AI Therapeutics reports considerable progress on LAM-001 and LAM-004 clinical trials

Children's Health

AI Therapeutics is a clinical-stage biopharmaceutical company that has created an artificial intelligence-driven drug development platform for matching drugs to new indications.

The company has made significant recent progress on its four clinical assets and proprietary Guardian Angel™ algorithm.


LAM-001 is the world’s first inhaled formulation of rapamycin to be studied in clinical trials. Through low-dose inhalation into the lung, LAM-001 has the potential to deliver efficacious doses of rapamycin while minimizing the systemic side effects observed with oral formulations of rapamycin.

LAM-001 has completed clinical trials in normal healthy volunteers and in patients with lymphangioleiomyomatosis (LAM disease).

LAM-001 may offer an opportunity for women with LAM disease to stabilize their lung function and reduce the side effects associated with oral rapamycin.”

Dr. Simon Johnson, Professor of Respiratory Medicine, University of Nottingham

Added Dr. Lewis Rubin, Professor of Medicine, Emeritus and former Director of the Division of Pulmonary and Critical Care Medicine at the University of California, San Diego School of Medicine, “I also see tremendous potential for LAM-001 as a new mechanistic approach to treat pulmonary arterial hypertension (PAH) since rapamycin has effects on two pathways – mammalian target of rapamycin (mTOR) and bone morphogenetic protein receptor type II (BMPR2) that are involved in the pathogenesis of PAH.”

LAM-001 has Orphan Drug Designations in the US for the therapy of LAM disease and PAH, as well as Orphan Drug Designation in Europe for the therapy of LAM disease.

AI Therapeutics is seeking a collaboration partner with commercial expertise to advance LAM-001 in a registrational trial for LAM disease and a Phase 2 trial for PAH.


LAM-004 is a proprietary formulation of topical rapamycin for patients with facial angiofibroma and other skin diseases.

LAM-004 has completed a Phase 1 trial in patients with facial angiofibroma. The drug has shown preliminary evidence of efficacy and appears to be well-tolerated. AI Therapeutics has licensed key intellectual property (IP) from the Universities of Michigan and Texas, in addition to its own filed IP.

The Phase 2 clinical data licensed from the University of Texas is published in JAMA Dermatology. The data showed excellent safety and efficacy for topical rapamycin in the largest randomized, placebo-controlled trial ever performed in facial angiofibroma patients.

There is a high unmet medical need for a topical rapamycin drug to treat facial angiofibroma, and AI Therapeutics has the most comprehensive knowledge base for understanding how to best design and develop topical rapamycin successfully for use in facial angiofibroma.”

Dr. Mary Kay Koenig, Professor of Pediatrics, University of Texas Health Science Center, Houston

LAM-004 has Orphan Drug Designation in the USA for facial angiofibroma.

AI Therapeutics is seeking a collaboration partner with commercial expertise to advance LAM-004 for facial angiofibroma and other skin indications.

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