NEW YORK – PTC Therapeutics on Monday said it inked an exclusive global license and collaboration agreement with Novartis Pharmaceuticals for PTC518, a messenger RNA degrader designed to treat Huntington's disease, and related molecules.
Under the terms of the deal, Basel, Switzerland-based Novartis will pay PTC $1 billion upfront in cash and up to $1.9 billion in development, regulatory, and sales milestones.
Novartis will be responsible for developing, manufacturing, and commercializing PTC518 after PTC completes the placebo-controlled portion of the ongoing Phase II PIVOT-HD trial. PTC will share profits and losses with Novartis for a product commercialized in the US on a 40/60 basis, respectively. PTC is also eligible to receive tiered double-digit royalties on net sales outside of the US.
"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," Novartis CEO Vas Narasimhan said in a statement.
The companies expect the transaction to close in Q1 2025. PTC expects to complete the placebo-controlled portion of the PIVOT-HD trial in the first half of 2025.
Huntington's disease is a neurodegenerative condition caused by certain mutations in the HTT gene. Warren, New Jersey-based PTC developed PTC518 as an oral medication that is designed to degrade HTT messenger RNA, and it is currently being tested in patients with genetically confirmed Huntington's disease with a CAG repeat length of 40 to 50.
PTC in June said that interim results from the PIVOT-HD trial demonstrated durable, dose-dependent reductions in mutant HTT protein levels in blood and cerebrospinal fluid after treatment, as well as a favorable safety and tolerability profile.
PTC said it will use proceeds from the transaction to expand the platform it has developed to identify small molecules that affect mRNA splicing, which it used to discover PTC518, and to support other commercial and development portfolio activities.
"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," PTC CEO Matthew Klein said in a statement. "This collaboration combines PTC's expertise in developing small molecule splicing therapies with Novartis' expertise in global development and commercialization of neuroscience therapies."