In anticipation of advancing VQ-101 into later stage clinical studies, Vanqua also announced the appointment of Dr Maurizio Facheris as the company’s CMO. Credit: Shutterstock/Kateryna Kon

Chicago-based Vanqua Bio’s Parkinson’s disease drug has shown proof-of-concept in a first-in-human study.

VQ-101 is an oral, small molecule allosteric activator targeting the lysosomal enzyme glucocerebrosidase (GCase) that is undergoing evaluation as a potential treatment for Parkinson’s disease in a Phase Ia/Ib trial.

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Interim data from the randomised, single and multiple ascending dose Phase Ia trial conducted at the Centre for Human Drug Research (CHDR) in the Netherlands showed VQ-101 achieved activation of lysosomal enzyme glucocerebrosidase (GCase) by over 75% in healthy volunteers, with sustained activation observed following multiple doses.

The outcome surpassed the study’s target engagement goal of 50% activation, which Vanqua said was informed by its preclinical studies in patient-derived dopaminergic neurons in which 50% activation of GCase resulted in significant blockade of the accumulation of insoluble alpha-synuclein, one of the pathologic hallmarks of Parkinson’s disease.

VQ-101, which was found to be well-tolerated, with no dose-limiting or serious adverse events (AE) reported in the Phase Ia trial, also achieved significant levels in the cerebrospinal fluid (CSF), indicating full penetration of the central nervous system (CNS).

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Vanqua CEO Jim Sullivan commented: “These results, paired with data from our preclinical patient-derived neuronal models, support the potential for VQ-101 to stop the accumulation of alpha-synuclein in Parkinson’s patients and slow or stop disease progression.

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“We are pleased to advance to the Phase Ib portion of the study in patients with PD,” Sullivan added.

The randomised, double-blind, placebo-controlled Phase Ib trial will investigate multiple doses of VQ-101 in PD patients with or without mutations in the glucocerebrosidase (GBA) gene. Topline data from Phase Ib of the trial is anticipated in 2025.

According to GlobalData, Phase I drugs for Parkinson’s disease have a 72% phase transition success rate (PTSR) indication benchmark for progressing into Phase II.

GlobalData is the parent company of Clinical Trials Arena.

In 2019, the World Health Organization (WHO) estimated that Parkinson’s disease, a brain condition that causes problems with movement and mental health, impacted more than eight million people worldwide, reflecting a doubling in the prevalence of the disease in the preceding 25 years.

Centogene recently published initial data from its Rostock International Parkinson’s Disease (ROPAD) genetic profiling study (NCT03866603), which found that mutations in genes including GBA and leucine-rich repeat kinase-2 (LRRK2) played a role in 15% of Parkinson’s disease cases.