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Ultragenyx Announces First Patient Dosed in Phase 1/2 Clinical Study of UX053, an mRNA Therapy for the Treatment of Glycogen Storage Disease Type III
NOVATO, Calif., Dec. 01, 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., Dec. 01, 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 first patient has been dosed in its Phase 1/2 study of UX053, an investigational messenger RNA (mRNA) therapy in development for the treatment of Glycogen Storage Disease Type III (GSDIII). “Initiation of this trial paves the way for UX053 to potentially become the first medicine for people living with GSDIII, who currently are burdened with managing a complex metabolic disorder with diet alone,” said Camille L. Bedrosian, M.D., Chief Medical Officer of Ultragenyx. “We believe mRNA is a promising treatment modality for rare diseases like GSDIII because this technology can deliver large genes to targeted cells and achieve a high uniformity of protein expression. We can also individualize mRNA therapies based on the needs of each patient by adjusting the dose level and dose frequency.” UX053 is Ultragenyx’s most advanced investigational mRNA therapy. The company is developing a preclinical pipeline of mRNA therapies through its long-term collaboration with Arcturus Therapeutics Inc., a clinical-stage messenger RNA medicines company. More information about Ultragenyx’s broad portfolio of rare disease medicines can be found here: https://www.ultragenyx.com/our-research/our-pipeline/ Phase 1/2 Clinical Trial Design The two-part Phase 1/2 clinical trial will evaluate the safety, tolerability and efficacy of UX053 in adults age 18 and older with GSDIII. Part 1 is open label and will enroll up to 10 patients who will receive a single ascending dose of UX053 administered via intravenous infusion. Part 2 is double-blind and will evaluate five repeat doses at escalating dose levels in up to 16 patients across four cohorts randomized 3:1 to UX053 or placebo. The primary endpoints are treatment-emergent adverse events (TEAEs), serious TEAEs and related TEAEs in both parts of the study. Secondary endpoints include pharmacokinetic parameters. Exploratory endpoints include clinician- and patient-reported outcomes, muscle strength, blood sugar, and biomarkers of liver, cardiac and muscle health. Further details can be referenced at: https://clinicaltrials.gov/ct2/show/NCT04990388. Ab...