When surrogate endpoints are used for accelerated approvals in advance of more compelling functional outcomes, how long should the duration of the confirmatory studies be?

Vertex and CRISPR look beyond beta thalassemia and sickle cell disease. Also, Bicycle gets $50m up front in Novartis deal, Regeneron commits $75m initially under a Treg collaboration with Sonoma, and Jounce gets a better deal from Concentra to wave off Redx merger.

‘Clear signal’ in the ATLAS study of presymptomatic, SOD1 mutation carriers could provide evidence needed to confirm tofersen’s benefit in symptomatic ALS patients, FDA’s Teresa Buracchio says, adding that getting confirmatory evidence from the VALOR open-label extension 'could be a little more tricky.'

Cytokinetics will not conduct the new trial in heart failure with reduced ejection fraction (HFrEF) required by the FDA’s complete response letter for omecamtiv mecarbil.