- 6-month GLP-tox study reinforces favorable safety profile of Tiprelestat
- Phase II Study in Pulmonary Arterial Hypertension (PAH) to be initiated by Stanford University in mid-2026
Kiel, GERMANY, December 2, 2025 – tiakis Biotech AG (“tiakis”), a clinical-stage biopharmaceutical company developing novel therapeutics for life-threatening pulmonary and cardiovascular diseases, today announced that the Company has successfully finalized a comprehensive data package to advance Tiprelestat into a Phase II clinical trial in Pulmonary Arterial Hypertension (PAH) by completing a preclinical GLP-tox study.
Tiprelestat is a first- and best-in-class biological neutrophil elastase inhibitor and BMPR2 amplifier developed as an investigational disease-modifying therapy for PAH. It has shown the ability to address the underlying inflammation and potentially reverse the vascular remodelling caused by this debilitating and fatal rare disease.
Tiprelestat has demonstrated an excellent safety profile in five clinical trials involving over 100 individuals. In addition, the positive results from the completed 6-month-GLP-tox study in rats reinforce the encouraging safety profile previously observed in pre-clinical and clinical studies. In this study, rats received daily subcutaneous (s.c.) doses of Tiprelestat at 5 mg or 20 mg per kilogram of body weight for 180 days vs. a placebo control group. No adverse effects were observed at any dose level demonstrating a significant therapeutic window for safe dosing for 24 weeks in the upcoming Phase II PAH trial.
Clinical observations, safety lab results, gross necropsy results and histopathology results did not reveal any safety concerns, even at the highest dose level, which corresponds to a Human Equivalent Dose (HED) based 20-fold safety window for the targeted dose in PAH patients. These results strongly support targeted exposure levels of Tiprelestat at fixed doses of 5 mg/day and 10 mg/day s.c.
Pending final review by the U.S. FDA, a Phase II trial in PAH is expected to be initiated in mid-2026 by Stanford University. In early 2025, the FDA had already issued a positive opinion on the study design of the “ATHENA” trial. In September 2025, a double-digit million US$ grant was awarded to Stanford University by the National Institutes of Health (NIH) to run the ATHENA trial.
Tiprelestat has been granted orphan designations in PAH in Europe and the U.S. and is eligible for biologics exclusivity in the U.S. upon approval.
“We are excited to have finalized a robust data package for the Phase II ATHENA trial of Tiprelestat in PAH,” said Martin Voss, Chief Executive Officer of tiakis. “Pulmonary arterial hypertension remains an area of urgent unmet need, with current registry data showing a 5-year survival rate of only 57% of diagnosed PAH patients. We believe Tiprelestat holds significant promise as a potential disease-modifying therapy.”
tiakis will be available for meetings with investors and biopharmaceutical companies during the JP Morgan Healthcare Conference in San Francisco, CA, January 12-15, 2026.
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About tiakis Biotech
tiakis Biotech AG is an innovative, clinical-stage pharmaceutical company specializing in groundbreaking approaches to protect human tissues and organ structures. The Company develops anti-inflammatory treatments with a primary focus on pulmonary arterial hypertension (PAH). tiakis´ lead candidate Tiprelestat is in clinical development and addresses unmet medical needs in life-threatening conditions. The Company is based in Kiel, Germany.
For further information, please visit https://tiakis.bio.
Contact
tiakis BIOTECH AG
Sophienblatt 40
24103 Kiel
Germany
phone: +49 431 8888-462
fax: +49 431 8888-463
email: info@tiakis.bio
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