Rare Disease Report

Eryaspase Fails to Meet Primary Endpoints in AML Study

DECEMBER 08, 2017
Mathew Shanley
Data presented at the European Society for Medical Oncology (ESMO) Annual Meeting in Madrid concluded that eryaspase (Graspa) could potentially exhibit clinical benefit in patients with metastatic pancreatic adenocarcinoma.

It was announced this morning, however, that top-line results from a Phase 2b study conducted by ERYTECH Pharma in collaboration with Orphan Europe (Recordati Group) that the drug showed an inability to meet its primary endpoint of overall survival (OS) in acute myeloid leukemia (AML).

AML is a cancer type characterized by its initial presentation in the bone marrow and spreads rapidly to the blood. It expands white blood cells, which then obstruct the production of typical cells. Occasionally, it can extend into other parts of the body, including the lymph nodes, liver, spleen, central nervous system (CNS), and testicles.

123 patients with a median age of 78 years were enrolled in the open-label, randomized clinical study at 30 European sites and were randomized 2-to-1 to receive eryaspase in combination with low-dose cytarabine (LDAC) versus LDAC alone. The study did not meet the OS primary endpoint, as the OS Hazard Ratio (HR) was 1.06 (95% confidence intervals [Cl]; 0.70, 1.61). When adjusting for age and other minor imbalances in the main prognostic factors at baseline, including karyotype and French-American-British (FAB) status, the OS HR was 0.98 (95% CI; 0.64, 1.50).

The median number of months on eryaspase was less then 2 months in both treatment arms. The toxicity profile was acceptable and consistent with previously reported data for the drug.

“These data reflect the complexity of this disease, particularly in the older age group.” commented Iman El-Hariry, MD, PhD, Chief Medical Officer of ERYTECH in a press release. “While we are disappointed with the outcome, we are reassured with the safety profile of eryaspase in these very frail and elderly patients.”

Current standard-of-care for AML commonly includes 2 chemotherapy phases, which usually need to be initiated immediately after diagnosis because of the cancer’s ability to progress quickly.

The condition primarily affects the adult and senior patient population, and the median age of patients diagnosed with AML is approximately 67 years. Per the American Cancer Society, 21,000 new cases of AML were diagnosed in the United States in 2016; nearly half resulted in death.

ERYTECH will hold a conference call and a webcast on Monday, December 11th at 10:00 am EST to further discuss the results of this study.

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