ATL-001: Congenital Erythropoietic Porphyria (CEP)

ATL-001 is a first-in-class oral pharmacological chaperone for CEP, an ultra-rare, life-threatening disorder with no approved treatment. The disease is caused by loss-of-function mutations in uroporphyrinogen III synthase (UROIIIS), an enzyme in the heme biosynthesis pathway, leading to a toxic buildup of porphyrins in skin, bone marrow, and other tissues.
ATL-001 restores UROIIIS folding and activity and acts as a moderate iron chelator, addressing porphyrin accumulation through two complementary mechanisms. The compound is a reformulation of ciclopirox, an approved topical antifungal, repositioned for oral chronic use through a proprietary formulation that resolves the gastrointestinal toxicity that previously blocked systemic administration.
This project is at the clinical phase of discovery.