BELGIUM – Novartis is putting US$150 million into a Parkinson’s disease drug candidate that is competing with drugs in development at companies such as Roche and AstraZeneca.
Novartis will pay the aforesaid sum upfront as part of the agreement, and the two companies will split the costs and responsibilities for developing the drug, known as UCB0599.
Novartis and UCB will collaborate on the development of an experimental pill that will target an abnormal protein that accumulates in Parkinson’s disease patients’ brains in order to slow their decline.
The agreement, also gives the Swiss pharmaceutical company the option to in-license a second experimental Parkinson’s treatment that targets the same protein.
If the drug is approved, UCB will market it in Europe, while Novartis will market it globally. In addition, Novartis has agreed to pay UCB up to US$1.5 billion in milestone payments in exchange for the opportunity to co-develop a midphase Parkinson’s prospect.
Drugmakers have attempted to prevent alpha-synuclein accumulation, as they did with the protein amyloid beta in Alzheimer’s disease, by using injectable or intravenous monoclonal antibodies that bind to alpha-synuclein.
UCB’s medication, on the other hand, is a small molecule option that can be taken as a pill. Novartis touted UCB0599 as having the “potential to transform care” as the first oral disease-modifying treatment.
UCB0599, which was originally in-licensed from privately held Neuropore Therapies, inhibits the “misfolding” of alpha-synuclein, which leads to its abnormal structure. Several companies have pursued alpha-synuclein, with varying degrees of success.
After seeing underwhelming phase 2 data, Biogen decided to sell its asset. Roche and Prothena delivered mixed midphase data but chose to press on, propelling themselves to the front of the pack.
While the alpha-synuclein approach has had setbacks, UCB and Novartis may face competition from partners Roche and Prothena, as well as Seelos Therapeutics, a neurological disease-focused biotech.
The experimental drug prasinezumab developed by Roche and Prothena failed in a Phase 2 study in early Parkinson’s patients, but the companies have launched a new trial in patients who are stable on levodopa, the standard treatment for the disease.
UCB approaches alpha-synuclein from a different angle than some of its key competitors, with a small-molecule approach that allows for oral dosing.
The Belgian biopharma firm published phase 1b data on the candidate earlier this year, indicating a manageable safety and tolerability profile.
In phase 1 testing, the drug reached brain tissue and showed promising biomarker signs in healthy volunteers. A Phase 2 trial is currently underway to see if it has any effect on movement disorders or cognitive decline in patients with early Parkinson’s disease.
Meanwhile, Seelos is using CRISPR gene editing technology to correct a mutation in the SNCA gene, which encodes alpha-synuclein. Although the drug has not been tested in humans, laboratory tests have shown that it can reduce genetic and protein expression in rodents.
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