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GeneTx and Ultragenyx Announce FDA has Removed Clinical Hold on Phase 1/2 Clinical Study of GTX-102 for the Treatment of Angelman Syndrome in the U.S.
SARASOTA, Fla. and NOVATO, Calif., Sept. 27, 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., Sept. 27, 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 U.S. Food and Drug Administration (FDA) has removed the clinical hold and GeneTx may begin dosing naïve patients in the Phase 1/2 study of GTX-102 in pediatric patients with Angelman syndrome. GeneTx previously received clearance, under a separate amendment, to begin the Phase 1/2 study in the United Kingdom and Canada. “The dosing protocol across the three regions is designed to provide a broad picture on dose response to inform loading and maintenance regimens as we move to the next phase of development,” said Scott Stromatt, M.D., Chief Medical Officer of GeneTx. “We are working with urgency to begin treating study participants in all three regions over the next several months recognizing that there is no approved therapy available for the Angelman community.” Phase 1/2 study design in the United StatesThe Phase 1/2 open-label, multiple-dose study evaluates the safety, tolerability, and plasma and cerebrospinal fluid concentrations of GTX-102 in pediatric patients with Angelman syndrome with a genetically confirmed diagnosis of full maternal UBE3A gene deletion. Naïve Patient Dose-loading PhaseUnder the amended U.S. protocol, eight patients (4 to Maintenance PhaseAll U.S. patients who have completed the dose-loading phase will then move to a maintenance phase during which they will receive 2 mg of GTX-102 every three months and continue to be monitored for response and safety. Phase 1/2 study design in U.K. and Canada Under the protocol approved in the U.K. and Canada, approximately 12 patients will be enrolled into two cohorts split by age: patients ages 4 to The starting doses in Cohorts 4 and 5 will be 3.3 and 5 mg, respectively. Patients will receive three to four monthly doses, titrated individually through smaller steps than the first three cohorts in the original study with dose increases based on response and enhanced safety monitoring. Patients will then move to a maintenance phase during which they will receive GTX-102 every three months and continue to be monitored for response and safety. In this phase, individual dose titration may continue i...