Vanqua Bio, a Chicago-based pharmaceutical company, has announced promising results from its Phase Ia trial of VQ-101, a novel oral treatment for Parkinson's disease. The positive data has led to the initiation of a Phase Ib trial to further evaluate the drug's efficacy and safety in Parkinson's patients.
VQ-101 is a small molecule allosteric activator targeting glucocerebrosidase (GCase), a lysosomal enzyme. The Phase Ia/Ib trial is designed to assess its potential as a disease-modifying therapy for Parkinson's disease.
Phase Ia Trial Results
Interim data from the randomized, single and multiple ascending dose Phase Ia trial, conducted at the Centre for Human Drug Research (CHDR) in the Netherlands, revealed that VQ-101 achieved over 75% activation of GCase in healthy volunteers. This outcome exceeded the study's target engagement goal of 50% activation. Vanqua's preclinical studies in patient-derived dopaminergic neurons indicated that 50% activation of GCase resulted in a significant blockade of insoluble alpha-synuclein accumulation, a key pathological hallmark of Parkinson's disease.
In addition to demonstrating significant GCase activation, VQ-101 was found to be well-tolerated, with no dose-limiting or serious adverse events reported. The drug also achieved significant levels in the cerebrospinal fluid (CSF), indicating full penetration of the central nervous system (CNS).
Advancement to Phase Ib
Based on these encouraging results, Vanqua Bio is moving forward with a Phase Ib trial in patients with Parkinson's disease. The randomized, double-blind, placebo-controlled Phase Ib trial will evaluate multiple doses of VQ-101 in Parkinson's patients, both with and without mutations in the glucocerebrosidase (GBA) gene. Topline data from this phase of the trial is anticipated in 2025.
"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," said Jim Sullivan, CEO of Vanqua.
Parkinson's Disease Context
Parkinson's disease is a progressive neurodegenerative disorder affecting millions worldwide. The World Health Organization (WHO) estimated in 2019 that over eight million people were living with Parkinson's disease, a number that has doubled in the preceding 25 years. The disease is characterized by motor symptoms such as tremor, rigidity, and bradykinesia, as well as non-motor symptoms including cognitive impairment and depression.
Genetic factors play a significant role in Parkinson's disease. A recent study, the Rostock International Parkinson’s Disease (ROPAD) genetic profiling study, found that mutations in genes such as GBA and leucine-rich repeat kinase-2 (LRRK2) contributed to 15% of Parkinson's disease cases.
According to GlobalData, Phase I drugs for Parkinson’s disease have a 72% phase transition success rate (PTSR) for progressing into Phase II, providing cautious optimism for VQ-101's future development.
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.
