OrphAI Therapeutics

Formerly known as AI Therapeutics and LAM Therapeutics, OrphAI Therapeutics is a developer of precision therapeutics technology designed to treat cancers and rare diseases. The company's technology works to identify and develop treatments for rare orphan conditions using data mining and deep learning to help accelerate drug development. The company also deploys technologies including next-generational gene sequencing, genome editing, chemical genomics, and combinational drug screening with machine learning for drug development.

In September 2023, OrphAI Therapeutics announced the company's name change (from AI Therapeutics), which was intended to reflect the company's evolution into a company with multiple Phase 2 clinical programs for the treatment of orphan diseases and capture the company's aspirational mission.

OrphAI Therapeutics' pipeline of drugs includes the company's three investigational therapies: AIT-101, LAM-001, and AIT-102.

AIT-101 is a proprietary, oral disintegrating formulation of a PIKfyve kinase inhibitor. The active drug substance in AIT-101 is an experimental therapy that has been evaluated and demonstrated to be safe in nearly 1,000 subjects. The potential for AIT-101 is the treatment of Amyotrophic Lateral Sclerosis (ALS) through the improvement of the survival of motor neurons in ALS patients. OrphAI Therapeutics has initiated a clinical trial of LAM-002A, a formulationof the AIT-101 program, in ALS patients with a C9orf72 mtuation to evaluate safety, tolerability, blood brain barrier penetration, and impact on target and disease biomarkers.

LAM-001 is a proprietary dry powder inhaled formulation of sirolimus, also known as rapamycin. Oral sirolimus was first approved as a treatment for kidney transplant rejection in 1999. LAM-001 has been designed to deliver therapeutic doses of rapamycin to the lungs without systemic exposures and concomitant toxicity seen with oral dosing to lead to a safer treatment. OrphAI Therapeutics has initiated clinical studies of LAM-001 in the rare lung diseases pulmonary hypertension (PH) and bronchiolitis obliterans syndrome (BOS), with plans to conduct studies in pulmonary sarcoidosis.

LAM-004 is a proprietary formulation of LAM-001, which has completed a Phase 1 trial in patients with facial angiofibroma. This came after the drug showed preliminary evidence of efficacy and appeared to be well-tolerated.

AIT-102 is a novel molecule selected from a series of analogues of the product mithramycin, which was first approved as a pharmaceutical agent in 1970. It has been used for the treatment of various cancers, although its manufacture was discontinued in 2000 due to its toxicity. AIT-102 is determined to have 20-40 times lower toxicity with equal or greater anti-tumor activity against multiple tumor cell lines.