Prothena discontinues AL amyloidosis candidate

Prothena Corp. plc (NASDAQ:PRTA) discontinued development of amyloid light-chain (AL) amyloidosis candidate NEOD001. The company said the humanized mAb targeting amyloid missed the primary and secondary endpoints in the Phase IIb PRONTO trial, and an IDMC recommended discontinuing the Phase III VITAL trial following a futility analysis.

The double-blind, international PRONTO trial evaluated NEOD001’s effect on cardiac best response as measured by N-terminal pro-brain natriuretic peptide (NT-proBNP) levels after 12 months of treatment in 129 previously treated AL amyloidosis patients with persistent cardiac dysfunction. Prothena said NEOD001 led to a 39.4% response rate vs. 47.6% for placebo. Secondary endpoints in PRONTO included Short Form-36v2 Physical Component Summary Score, 6-minute walk test (6MWT) and NT-proBNP rate of change...