Vanqua Bio Announces Positive Interim Results from Phase 1 Clinical Trial of VQ-101, an Orally Administered, Brain-Penetrant, Allosteric Activator of GCase for the Treatment of GBA-Parkinson’s and Related Disorders

• VQ-101 is the first small molecule to demonstrate > 75% activation of the lysosomal enzyme glucocerebrosidase (GCase) in humans
• In healthy volunteers, VQ-101 was well tolerated and achieved peripheral and cerebrospinal fluid (CSF) exposures supporting once daily dosing
• Vanqua has initiated the Phase 1b portion of the study in patients with Parkinson’s disease (PD) with and without GBA mutations; data are expected in 2025
• Vanqua announces the expansion of its development team with the appointment of Maurizio Facheris, M.D., as Chief Medical Officer

CHICAGO, Oct. 09, 2024 (GLOBE NEWSWIRE) — Vanqua Bio, a clinical-stage biopharmaceutical company dedicated to discovering and developing next-generation medicines for the treatment of neurodegenerative diseases, announced positive results from its Phase 1 clinical trial of VQ-101, an orally administered, brain-penetrant, allosteric activator of GCase. The Phase 1a portion of the study evaluated safety, pharmacokinetics, and pharmacodynamics across single and multiple ascending doses in healthy volunteers.

“We are excited to announce that VQ-101 demonstrated robust activation of lysosomal GCase and high levels of CSF exposure at doses that were safe and well tolerated in healthy volunteers. These data reflect the considerable efforts of an experienced team that is passionate about making a difference in the lives of people living with PD,” said Jim Sullivan, Ph.D., CEO of Vanqua Bio. “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. We are pleased to advance to the Phase 1b portion of the study in patients with PD.”

The Phase 1a portion of the clinical trial, conducted at the Centre for Human Drug Research (CHDR) in the Netherlands, consisted of randomized, double-blind, placebo-controlled single and multiple ascending doses in healthy volunteers. The Phase 1b portion of the trial will include 28-day, randomized, double-blind, placebo-controlled treatment with multiple ascending doses of VQ-101 in patients with PD with and without GBA mutations. In healthy volunteers, VQ-101 activated lysosomal GCase by more than 75% in single dose cohorts, and activation was sustained following multiple doses. This exceeded the study’s target engagement goal of 50% activation, which was informed by human genetics and Vanqua’s preclinical studies in patient-derived dopaminergic neurons in which 50% activation of GCase resulted in significant blockade of the accumulation of insoluble alpha synuclein, the pathologic hallmark of PD.

In addition to robust target engagement, VQ-101 achieved significant levels in the CSF; the mean CSF:unbound plasma ratios were ≥1, indicating full central nervous system penetrance. Lastly, in both the single and multiple ascending dose cohorts of this Phase 1a study, VQ-101 was well-tolerated, with no dose-limiting or serious adverse events reported. All treatment-emergent adverse events were mild or moderate in nature and there were no study discontinuations due to adverse events. Detailed data from the Phase 1 study will be presented at a future medical conference.

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. Vanqua’s acting CMO, Dr. Jesse Cedarbaum, will continue to support the company as a senior clinical advisor.

“We are grateful for Jesse’s contributions to Vanqua’s progress and are pleased that he will remain a key part of the Vanqua team,” said Jim Sullivan, Ph.D. “Maurizio brings first-hand experience of taking PD therapies from early phase development to approval and will be an excellent addition to our team.”  

Before joining Vanqua, Dr. Facheris was Senior Medical Director in Neuroscience Development at AbbVie, where he oversaw the clinical development of therapeutic approaches for PD, including successfully bringing foslevodopa/foscarbidopa for subcutaneous infusion to approval. Prior to that, he was Senior Associate Director of Research Programs at the Michael J. Fox Foundation for Parkinson’s Research. Dr. Facheris received his M.D. from the University of Brescia, Italy and attended his residency in Neurology at the University of Milano, Italy.

About VQ-101

VQ-101 is a novel brain-penetrant small molecule allosteric activator of GCase. VQ-101 activates lysosomal GCase in a live-cell assay in vitro, ex vivo, and in vivo, and blocks the accumulation of insoluble alpha synuclein, which is the pathological hallmark of PD, in patient-derived neurons.

About Vanqua Bio

Founded in 2019 and headquartered in Chicago, Vanqua Bio is a biopharmaceutical company dedicated to discovering and developing next-generation medicines that have the potential to transform the lives of patients with neurodegenerative diseases. Our technology platform utilizes human genetics and patient-derived neuronal cells to identify, validate, and clinically translate novel disease pathways associated with lysosomal dysfunction or aberrant activation of the innate immune system. Initially, we are targeting glucocerebrosidase (GCase) as a potential treatment for Parkinson’s disease (PD). Additional programs address overactivation of the innate immune system in central and peripheral neurodegenerative disorders, including Alzheimer’s disease. For more information, go to www.vanquabio.com.

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