Swiss pharmaceutical giant Novartis (NYSE: NVS) has entered into a global license and collaboration agreement with US firm PTC Therapeutics (NASDAQ: PTCT), obtaining development, manufacturing, and commercialization rights to PTC518, an HTT mRNA splice modulator targeting Huntington’s disease.
Financial Terms and Milestones of the Agreement
Under the terms of the agreement, PTC Therapeutics will receive an upfront payment of USD 1.0 billion and is eligible for up to USD 1.9 billion in development, regulatory, and sales milestones. Additionally, PTC Therapeutics will share profits in the US and receive tiered royalties on ex-US sales, highlighting the significant investment and potential returns associated with the development of PTC518.
Huntington’s Disease and the Potential of PTC518
Huntington’s disease is a rare autosomal dominant inherited neurodegenerative disorder caused by an expanded CAG repeat tract in the HTT gene. PTC518 has the potential to reduce the accumulation of mutant huntingtin protein by decreasing HTT gene expression, thereby delaying nerve damage and disease progression. The drug was awarded fast-track status in the US in October this year, indicating its potential to become the first oral disease-modifying therapy for Huntington’s disease.
Phase II PIVOT-HD Study and Novartis’ Role
PTC518 is currently subject to the Phase II PIVOT-HD study. Upon completion of the placebo-controlled part during the first half of 2025, Novartis is expected to take over the development, manufacturing, and commercialization of PTC518, further expanding its pipeline in the treatment of rare diseases.-Fineline Info & Tech
