PTC And Novartis Collab For Huntington's Disease Therapy

PTC Therapeutics, Inc. announced an exclusive global licensing and collaboration agreement with Novartis Pharmaceuticals Corporation, a subsidiary of Novartis AG, for its PTC518 Huntington's disease program. This agreement, which includes related molecules, provides PTC with an upfront payment of $1 billion, up to $1.9 billion in potential development, regulatory, and sales milestone payments, a profit-sharing arrangement in the U.S., and tiered double-digit royalties on sales outside the U.S.

Matthew B. Klein, M.D., Chief Executive Officer, PTC Therapeutics, said in a statement, "PTC518 is the leading oral disease-modifying therapy in development for Huntington's disease, and the economics of this agreement are consistent with the promise of this treatment. This collaboration combines PTC's expertise in developing small molecule splicing therapies with Novartis's expertise in global development and commercialisation of neuroscience therapies. 

He further added, “We are excited to collaborate with Novartis to accelerate the potential of PTC518 for the hundreds of thousands of HD patients worldwide in need of a therapy designed to be well-tolerated and an effective disease-modifying therapy. PTC will use the proceeds of this transaction to expand our splicing platform and support commercial and development portfolio activities."

PTC518, developed using PTC’s validated splicing platform, is currently under evaluation in the Phase 2 PIVOT-HD trial. Interim results from June 2024 revealed significant findings, including a durable, dose-dependent reduction in mutant Huntingtin protein (HTT) levels in blood and cerebrospinal fluid (CSF). Additionally, early signals of clinical benefit were observed after 12 months of treatment. The therapy has maintained a strong safety and tolerability profile.

Vas Narasimhan, CEO of Novartis, commented, "Huntington's Disease is a devastating, fatal, familial disease. This agreement with PTC is intended to bolster our neuroscience pipeline and reflects our strategic focus and commitment to explore new and potentially transformative approaches for neurodegenerative diseases with high unmet needs. We look forward to building on our expertise in neurodegenerative diseases and experience in HD to advance this potential first-in-class oral therapy for the HD community."

Once the placebo-controlled segment of the PIVOT-HD trial concludes, expected in the first half of 2025, Novartis will oversee the development, manufacturing, and commercialisation of PTC518. In the U.S., profits and losses will be shared on a 40/60 basis, with PTC receiving 40% and Novartis 60% of the profits. The agreement is subject to customary closing conditions, including regulatory approvals, and is anticipated to finalise in the first quarter of 2025.