Novartis takes aim at risky Parkinson's target with $1.5bn UCB deal

Novartis has licensed a potential drug for Parkinson's from Belgium's UCB that it thinks could be the first oral, disease-modifying drug for the disease – if it can avoid the fate of earlier drugs in the class.

The drug – called UCB0599 – targets a protein called alpha synuclein that tends to get misfolded and accumulates into clumps in the brains of Parkinson's patients, and is thought to damage neurons – somewhat analogous to amyloid and tau proteins in Alzheimer's disease.

The Swiss group is paying $150 million upfront for co-development rights to UCB0599 in a deal that could be worth $1.5 billion if the drug reaches the market and hits sales objectives.

UCB0599 is in phase 2 development, and Novartis is also taking an option on a follow-up compound called UCB7853 in phase 1. The two companies will split the cost of development and – if approved – the drug will be sold by UCB in Europe and Novartis in the rest of the world.

Novartis says alpha-synuclein misfolding is "the most prominent neuropathological hallmark of [Parkinson's] and a primary step in disease progression." In animal models of the disease, UCB0599 reduced alpha-synuclein pathology and neurodegeneration, and improved motor function.

It all sounds great in theory, and if UCB0599 works in trials it could offer a real advance on current therapies for Parkinson's which remain largely based on levodopa, which replaces the neurotransmitter dopamine that is diminished as neurons in the brain die off.

The track record of drugs targeting alpha-synuclein isn't that good however. Biogen jettisoned cinpanemab, an antibody against the target, earlier this year after it missed the mark in a phase 2 trial.

Another antibody from Roche and Prothena – prasinezumab – also disappointed in one phase 2 trial, but is still being developed in another phase 2b study called PADOVA involving early Parkinson’s disease patients who are on stable symptomatic medication.

AbbVie’s antibody ABBV-0805 cleared a phase 1 trial but seems to have been side-lined for strategic reasons, and it also abandoned a gene therapy candidate licensed from Voyager Therapeutics.

Other companies with active alpha-synuclein antibodies include AstraZeneca and Takeda with MEDI 1342 in phase 2 for Parkinson's and Lundbeck with Lu AF82422 – although it is initially targeting another neurodegenerative disease called multiple system atrophy.