Phase 2a results for PORT-77 show rapid, dose-dependent reductions in plasma protoporphyrin and a favorable safety profile in adults with erythropoietic protoporphyria (EPP), according to dermatologist Robert Sarkany. At the higher dose, levels begin falling within six hours and reach their full decrease within four days; the lowest reported reduction is 57%, while the average is 79%.
GondolaBio reports positive results from the Phase IIa GATEWAY study of PORT-77. The investigational oral ABCG2 inhibitor is aimed at treating erythropoietic protoporphyria (EPP) and X-linked protoporphyria (XLP). GondolaBio announces the findings on behalf of its affiliate, Portal Therapeutics.
GondolaBio, on behalf of affiliate Portal Therapeutics, reports positive Phase 2a GATEWAY results for PORT-77 in patients with erythropoietic protoporphyria (EPP). The trial meets its primary endpoint of plasma protoporphyrin IX (PPIX) reduction, with a mean decrease of 79% in the high-dose cohort and 63% in the low-dose cohort.
Gondolabio, through its affiliate Portal Therapeutics, receives FDA orphan drug and fast track designations for PORT-77, an oral small-molecule therapy under development for erythropoietic protoporphyria (EPP) and X-linked protoporphyria (XLP).