Aeglea BioTherapeutics Announces Orphan Drug Designations for Homocystinuria

Aeglea BioTherapeutics announced the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to ACN00177 for the treatment of Homocystinuria, a serious metabolic disorder characterized by elevated plasma homocysteine which leads to a wide range of life-altering complications and reduced life expectancy. Additionally, the European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP) has issued a positive opinion recommending Orphan Drug Designation for ACN00177 for the treatment of Homocystinuria in the European Union. ACN00177 is a novel engineered human enzyme therapy designed to lower the total level of homocysteine in the plasma.

"People with Homocystinuria are living with serious complications, including severe vision issues, skeletal abnormalities, intellectual disability and a high risk of thrombosis which is a common cause of early death, yet many patients do not have effective treatments. These designations underscore the very high unmet need for new treatments for this progressive disease and reaffirms our belief that ACN00177 has the potential to change the lives of people with Homocystinuria," said Anthony Quinn, M.B Ch.B, Ph.D., president and chief executive officer of Aeglea. "We look forward to dosing the first person in our Phase 1/2 clinical trial of ACN00177 which will move us one step closer to our goal of providing an impactful therapy for people with Homocystinuria who are in need of new treatment options."

The FDA grants Orphan Drug Designation to drugs and biologics that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the U.S. The designation provides certain benefits, including the potential for up to seven years of market exclusivity upon FDA approval, prescription drug user fee waivers and tax credits for qualified clinical trials. The EMA's Orphan Drug Designation provides benefits, such as protocol assistance, fee reductions and up to ten years of market exclusivity upon regulatory approval, to companies working to develop treatments for life-threatening or chronically debilitating conditions that affect no more than five in 10,000 people in the EU and where no satisfactory treatments are available.

Aeglea is developing ACN00177 for the treatment of patients with cystathionine beta synthase (CBS) deficiency, also known as Classical Homocystinuria. Homocysteine accumulation plays a key role in multiple progressive and serious disease-related complications, including thromboembolic vascular events, skeletal abnormalities including severe osteoporosis, developmental delay, intellectual disability, lens dislocation and severe near-sightedness. ACN00177 has been designed as a novel recombinant human enzyme, which degrades the amino acid homocysteine and its related homocystine dimer. With this mechanism, ACN00177 is intended to lower the abnormally high blood levels of homocysteine in patients with Homocystinuria. Preclinical data demonstrated that ACN00177 improved important disease-related abnormalities and survival in a mouse model of Homocystinuria. The Company initiated a Phase 1/2 trial in the second quarter of 2020 and continues patient identification and administrative activities.