Pipeline

Programs
LAM–001

Lymphangioleiomyomatosis

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LAM-001 is the world’s first inhaled mTOR inhibitor and designed to treat the rare lung disease, lymphangioleiomyomatosis. LAM is a genetic-based disease found primarily in women and characterized by hyperactivation of mTOR signaling. LAM-001 has completed clinical trials in normal healthy volunteers and in patients with LAM disease.
Preclinical
Phase 1
Phase 2
Phase 3

monotherapy

Programs
LAM–002

B-cell non-Hodgkin Lymphoma

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LAM-002 (apilimod) is an exquisitely selective first-in-class, orally-administered PIKfyve kinase inhibitor that kills specific cancers and has little to no effect on normal cells. LAM-002 has completed the dose escalation portion of a Phase 1 trial in relapsed or refractory B-cell non-Hodgkin lymphoma, and a recommended Phase 2 dose has been selected. LAM-002 was found to be safe and well-tolerated, and anti-tumor activity was observed in patients who had failed multiple prior lines of therapy. Patient accrual has been completed for LAM-002 as single agent and in combination with either atezolizumab or rituximab in patients with previously treated follicular lymphoma.
Clinical Trial
Preclinical
Phase 1
Phase 2
Phase 3

monotherapy

atezolizumab combination

rituximab combination

Programs
LAM-002

Amyotrophic Lateral Sclerosis

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LAM-002 (apilimod) is a first-in-class, PIKfyve kinase inhibitor that activates transcription factor EB (TFEB), the master regulator of lysosomal biogenesis.  TFEB activation clears toxic aggregates that drive neurodegenerative disorders. A wealth of new data points to the accumulation of toxic proteins as a common mechanism in neurodegenerative conditions disorders including amyotrophic lateral sclerosis (ALS), Parkinson’s disease and Alzheimer’s disease. LAM-002 is preparing to enter a Phase 2 clinical trial for patients suffering from ALS.
Preclinical
Phase 1
Phase 2
Phase 3

Programs
LAM–003

Acute Myeloid Leukemia

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LAM-003 is a once a day, orally bioavailable inhibitor of heat-shock protein 90 (HSP90) that has shown remarkable efficacy in preclinical models of acute myeloid leukemia (AML) as a single agent and when combined with venetoclax or azacitidine. LAM-003 also retains anti-leukemic activity against AML cells rendered resistant to FMS-like tyrosine kinase 3 (FLT3) kinase inhibitors. The preclinical efficacy of LAM-003 was recently highlighted in Blood Advances and at the American Society for Hematology 2019. LAM-003 is currently being evaluated in a Phase 1 trial enrolling patients with previously treated AML.
Clinical Trial
Preclinical
Phase 1
Phase 2
Phase 3

monotherapy

Programs
LAM–004

Facial Angiofibroma

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LAM-004 is a proprietary formulation of topical rapamycin for patients with tuberous sclerosis complex (TSC)-facial angiofibroma. A Phase 1 clinical trial of LAM-004 in patients with facial angiofibroma has been completed. The drug has shown preliminary evidence of efficacy and appears to be well-tolerated. AI Therapeutics has also licensed an associated patent application and Phase 2 clinical data from the University of Texas that 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.
Preclinical
Phase 1
Phase 2
Phase 3

Programs
LAM–005

Rare disease

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LAM-004 is a proprietary formulation of topical rapamycin for patients with tuberous sclerosis complex (TSC)-facial angiofibroma. A Phase 1 clinical trial of LAM-004 in patients with facial angiofibroma has been completed. The drug has shown preliminary evidence of efficacy and appears to be well-tolerated. AI Therapeutics has also licensed an associated patent application and Phase 2 clinical data from the University of Texas that 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. LAM-004 has Orphan Drug Designation in the USA for facial angiofibroma.
Preclinical
Phase 1
Phase 2
Phase 3

A new generation of medicines.

Our aim is to accelerate drug development and increase clinical success by matching drugs to the right patients. We utilize model systems, patient samples derived from our clinical trials, next-generation sequencing, genome editing, chemical genomics, expression profiling and combinatorial drug screening to continually teach the Guardian Angel™ algorithm how to match drugs to patients. AI Therapeutics has advanced four drugs into clinical trials: LAM-001 for lymphangioleiomyomatosis, LAM-002 for B-cell non-Hodgkin lymphoma and ALS, LAM-003 for acute myeloid leukemia, and LAM-004 for facial angiofibroma.