Sometimes intent-to-treat analyses don't tell the full story. Though tafamidis missed the co-primary endpoints of a Phase II/III trial last July, FoldRx Pharmaceuticals Inc. argued the results would support approval of the small molecule to treat familial amyloid polyneuropathy (FAP).

Eleven months later, a group of VCs led by new investors Novo Ventures and Morgenthaler Ventures has agreed. Last week, they ponied up $29 million in a round designed to carry the program to