Novartis bolsters Neuroscience pipeline with in-licensing of PTC518 for Huntington’s disease

Novartis announced that it has entered into a global license and collaboration agreement with PTC Therapeutics for PTC518, an HTT mRNA splice modulator with the potential to become the first oral disease-modifying therapy for Huntington’s disease.

The agreement bolsters the company’s Neuroscience pipeline and reflects its strategic focus on neurodegenerative diseases with high unmet needs.

Novartis will assume responsibility for PTC518’s development, manufacturing and commercialization following the completion of the placebo-controlled portion of the ongoing Phase II PIVOT-HD study, expected in H1 2025.

Under the terms of the agreement, Novartis will pay USD 1.0 billion upfront and up to $1.9 billion in development, regulatory and sales milestones. Novartis will also share profits in the US and pay tiered royalties on ex-US sales.

The transaction is subject to customary closing conditions.