PTC Therapeutics, Inc. has just announced a significant exclusive global license and collaboration agreement with Novartis Pharmaceuticals Corporation for its PTC518 Huntington's disease program. As part of this deal, PTC will receive an upfront payment of $1.0 billion and is eligible to receive up to $1.9 billion in development, regulatory, and sales milestones. Additionally, PTC will share profits in the U.S. and receive double-digit tiered royalties on ex-U.S. net sales.
The PTC518 program is a leading oral disease-modifying therapy in development for Huntington's disease and is currently being studied in the ongoing Phase 2 Pivot-HD trial. Interim results reported in June 2024 demonstrated that PTC518 treatment resulted in a durable, dose-dependent reduction in blood and cerebrospinal fluid mutant huntingtin protein levels, as well as early signals of dose-dependent benefit on key clinical measurements at 12 months. Importantly, PTC518 continues to demonstrate a favorable safety and tolerability profile.
Novartis will assume responsibility for PTC518’s development, manufacturing, and commercialization following the completion of the ongoing placebo-controlled portion of Pivot-HD, expected in the first half of 2025. The companies will share U.S. profits and losses on a 40/60 basis (40% PTC and 60% Novartis).
The closing of the transaction is subject to customary closing conditions, including regulatory clearance, with the anticipated completion in the first quarter of 2025.
This collaboration with Novartis reflects PTC's expertise in developing small molecule splicing therapies and Novartis’s expertise in global development and commercialization of neuroscience therapies. PTC intends to use the proceeds of this transaction to expand its splicing platform and support commercial and development portfolio activities.
Huntington's disease is a fatal, hereditary, genetic disorder of the central nervous system caused by a defective gene that produces a toxic and damaging protein. There are currently no approved drugs that delay the onset or slow disease progression, making the potential of PTC518 significant for the HD community.
PTC Therapeutics is a global biopharmaceutical company focused on the discovery, development, and commercialization of clinically differentiated medicines for patients with rare disorders. The company's mission is to provide access to best-in-class treatments for patients who have little to no treatment options, and this agreement with Novartis represents a significant step forward in fulfilling that mission. As a result of these announcements, the company's shares have moved 14.3% on the market, and are now trading at a price of $50.17. For more information, read the company's full 8-K submission here.
