tiakis Biotech AG, a leading clinical-level biopharmaceutical company conquering novel therapeutics for cardiovascular and life-threatening pulmonary disease, confirmed the completion of its robust data package to upgrade Tiprelestat into a Phase 2 clinical trial in Pulmonary Arterial Hypertension (PAH) by achieving a preclinical GLP-tox study. With the completion of the data package, the trial will also get the required attention and a boost to succeed further.
The Tiprelestat is the first-ever excellent BMPR2 amplifier and biological neutrophil elastase inhibitor established as an investigational disease-alter therapy for PAH. It has highlighted its potential to identify the cause of reverse and underlying inflammation, the vascular remodelling caused due to fatal and debilitating rare disease. The Tiprelestat has illustrated a commendable safety profile in five clinical trials with 100 individuals.
The trial achieved positive and convincing results from the completed 6-month GLP-tox study in mice, strengthening the empowering safety profile that was previously noticed in clinical and preclinical studies. This study covered the rats' intake of daily subcutaneous (s.c.) doses of Tiprelestat at 20 mg or 5 mg per kilogram of body weight for around 180 days under a placebo control group.
There were no such negative effects noticed at any of the dose levels. This illustrated a noteworthy therapeutic window for reliable and safe dosing for around 24 weeks in the forthcoming Phase 2 PAH trial. Furthermore, the final review by the U.S. FDA for the Phase 2 trial in PAH might be evaluated in mid-2026 by Stanford University. The FDA has already given positive feedback and opinion on the study structure of the ‘ATHENA’ trial in 2025.
The Tiprelestat has been rewarded with orphan designations in PAH in the U.S and Europe and is also qualified for the biologics exclusivity based on the U.S approval. The millions of US$ reward for handed to Stanford University by the National Institutes of Health (NIH) to work on the ATHENA trial in September 2025.
The Chief Executive Officer of tiakis, Martin Voss, said, “We are thrilled and proud to finalise a comprehensive data package for the Phase 2 ATHENA trial of Tiprelestat in PAH. Pulmonary arterial hypertension has always been the area of urgent unmet need, with the latest registry data confirming the 5-year survival rate of just 57% of treated PAH patients. We think Tiprelestat holds a meaningful commitment as a capable disease-altering therapy.”
Holding M.Pharm in Pharmaceutical Chemistry, Chandni crafts cutting-edge, research-driven healthcare news for Towards Healthcare, combining scientific depth with innovative storytelling to simplify complex topics for global readers.
