All articles tagged with #ultragenyx
Ultragenyx's Phase III Aspire trial of apazunersen in Angelman syndrome missed both its primary endpoint and a key secondary endpoint, prompting a ~43% after-hours stock drop; the company will review the program and pursue significant expense reductions, while analysts warn commercialization could be challenging even if other Angelman studies meet endpoints.
Ultragenyx said its experimental Angelman syndrome therapy GTX-102 showed no benefit versus a sham in a large Phase 3 trial, ending earlier hopes from earlier results and creating a major hurdle for the company's profitability plans in the rare-disease space.
Shares of Ultragenyx and Mereo BioPharma plummeted after their Phase 3 trial for the brittle-bone disease treatment setrusumab failed to meet primary endpoints, despite secondary improvements in bone density, leading to significant market value losses and cautious outlooks from analysts.
Ultragenyx Pharmaceutical's experimental drug setrusumab for osteogenesis imperfecta failed to reduce fracture rates in late-stage studies, leading to a 43.5% drop in its stock price and potential discontinuation of the program, despite improvements in bone mineral density.
Ultragenyx and Mereo's late-stage clinical trial for a rare bone disease drug has failed, marking a significant setback for the development program.
The FDA has declined to approve Ultragenyx's gene therapy, highlighting ongoing challenges in biotech approvals, while other news includes AstraZeneca's promising hypertension drug and insights into FDA commissioner Marty Makary's initiatives.