Ultragenyx mulls fate of rare disease programme on Phase III flop

Analysts are raising concerns around ASPIRE's potential readthrough to another ongoing study in Angelman syndrome involving apazunersen. Following the late-stage miss, analysts raise concerns around potential readthrough to another ongoing study in Angelman syndrome involving apazunersen. Ultragenyx will now evaluate the development programme for apazunersen, or GTX-102, in Angelman syndrome. Credit: VZ_Art / Shutterstock.com. Ultragenyx Pharmaceutical is deliberating the fate of its Angelman syndrome candidate, apazunersen, after the drug failed to show benefit in a Phase III trial. During the late-stage, single-arm ASPIRE trial (NCT06415344), the antisense oligonucleotide (ASO) therapy did not trigger a significant change from baseline in cognition…

Source: Clinical Trials Arena