Ai Therapeutics Initiates Phase 2 Clinical Trial to Evaluate LAM-001 for Pulmonary Arterial Hypertension (PAH) Treatment
Friday, September 01, 2023
AI Therapeutics, a clinical-stage biopharmaceutical company specializing in rare disease therapeutics, has unveiled the initiation of a Phase 2 study for LAM-001, its innovative inhaled form of sirolimus. This development aims to address pulmonary arterial hypertension (PAH), a rapidly progressive cardiopulmonary disease that affects an estimated 30,000 patients in the US. PAH's five-year survival rate is approximately 57%.
Dr. Aaron Waxman, Principal Investigator of the study and Director of the Pulmonary Vascular Disease Program at Brigham and Women’s Hospital, emphasized the limitations of current PAH therapies that primarily target vasoconstriction. These treatments do not address the underlying cellular remodeling driving the disease. In contrast, LAM-001 operates by modulating both the mTOR and BMPR2 pathways. This approach could potentially reduce smooth muscle cell hyperproliferation and alleviate endothelial cell dysfunction in the pulmonary vasculature, thereby altering the long-term disease course. Dr. Waxman suggested that LAM-001 might complement the existing drug arsenal for treating this challenging disease.
LAM-001 delivers sirolimus directly to the lungs, potentially minimizing systemic exposure and side effects. Dr. Brigette Roberts, CEO of AI Therapeutics, emphasized the need for additional treatment options for PAH patients who are unresponsive to current therapies. The Phase 2 study, spanning 24 weeks, will involve 15 adults with advanced PAH symptoms despite ongoing treatment. The study will assess the safety, tolerability, and efficacy of LAM-001. The primary endpoint is safety and tolerability, with a focus on changes in peak oxygen uptake (VO2 max) recorded through invasive cardiopulmonary exercise testing (iCPET) at week 24. Secondary measures include various hemodynamic metrics, cardiac structure and function, and additional indicators.
LAM-001 is an innovative inhaled formulation of sirolimus, also known as rapamycin. It holds promise due to its potential to inhibit mTOR-mediated pulmonary arterial smooth muscle cell proliferation and improve BMPR2-mediated endothelial cell function. These mechanisms have shown promise in preclinical studies and animal models of PAH. The trial's details are available on the clinicaltrials.gov website under the identifier NCT05798923.
