Pharma BD Deal Intelligence
Novartis licensed PTC518 (now votoplam), an oral small-molecule HTT-lowering splicing modifier for Huntington's disease then in Phase II, paying $1B upfront in a deal worth up to $2.9B. The license closed March 31, 2025, after which Novartis assumed development, manufacturing and commercialization. Novartis advanced the program into the global Phase 3 INVEST-HD study (~770 early HD patients, cUHDRS at Month 36), dosing its first patient in Q2 2026 and triggering a $50M milestone payment to PTC. Phase 3 was supported by the Month-24 PIVOT-HD long-term-extension interim, which showed dose-dependent slowing of disease progression on cUHDRS (52% at 10mg, 28% at 5mg) vs. natural-history controls.