UCB’s Novartis-partnered investigational small molecule has failed to improve symptoms of Parkinson’s disease in a phase 2a trial.
The oral asset—dubbed minzasolmin—missed the study’s primary and secondary clinical endpoints, according to a Dec. 16 release.
The proof-of-concept study ORCHESTRA enrolled 496 patients with early-stage Parkinson’s, evaluating the main measure using a disease rating scale compared to baseline following up to 18 months of the investigational treatment.
Secondary endpoints included subscale ratings of symptoms and incidence of treatment-emergent adverse events (TEAEs), among others.
After seeing the results, the Belgium-based company has decided to terminate the program’s extension phase. The company is still analyzing disease biomarker data, with the study findings submitted for publication in a peer-reviewed journal.
The incidence of TEAEs was comparable across all study groups, with no new safety risks identified, according to UCB.
Minzasolmin is an alpha-synuclein misfolding inhibitor being developed in partnership with Novartis for early Parkinson’s. The Big Pharma put down $150 million in cash and offered up to $1.5 billion biobucks to co-develop minzasolmin, which differentiated itself from other alpha-synuclein candidates via oral dosing.
Multiple companies have gone after alpha-synuclein, with Biogen dumping its investigational injection cinpanemab in 2021 after missing both primary and secondary endpoints in a phase 2 trial. But the Big Biotech still touts an investigational asset targeting alpha-synuclein in its pipeline; called BIIB101, the Ionis-partnered candidate is being studied in a phase 1 multiple system atrophy trial.
Roche and Prothena delivered mixed phase 2 data for their candidate prasinezumab but pushed on, with a large phase 2b study’s primary readout expected this past September. The data have not been made publicly available yet.
UCB has yet to give up on the alpha-synuclein pathology, turning its attention to UCB7583, which is being evaluated as an inhibitor of extracellular alpha-synuclein spread. The asset is not yet listed in UCB’s online pipeline, which currently only includes clinical-stage assets.
The company also has another clinical candidate targeting Parkinson’s. The asset, dubbed glovadalen (UCB0022), is an oral small molecule designed to activate the dopamine D1 receptor and improve symptom control. Glovadalen is currently being studied in a phase 2 trial, with initial results expected in March 2025, according to ClinicalTrials.gov.