Pegtibatinase is an investigational therapeutic candidate for the treatment of classical homocystinuria
Pegtibatinase is a novel investigational human enzyme replacement candidate being evaluated in Phase 3 development for the treatment of classical homocystinuria (HCU). Classical HCU is a rare metabolic disorder characterized by elevated levels of plasma homocysteine that can result in life-threatening thrombotic events such as stroke, pulmonary embolism and deep vein thrombosis, ophthalmologic and skeletal complications, as well as developmental delay. Learn more about classical HCU.
Current treatment options for people with classical HCU, including severe dietary restrictions and use of vitamin B6 and betaine, are often ineffective in managing homocysteine levels and a significant unmet need remains.
Pegtibatinase is an investigational PEGylated, recombinant enzyme replacement candidate designed to address the underlying cause of classical HCU — a deficiency in the naturally occurring enzyme cystathionine beta synthase (CBS). The goal of the pegtibatinase program is to develop a well-tolerated, safe and effective treatment that sustainably replaces CBS activity to reduce levels of homocysteine.
Pegtibatinase is currently advancing in the pivotal Phase 3 HARMONY Study to support the potential approval of the investigational therapy for the treatment of classical HCU. The HARMONY Study, which is now enrolling, is a global, randomized, multi-center, double-blind, placebo-controlled Phase 3 clinical trial designed to evaluate the efficacy and safety of pegtibatinase as a novel treatment to reduce total homocysteine (tHcy) levels.
The Company announced that data from the highest dosed cohort in the Phase 1/2 COMPOSE Study showed a clinically meaningful reduction in total homocysteine from baseline. Pegtibatinase was generally well-tolerated across all doses.
Pegtibatinase has been granted Breakthrough Therapy, Rare Pediatric Disease and Fast Track designations by the FDA, as well as Orphan Drug designation in the U.S. and Europe. It is an investigational therapy that has not been approved by the FDA or any other regulatory authority.