Vasa Therapeutics Announces Successful Completion of First-in-Human Study for Investigational Therapy VS-041, a Small Molecule Drug Candidate for Heart Failure with Preserved Ejection Fracture

• VS-041 safe and well tolerated in healthy patients
• Biomarker trial in patients with HFpEF targeted to initiate in 2025

ENCINITAS, Calif. and WROCLAW, Poland, June 4, 2025 /PRNewswire/ -- Vasa Therapeutics ("Vasa"), a clinical stage biopharmaceutical company developing novel therapies for cardiovascular and metabolic aging, today announced the successful completion of its first-in-human phase 1 clinical study evaluating the safety, tolerability, and pharmacokinetics of VS-041, a narrow spectrum small molecule inhibitor of matrix metalloproteinases (MMPs). 

The phase 1 study, conducted in 70 healthy adult participants in a single and multiple ascending dose design, demonstrated that orally administered VS-041 was safe and well tolerated at all dose levels tested. No serious adverse events were reported, and all treatment-emergent adverse events were mild to moderate in severity and not attributed to the investigational agent. Additionally, the trial achieved its pharmacokinetic objectives, with target serum levels commensurate with the projected target efficacious dose.  Results of the study will be presented at upcoming scientific meetings.

"We are very encouraged by the safety and tolerability profile of VS-041.  VS-041 inhibits specific MMPs that contribute to the pathophysiology of heart failure with preserved ejection fraction (HFpEF)," said Noreen Henig, MD, Chief Medical Officer of Vasa Therapeutics. "We plan to advance development of VS-041 and test its effects on clinical and prognostic biomarkers of HFpEF, including endotrophin, in the target patient population later this year."

"Completion of our phase 1 study with VS-041 marks a significant milestone in our mission to advance a pipeline of drug candidates to address unmet medical needs in large groups of patients," added Artur Plonowski, MD, PhD, Chief Executive Officer and co-founder of Vasa Therapeutics.  "We are on track to have two assets in clinical development in 2026, and I want to thank the entire Vasa team for putting their hearts and science into innovative therapies for cardiovascular and skeletal muscle diseases." 

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 diseases, such as chronic kidney disease. VS-041 robustly reduces cardiac fibrosis and significantly improves diastolic heart functions in a preclinical HFpEF models. The compound also inhibits the release of endotrophin from primary human cardiac fibroblasts. In GLP toxicology studies, VS-041 demonstrated a highly favorable safety and tolerability profile. Development of VS-041 was co-funded by the European Regional Development Fund and the Polish National Centre for Research and Development (POIR.01.01.01-00-1210/19-01).

About Vasa Therapeutics

Vasa is a privately held biopharmaceutical company developing therapeutics that target pathophysiologies of cardiovascular and metabolic aging. In addition to VS-041, Vasa developed a cutting-edge platform for long-acting apelin for combination treatment with incretins in obesity, especially for patients at risk for skeletal muscle loss or cardiovascular disease. As a monotherapy, long-acting apelin agonists could also be beneficial in treating neuromuscular diseases, heart failure, chronic kidney disease, or peripheral artery disease. Vasa's preclinical pipeline also includes a best-in-class inhibitor of CamKIId for heart failure and life-threatening arrhythmias.

For more information, please visit www.vasatherapeutics.com

SOURCE Vasa Therapeutics