Business
Ultragenyx Announces Positive Data from Confirmatory Cohort of Phase 1/2 Study of DTX401 Gene Therapy for Glycogen Storage Disease Type Ia (GSDIa)
All Patients in Cohort 3 Demonstrate Increased Time to Hypoglycemia and a Substantial Reduction in Cornstarch Use Introduction of Continuous Glucose
About this update from Ultragenyx Pharmaceutical Inc.
[{"type":"text","content":"All Patients in Cohort 3 Demonstrate Increased Time to Hypoglycemia and a Substantial Reduction in Cornstarch Use\n Introduction of Continuous Glucose Monitoring Reveals Early Transgene Expression and Enables More Rapid Reduction in Cornstarch Ultragenyx to Host ASGCT Recap Investor Conference Call today at 8:30 a.m. Eastern Time NOVATO, Calif., May 15, 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 initial data from the confirmatory third cohort and longer-term data from the first two cohorts of the ongoing Phase 1/2 study of DTX401, an adeno-associated virus (AAV) based gene therapy for the treatment of glycogen storage disease type Ia (GSDIa). All three patients in Cohort 3 responded to therapy and demonstrated more rapid reductions in cornstarch requirements compared to the first two cohorts. Across all cohorts, 100 percent of patients (n=9) demonstrated meaningful and sustained cornstarch reductions over time and significant increases in time to hypoglycemia. Data from the Phase 1/2 study are being presented today at the American Society of Gene & Cell Therapy (ASGCT) virtual meeting. “In the confirmatory third cohort, continuous glucose monitoring showed early transgene expression and enabled faster and more accurate reductions in cornstarch compared with prior cohorts,” said Eric Crombez, M.D., Chief Medical Officer of the Ultragenyx Gene Therapy development unit. “The use of continuous glucose monitoring offers the ability to better understand the improvement in glucose metabolism after treatment with DTX401 over the course of the entire day and on an ongoing basis.” \"We are extremely excited by the results being obtained in this first in human gene therapy trial for glycogen storage disease type Ia,” said David Weinstein, M.D., M.M.Sc., Professor and Director, Glycogen Storage Disease Program at Connecticut Children’s Medical Center and UConn Health. “The third cohort of patients is experiencing more rapid improvement with fasting and tolerating quicker reduction in their cornstarch therapy when compared with the prior cohorts. Most importantly, the benefits seen for patients are being maintained, and the majority of treated patients are now o...