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Phase 3 INVEST-HD: randomized, placebo-controlled study of votoplam in Huntington's disease; sponsor Novartis; ~770 participants; start 2026-03-24; primary…
Month-24 PIVOT-HD LTE: dose-dependent slowing on cUHDRS in Stage 2 HD (52% at 10mg, 28% at 5mg) vs natural-history cohort; votoplam = former PTC518.
First patient dosed in Phase 3 INVEST-HD study, triggering $50 million milestone payment from Novartis to PTC in Q2 2026.
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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.
| Deal | Year | Value | Outcome |
|---|---|---|---|
| Novartis AG / PTC Therapeutics Inc. (this deal) | 2024 | $2.9B | 80 |
| Novartis AG / Advanced Accelerator Applications S.A. | 2017 | $3.9B | 98 |
| Novartis AG / Endocyte, Inc. | 2018 | $2.1B | 96 |
| Novartis AG / Lek Pharmaceuticals d.d. | 2002 | $876M | 88 |
| Novartis AG / GlaxoSmithKline plc | 2014 | $16.0B | 81 |
| Novartis AG / Chinook Therapeutics Inc. | 2023 | $3.5B | 78 |
| Novartis AG / Hexal AG | 2002 | $8.3B | 75 |
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