UCB, a biopharmaceutical company based in Belgium, has experienced a significant setback in its efforts to develop a treatment for Parkinson's disease.
The company's investigational small molecule, minzasolmin, which was being developed in collaboration with Novartis, did not meet its primary and secondary clinical endpoints in a phase 2a trial.
The trial, called ORCHESTRA, involved 496 patients with early-stage Parkinson's disease and aimed to assess the drug's efficacy over a period of up to 18 months.
Unfortunately, the results showed that minzasolmin did not improve symptoms as intended, leading UCB to terminate the program's extension phase.
Despite the disappointing outcome, UCB reported that the incidence of treatment-emergent adverse events (TEAEs) was similar across all study groups, and no new safety risks were identified.
The company is currently analyzing disease biomarker data from the trial, and the findings will be submitted for publication in a peer-reviewed journal.
The failure of minzasolmin has significant financial implications, considering the substantial investment from Novartis.
UCB had received an initial cash infusion of $150 million and the potential for up to $1.5 billion in co-development biobucks.
Despite this setback, UCB remains committed to exploring the alpha-synuclein pathology.
The company is now focusing on UCB7583, an asset being evaluated as an inhibitor of extracellular alpha-synuclein spread.
UCB7583 is not yet listed in the company's online pipeline, but it represents continued investment in the pursuit of effective treatments for Parkinson's disease.
Additionally, UCB is advancing another clinical candidate, glovadalen (UCB0022), which aims to activate the dopamine D1 receptor to improve symptom control.
This oral small molecule is currently undergoing a phase 2 trial, and initial results are expected in March 2025.
UCB's challenges in Parkinson's disease research are not unique, as other companies have also faced setbacks.
Biogen, for example, discontinued its investigational injection cinpanemab in 2021 after it failed to meet primary and secondary endpoints in a phase 2 trial.
However, Biogen continues to explore other avenues, including an investigational asset called BIIB101, which targets alpha-synuclein and is currently being studied in a phase 1 trial for multiple system atrophy.
Roche and Prothena have also encountered mixed results with their candidate prasinezumab, but they have chosen to continue their research.
A large phase 2b study's primary readout for prasinezumab is expected to be released soon.
The competitive landscape in Parkinson's disease research remains dynamic, with various companies striving to develop effective therapies targeting alpha-synuclein, a protein implicated in the pathology of the disease.
As the industry grapples with these challenges, innovative approaches and the exploration of new candidates will be crucial in the ongoing battle against Parkinson's disease.