Vanqua Bio has announced positive interim results from its Phase 1 clinical trial of VQ-101, a novel drug designed to treat GBA-Parkinson’s and related disorders. This breakthrough could potentially slow or even halt the progression of Parkinson’s disease, offering hope to millions of patients worldwide.

Key Highlights:

  1. Impressive Enzyme Activation: VQ-101 demonstrated over 75% activation of the lysosomal enzyme glucocerebrosidase (GCase) in humans, exceeding the study’s target of 50%.
  2. Safety and Tolerability: The drug was well-tolerated in healthy volunteers, with no serious adverse events reported.
  3. Promising Brain Penetration: VQ-101 achieved significant levels in the cerebrospinal fluid, indicating full central nervous system penetrance.
  4. Advancement to Patient Trials: Vanqua Bio has initiated the Phase 1b portion of the study in Parkinson’s patients, with results expected in 2025.

Why It Matters

Parkinson’s disease affects millions of people globally, and current treatments only address symptoms without slowing disease progression. VQ-101’s ability to activate GCase and potentially stop the accumulation of alpha-synuclein (a hallmark of PD) could represent a game-changing approach to treatment.

Dr. Jim Sullivan, CEO of Vanqua Bio, expressed excitement about the results, stating, “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.”

Looking Ahead

As Vanqua Bio prepares for later-stage clinical studies, they’ve strengthened their team by appointing Dr. Maurizio Facheris as Chief Medical Officer. Dr. Facheris brings valuable experience in taking Parkinson’s therapies from early development to approval.


Note: This article has been written based on a press release from Vanqua Bio dated October 09, 2024.