ARTICLE | Deals
Biotech retains upside in licensing agreement for oral therapy derived from same splicing platform as SMA drug
By Paul Bonanos, Director of Biopharma Intelligence
December 2, 2024 11:06 PM UTC
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…
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- Source: https://www.biocentury.com/article/654354/ptc-adds-1b-up-front-more-muscle-for-huntington-program-via-novartis-deal