Novartis will be gaining exclusive global rights to PTC Therapeutics’ mid-stage Huntington’s disease (HD) programme in a deal worth up to $2.9bn.
The candidate, PTC518, was discovered from PTC’s splicing platform and is currently being evaluated in the phase 2 PIVOT-HD trial.
Approximately one in every 10,000 to 20,000 people in the US are affected by HD, a fatal genetic disorder of the central nervous system caused by a defective gene that produces a mutated protein called HTT.
The disease primarily affects the brain and results in abnormal movements, difficulties with speech, swallowing and walking, and also causes behavioural, cognitive and motor symptoms.
There is no cure for HD and, while there are therapies approved for specific disease symptoms, there are none that delay the onset or slow disease progression.
Interim data from PIVOT-HD reported by PTC in June showed that PTC518 resulted in durable, dose-dependent reductions in blood and cerebrospinal fluid mutant HTT levels, and demonstrated early signals of dose-dependent benefit on key clinical measurements at one year.
Under the global licence and collaboration agreement, Novartis will assume responsibility for the drug’s development, manufacturing and commercialisation, following the completion of the on-going placebo-controlled portion of the study, expected to occur in the first half of 2025.
In exchange, PTC will receive an upfront payment of $1bn and will be eligible for up to $1.9bn in development, regulatory and sales milestones, a 40% share of profits and losses in the US, as well as royalties on sales outside the US.
Vas Narasimhan, Novartis’ chief executive officer, said the agreement is intended to “bolster” the company’s neuroscience pipeline and “reflects [its] strategic focus and commitment to explore new and potentially transformative approaches for neurodegenerative diseases with high unmet needs”.
Also commenting on the alliance, PTC’s chief executive officer, 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.”