Novartis has entered a worldwide licensing and partnership agreement with PTC Therapeutics to develop PTC518, a potential oral therapy for Huntington's disease (HD).

The PTC518 Huntington's disease programme also encompasses related molecules.

The partnership is set to enhance Novartis' focus on neurodegenerative conditions.

PTC Therapeutics will receive an upfront payment of $1bn, with a possible further $1.9bn based on development, regulatory and sales milestones.

The partnership includes US profit sharing, with PTC receiving a 40% share and Novartis 60%, and tiered royalties on sales outside the US.

Novartis will handle PTC518’s development, production and commercialisation after the completion of the ongoing placebo-controlled segment of the PIVOT-HD study, expected in the first half of 2025.

The transaction is expected to close in the first quarter of 2025.

Novartis CEO Vas Narasimhan stated: "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."

PTC518, originating from PTC's splicing platform, is currently under investigation in the Phase II PIVOT-HD trial. Interim data announced in June 2024 showed that the treatment led to a sustainable, dose-dependent reduction in mutant Huntington protein levels in blood and cerebrospinal fluid, alongside preliminary signs of clinical benefit after 12 months.

PTC Therapeutics CEO Matthew Klein stated: "PTC will use the proceeds of this transaction to expand our splicing platform as well as to support commercial and development portfolio activities.”

In November 2024, Novartis acquired Kate Therapeutics to advance gene therapy development.