Pipeline

A new generation of medicines.

AI Therapeutics is advancing two drugs into clinical trials: AIT-101 for ALS and LAM-001 for Bronchiolitis Obliterans (BOS), Pulmonary Arterial Hypertension (PAH) and Pulmonary Sarcoidosis.

Program
Preclinical
Phase 1
Phase 2
Phase 3
Programs
AIT–101

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 ALS, FTD, Parkinson’s disease and Alzheimer’s disease. LAM-002 is preparing to enter a Phase 2 clinical trial for patients suffering from ALS or FTD with a mutation in the gene c9ORF72.
Preclinical
Phase 1
Phase 2
Phase 3

Preclinical
Phase 1
Phase 2
Phase 3

Programs
LAM–001

Pulmonary Sarcoidosis

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Details
Preclinical
Phase 1
Phase 2
Phase 3

Programs
LAM-001

Pulmonary Arterial Hypertension

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Details
Preclinical
Phase 1
Phase 2
Phase 3

Programs
AIT–102

Pediatric Oncology

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Preclinical
Phase 1
Phase 2
Phase 3