Plks Inhibitor Volasertib Effective in Phase II Trial Against AML

Results from a Phase II study demonstrated 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), a form of chemotherapy, compared to LDAC alone.

Remission rates were also higher with Volasertib and LDAC compared to LDAC alone.

"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," commented Prof. Döhner from the Department of Internal Medicine III of the University Hospital Ulm and principal investigator of the Phase II trial. "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 Phase II clinical trial showed patients treated with volasertib combined with LDAC had a median overall survival of 8 months versus 5.2 months in patients treated with LDAC alone. The response rate (complete remission or complete remission with incomplete blood count recovery) was more than doubled for patients receiving volasertib* and LDAC versus LDAC alone (31% versus 13.3%).

Volasertib is an investigational compound. It works by inhibiting enzymes known as Polo-like kinases (Plks). Plk1 is the best characterized kinase of the Plk family. By inhibiting Plk1, volasertib causes cell death. AML is characterized by very high cell division, and researchers believe that the inhibition of Plk1 activity can lead to cancer regression.

"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." commented 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."

Volasertib has the US FDA's Breakthrough Therapy Designation along with Orphan Drug Designation by both the FDA and the European Commission.

The results were reported in Blood, the journal of the American Society of Hematology.

Source: Blood