AI Therapeutics Announces Name Change to OrphAI Therapeutics

GUILFORD,CT, September 28, 2023 — AI Therapeutics, Inc., a clinical-stage biopharmaceutical company developing novel therapeutics for rare diseases, announced today that it has changed its name to OrphAI Therapeutics Inc.  As part of the name change, the company is launching a new website at “”.  The new name reflects the company’s evolution into a company with multiple Phase 2 clinical programs for the treatment of orphan diseases, and captures its aspirational mission of bringing life-saving therapeutics to patients in need.

“With three agents in development for rare neurodegenerative, pulmonary and oncologic indications, our company has now grown into a clinically focused company committed to bringing life-saving drugs to patients with rare diseases. We are proud of our roots as a company leveraging the power of artificial intelligence in drug development, and continue to explore applications which can inform our orphan drug programs. OrphAI Therapeutics is focused on meeting our ultimate goal of bringing to market innovative disease-altering treatments. Our new name reflects the progress we have made toward that end,” said Brigette Roberts, M.D., Chief Executive Officer and Director of OrphAI Therapeutics.

About OrphAI Therapeutics

OrphAI Therapeutics’ mission is to transform the lives of patients with rare diseases. The company is currently developing three investigational therapies across multiple orphan indications: AIT-101, a first-in-class PIKfyve inhibitor, which recently completed a Phase 2a clinical trial in amyotrophic lateral sclerosis (ALS); LAM-001, a proprietary inhaled form of rapamycin, currently in Phase 2 for bronchiolitis obliterans syndrome (BOS) and pulmonary arterial hypertension (PAH); and AIT-102, a proprietary analogue of mithramycin, in preclinical development for the treatment of SWI/SNF mutated or dysregulated tumors.  To learn more, please visit: