NasdaqGS:RAREBiotechs
Trial Setback Might Change The Case For Investing In Ultragenyx Stock (RARE)
Ultragenyx Pharmaceutical reported that the Phase 3 Aspire trial of apazunersen in Angelman syndrome did not meet primary or key secondary endpoints. The company confirmed a safety profile consistent with earlier studies and signaled plans to cut expenses and reassess the program.
At the same time, Ultragenyx secured FDA approval and a priority review voucher for its GENGLYCOS gene therapy. The company also published 96 week Phase 3 data showing large cornstarch reductions with maintained...