- VQ-101 is the first small molecule to demonstrate >70% activation of the lysosomal enzyme glucocerebrosidase (GCase) in GBA-Parkinson’s (GBA-PD) patients
- In the ongoing Phase 1b study in patients with GBA-PD, VQ-101 was well tolerated and achieved peripheral and cerebrospinal fluid (CSF) exposures supporting once daily oral dosing
- The open label extension portion of the Phase 1b study in Parkinson’s patients is ongoing; additional data are expected in early 2026
CHICAGO, Oct. 02, 2025 (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 interim results from the Phase 1b portion of its clinical trial of VQ-101, an orally administered, fully brain-penetrant, allosteric activator of GCase. The Phase 1b portion of the study is evaluating safety, pharmacokinetics, and pharmacodynamics across multiple ascending doses in Parkinson’s patients with and without GBA mutations.
“We are pleased to announce that VQ-101 demonstrated robust activation of lysosomal GCase and full CNS penetrance at doses that were safe and well tolerated following 28 days of dosing in patients with GBA-PD,” said Jim Sullivan, Ph.D., CEO of Vanqua Bio. “By restoring GCase activity to at least healthy volunteer levels, we believe that VQ-101 has the potential to positively impact the lives of patients following longer term dosing. These results support the continued development of VQ-101 in Parkinson’s disease and we look forward to sharing additional data from the open label extension portion of the study in 2026.”
In the Phase 1b portion of this clinical trial conducted in GBA-PD subjects, individuals were randomized in a double-blind fashion to receive once daily VQ-101 or placebo for 28 days with the opportunity of enrolling into an open-label extension for up to 6 months. Following 28 days of dosing, VQ-101 exceeded the study’s target engagement goal, 50% lysosomal GCase activation, at all doses tested. Based on human genetics and Vanqua’s preclinical studies in patient-derived dopaminergic neurons, 50% activation of lysosomal GCase is expected to restore patients’ lysosomal GCase activity to healthy volunteer levels and block the accumulation of insoluble alpha synuclein, the pathologic hallmark of PD.
Consistent with the robust target engagement observed in the study, VQ-101 demonstrated a favorable safety and pharmacokinetics profile. No dose-limiting or serious adverse events were reported, and all treatment-emergent adverse events were mild or moderate in severity. There were no study discontinuations due to adverse events. VQ-101 was fully CNS penetrant, with mean CSF:unbound plasma ratios ≥1.
In addition to presenting the positive interim Phase 1b results at the upcoming International Congress of Parkinson’s Disease and Movement Disorders® in Honolulu, Hawaii October 5th - 9th , Vanqua will also present an analysis comparing progression of motor function and cognitive abilities in Parkinson’s patients with and without GBA1 mutations using the Michael J. Fox Foundation’s Parkinson's Progression Markers Initiative (PPMI) study database. Motor function and cognitive abilities have been shown to decline more rapidly in GBA-PD compared with idiopathic Parkinson’s disease (iPD) participants, including in time-to-event outcomes that can be used as potential endpoints for late phase clinical trials.
The International Congress of Parkinson’s Disease and Movement Disorders® presentation information:
Presentation Title: VQ-101, a CNS-Penetrant Small Molecule Allosteric Activator of Glucocerebrosidase (GCase), Demonstrates Favorable Tolerability and Sustained Activation of Lysosomal GCase in Individuals with Parkinson's Disease
Presenter: Dr. Maurizio Facheris, CMO
Session: Tuesday October 7th, 12:00-2:30pm HST
Presentation Title: Decline in Motor Function and Cognitive Abilities in GBA-PD Compared to idiopathic PD (iPD): An Analysis of PPMI
Presenter: Omer Siddiqui, CDO
Session: Wednesday October 8th, 1:30-2:00pm HST
About VQ-101
VQ-101 is a novel, orally administered, fully CNS-penetrant allosteric activator of glucocerebrosidase (GCase). VQ-101 is initially being investigated in GBA1-associated Parkinson’s disease (GBA-PD) and idiopathic Parkinson’s disease (iPD). By restoring GCase activity to healthy levels, VQ-101 aims to address the underlying genetic mechanism of disease in GBA-PD and slow or stop the progression of disease. Initial Phase 1 results with VQ-101 demonstrated sustained lysosomal GCase activation in healthy volunteers and Parkinson’s patients. In preclinical studies in patient derived neurons, 50% GCase activation blocked the accumulation of alpha synuclein, the pathogenic hallmark of PD. The Phase 1b study in Parkinson’s patients with and without GBA-mutations is ongoing.
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.
info@vanquabio.com
