UCB Parkinson’s candidate minzasolmin misses goal

Belgian drugmaker UCB (Euronext: UCB) today announced the ORCHESTRA proof-of-concept study of minzasolmin - an investigational, oral small molecule, alpha-synuclein misfolding inhibitor - developed in partnership with Novartis (NOVN: VX) for early Parkinson’s disease, did not meet its primary and secondary clinical endpoints.

UCB noted that it has a portfolio of early phase pre-clinical and clinical programs evaluating multiple and distinct potential new treatment approaches in Parkinson’s disease. The misfolding and aggregation of alpha-synuclein is a key pathological driver of Parkinson’s disease, therefore, in addition to minzasolmin, which focused on inhibiting intracellular alpha-synuclein misfolding, UCB is progressing UCB7583, currently under investigation for preventing extracellular alpha-synuclein spread. Going beyond alpha-synuclein pathology, UCB is progressing glovadalen (UCB0022), an orally available, brain-penetrant, small molecule, designed to enhance the potency of dopamine ‘when and where needed’ to activate the dopamine D1 receptor and thereby improve symptom control.

UCB paid $150 million in upfront cash to Novartis and offered up to $1.5 billion to co-develop minzasolmin, which differentiated itself from other alpha-synuclein candidates via oral dosing.