VQ-101 / Vanqua Bio - LARVOL DELTA

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 (MDS Congress 2025) - "VQ-101 was well tolerated at all dose levels evaluated in both HVs and participants with PD, with and without a GBA1 mutation. VQ-101 has a favorable CSF and plasma PK profile, supporting once daily oral dosing and demonstrated dose-dependent target engagement. These results, together with preclinical data, support the potential for VQ-101 to slow or stop the progression of PD by increasing GCase activity and blocking the accumulation of misfolded aSyn."

Clinical • CNS Disorders • Movement Disorders • Parkinson's Disease • GBA • GBA1

Vanqua Bio Highlights Positive Interim Data from its Ongoing Phase 1b Clinical Trial of VQ-101, an Allosteric Activator of GCase for the Treatment of GBA-Parkinson’s and Related Disorders, at The International Congress of Parkinson’s Disease and Movement Disorders (GlobeNewswire) - "Following 28 days of dosing, VQ-101 exceeded the study’s target engagement goal, 50% lysosomal GCase activation, at all doses tested....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....The open label extension portion of the Phase 1b study in Parkinson’s patients is ongoing; additional data are expected in early 2026."

P1 data • Parkinson's Disease

The allosteric activator of glucocerebrosidase VQ-101 shows sustained activation of lysosomal GCase in humansThe allosteric activator of glucocerebrosidase VQ-101 shows sustained activation of lysosomal GCase in humans (EAN 2025) - "VQ-101 is safe and well tolerated at all tested dose levels. VQ-101 has a favorable CSF and plasma PK profile, supporting once daily oral dosing and demonstrates dose-dependent target engagement in HVs. These results, together with preclinical data, support the potential for VQ-101 to slow or stop the progression of PD by increasing GCase activity and blocking the accumulation of misfolded aSyn."

CNS Disorders • Movement Disorders • Parkinson's Disease • GBA • GBA1

VQ-101, A SMALL MOLECULE ALLOSTERIC ACTIVATOR OF GLUCOCERBROSIDASE (GCASE), DEMONSTRATES ROBUST AND SUSTAINED TARGET ENGAGEMENT IN HUMANS (ADPD 2025) - "These Results , together with preclinical data, support the potential for VQ-101 to stop the accumulation of α-syn in PD patients and slow or stop the progression of disease. Enrollment of PD patients in the phase 1b part evaluating multiple doses of VQ101 is ongoing."

CNS Disorders • Movement Disorders • Parkinson's Disease • GBA • GBA1

Vanqua Bio to Present at the AD/PD 2025 International Conference (GlobeNewswire) - P1a/1b | N=N/A | "Vanqua Bio...announced that the company will present at AD/PD 2025 International Conference on Alzheimer’s and Parkinson’s Diseases and Related Neurological Disorders, taking place in Vienna, Austria on April 1-5....The presentation will detail interim results from the ongoing Phase 1 clinical trial of VQ-101 in healthy volunteers and patients with Parkinson’s disease (PD)....In Phase 1a, VQ-101 achieved robust peripheral and central exposure, was well tolerated, and demonstrated sustained activation of live-cell lysosomal GCase by more than 75%....The Phase 1b portion of the trial in patients with PD with and without GBA mutations is ongoing and expected to complete later this year."

P1 data • Trial status • Parkinson's Disease

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 (GlobeNewswire) - "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...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."

P1 data • CNS Disorders • Parkinson's Disease

Vanqua Bio to Present at Inaugural GBA1 2024 Meeting (GlobeNewswire) - "Vanqua Bio...announced today that it will participate in the inaugural GBA1 meeting which will bring together health professionals and stakeholders from both academia and industry engaged in GBA1 research. The meeting will take place from June 27 - 29, 2024, at The Montreal Neurological Institute-Hospital...The GBA1 meeting aims to advance the collective understanding and treatment of GBA1-associated neurodegenerative diseases."

Preclinical • CNS Disorders • Parkinson's Disease