Boehringer Ingelheim has reported results from a Phase II trial of volasertib in combination with low-dose cytarabine (LDAC) in patients with previously untreated acute myeloid leukemia (AML) aged 65 or older and ineligible for intensive remission induction therapy.
Results showed that AML patients lived longer when treated with volasertib combined with LDAC, a form of chemotherapy, compared with LDAC alone.
The data also demonstrated that volasertib, when used in combination with LDAC, increased the percentage of older AML patients who achieved remission.
University Hospital Ulm Department of Internal Medicine III professor and principal Phase II trialinvestigator Konstanze Döhner said: "Despite being a rare disease, AML is one of the most common leukaemias in adults and predominantly affects older people.
"The established approach to treat younger AML patients is an intensive chemotherapy regimen, called intensive induction therapy.
"However, older patients often cannot tolerate these chemotherapy doses, and have very limited treatment options.
"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."
The trial showed patients treated with the combination of volasertib and LDAC had a median overall survival of eight months versus 5.2 months in patients treated with LDAC alone.
According to the company, the response rate (complete remission or complete remission with incomplete blood count recovery) was more than doubled for patients receiving volasertib and LDAC combination versus LDAC alone (31% versus 13.3%).
Volasertib is an investigational compound that inhibits enzymes called Polo-like kinases (Plks).
Plk1 is the best characterised kinase of the Plk family and inhibition of it by volasertib ultimately results in cell death (apoptosis).
Boehringer Ingelheim chief medical officer Klaus Dugi said: "Boehringer Ingelheim is committed to research in areas of unmet medical need, including those in rare diseases.
"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."
The most common non-haematological adverse events for patients receiving volasertib in combination with LDAC were decreased white blood cells with fever and infections and gastrointestinal side effects.
In 2013, volasertib secured breakthrough therapy designation by the US Food and Drug Administration (FDA) and orphan drug designation by the FDA and the European Commission in 2014.
Volasertib is currently being evaluated in combination with LDAC in a randomised, double-blind, multi-centre, controlled Phase III clinical trial for AML called POLO-AML-2.
Image: Boehringer Ingelheim Center (BIC), corporate headquarters building in Ingelheim, Germany. Photo: courtesy of Boehringer Ingelheim GmbH.