Cyprus²⁺ program, which moves us one step closer to beginning Stage 2, the pivotal, randomized placebo-controlled stage of the study,” said Eric Crombez, M.D., chief medical officer at Ultragenyx. “Beyond the seamless nature of this study, another important differentiator of this program is that it leverages our Pinnacle PCL™ platform, which enabled a single run to support Stage 1, demonstrating that the productivity improvements generated by our platform are able to support larger-scale clinical programs.”

Data presented in October 2023 at a Company Analyst Day demonstrated that UX701 has been well tolerated in the first dose cohort, with no unexpected related treatment emergent adverse events observed as of the data cut-off date. Four of five patients enrolled in Cohort 1 had started tapering standard-of-care treatment, including two that came completely off of chelators and/or zinc therapy. Additional interim data from all three Stage 1 dose cohorts are expected in the first half of 2024, which will be followed by dose selection and initiation of Stage 2 in the second half of 2024.