- Novartis and UCB will co-develop an experimental pill that aims to slow the decline of Parkinson's disease patients by targeting an abnormal protein that accumulates in their brains. The deal, announced Thursday, also gives the Swiss drugmaker an option to in-license a second experimental Parkinson's treatment that acts on the same protein.
- Under the deal, Novartis will pay UCB $150 million upfront and the two companies will split the costs and responsibilities for developing the drug, called UCB0599. If the drug gains approval, UCB will commercialize it in Europe while Novartis markets it in the rest of the world. UCB could also earn up to $1.5 billion in conditional payments tied to development, regulatory and sales milestones.
- UCB0599 is one of several experimental projects looking to block the effects of abnormal alpha-synuclein in the brains of Parkinson's patients. Some have already faced setbacks or outright failures, including projects from Biogen, Prothena and Voyager Therapeutics.
Physicians can primarily treat the symptoms of Parkinson's disease with drugs like levodopa, but have no pharmaceutical options for tackling the underlying cause of the disorder. In recent years, the focus has been on targeting alpha-synuclein, which in many Parkinson's patients is abnormal and accumulates in brain tissues. The protein is also present in the brains of some patients with dementia and Alzheimer's disease.
As with the protein amyloid beta in Alzheimer's, drugmakers have sought to block accumulation through injectable or intravenous monoclonal antibodies that bind to alpha-synuclein. UCB's drug, on the other hand, is small molecule option that can be administered as a pill. In its research and development day Thursday, Novartis touted UCB0599 as having the "potential to transform care" as a first oral disease-modifying treatment.
Originally in-licensed from privately held Neuropore Therapies, UCB0599 inhibits the "misfolding" of the alpha-synuclein that leads to its abnormal structure. Phase 1 testing in healthy volunteers revealed the drug reached brain tissue and showed encouraging biomarker signs. A Phase 2 trial now underway will measure whether it has any affect on movement disorders or cognitive decline in early Parkinson's patients.
While the alpha-synuclein approach has seen setbacks, UCB and Novartis could face competition from partners Roche and Prothena, as well as a neurological disease-focused biotech called Seelos Therapeutics.
Roche and Prothena's experimental drug, called prasinezumab, failed in a Phase 2 study in early Parkinson's patients, but the pair have initiated another trial in patients who are stable on levodopa, the mainstay of treatment for the disease.
Seelos, meanwhile, is using CRISPR gene editing technology to alter the mutation in a gene called SNCA that encodes alpha-synuclein. That project has not been tested in humans, but laboratory tests have revealed that the drug can reduce genetic and protein expression in rodents.