PTC Therapeutics Inc. has signed an exclusive global license and collaboration agreement with Novartis Pharmaceuticals Corporation, a subsidiary of Novartis AG, for its PTC518 Huntington’s disease program, which includes related molecules.
 
Under the agreement, PTC will receive an upfront payment of $1.0 billion, up to $1.9 billion in development, regulatory and sales milestones, a profit share in the U.S., and double-digit tiered royalties on ex-U.S. sales.
 
“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,” said Matthew B. Klein, M.D., CEO, PTC Therapeutics. “This collaboration combines PTC’s expertise in developing small molecule splicing therapies with Novartis’s expertise in global development and commercialization of neuroscience therapies. 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 as well as to support commercial and development portfolio activities.”
 
“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,” said Vas Narasimhan, CEO of Novartis. “We look forward to building on our expertise in neurodegenerative diseases and experience in HD with the intention to advance this potential first in class oral therapy for the HD community.”
 
PTC518 is under investigation in the ongoing Phase 2 PIVOT-HD trial. Interim results released in June 2024 indicated that treatment with PTC518 led to a sustained, dose-dependent decrease in both blood and cerebrospinal fluid (CSF) levels of the mutant Huntingtin protein (HTT), along with initial indications of a dose-dependent advantage in key clinical metrics after 12 months. Notably, 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 on-going placebo-controlled portion of PIVOT-HD, which is expected to occur in H1 2025. 
 
The closing of the transaction is subject to customary closing conditions, including regulatory clearance. The parties anticipate that the agreement will close in the first quarter of 2025.