PTC Therapeutics has inked a global license and collaboration agreement with Novartis for its PTC518 Huntington's disease program, including related compounds.
The agreement grants PTC an upfront payment of $1 billion, with potential milestone payments of up to $1.9 billion. Additionally, PTC will share U.S. profits at a 40/60 split with Novartis and receive tiered double-digit royalties on sales outside the U.S.
Novartis will take over global development, manufacturing, and commercialization of PTC518 following the completion of the ongoing placebo-controlled portion of the PIVOT-HD trial, expected in the first half of 2025. Interim results from the phase 2 trial, reported earlier this year, showed dose-dependent reductions in mutant Huntingtin protein levels and early clinical benefits, alongside a favorable safety profile.
The transaction is subject to regulatory approvals and other customary closing conditions, with the parties anticipating completion in the first quarter of 2025. Under the agreement, PTC plans to use the proceeds to expand its splicing platform and support its commercial and development activities.
PTC518, an oral therapy designed to modify Huntington's disease progression, has demonstrated promise in clinical studies and represents a potential advance for patients with this unmet medical need.