We have conducted clinical trials of investigational medicines for the treatment of people with Homocystinuria and Arginase 1 Deficiency.

Developing new therapies takes years of research and clinical trials are a vital part of this process. Data gathered during a clinical trial is necessary to determine if a new drug or biologic is safe and effective in treating human diseases. Regulatory authorities then use the results of clinical trials and other information to decide if a new medicine should be approved.

Disease Area Clinical Trial Overview
The multicenter, open label, dose escalation Phase 1/2 trial is anticipated to enroll 16-20 patients diagnosed with Classical Homocystinuria aged 12 years or older at sites located in the United Kingdom and Australia and aged 18 years and older in the United States. The primary endpoint is the safety and tolerability of pegtarviliase (formerly known as AGLE-177). Secondary endpoints include pharmacokinetic assessments as well as reduction in plasma total homocysteine levels. Patients will be dosed weekly for four weeks.

More information about the trial can be found on

Arginase 1 Deficiency
PEACE (Pegzilarginase Effect on Arginase 1 Deficiency Clinical Endpoints) is a global, randomized, double-blind, placebo-controlled trial enrolled 32 Arginase 1 Deficiency (ARG1-D) patients aged two years and older, and was designed to assess the effects of treatment with pegzilarginase versus placebo over 24 weeks. The study achieved the primary endpoint of plasma arginine reduction from baseline levels. Pronounced and sustained plasma arginine reduction was accompanied by a positive trend in the Gross Motor Function Measure Part E (GMFM-E), a key clinical assessment of a patient’s mobility. Pegzilarginase was well tolerated and safety data was consistent with results from previous clinical trials.

All 31 patients who completed the double-blind placebo-controlled portion of the trial rolled over into the ongoing long-term extension study.

More information about the trial can be found on

Expanded Access Policy

At Aeglea, we are committed to patient health, safety and well-being. We focus our research and development on diseases where we can make a difference in the lives of patients who have few or no effective therapeutic options. As part of the drug development process, we work closely with patients to help us better understand how disease affects their everyday lives, which enables us to find creative solutions for bringing potentially life-changing treatments to patients faster. We are committed to developing potential new therapies and making them available to patients around the world through a variety of mechanisms allowed by regulatory authorities. We work with a sense of urgency to deliver these therapies through the approval process to make them more widely available. Our goal is to provide access to our investigational therapies at the appropriate time and in the correct manner for patients.

Aeglea is creating and developing innovative medicines to treat serious diseases with high unmet medical need. Clinical trials are required to establish whether an investigational medical product is safe and effective, and participants in clinical trials play an important role in helping to improve the understanding and treatment of serious diseases. At present, we believe that participation in one of our clinical trials is the most appropriate way to access our investigational therapies and will provide expanded access only on a limited basis.

Advancing Multiple Development Programs