Maternal outcomes among pregnant women with shunt-related congenital heart disease-associated pulmonary hypertension: a retrospective study
Stanford University Secures NIH Grant To Advance Tiakis Biotech´s Elafin (Tiprelestat) Through A U.S. Phase II Trial In Pulmonary Arterial Hypertension
- Novel, disease-modifying treatment option for Pulmonary Arterial Hypertension (PAH), an orphan indication with a significant unmet medical need
Kiel, GERMANY, September 8, 2025 – tiakis Biotech AG ("tiakis"), a clinical-stage biopharmaceutical company developing novel therapeutics for life-threatening pulmonary and cardiovascular diseases, today announced that its long-standing collaboration partner Stanford University has been awarded a US$ double-digit million grant from the National Institutes of Health (NIH) - National Heart Lung and Blood Institute (NHLBI) - the Lung Division, to fund a U.S. Phase II trial of Tiprelestat for the treatment of Pulmonary Arterial Hypertension (PAH). In early 2025, the U.S. FDA has issued positive scientific advice on the planned trial design. The first patients are expected to be treated by Stanford University in mid-2026.
Tiprelestat has been developed by tiakis as an investigational disease-modifying therapy for PAH that has the ability to address the underlying inflammation and potentially reverse the vascular remodeling caused by this debilitating and fatal rare disease. Tiprelestat has already demonstrated an excellent safety profile in five clinical trials involving over 100 subjects. Tiprelestat has received orphan designation for PAH in the U.S. And in Europe. Tiakis will provide Stanford with the clinical trial materials for this U.S. Study at its own expense.
Existing PAH treatments aim to restore the balance of vasoconstriction and vasodilation, targeting pathways such as endothelin, nitric oxide, and prostacyclin. Nevertheless, the 5-year survival rate in PAH is only 57%, according to registry data. Therefore, there is an urgent medical need for new therapies specifically targeting pulmonary vascular remodeling and inflammation.
"The NIH grant is an outstanding validation and recognition of Tiprelestat's robust science and its potential to deliver a truly transformative therapy for PAH patients. The investigative team thanks tiakis, our long-standing collaboration partner, for its commitment to supporting Stanford University in this U.S. Phase II trial," said Professor Roham Zamanian, MD, Director of the Adult Pulmonary Hypertension Program at Stanford University and Principal Investigator of the trial.
"Tiprelestat holds the promise of a disease-modifying therapy because of its unique mechanism of action which addresses major pathological processes in PAH, specifically inflammation and BMPR2 deficiency," added Professor Marlene Rabinovitch, MD, the Dwight and Vera Dunlevie Professor of Pediatric Cardiology, and Director of the Basic Science and Engineering Initiative of the Children's Heart Center at Stanford University and Principal Investigator of the trial. "Data from the planned U.S. Phase II trial are expected to confirm this hypothesis."
"We would like to congratulate Drs. Roham Zamanian and Marlene Rabinovitch on receiving this prestigious NIH award. We also congratulate the leader of the companion award to the Data Coordinating Center for this trial, Dr. Cathie Spino at the SABER unit of the University of Michigan. This award recognizes Tiprelestat's potential and pioneering approach by scientific and clinical experts at this well-respected institute," said Martin Voss, CEO of tiakis Biotech AG.
Research reported in this publication will be supported by the National Heart, Lung, And Blood Institute of the National Institutes of Health under Award Number UG3HL180990. The content is solely the responsibility of the authors and does not necessarily represent the official views of the National Institutes of Health.
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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 AGSophienblatt 4024103 KielGermanyphone: +49 431 8888-462fax: +49 431 8888-463email: info@tiakis.Bio
Media InquiriesakampionDr. Ludger Wess / Ines-Regina ButhManaging Partnersinfo@akampion.ComTel. +49 40 88 16 59 64 / +49 30 23 63 27 68
Stanford UniversityRoham T. Zamanian, MD, FCCPProfessor of MedicineDirector, Adult Pulmonary Hypertension ProgramAssociate Director, Vera Moulton Wall Center for Pulmonary Vascular DiseaseDivision of Pulmonary & Critical Care MedicineStanford University School of Medicineemail: zamanian@stanford.Edu
Clinical Impact Of Mean Pulmonary Arterial Pressure After Balloon Pulmonary Angioplasty For Inoperable Chronic Thromboembolic Pulmonary Hypertension
This study investigated the effects of mPAP after BPA on clinical outcomes and long-term prognosis. We compared clinical parameters, withdrawal from pulmonary vasodilators or oxygen therapy and prognosis across three groups: mPAP≤20, >20–<30 and ≥30 mm Hg. Key findings were: (1) patients with mPAP ≤20 mm Hg showed greater improvements in symptoms, exercise tolerance, plasma BNP levels and RVEF. (2) Fewer patients in this group continued treatment with pulmonary vasodilators and oxygen therapy. (3) Although patients with mPAP <30 mm Hg had a favourable long-term prognosis, outcomes were similar between the mPAP ≤20 mm Hg and >20–<30 mm Hg groups.
In this study, the overall prognosis after BPA was favourable, although a small number of patients did not achieve the general treatment goal of an mPAP of <30 mm Hg. The group with an mPAP of ≥30 mm Hg had a significantly poorer prognosis than the other groups, which is similar to the findings of previous reports describing the natural history of CTEPH.22 Patients who achieved an mPAP of <25 mm Hg after BPA were reportedly less likely to experience recurrent pulmonary hypertension and had a better prognosis.21 In the current study, prognosis was similar between groups with an mPAP of ≤20 mm Hg and >20–<30 mm Hg. This suggests that mild pulmonary hypertension after BPA is acceptable for selected patients—such as older individuals with reduced activity and those unable to undergo frequent treatment due to psychiatric or other disorders—when the primary goal is to improve prognosis rather than to relieve symptoms or improve exercise tolerance. However, since the results may not have been fully adjusted for patient background, the findings should be interpreted cautiously. Although the median observation period was 44 months, it might still be insufficient for assessing the long-term prognosis of patients with mild pulmonary hypertension.
The group with normalised haemodynamics after BPA (a group with an mPAP of ≤20 mm Hg) had milder symptoms, better exercise tolerance, lower plasma BNP levels and a higher RVEF. This suggests that BPA aimed at lowering mPAP leads to improved clinical parameters, including symptoms, exercise tolerance and right ventricular function. The decrease in mPAP after BPA may have reduced afterload in the right heart system and contributed to the improvements in symptoms and exercise tolerance. Although no trend in the CI, considered a factor contributing to exercise tolerance, was found across the three groups, the heart rate was lower in the group with a lower mPAP than in the other groups. On CMR images, no trends in right ventricular end-diastolic volume index or right ventricular stroke volume index were found across the three groups; however, RVEF was better in the lower mPAP group than in the other groups, reflecting a decrease in afterload. However, further research is required to determine whether this favourable RVEF and low resting heart rate are beneficial during exercise. Some reports indicate that improvement in resting mPAP alone is insufficient to improve symptoms and exercise tolerance.23 A previous report suggested that although resting mPAP did not correlate with 6MWD, peak oxygen consumption in exercise testing correlated with 6MWD.24 However, the present study did not evaluate exercise-induced pulmonary hypertension, and it may be insufficient to evaluate symptoms and exercise tolerance based on resting haemodynamic assessment alone.
Fewer patients with lower mPAP required oxygen therapy or pulmonary vasodilators. We speculate that improved oxygenation and haemodynamics in this group prompted treatment withdrawal. Given that oxygen therapy restricts daily life, achieving an mPAP of ≤20 mm Hg—and thus reducing treatment reliance—may enhance the quality of life. However, as treatment withdrawal was at each physician's discretion, the findings may not be generalisable. In Japan, pulmonary vasodilators, including riociguat25 and selexipag,26 are used to treat microvasculopathy in patients with CTEPH. A recent report suggests that pulmonary vasodilators after BPA are effective, particularly in older patients,27 and further studies are warranted to determine the criteria for the continuation or discontinuation of pulmonary vasodilators after BPA.
This study explored the clinical impact of mPAP after BPA and suggested goals for mPAP after BPA. Normalisation of mPAP after BPA is required to improve clinical parameters, such as symptoms and exercise tolerance. A previous report has shown that additional BPA after BPA treatment can be safely performed and can improve symptoms and 6MWD.28 In cases of mild pulmonary hypertension—defined as an mPAP >20–<30 mm Hg with lesions suitable for additional BPA—further BPA may be considered to improve symptoms and exercise tolerance. Further studies are needed to assess the haemodynamic benefits of adding pulmonary vasodilators in patients without BPA-treatable lesions. Moreover, depending on the patient's daily activities and treatment tolerability, mild pulmonary hypertension with an mPAP >20–<30 mm Hg after BPA may be acceptable solely to improve prognosis. Consequently, BPA treatment should be tailored to the individual patient's condition.
Study limitationsThis study has some limitations. First, this was a single-centre retrospective study with few cases, limiting the generalisability of its findings; thus, prospective multicentre studies are needed. Second, although the findings suggest an association between mPAP after BPA and clinical parameters as well as long-term prognosis, establishing a causal relationship between BPA-induced mPAP reduction and these outcomes was hindered by inadequate adjustment for patient background and other factors. Moreover, as an exploratory study, robust causal inferences were inherently challenging. Third, this study included only patients who underwent follow-up RHC, and 53 patients were excluded for this reason. Among these excluded patients, 11 died during the follow-up period, raising the possibility that selection bias cannot be completely ruled out. In the analytic cohort (n=304), pneumonia and unknown cause were frequent among the 13 deaths; by contrast, in the excluded group (n=53), of the 11 deaths only one was due to pneumonia and two were of unknown cause. The remaining causes included malignancy (n=4), ischaemic heart disease (n=1), senility (n=1), cellulitis (n=1) and intestinal obstruction (n=1). This distribution suggests that deaths in the excluded cohort were not predominantly attributable to CTEPH, and comorbidities may have limited the feasibility of follow-up RHC. Fourth, CMR images were performed as close as possible to the first follow-up RHC, with a median interval of 0 months (IQR 0–2). However, the variability in timing between CMR and RHC may have led to inconsistencies in the assessment of right ventricular function relative to haemodynamic status, potentially introducing measurement bias. Finally, the criteria for withdrawing oxygen therapy and pulmonary vasodilators were unclear and based on each physician's discretion.
Back to topPulmonary Arterial Hypertension Pipeline Outlook 2025: Clinical Trial Studies, EMA, PDMA, FDA Approvals, MOA, ROA, NDA, IND, And Companies
DelveInsight's, "Pulmonary Arterial Hypertension Pipeline Insight 2025" report provides comprehensive insights about 55+ companies and 55+ pipeline drugs in Pulmonary Arterial Hypertension pipeline landscape. It covers the Pulmonary Arterial Hypertension pipeline drug profiles, including clinical and nonclinical stage products. It also covers the therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.
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Get insights into Pulmonary Arterial Hypertension Clinical Trials, emerging therapies, and leading companies with DelveInsight @ Pulmonary Arterial Hypertension Treatment Drugs
Pulmonary Arterial Hypertension Emerging Drugs Profile
Sotatercept is a first-in-class therapeutic fusion protein comprised of the extracellular domain of human activin receptor type IIA, fused to the Fc domain of human immunoglobulin G1 (IgG1). It provides balance of the growth-promoting activin growth differentiation factor pathway, and the growth-inhibiting BMP pathway by serving as a ligand trap for the TGF-β superfamily. The United States Food and Drug Administration (FDA) has granted Orphan Drug designation and Breakthrough Therapy designation to sotatercept for the treatment of PAH; the European Medicines Agency (EMA) has granted Priority Medicines (PRIME) designation to sotatercept for the treatment of PAH. Sotatercept is in Phase III clinical trial for the treatment of PAH.
LIQ861 is an investigational, inhaled dry powder formulation of treprostinil designed using the Company's novel PRINT technology and engineered with the goal of enhancing deep-lung delivery of treprostinil in PAH patients by means of a convenient, palm-sized dry powder inhaler. Liquidia resubmits New Drug Application for LIQ861 under the 505(b)(2) regulatory pathway for the treatment of pulmonary arterial hypertension (PAH). The Pulmonary Arterial Hypertension Pipeline Report Provides Insights into
Explore groundbreaking therapies and clinical trials in the Pulmonary Arterial Hypertension Pipeline. Access DelveInsight's detailed report now! @ New Pulmonary Arterial Hypertension Drugs
Pulmonary Arterial Hypertension Companies
Merck Sharp & Dohme, Acceleron Pharma, Liquidia Technologies, Gossamer Bio, Resverlogix, PhaseBio Pharmaceuticals, Pharmosa BioPharm, Complexa, Gmax Biopharm Australia, Mezzion, Radikal Therapeutics, Galectin Therapeutics, Altavant Sciences, Ribomic and others.
Pulmonary Arterial Hypertension pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as
Pulmonary Arterial Hypertension Products have been categorized under various Molecule types such as
Learn about new Pulmonary Arterial Hypertension drugs, pipeline developments, and key companies with DelveInsight's expert analysis @ Pulmonary Arterial Hypertension Market Drivers and Barriers
Scope of the Pulmonary Arterial Hypertension Pipeline Report
Download DelveInsight's in-depth Pulmonary Arterial Hypertension Pipeline report today! @ Pulmonary Arterial Hypertension Companies, Key Products and Unmet Needs
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