AI Therapeutics is now OrphAI Therapeutics

Pipeline

A new generation of medicines.

OrphAI Therapeutics is advancing three drugs into clinical trials/development: AIT-101 for ALS; LAM-001 for Pulmonary Hypertension (PH), Bronchiolitis Obliterans Syndrome (BOS) and Pulmonary Sarcoidosis; AIT-102 for oncology indications including Ewing Sarcoma (ES), Rhabdoid Tumor (RT), and SWI/SNF mutated/dysregulated cancer.

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

Amyotrophic Lateral Sclerosis

Hide Details
Details
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 has completed a Phase 2 clinical trial for patients suffering from ALS  with a mutation in the gene c9ORF72.
Preclinical
Phase 1
Phase 2
Phase 3

Programs
LAM-001

Pulmonary Hypertension

Hide Details
Details
Preclinical
Phase 1
Phase 2
Phase 3

Preclinical
Phase 1
Phase 2
Phase 3

Programs
LAM–001

Pulmonary Sarcoidosis

Hide Details
Details
Preclinical
Phase 1
Phase 2
Phase 3

Programs
AIT–102

Oncology Indications

Hide Details
Details
Preclinical
Phase 1
Phase 2
Phase 3