Abstracts illustrate the drivers and progression of ARG1-D including the long-term challenges faced by patients, caregivers and healthcare professionals
Phase 3 PEACE study baseline characteristics provide important demographic and disease insights on plasma arginine, spasticity, seizures and mobility deficits
Phase 3 PEACE study topline data expected in December 2021
AUSTIN, Texas, Nov. 4, 2021 /PRNewswire/ -- Aeglea BioTherapeutics, Inc. (Nasdaq: AGLE), a clinical-stage biotechnology company developing a new generation of human enzyme therapeutics to benefit people with rare metabolic diseases, today announced that seven abstracts will be presented at the 14th International Congress of Inborn Errors of Metabolism (ICIEM) 2021 being held November 21-23, 2021. The series of abstracts illustrate the building interest and knowledge around the clinical impact of Arginase 1 Deficiency (ARG1-D), the importance of increased disease awareness to drive more rapid and accurate diagnosis and the need for improved treatments given the significant limitations of the current standard of care. The data will be presented at ICIEM in advance of the topline data readout for the pivotal Phase 3 PEACE (Pegzilarginase Effect on Arginase 1 Deficiency Clinical Endpoints) study expected in December.
Key highlights include the first presentation of the baseline characteristics of patients participating in the PEACE study (Enns et al), a growing body of evidence that implicates high arginine as the primary driver of disease pathology (Diaz et al), and several individual patient case studies that reveal the long-term challenges faced by patients with ARG1-D from diagnosis to death. Taken together, the presentations at ICIEM emphasize the need for therapies that reduce and maintain control of plasma arginine levels in patients with ARG1-D.
"We are extremely pleased to have such a strong presence at ICIEM this year. This collection of abstracts provides a clear framework for understanding the devastating impact of ARG1-D and the challenges diagnosing and treating patients with this condition," said Eric Bradford, M.D., M.B.A., chief development officer at Aeglea. "The long-term studies of individual patients and other presentations, which highlight the role of arginine and define the hallmarks of the disease, tell a compelling story about the unmet need, the importance of managing plasma arginine levels and the potential advance that pegzilarginase represents. In addition, the presentation of the baseline characteristics at ICIEM sets the stage for the topline results from PEACE, which will be the first controlled study of an investigative therapy for this progressive and debilitating disorder."
Highlighted Presentations from ICIEM
Title: Patient characteristics in the pivotal Phase 3 PEACE trial of pegzilarginase human enzyme therapy for Arginase 1 Deficiency
Abstract Number: 453
Oral Presentation Session: Novel Therapeutics/Mechanisms
Presentation Date and Time: November 22, 3:15-3:30pm AEDT
Presenter: Gregory Enns, Stanford University School of Medicine
Summary: Thirty-two patients in the PEACE Phase 3 study represent the largest well characterized ARG1-D trial cohort to date. The demographic and disease features of the cohort are generally consistent with insights from the previous Phase 1/2 cohort and published case series, with markedly elevated plasma arginine (despite treatment with standard of care), spasticity, seizures, and mobility deficits.
Highlights include:
- Median plasma arginine was 407 µM, which was similar to baseline levels observed in the Phase 1/2 study of pegzilarginase (median 389 µM)
- Sixty-six percent of randomized patients had a history of spasticity, including 38% who had moderate-to-severe spasticity
- Mean age of participants was 11 years old (standard deviation ±6.5) and the age range was 2-29 years old
- Forty-four percent of patients were classified as Gross Motor Function Classification System (GFMCS) Level 1 and 56% were GMFCS Level 2 or greater (higher classification level indicates more significant functional impairment)
- Patients demonstrated a baseline deficit on mobility assessments including:
- Eighty-four percent on the Gross Motor Function Measure (GMFM) Part E (walking, running and jumping), with deficit defined as a score