A phase 2 trial of the investigational medicine Venetoclax met its primary endpoint ( overall response rates ) in patients with relapsed / refractory or previously untreated chronic lymphocytic leukemia ( CLL ) with 17p deletion.
The open-label study evaluated the efficacy and safety of Venetoclax, an inhibitor of the B-cell lymphoma-2 ( BCL-2 ) protein.
The BCL-2 protein prevents apoptosis of some cells, including lymphocytes, and can be expressed in some cancer types. Venetoclax is designed to selectively inhibit the function of the BCL-2 protein.
The safety profile was similar to previous studies and no unexpected safety signals were reported for Venetoclax.
In 2015, the FDA ( Food and Drug Administration ) granted Breakthrough Therapy Designation to Venetoclax for the evaluation of treatment of chronic lymphocytic leukemia in previously treated patients with the 17p deletion.
Chronic lymphocytic leukemia is a slow-progressing cancer of the bone marrow and blood in which the bone marrow makes too many lymphocytes, a type of white blood cell. It is the most common leukemia diagnosed in adults in western countries.
In the United States, chronic lymphocytic leukemia accounts for approximately 14,620 new cases of leukemia diagnosed each year.
Approximately 3-10% of patients with chronic lymphocytic leukemia have 17p deletion at diagnosis, and it occurs in 30-50% of patients with relapsed / refractory chronic lymphocytic leukemia.
The 17p deletion mutation is a genomic alteration in which a part of chromosome 17 is absent.
The median life expectancy for CLL patients with 17p deletion is less than 2-3 years.
The phase 2 trial has enrolled 157 patients, 107 in the main study cohort evaluating efficacy, and 50 patients in the safety expansion cohort.
The primary efficacy endpoint is overall response rate and the primary safety endpoints are the number and percentage of patients who experienced treatment-related adverse events, changes in physical exam findings, including vital signs, changes in clinical laboratory test results and changes in cardiac assessment findings.
Secondary efficacy outcome measures include complete remission rate, partial remission rate, duration of response, overall survival and progression-free survival, among others. ( Xagena )
Source: Abbvie, 2015