Vanqua Bio Commences Phase 1 Clinical Study with First Patient Administered VQ-101, a GCase Activator for GBA-PD and Associated Conditions

April 9, 2024 — Vanqua Bio, a clinical-stage biopharmaceutical company, has dosed the first patient in a Phase 1 clinical trial for VQ-101, an oral, brain-penetrating small molecule allosteric activator of glucocerebrosidase (GCase), in healthy volunteers and patients with different forms of Parkinson's disease (PD). The study aims to establish the pharmacodynamic effects of VQ-101, which has shown potential in preclinical research to be a promising treatment for PD, particularly for those with GBA1 mutations, the most prevalent genetic risk factor for the disease. 

Vanqua Bio's CEO, Jim Sullivan, PhD, highlighted the significance of the Phase 1 study launch, reflecting the company's commitment to clinical development based on expertise in lysosomal biology and medicinal chemistry. 

VQ-101 is being developed initially for GBA-PD, representing a precision-medicine approach targeting the largest genetically defined segment of PD patients. The compound has demonstrated the ability to activate GCase, engage therapeutic pathways, and prevent the buildup of alpha synuclein, a key protein associated with PD pathology. 

The Phase 1 trial is a randomized, double-blind, placebo-controlled study designed to assess VQ-101's safety, tolerability, pharmacokinetics, and pharmacodynamics in both healthy individuals and PD patients, regardless of GBA1 mutation status. Vanqua Bio, established in 2019 and based in Chicago, is focused on creating innovative treatments for neurodegenerative diseases, utilizing human genetics and patient-derived cellular models to identify and validate novel disease pathways. The company's efforts extend to targeting GCase for PD and addressing the overactivation of the innate immune system in neurodegenerative disorders like Alzheimer's disease.

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