Novartis bolsters Neuroscience pipeline with in-licensing of PTC518 for Huntington's disease
Basel: Novartis has announced that it has entered into a global license and collaboration agreement with PTC Therapeutics for PTC518.
PTC518 is an HTT mRNA splice modulator with the potential to become the first oral disease-modifying therapy for Huntington's disease, according to Novartis.
"The agreement bolsters the company’s Neuroscience pipeline and reflects its strategic focus on neurodegenerative diseases with high unmet needs," the Company stated.
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.
Huntington's disease is a progressive, inherited neurodegenerative disorder caused by a mutation in the HTT gene, leading to the production of a toxic protein that damages brain cells. It is characterized by motor symptoms such as involuntary movements (chorea), impaired coordination, and difficulty swallowing; cognitive decline affecting memory, problem-solving, and decision-making; and psychiatric symptoms like depression, irritability, and anxiety. The disease typically manifests in adulthood, usually between ages 30 and 50, and is inherited in an autosomal dominant manner.
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.
Medical Dialogues team had earlier reported that the European Commission (EC) approved Novartis's Kisqali (ribociclib) in combination with an aromatase inhibitor (AI) for the adjuvant treatment of patients with hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2)-negative early breast cancer (EBC) at high risk of recurrence.
