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Ultragenyx Announces FDA Clearance of Investigational New Drug (IND) Application for UX053, an mRNA for the Treatment of Glycogen Storage Disease Type III
NOVATO, Calif., March 08, 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., March 08, 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 UX053, an investigational mRNA therapy being evaluated for the treatment of Glycogen Storage Disease Type III (GSDIII). Enrollment in a Phase 1/2 study is expected to begin in the second half of 2021. “FDA IND clearance paves the way for UX053 to enter the clinic as the first possible pharmacologic treatment option for patients with GSDIII,” said Camille L. Bedrosian, M.D., Chief Medical Officer of Ultragenyx. “UX053 is the most advanced of our investigational mRNA therapies, with multiple others in earlier preclinical development, and will be one of the first mRNA programs to enter clinical development for rare genetic diseases.” Ultragenyx is developing UX053 and a number of other mRNA therapies in the preclinical stage for undisclosed indications. These mRNA therapies come out of a long-term collaboration with Arcturus Therapeutics, a clinical-stage messenger RNA medicines company. Study Design The two-part Phase 1/2 clinical trial will evaluate the safety, tolerability, and efficacy of UX053 in adults with GSDIII. Part 1 is open label and will enroll up to 12 patients who receive a single ascending dose of UX053. Part 2 is a randomized, double-blind, placebo-controlled multi-ascending study of 5 doses. It will enroll up to 16 patients across four cohorts randomized 3:1 to UX053 or placebo. In addition to safety, tolerability, and pharmacokinetics, study endpoints include clinician- and patient-reported outcomes, muscle strength, and biomarkers of liver, cardiac, and muscle health. About Glycogen Storage Disease Type III Our preclinical candidate UX053 is being developed for the treatment of GSDIII, a disease caused by a glycogen debranching enzyme (AGL) deficiency that results in glycogen accumulation in the liver and muscle. GSDIII can cause hepatomegaly, hypoglycemia, hyperlipidemia, some progressive liver cirrhosis, and skeletal and cardiac muscle disease. There are no approved treatment options for GSDIII and the current standard of care is a strict diet, i...