The US firm’s oral plasma kallikrein inhibitor was associated with liver elevations in a number of Phase II patients, prompting a trial termination and shift in focus to an on-demand program. 

Having led $30bn of acquisitions of companies at Sanofi, the new challenge for its former global head of partnering is to help make Owkin, “the most disruptive AI biotech company in the world,” he tells Scrip.

With the expectation for increasing amounts of patient experience data comes the need for the FDA and sponsors to consider, and account for, the degree to which open-label bias influences oncology trial PROs; agency staff suggest trial design elements and analytic approaches for dealing with bias at a meeting on cancer clinical outcomes assessments.

While many HAE patients converted from injectable prophylaxis to BioCryst’s oral Orladeyo last year, physicians said a promising injectable candidate from Ionis could benefit from varied patient preference.