PTC adds $1B up front, ‘more muscle’ for Huntington program via Novartis deal

Novartis’ decision to spend $1 billion up front for rights to PTC’s therapy for Huntington disease is a wager on a small molecule built from the same platform as Roche’s blockbuster Evrysdi — now the best-selling of three therapies for SMA from different modalities.

One of multiple approaches to treating Huntington disease, PTC518 from PTC Therapeutics Inc. (NASDAQ:PTCT) modulates pre-mRNA splicing to correct repeats in the HTT gene. The treatment has a chance to be “the first approved disease-modifying therapy for HD,” possibly via the accelerated approval pathway, CEO Matthew Klein said on a conference call Monday morning...