BioCryst starts subject enrolment in trial of hereditary angioedema therapy

The trial will assess the safety and pharmacokinetics of Orladeyo in paediatric HAE patients.

Bristol Myers Squibb reports data from trial of Breyanzi for CLL

Data from the TRANSCEND CLL 004 trial showed that the study met the primary endpoint of complete response rate.

AC Immune reports positive data from Alzheimer’s disease vaccine trial

At week six, the ACI-24.060 vaccine elicited an anti-Abeta antibody response in ABATE’s first, low dose cohort of AD patients.

FDA accepts Intercept’s resubmitted NDA for obeticholic acid

According to GlobalData’s Pharmaceutical Intelligence Center, obeticholic acid currently has a 64% likelihood of FDA approval.

Mapping endpoints: building gene therapy trials for the future

How can gene therapy trials choose the best endpoints to navigate development and demonstrate patient value?