German drugmaker Boehringer Ingelheim recently announced that its breakthrough treatment for a rare form of leukemia prolonged survival and beat out chemotherapy in a mid-stage study.
The company said that results from a Phase II study showed that patients with previously untreated acute myeloid leukemia (AML) aged 65 or older and ineligible for intensive remission induction therapy, lived longer when treated with volasertib combined with low dose cytarabine (LDAC) compared to LDAC alone. Data on overall survival showed that the addition of volasertib to LDAC increased the percentage of older AML patients who achieved remission. Results from the Phase II study were published in the American Society of Hematology journal, Blood.
AML is an aggressive and devastating blood cancer. Despite being a rare disease, AML is one of the most common leukemias in adults and predominantly affects people over the age of 60. Currently, the established approach to treat younger AML patients is an intensive chemotherapy regimen, called intensive induction therapy. However, many older patients cannot tolerate these chemotherapy doses, and have very limited treatment options and poor prognosis.
“These clinical trial results that evaluated volasertib in combination with a lower intensity chemotherapy are important and have informed future research for this rare disease, where new treatment options are greatly needed,” said Prof. Döhner from the Department of Internal Medicine III of the University Hospital Ulm and principal investigator of the Phase II trial.
The Phase II trial evaluated 87 patients with a median age of 75 years. Results showed that patients treated with volasertib in combination with LDAC had a median overall survival (OS) of 8 months compared to 5.2 months in patients treated with LDAC alone. Additionally, data showed that the cancer was absent in 31 percent of patients after being treated with volasertib with LDAC compared to 13.3 percent of patients who were treated with LDAC alone. The most common non-hematological adverse events reported in patients receiving volasertib were decreased white blood cells with fever and infections and gastrointestinal side effects. These side effects were clinically manageable and were expected, due to volasertib’s mechanism of action.
Volasertib inhibits enzymes called Polo-like kinases (Plks) to spur cancer cell death. The drug’s inhibition of Plk1 activity should block the extremely high cell division that is a characteristic of AML, which may result in cancer regression. The US Food and Drug Administration (FDA) granted volasertib Breakthrough Therapy Designation in 2013 and Orphan Drug Designation in 2014. Boehringer Ingelheim is currently evaluating the drug in combination with LDAC in the Phase III POLO-AML-2 clinical trial for treatment of AML.
“As with other rare and life threatening diseases, the need for new treatment options in AML is very high. Boehringer Ingelheim is committed to research in areas of unmet medical need, including those in rare diseases,” said Prof. Klaus Dugi, Chief Medical Officer, Boehringer Ingelheim. “We are pleased to see that volasertib has shown promising overall survival results in this clinical trial and we are optimistic that the drug will further demonstrate its potential benefit in this rare disease in the ongoing Phase III study.”
Source: Boehringer Ingelheim Pharmaceuticals, Inc.
Last updated: 7/9/14; 11:35am EST