Vasa Therapeutics Begins Phase 1 Trial of VS-041 for Heart Failure with Preserved Ejection Fraction

Vasa Therapeutics, a biopharmaceutical company focused on developing innovative cardiovascular and metabolic therapies, has announced authorization from the U.K. Medicines and Healthcare products Regulatory Agency (MHRA) to begin a Phase 1 clinical trial of its investigational drug, VS-041. This trial will evaluate the safety, tolerability, and pharmacokinetics of VS-041 in healthy adult participants. The drug is intended for the treatment of heart failure with preserved ejection fraction (HFpEF) and represents a novel approach in this therapeutic area.

Dr. Artur Plonowski, CEO of Vasa Therapeutics, emphasized the significance of this milestone. He noted that there are currently no approved treatments for HFpEF that effectively improve the structural properties of the myocardium and provide long-lasting clinical benefits. VS-041 aims to address this gap by inhibiting the release of endotrophin, a biomarker associated with fibrotic and inflammatory responses in the heart. This mechanism could offer a personalized treatment option for patients with elevated endotrophin levels who are at high risk of adverse outcomes.

The start of the Phase 1 trial coincides with the expansion of Vasa Therapeutics' seed funding, which now totals $11 million. The funding round was led by Orphinic Scientific SA, with additional participation from i&i Biotech Fund I SCSp. The proceeds will be used to support the clinical development of VS-041 and further advance Vasa's preclinical programs.

Heart failure, a condition affecting over 64 million people worldwide, is a growing concern, particularly in the United States where it currently affects approximately 6.7 million individuals. This number is projected to rise to over 8.5 million by 2030. Nearly half of these cases are HFpEF, a condition that is becoming increasingly prevalent. Patients with HFpEF face dire prognoses, with approximately 75% dying within five years of their initial hospitalization and 84% being rehospitalized. Current HFpEF therapies offer symptomatic relief but do not modify the disease or provide durable benefits once treatment stops.

Endotrophin, a peptide derived from collagen type VI, has been linked to metabolic dysregulation, inflammation, and fibrosis in the heart. Studies have shown that baseline levels of plasma endotrophin are strongly associated with poor outcomes in HFpEF patients, including increased risk of death and rehospitalization.

VS-041, an oral compound developed by Vasa Therapeutics, targets these issues by significantly reducing cardiac fibrosis and improving diastolic heart functions in preclinical models of HFpEF. The drug also inhibits the release of endotrophin from human cardiac fibroblasts. In toxicology studies conducted under Good Laboratory Practice (GLP) conditions, VS-041 has shown a highly favorable safety and tolerability profile. The preclinical development of VS-041 was co-funded by the European Regional Development Fund and the Polish National Centre for Research and Development.

Vasa Therapeutics is a privately held company dedicated to creating therapies that address cardiovascular and metabolic aging. Besides VS-041, the company is working on a platform for long-acting apelin, intended for use in combination with incretins to treat obesity, especially in patients at risk for muscle loss or cardiovascular disease. Long-acting apelin agonists also hold potential as monotherapies for conditions such as muscle atrophy, heart failure, chronic kidney disease, and peripheral artery disease. The company’s preclinical pipeline includes a state-of-the-art inhibitor of CamKIId, designed to treat heart failure and life-threatening arrhythmias.

Vasa Therapeutics' advancements are pivotal in the ongoing battle against heart failure and its associated complications, potentially offering new hope for millions of patients worldwide.