Boehringer's 'breakthrough' cancer drug comes through in Phase II

Boehringer Ingelheim CMO Klaus Dugi

Boehringer Ingelheim's treatment for a rare form of leukemia prolonged survival and beat out chemotherapy in a midstage study on elderly patients, the company said, results that will help make the case for a drug that has already received the FDA's breakthrough therapy designation.

In a Phase II study on patients over 65 with acute myeloid leukemia (AML), Boehringer's volasertib combined with a low dose of chemo led to a median overall survival of 8 months, beating out 5.2 months on chemo alone. Patients in the treatment arm charted more than double the response rate of the control group--31% to 13.3%--and event-free survival came in at 5.6 months compared to 2.3 months.

The drug, which inhibits polo-like kinase (Plk) to spur cancer cell death, targets patients for whom intensive chemo is not an option. AML predominantly affects the elderly, according to Boehringer, and volasertib's success in Phase II is an encouraging sign for sufferers of a rare and deadly disease, CMO Klaus Dugi said in a statement.

"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," Dugi said.

Boehringer is currently working through a double-blind, multicenter trial testing volasertib's effects on a similar patient population, planning to enroll 660 AML sufferers and wrap up studies in 2016.

The drug picked up the FDA's coveted breakthrough tag last year in light of strong midstage results, guaranteeing a front-row seat with regulators and an expedited trip to a final decision. Boehringer said in an email it will "work closely with the FDA to determine the most efficient path forward" for volasertib, and the company plans to hold off on submitting a new drug application until it has Phase III data in hand..

The latest results were published in the American Society of Hematology journal Blood.

- read the statement
- here's the study abstract