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Ultragenyx Announces Positive Longer-term Results from First Three Cohorts of Phase 1/2 Study of DTX301 Gene Therapy in Ornithine Transcarbamylase (OTC) Deficiency
All three patients in Cohort 3 now confirmed responders All three complete responders in the study remain clinically and metabolically stable after
About this update from Ultragenyx Pharmaceutical Inc.
[{"type":"text","content":"All three patients in Cohort 3 now confirmed responders\n All three complete responders in the study remain clinically and metabolically stable after longer-term follow-up Ultragenyx to host ASGCT recap investor conference call Friday, May 15 at 8:30 a.m. Eastern Time NOVATO, Calif., May 13, 2020 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel products for rare and ultra-rare diseases, today announced positive longer-term safety and efficacy data from the first three cohorts of the ongoing Phase 1/2 study of DTX301, an investigational adeno-associated virus (AAV) gene therapy for the treatment of ornithine transcarbamylase (OTC) deficiency. Six of nine patients in the study have responded to the gene therapy (three female, three male), including all three patients in Cohort 3 who are now confirmed responders. The three previously disclosed complete responders, who have discontinued all ammonia scavengers and liberalized their diet, remain clinically and metabolically stable after longer-term follow-up. Data from the Phase 1/2 study were presented today at the American Society of Gene & Cell Therapy (ASGCT) virtual 2020 Annual Meeting. “We are seeing durable and clinically meaningful responses to DTX301. We are extremely encouraged that the patients who have stopped alternate pathway medications and liberalized dietary restrictions continue to do very well over these longer periods of time,” said Eric Crombez, M.D., Chief Medical Officer of the Ultragenyx Gene Therapy development unit. “The recent data also reinforces the higher response rate seen with the Cohort 3 dose of 1x10^13 GC/kg. All three patients in Cohort 3 have responded to DTX301 and the treating physician of one of these patients reported that this is the best she has been health wise. This dose has been selected for the Phase 3 study, which is currently expected to initiate in the first half of 2021.” Cohort 3 Updated Data: All three patients at 1x1013 GC/kg dose now confirmed responders Patient 9 (newly confirmed responder, male):Patient 9 showed a 188 percent increase in rate of ureagenesis, from 25 percent of normal at baseline to 73 percent of normal at Week 24. He now has shown two significantly increased ureagenesis measures and is confirmed as a responder...