Rare Genetic Diseases Resources

Patient Advocacy, Clinical Trials and Additional Information

At Aeglea we are dedicated to changing patients’ lives. Our credo is to put patients first. Successful research and drug development depends on patient advocacy and close collaboration with all members of a disease community including:

  • Patients and their families and/or caregivers
  • Physicians
  • Metabolic dietitians
  • Allied healthcare providers
  • Advocacy organizations
  • Research institutions

Currently, our patient advocacy programs focus on people affected by a rare, genetic urea cycle disorder called Arginase I deficiency. The Aeglea patient advocacy team connects with patient communities and organizations to ensure their voices are heard within our company and to better understand the needs of people living with Arginase I deficiency.

Together we are conducting a retrospective natural history study to increase our understanding of Arginase I deficiency and its impact. We also are working to improve detection of the disease through newborn screening, which is currently available in many, but not all, states.

Current Clinical Trials

We are conducting a clinical trial to assess the safety and efficacy of our enzyme replacement therapy in patients with Arginase I deficiency.

A Phase 1/2 Study of AEB1102 in Patients with Arginase I Deficiency

  • Drug: AEB1102(Pegzilarginase) – Co-ArgI-PEG modified human Arginase I
  • Phase: Phase 1, Phase 2
  • Study Type: Interventional
  • Endpoint Classification: Safety Study/Efficacy Study
  • Intervention Model: Single Group Assignment
  • Masking: Open Label
  • Primary Purpose: Treatment
  • Indication: Arginase I Deficiency, Hyperargininemia
  • Status: Active
  • Additional Information: https://clinicaltrials.gov/ct2/show/NCT02488044?term=aeglea&rank=3

At present, Aeglea Biotherapeutics is not providing experimental therapeutic agents outside of the clinical trial setting. As part of our dedication to changing patients’ lives, we are currently developing a new policy that will address Aeglea’s process to determining patient access to investigational therapeutic agents for compassionate use.