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Ultragenyx Announces FDA Clearance of Investigational New Drug (IND) Application for UX016, a Sialic Acid Prodrug for the Treatment of GNE Myopathy
Program to be externally funded by a venture philanthropy agreement through clinical proof-of-concept, including the Phase 1/2 study planned to begin in the
About this update from Ultragenyx Pharmaceutical Inc.
[{"type":"text","content":"Program to be externally funded by a venture philanthropy agreement through clinical proof-of-concept, including the Phase 1/2 study planned to begin in the second half of 2026\nNOVATO, Calif., March 30, 2026 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today announced that the U.S. Food and Drug Administration (FDA) has cleared the Investigational New Drug (IND) application for UX016, an investigational small molecule prodrug of sialic acid (SA) being evaluated as a substrate replacement therapy for GNE myopathy (GNEM). GNEM is a rare, severely debilitating, inherited neuromuscular disorder caused by mutations in the GNE gene that lead to deficient SA production. The UX016 program is externally funded by a patient group through clinical proof-of-concept, including a Phase 1/2 study expected to begin in the second half of 2026. “People living with GNE myopathy face a profound and progressive loss of muscle function that affects every aspect of daily life, yet there are currently no approved treatment options to slow or alter the course of the disease,” said Coleman Kennedy, chief executive officer of the Neuromuscular Disease Foundation (NDF). “We appreciate Ultragenyx’s engagement with our community and are grateful for the innovative philanthropic support from others helping to move this program into the clinic. We look forward to continued collaboration as this study begins.” UX016 is a prodrug composed of SA and a hydrophobic fatty acid tail that enhances efficient delivery to muscle as compared to naturally occurring SA. UX016 aims to address challenges that have historically limited the efficacy of prior substrate replacement therapies. Based on preclinical data, the fatty acid tail improves UX016 distribution to muscle and other tissues and supports more efficient uptake and release of SA within muscle cells. “Advancing UX016 into the clinic marks an important milestone for the GNE myopathy community and reflects our commitment to developing an innovative approach to increase sialic acid uptake in muscle and to addressing the significant medical needs faced by this community,” said Emil Kakkis, M.D., Ph.D., chief executive officer and president of Ultragenyx. “We are pleased to initiate this work with the support of an innovative external funding model and look forward to working closely with physicia...