Pipeline
Atlas develops oral pharmacological chaperones for rare and ultra-rare diseases caused by destabilized or misfolded proteins. The pipeline has four programs. ATL-001, the most advanced, is in Phase 1/2 for Congenital Erythropoietic Porphyria. The other three are in discovery, supported by our NMR-based Chassys® platform.
ATL-001
Congenital Erythropoietic Porphyria (CEP)
Stage Phase 1/2, ongoing in the US
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.
ATL-002
Tyrosinemia Type I
Stage Discovery, hit validation
Mutations in the fumarylacetoacetate hydrolase (FAH) gene cause human tyrosinemia type I, an inborn error of metabolism characterized by the accumulation of multiple abnormal compounds in both the blood and urine: tyrosine, methionine, tyrosine metabolites, and succinylacetone. We are developing pharmacological chaperones for this disease.
ATL-003
Prion Disease
Stage Discovery, hit identification
Prion disease (e.g., Creutzfeldt-Jakob disease) results from the progressive accumulation of an abnormal form of the naturally occurring cellular prion protein leading to subsequent adverse effects on brain function and ultimately death.
ATL-001b
Porphyria Cutanea Tarda (PCT)
Stage Discovery, lead validation
PCT is the most common of porphyrias and it encompasses a group of acquired and familial disorders in which activity of the uroporphyrinogen decarboxylase (UROD) is deficient. We are currently exploring the possibility of applying pharmacological chaperones to improve heme homeostasis in PCT patients.