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Ultragenyx Announces Positive Multi-Year Durability Data from Ongoing Phase 1/2 Gene Therapy Studies and Data on HeLa 3.0 Manufacturing Platform at American Society of Gene & Cell Therapy 2021 Annual Meeting
Durable and Clinically Meaningful Responses Maintained at More than 2.5 and 3 Years Post-Treatment in Phase 1/2 Studies of DTX401 for GSDIa and DTX301 for OTC
About this update from Ultragenyx Pharmaceutical Inc.
[{"type":"text","content":"Durable and Clinically Meaningful Responses Maintained at More than 2.5 and 3 Years Post-Treatment in Phase 1/2 Studies of DTX401 for GSDIa and DTX301 for OTC Next-Generation HeLa Producer Cell Line Manufacturing Platform Results in Significant Product Yield Increases NOVATO, Calif., May 14, 2021 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for rare and ultra-rare diseases, today announced positive longer-term data from the Glycogen Storage Disease Type Ia (GSDIa) and Ornithine Transcarbamylase (OTC) Deficiency Phase 1/2 studies demonstrating ongoing durability of response, as well as data highlighting further advancements to the company’s proprietary HeLa producer cell line (PCL) manufacturing platform. Data were presented this week at the American Society of Gene & Cell Therapy (ASGCT) 24th Annual Meeting. “In the GSDIa and OTC programs, the durable responses coupled with the successful implementation of prophylactic steroids are encouraging as we move into our pivotal Phase 3 studies for these programs this year,” said Emil D. Kakkis, M.D., Ph.D., Chief Executive Officer and President of Ultragenyx. “We have also made significant progress with the latest 3.0 generation of our proprietary HeLa PCL manufacturing platform that will allow even greater product yield at the 2000 liter scale while maintaining or even improving product quality. This enables us to study larger disease indications and expand into central nervous system and muscle disorders that may require higher doses, all while continuing to drive down AAV production costs.” DTX401 (GSDIa) ProgramLonger-term Phase 1/2 data demonstrate durability of response, with sustained responses lasting more than 2.5 years since treatment All nine patients continue to demonstrate improved glucose control while tapering or discontinuing oral glucose replacement with cornstarch and improvements in energy metabolism pathways over the long term, with patients in the first cohort sustaining responses more than 2.5 years since treatment. Patients continue to taper the amount and frequency of cornstarch dosing reaching an overall mean reduction of cornstarch intake of 79% (p-value","length":2598,"tagName":"div"}]