Press release

AI Therapeutics Announces Significant Progress on LAM-001 and LAM-004 Programs

Sponsored by Businesswire

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). Stated Dr. Simon Johnson, Professor of Respiratory
Medicine at the University of Nottingham, “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.” 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.1 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,”
said Dr. Mary Kay Koenig, Professor of Pediatrics at the University of
Texas Health Science Center at 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

1Koenig, M. JAMA Dermatol. 2018 Jul1;154(7) :773-780.

About AI Therapeutics

 has built a state-of-the-art deep learning
platform, Guardian AngelTM, that synthesizes public and
proprietary data on drugs and diseases and finds new indications for our
library of new chemical entities. Aided by Guardian AngelTM
we now have four drugs in the clinic, including drugs that shrink
cancers, the most promising candidate to treat ALS (Lou Gehrig’s
disease), and drugs with applications for longevity.

AI Therapeutics is a 4Catalyzer company, and benefits from the
advantages of being part of an incubator with >300 scientists and
engineers, and offices in Connecticut, New York, Silicon Valley, and
Taiwan. The mission of 4Catalyzer is to save the lives of people we love
and make healthcare more accessible.