Vanqua Bio Advances Experimental Parkinson’s Therapy with VQ-101, Bolsters Leadership Team

VQ-101 Clinical Trial Initiation:
Vanqua Bio has initiated a Phase 1 clinical trial for VQ-101, an orally administered brain-penetrant small molecule allosteric activator of glucocerebrosidase (GCase), aimed at treating Parkinson's disease with mutations in the GBA1 gene (GBA-PD).

Leadership Expansion:
To support clinical development, Vanqua Bio has expanded its leadership team by appointing Jesse Cedarbaum, MD, as Chief Medical Officer, Omer Siddiqui as Chief Development Officer, and Rand Sutherland, MD, MPH, to the Board of Directors.

Therapeutic Hypothesis:
VQ-101 is designed to restore lysosomal function by activating GCase, thereby preventing the accumulation of toxic alpha-synuclein, a hallmark of Parkinson's disease.

Preclinical Success:
Preclinical studies have demonstrated VQ-101's efficacy in activating GCase in live-cell assays, reducing lipid substrates, and blocking the accumulation of pathogenic alpha-synuclein in patient-derived neuronal cells and animal models.

Clinical Trial Design:
The Phase 1 study is a randomized, double-blind, placebo-controlled single- and multiple-ascending dose study in healthy volunteers and PD patients with or without GBA1 mutations, aiming to evaluate VQ-101's safety, tolerability, pharmacokinetics, and pharmacodynamics.