Business
GeneTx and Ultragenyx Announce First Patient Dosed in Canada in Phase 1/2 Clinical Trial of GTX-102 in Patients with Angelman Syndrome
SARASOTA, Fla. and NOVATO, Calif., Oct. 07, 2021 (GLOBE NEWSWIRE) -- GeneTx Biotherapeutics LLC and Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), companies
About this update from Ultragenyx Pharmaceutical Inc.
[{"type":"text","content":"SARASOTA, Fla. and NOVATO, Calif., Oct. 07, 2021 (GLOBE NEWSWIRE) -- GeneTx Biotherapeutics LLC and Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), companies partnered in the development of GTX-102, an investigational treatment for Angelman syndrome, today announced that the first patient has been dosed in the Phase 1/2 study in Canada with three other patients enrolled and scheduled to begin dosing. “We have been actively working with our principal investigators across all three regions over the past several months to efficiently execute this Phase 1/2 trial and can now move forward rapidly with study enrollment,” said Scott Stromatt, M.D., Chief Medical Officer of GeneTx. “This is an in-depth study to evaluate both safety and measures of efficacy across multiple domains in Angelman Syndrome that will inform us on the optimal endpoints to evaluate in the registrational program.\" The open-label, multiple-dose, dose-escalating Phase 1/2 study is evaluating the safety, tolerability and efficacy of GTX-102 in pediatric patients with Angelman syndrome who have a genetically confirmed diagnosis of full maternal UBE3A gene deletion. The study will evaluate the overall clinical global impression scale of change after the first two doses, along with a panel of other efficacy assessments for the domains impacted in Angelman, including communication, sleep, behavior, gross motor skills, fine motor skills, and seizures, which will be measured at Day 128. “The dosing schedule across the three arms of this study, in the U.K., Canada, and the U.S., is designed to achieve a cumulative dose range similar to the original study where we saw impact across domains beginning at the lowest dose of 3.3 mg and meaningful improvements at 13-20 mg of cumulative dosing in younger patients,” said Emil D. Kakkis, M.D., Ph.D., Chief Executive Officer and President of Ultragenyx. “Based on the prior data, we believe this study should contribute meaningfully to our understanding of dose response and safety in this program.” About the GTX-102 Phase 1/2 Study Design in Canada and the U.K. Under the protocol approved in the U.K. and Canada, 12 patients will be enrolled into two cohorts split by age: patients ages 4 to","length":2824,"tagName":"div"}]