UCB, in collaboration with Novartis, has announced that minzasolmin, an investigational oral small molecule designed to inhibit alpha-synuclein misfolding, failed to meet its primary and secondary clinical endpoints in the Phase 2 ORCHESTRA trial (NCT04658186) for early Parkinson's disease.
The ORCHESTRA study was a Phase 2a, randomized, placebo-controlled, 18-month trial involving over 450 patients with early-stage Parkinson's disease. The study aimed to evaluate the efficacy, safety, tolerability, and pharmacokinetics of oral minzasolmin. The primary endpoint was the progression in the Movement Disorder Society-Unified PD Rating Scale (MDS-UPDRS) Parts I–III sum score.
Future Directions for UCB in Parkinson's Research
Following the disappointing results, UCB has decided to terminate the extension phase of the minzasolmin program. However, the company remains committed to Parkinson's disease research and is shifting its focus to other potential treatments. According to Alistair Henry, Chief Scientific Officer at UCB, the company will continue its commitment to a science and patient driven strategy to address both its causes and symptoms.
UCB is progressing UCB7583, which is under investigation for preventing extracellular alpha-synuclein spread. Additionally, UCB is advancing glovadalen (UCB0022), an orally available, brain-penetrant, small molecule designed to enhance the potency of dopamine to activate the dopamine D1 receptor and thereby improve symptom control.
Safety and Biomarker Analysis
The safety profile of minzasolmin in the ORCHESTRA study was consistent with previous knowledge, and no new safety concerns were identified. The incidence of treatment emergent adverse events was comparable across all three treatment groups (180mg/day, 360mg/day, or placebo). Disease biomarker data, in which there was a preliminary signal, is still being analyzed, and the findings from the study have been submitted to an upcoming scientific meeting and will be submitted for publication in a peer-reviewed journal.
