At Orphai Therapeutics, we are focused on developing treatments for pulmonary conditions with high unmet medical need.

Pulmonary diseases driven by mTOR dysregulation

Dysfunctional mechanistic Target of Rapamycin (mTOR) signaling is a central pathological driver of inflammation, fibrosis and vasculature and airway remodeling across multiple severe lung diseases such as pulmonary hypertension (PH) and bronchiolitis obliterans syndrome (BOS).

Pulmonary hypertension, including PH associated with interstitial lung diseases (PH ILD) and sarcoidosis-associated PH (SAPH), are driven by progressive inflammation, vascular remodeling, declining lung function, rising pulmonary vascular resistance, and increasing strain on the heart. Yet, no approved therapies fully address the underlying pathophysiology of these diseases.

In BOS, small airway obstruction due to fibrosis and immune driven injury ultimately compromise lung function. As a progressive and often life-threatening form of chronic lung allograft dysfunction, BOS leads to irreversible airflow limitation and remains one of the most serious complications of lung transplantation leading to graft failure. No approved therapies exist today.

Our approach

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 diseases stems from its ability to inhibit mTOR-mediated pulmonary dysfunction. By dampening this aberrant signaling, LAM-001 is designed to exert anti-proliferative, anti-fibrotic and immunomodulatory effects. Together, these properties form the biological rationale for exploring mTOR inhibition across pulmonary indications where unchecked cellular growth and tissue remodeling contribute to disease progression and clinical worsening.

Ongoing and planned clinical studies

LAM-001 is currently being studied in multiple indications including PH-ILD, a serious and progressive condition affecting an estimated ~86K patients in the U.S. and ~120K in Europe. Based on compelling Phase 2a data presented at the American Thoracic Society (ATS) in May 2026, the company is advancing LAM-001 into a Phase 2b trial in PH-ILD, with initiation planned for mid-2026 and data anticipated in the first quarter of 2028. LAM-001 is also being evaluated in a Phase 2 study in BOS, a serious complication following lung transplantation affecting an estimated ~17K patients in the U.S. and ~11K in Europe, with data anticipated in the first quarter of 2027. In late 2026, the company plans to initiate a Phase 2 study of LAM-001 in SAPH, a severe complication of sarcoidosis with no approved therapy affecting an estimated ~60K patients in the U.S. and Europe.