Vasa Therapeutics Receives FDA IND Clearance to Advance Novel Small Molecule VS-041 for the Treatment of Heart Failure with Preserved Ejection Fraction (HFpEF)

Phase 1c Clinical Trial to Begin Immediately

ENCINITAS, Calif. and WROCLAW, Poland, Oct. 13, 2025 /PRNewswire/ -- Vasa Therapeutics, ("Vasa"), a private, clinical stage biotechnology company developing novel therapies for cardiovascular and metabolic aging, today announced that the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) application for VS-041, a narrow spectrum small molecule inhibitor of matrix metalloproteinases. The company plans to immediately initiate a Phase 1c clinical trial in the US for participants with Heart Failure with Preserved Ejection Fraction (HFpEF).

"After successful completion of our Phase 1 trial in healthy volunteers earlier this year, we are excited to initiate a Phase 1c study of VS-041 in participants affected with HFpEF and elevated serum levels of endotrophin. This IND clearance marks a major milestone for Vasa, underscores the strength of our science and development strategy, and demonstrates exceptional execution," said Artur Plonowski, MD, Chief Executive Officer of Vasa Therapeutics. "This next study will evaluate potential important effects of VS-041 on clinical and prognostic biomarkers of HFpEF, including endotrophin which is associated with excess morbidity and mortality." 

The Phase 1c study will assess the safety and tolerability of VS-041 in participants with HFpEF and elevated serum endotrophin. Data from this study could demonstrate target engagement and support dose selection for future clinical development.

"Endotrophin is recognized as a strong predictor of outcomes in HFpEF and may be an important contributor to its pathophysiology. A Phase 1c study of VS-041 is the first step to evaluate prospectively the therapeutic impact of interrupting the generation of endotrophin, " said Julio Chirinos, MD, PhD, Professor of Medicine in the Cardiovascular Division at the Perelman School of Medicine at the University of Pennsylvania, an expert in HFpEF and endotrophin, who also co-leads a Global Heart Failure biomarker consortium. 

About VS-041

VS-041 is an oral compound that was discovered and developed by Vasa for the potential treatment of HFpEF and other chronic disease characterized by fibroinflammation such as hypertrophic cardiomyopathy and chronic kidney disease. VS-041 robustly reduces cardiac fibrosis and significantly improves diastolic heart functions in preclinical HFpEF models. VS-041 is demonstrated to inhibit ex-vivo release of endotrophin from primary human cardiac fibroblasts. Development of VS- 041 was co-funded by the European Regional Development Fund and the Polish National Centre for Research and Development (POIR.01.01.01-00-1210/10-01).

About Heart Failure with Preserved Ejection Fraction (HFpEF)

HFpEF is a complex and progressive form of heart failure characterized by by stiffening and fibroinflammation of the heart muscle and impaired relaxation despite normal contractile function. HFpEF affects ~3 million individuals in the US and ~10 million globally. HFpEF is characterized by fibroinflammation that is modulated by MMPs. Currently approved therapies for HFpEF provide clinical improvement but do not result in disease modification and durable improvement upon discontinuation of the treatment.

The University of Pennsylvania is the owner of certain patent applications covering diagnostic methods that use plasma and urine proteins to predict outcomes in patients with HFpEF.

About Vasa Therapeutics

Vasa Therapeutics is a clinical-stage biopharmaceutical company developing novel therapeutics that target the pathophysiology of cardiovascular and muscle aging. The company has developed a pipeline of four unique assets targeting unmet medical needs in heart failure, sarcopenia, rare neuromuscular diseases, peripheral arterial disease and life-threatening arrhythmias.

In addition to the lead program VS-041, Vasa developed a library of long-acting apelin programs targeting rare neuromuscular diseases, peripheral artery disease (PAD) and sarcopenia that have shown the potential to preserve muscle function, increase cardiac output and reverse age-related sarcopenia in preclinical models. Vasa's long-acting apelin VS-241 will begin clinical testing in 2026.

Vasa recently won the 2025 TOP10 Longevity Breakthrough Award. Led by a world-class team with an outstanding track record of successful drug development in large pharma and small biotech companies, Vasa Therapeutics puts heart and science in everything we do. 

For more information, please visit vasatherapeutics.com

SOURCE Vasa Therapeutics