Business
Ultragenyx to Present GTX-102 Angelman Syndrome Program Update at the ASF Family Conference and Research Symposium
Presentations will include an encore of the positive interim Phase 1/2 results presented in April at the 76th Annual American Academy of Neurology (AAN)
About this update from Ultragenyx Pharmaceutical Inc.
[{"type":"text","content":"Presentations will include an encore of the positive interim Phase 1/2 results presented in April at the 76th Annual American Academy of Neurology (AAN) Meeting\nNOVATO, Calif., July 24, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today announced that the company will share the latest clinical data, regulatory progress and program next steps for GTX-102, its investigational antisense oligonucleotide for Angelman syndrome, on Wednesday, July 24, at the 2024 Angelman Syndrome Foundation (ASF) Family Conference and Research Symposium in Sandusky, Ohio. “Since our positive interim Phase 1/2 data presentation at AAN in April, we have continued to see the patients in our study develop new skills across multiple domains with no new serious adverse events,” said Eric Crombez, M.D., chief medical officer at Ultragenyx. “We look forward to working with the ASF and the broader community as we prepare to initiate a global randomized study by the end of this year.” The ASF data presentation will review the previously disclosed Phase 1/2 results presented at AAN and an update on plans to initiate a Phase 3 pivotal trial by the end of the year. ASF 2024 Presentation Details: Title: Developing Treatments for Complex Rare DiseasesSession: Industry Introduction with UltragenyxPresenter: Emil D. Kakkis, M.D., Ph.D., chief executive officer and president of UltragenyxDate / Time: Wednesday, July 24, 2024, at 3:10 pm Eastern Time Title: GTX-102 Angelman Program UpdateSession: Industry Overview and UpdatesPresenter: Kim Goodspeed, M.D., medical director, global clinical development at UltragenyxDate / Time: Wednesday, July 24, 2024, at 3:40 pm Eastern Time Both presentations will be livestreamed from the ASF website: https://asfconference.org/family-conference/ About GTX-102GTX-102 is an investigational antisense oligonucleotide delivered via intrathecal administration and designed to target and inhibit expression of UBE3A-AS. Nonclinical studies have shown that GTX-102 reduces levels of UBE3A-AS and reactivates expression of the paternal UBE3A allele in neurons of the central nervous system (CNS). Reactivation of paternal UBE3A expression in animal models of Angelman syndrome has been associated with improvements in some of the neurological symptoms associated with the condition. GTX-102 has been granted Orphan Drug Design...