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AbbVie to Seek Approval for Potential Blockbuster Leukemia Drug

Venetoclax meets primary endpoint in midstage trial

A phase II trial of venetoclax (AbbVie/Genentech/Roche), an investigational oral B-cell lymphoma-2 (BCL-2) inhibitor, has met its primary endpoint of achieving overall response rates in patients with relapsed/refractory or previously untreated chronic lymphocytic leukemia (CLL) with the 17p deletion genetic mutation. The findings from this study will serve as the pivotal registration data for a new drug application to the FDA.

In 2015, the FDA granted a “breakthrough therapy” designation to venetoclax for the treatment of CLL in previously treated (relapsed/refractory) patients with the 17p deletion.

The international, open-label clinical trial was designed to evaluate the efficacy and safety of venetoclax in CLL patients with the 17p deletion who relapsed, were refractory to existing therapies, or were previously not treated for their CLL. The study enrolled 157 patients –– 107 in the main cohort evaluating efficacy, and 50 in the safety expansion cohort.

The trial’s primary efficacy endpoint was the overall response rate, and the primary safety endpoints were the number and percentage of patients who experienced treatment-related adverse events, changes in physical examination findings (including vital signs), changes in clinical laboratory test results, and changes in cardiac assessment findings. Secondary efficacy outcome measures included the complete remission rate, the partial remission rate, the duration of response, overall survival, and progression-free survival, among others.

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. It is currently being evaluated in phase III clinical trials for the treatment of relapsed/refractory CLL, along with studies in several other cancers. Venetoclax is an investigational compound, and its safety and efficacy have not been evaluated by the FDA or any other health authority.

CLL is the most common leukemia diagnosed in adults in western countries. In the U.S., CLL accounts for approximately 14,620 new cases of leukemia diagnosed each year.

Approximately 3% to 10% of CLL patients have the 17p deletion at diagnosis, and this mutation occurs in 30% to 50% of patients with relapsed/refractory CLL. The 17p deletion is a genomic alteration in which a part of chromosome 17 is absent. The median life expectancy for CLL patients with this mutation is less than 2 to 3 years.

Source: AbbVie; August 12, 2015.

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