Novartis, through subsidiary Novartis Pharmaceuticals Corporation, has signed an exclusive global license and collaboration agreement with PTC Therapeutics for its Huntington's disease program.
Per the deal, Novartis will hand PTC an upfront payment of $1 billion and 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.
The deal centers on PTC518, a potential first in class oral therapy currently being studied in the ongoing phase 2 PIVOT-HD trial. PTC518 was discovered from PTC's validated splicing platform, which was also used to identify the first-ever approved small molecule splicing modifier, Evrysdi, which got the green light in 2020 to treat spinal muscular atrophy. Interim results reported back in June demonstrated that treatment with PTC518 resulted in 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.
Novartis says it will take over PTC518's development, manufacturing and commercialization, following the completion of the ongoing placebo-controlled portion of PIVOT-HD, which is expected to occur in early 2025.
The deal is good news for PTC, who just announced last week that it is halting development of its oral small molecule, utreloxastat, after the drug failed to meet its primary endpoint in a phase 2 ALS study.