Swiss pharmaceutical company Novartis has entered into a global license and collaboration agreement with US drugmaker PTC Therapeutics for PTC518 to treat Huntington’s disease.
Under the terms of the agreement, PTC will receive $1bn in upfront payment, and up to $1.9bn in development, regulatory and sales milestone payments.
Novartis will get rights to develop, manufacture and commercialise PTC518, after completing the ongoing Phase 2 PIVOT-HD study, which is expected in the first half (H1) of 2025.
Also, the Swiss drugmaker will share 40% of its profits in the US with PTC, along with double-digit tiered royalties on net sales of the drug outside the US.
The transaction is expected to be completed in the first quarter of 2025, subject to customary closing conditions, including regulatory approvals.
Novartis said the licence agreement will support its neuroscience pipeline and reflects its strategic focus on neurodegenerative diseases with high unmet needs.
Novartis CEO Vas Narasimhan said: “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 exploring 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 with the intention to advance this potential first-in-class oral therapy for the HD community.”
PTC518 is an oral disease-modifying therapy currently in development for Huntington’s disease, with the potential to become the first oral disease-modifying therapy.
PTC discovered PTC518 from its validated splicing platform and is currently being studied in the ongoing Phase 2 PIVOT-HD clinical trial.
In the interim results reported in June this year, PTC518 showed a durable, dose-dependent reduction in blood and cerebrospinal fluid (CSF) mutant Huntingtin protein (HTT) levels.
The drug also showed early signals of dose-dependent benefit on key clinical measurements at 12 months, along with a favourable safety and tolerability profile, said the US drugmaker.
PTC Therapeutics CEO Matthew Klein said: “This collaboration combines PTC’s expertise in developing small molecule splicing therapies with Novartis’s expertise in global development and commercialisation of neuroscience therapies.
“We are excited to collaborate with Novartis to accelerate the potential of PTC518 for the hundreds of thousands of HD patients worldwide in need of a therapy designed to be well-tolerated and an effective disease-modifying therapy.
“PTC will use the proceeds of this transaction to expand our splicing platform as well as to support commercial and development portfolio activities.”
