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Novartis and PTC Therapeutics Forge $2.9 Billion Partnership for Huntington’s Drug PTC518

NextEdge Admin

03 Dec 2024

2 min 22 sec

PTC Therapeutics,Huntington’s,Rare disease

Novartis and PTC Therapeutics have entered into a global licensing agreement to advance PTC518, a Huntington’s disease (HD) drug candidate. Novartis will pay $1 billion upfront and up to $1.9 billion in developmental, regulatory, and sales milestones. PTC will retain a 40% profit share in the U.S. and receive double-digit tiered royalties on ex-U.S. sales. The deal is expected to close in early 2025, pending regulatory approval.

PTC518 is a small molecule drug designed to reduce mutant huntingtin protein production, aiming to prevent neuronal death and brain injury, hallmarks of HD. The orally administered drug has shown promise in the ongoing Phase II PIVOT-HD trial. Interim results in June 2024 indicated dose-dependent reductions in mutant huntingtin levels in the blood and cerebrospinal fluid, along with “favorable trends” in clinical assessments such as Total Motor Score. The FDA granted PTC518 Fast Track designation in September 2024.

PTC will complete the PIVOT-HD trial by mid-2025 before transferring responsibility for development, manufacturing, and commercialization to Novartis. This partnership reinforces Novartis’ interest in HD therapies. Previously, Novartis explored branaplam, an mRNA modulator, for HD, but discontinued the program in 2023 due to side effects. In 2024, Novartis partnered with Voyager Therapeutics in a $1.3 billion deal to develop gene therapies for HD using advanced capsid delivery platforms.

The agreement comes shortly after PTC discontinued its ALS candidate utreloxastat due to poor Phase II results. Following the announcement, PTC shares surged 20% pre-market.