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. The Phase 1 trial of LAM-001 has been completed and a Phase 2 trial in LAM patients has been initiated.
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. LAM-002 is now being evaluated in a Phase 2 trial as single agent and in combination with either atezolizumab or rituximab in specific lymphoma subtypes.
Clinical Trial
Preclinical
Phase 1
Phase 2
Phase 3

monotherapy

atezolizumab combination

rituximab combination

Programs
LAM–003

Acute Myeloid Leukemia

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LAM-003 is an orally-administered first-in-class small molecule immuno-modulatory drug for a genomically-defined population of acute myeloid leukemia patients. LAM-003 has potent anti-leukemic activity in preclinical models and a Phase 1 trial of LAM-003 has been initiated.
Clinical Trial
Preclinical
Phase 1
Phase 2
Phase 3

monotherapy

Programs
LAM–004

Rare disease

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Preclinical
Phase 1
Phase 2
Phase 3

Programs
LAM–005

Rare disease

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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 three drugs into clinical trials: LAM-001 for lymphangioleiomyomatosis, LAM-002 for B-cell non-Hodgkin lymphoma, and LAM-003 for acute myeloid leukemia.