tiakis Biotech AG has announced that it has received positive scientific advice from the U.S. FDA regarding its planned Phase II clinical trial for Tiprelestat in the treatment of Pulmonary Arterial Hypertension (PAH). The FDA's feedback supports the study design, including primary and secondary endpoints, for the ATHENA trial.
The ATHENA trial is designed as a randomized, double-blind study involving three cohorts and a total of 90 patients. The trial aims to evaluate the safety and efficacy of Tiprelestat in combination with standard of care, compared to placebo, in patients diagnosed with PAH. A key element of the trial design is a 4-week withdrawal period, intended to explore Tiprelestat's potential for disease modification.
The primary endpoint for the ATHENA trial is Pulmonary Vascular Resistance (PVR), a critical measure of the disease's severity. The secondary endpoint is the 6-Minute Walk Distance (6MWD), an indicator of functional capacity and exercise tolerance in PAH patients.
Tiprelestat, also known as human recombinant Elafin, is an anti-inflammatory protein that functions as a reversible inhibitor of human neutrophil elastase and proteinase 3. It also inhibits the formation of neutrophil exosomes and neutrophil extracellular traps. Preclinical studies in animal models have demonstrated Tiprelestat's ability to promote vascular remodeling. Research has identified a significant imbalance between neutrophil elastase levels and Elafin deficiency in PAH patient cohorts.
"It is exciting to evaluate the therapeutic potential of Tiprelestat in PAH in a Phase 2 clinical trial," said Dr. Roham Zamanian, Professor of Medicine at Stanford University and Principal Investigator of the ATHENA trial. "Given its unique mechanism of action addressing major pathological processes in PAH, specifically inflammation and BMPR2 deficiency, Tiprelestat holds the promise of a disease-modifying therapy for PAH. Data from the planned 4-week withdrawal period are expected to confirm this hypothesis."
tiakis is currently preparing for the initiation of the ATHENA trial, which includes conducting a six-month repeat-dose toxicology study in rats. Tiprelestat has already been granted orphan drug designation for PAH in both the U.S. and Europe.
