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Ultragenyx Announces FDA Clearance of Investigational New Drug (IND) Application for UX701, a New Gene Therapy for the Treatment of Wilson Disease
Clinical trial will utilize a single-protocol Phase 1/2/3 design UX701 manufacturing complete at commercial quality and scale using HeLa PCL technology First
About this update from Ultragenyx Pharmaceutical Inc.
[{"type":"text","content":"Clinical trial will utilize a single-protocol Phase 1/2/3 design UX701 manufacturing complete at commercial quality and scale using HeLa PCL technology First patient to be dosed in the first half of 2021 NOVATO, Calif., Jan. 21, 2021 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultra-rare genetic diseases, today announced that the U.S. Food and Drug Administration (FDA) has cleared the Investigational New Drug (IND) application for UX701, an investigational AAV9 gene therapy being evaluated for the treatment of Wilson Disease. Enrollment in a seamless single-protocol Phase 1/2/3 study is expected to begin in the first half of 2021. This will be the company’s third in-house clinical gene therapy program and the second program in the clinic with the HeLa producer cell line manufacturing system. “FDA IND clearance allows for the advancement of this new gene therapy into the clinic and brings forward the hope for a new treatment for patients with Wilson Disease. UX701 has the potential to directly address the underlying basis of disease by restoring the normal transport and excretion of copper,” said Eric Crombez, M.D., Chief Medical Officer of the Ultragenyx Gene Therapy development unit. “The seamless Phase 1/2/3 clinical trial design will allow us to efficiently evaluate safety and efficacy of UX701 before studying an optimal dose in a larger number of patients to support registration. We appreciate the agency’s support for a novel clinical trial design that could bring this important potentially new treatment to the greatest number of patients as efficiently as possible.” Study Design UX701 will be studied in a seamless, single-protocol Phase 1/2/3 clinical trial. Manufacture and testing of GMP-grade drug product to supply the clinical study are complete using the company’s proprietary HeLa 2.0 producer cell line (PCL) process at the 2,000 liter scale. Stage 1 (evaluation of initial safety and dose finding) In the first stage of the study, the safety and efficacy of three dose levels of UX701 will be evaluated in 27 patients (nine per cohort), randomized 2-to-1 (gene therapy versus placebo). The dose cohorts will be enrolled sequentially using ascending doses. The patients will be followed for...