Vasa Therapeutics has reported positive topline findings demonstrating clinical proof of concept for its lead therapy, VS-041, in a Phase Ic study in patients with heart failure with preserved ejection fraction (HFpEF).
The randomised, placebo-controlled trial (NCT07219511) evaluated the narrow-spectrum matrix metalloproteinase (MMP) inhibitor at two dose levels in patients receiving background guideline-directed medical therapy for the heart condition.
Go deeper with GlobalData
Discover B2B Marketing That Performs
Combine business intelligence and editorial excellence to reach engaged professionals across 36 leading media platforms.
VS-041 proved safe and well tolerated while lowering NT-proBNP, an established biomarker of cardiac wall stress and heart failure, among the treated patients.
The study showed the utility of serum endotrophin via the NordicProC6 assay to detect patients with fibroinflammation. Analyses of extracellular matrix collagen turnover and exploratory clinical measures will follow in early 2027.
Vasa Therapeutics CEO and co-founder Artur Plonowski said: “Vasa had three important goals for the recently completed Phase Ic trial in patients with HFpEF.
“First, we showed that narrow-spectrum MMP inhibition is safe and well tolerated in a population of HFpEF patients with increased risk of unfavourable outcomes, and the reduction in NT-proBNP, achieved within 28 days, suggests a compelling path forward in the clinic.
“Secondly, we demonstrated that serum endotrophin can be used prospectively to identify an endotype of HFpEF patients that cannot be characterised by clinical data alone. This novel enrichment strategy should translate to smaller and potentially faster development.”
The oral compound gained fast track designation (FTD) from the US Food and Drug Administration (FDA) in November 2025, following a Phase I trial in healthy volunteers (ISRCTN15814719).
That early study confirmed safety at doses matching preclinical efficacy, where the molecule ameliorated cardiac fibrosis and improved diastolic relaxation in models of the progressive heart disorder.
Co-funded by the European Regional Development Fund and the Polish National Centre for Research and Development, VS-041 also targets fibroinflammation in chronic kidney disease and hypertrophic cardiomyopathy.
Privately held Vasa Therapeutics focuses on cardiovascular and muscle ageing, advancing a pipeline of four assets addressing heart failure, life-threatening arrhythmias, peripheral arterial disease, rare neuromuscular diseases, and sarcopenia.
