NEW HAVEN, Conn., Jan. 20, 2026 (GLOBE NEWSWIRE) -- Orphai Therapeutics Inc. (“Orphai”), a clinical-stage biopharmaceutical company dedicated to advancing novel therapeutics for patients with diseases of significant unmet need, today announced that results from its Phase 2a study of LAM-001 in pulmonary hypertension have been slated for oral presentation at the American Thoracic Society (ATS) 2026 International Conference taking place May 15 – 20, 2026 in Orlando, FL.
In this multicenter, open-label Phase 2a study, LAM-001 was evaluated as an add-on therapy to standard of care in adults with functional class III WHO Group 1, Group 3 or Group 5 pulmonary hypertension. Patients were evaluated at baseline and at 24 weeks for changes in multiple parameters including 6MWD, oxygen consumption, pulmonary vascular resistance (PVR), WHO functional class, plasma NT-proBNP, pulmonary function, and safety.
Details of the presentation are as follows:
| Title | An Open Label, Phase 2a Exploratory Study of Inhaled Sirolimus (LAM-001) for the treatment of Pulmonary Hypertension |
| Abstract # | 13605 |
| Session: | A19 - Mini Symposium: Clinical Trials, Targeted Therapies, Novel Approaches, and Global Landscape of Pulmonary Arterial Hypertension |
About LAM-001
LAM-001 is a proprietary, investigational, once-daily inhaled formulation of sirolimus, also known as rapamycin. LAM-001’s potential as a disease modifying agent in pulmonary hypertension stems from its ability to inhibit mTOR-mediated pulmonary arterial smooth muscle cell proliferation. The mTOR pathway has been shown to be activated in the pulmonary arterial smooth muscle cells of patients with pulmonary hypertension, and mTOR inhibition with rapamycin reverses smooth muscle cell hyperproliferation and attenuates pulmonary vascular remodeling and cardiopulmonary dysfunction in multiple non‑clinical models. By enhancing pulmonary delivery and reducing systemic exposure, LAM-001 offers a promising new potential disease modifying therapy for pulmonary hypertension patients in need.
About Orphai Therapeutics
Orphai Therapeutics is committed to transforming the lives of patients facing serious, underserved diseases by developing disease-modifying therapies to treat their conditions. The company is currently developing LAM-001 for the treatment of pulmonary hypertension associated with interstitial lung disease (PH-ILD), bronchiolitis obliterans syndrome (BOS), and sarcoidosis associated pulmonary hypertension (SAPH). A Phase 2 clinical study in BOS patients is ongoing, and Phase 2 studies in PH-ILD and SAPH are anticipated to begin in 2026. By pioneering innovative approaches, the company aims to offer new hope and improved quality of life to patients worldwide. Discover more about our research and clinical trials at: Orphai-Therapeutics.com
MEDIA CONTACT: info@orphai-therapeutics.com
