Pegtarviliase is the second clinical development program to emerge from our human enzyme therapeutics platform.

We are developing pegtarviliase (formerly known as AGLE-177) for the treatment of patients with cystathionine beta synthase (CBS) deficiency, also known as Classical Homocystinuria. Homocystinuria is a serious, progressive disorder characterized by elevated levels of total homocysteine. The accumulation of homocysteine leads to a wide range of life-altering complications, including thromboembolic vascular events, skeletal abnormalities (including severe osteoporosis), developmental delay, intellectual disability, lens dislocation and severe near sightedness.

By addressing the underlying cause of Homocystinuria, our goal is to change the course of the disease and change the course of the patient’s life.

Pegtarviliase is a novel engineered human enzyme designed to degrade both homocysteine and homocystine (two homocysteine molecules bound together) to lower abnormally high levels of homocysteine in the blood. We have initiated a first-in-human Phase 1/2 clinical trial to investigate the effectiveness of pegtarviliase in lowering levels of total homocysteine (homocysteine plus homocystine) in people with Homocystinuria. For more information, please visit the Clinical Trials page.

Pegtarviliase has been granted Rare Pediatric Disease and Orphan Drug Designations by the U.S. FDA and received Orphan Drug Designation from the European Medicines Agency.

Pegtarviliase is investigational. Safety and efficacy have not been established by any agency.

Advancing Multiple Development Programs