Genentech reports positive Phase II trial results of leukemia drug venetoclax

The trial met its primary endpoint, showing that venetoclax monotherapy resulted in a clinically meaningful reduction in the number of cancer cells in a pre-defined proportion of people with previously treated chronic lymphocytic leukemia (CLL) harboring the 17p deletion.

The company noted that no unexpected safety signals were reported for venetoclax.

Earlier the US Food and Drug Administration (FDA) has granted breakthrough therapy designation (BTD) for venetoclax for the treatment of previously treated CLL with the 17p deletion.

Roche Global Product Development head and chief medical officer Sandra Horning said: "Approximately 30% to 50% of people with relapsed or refractory chronic lymphocytic leukemia have the 17p deletion that makes their disease difficult to treat.

"Venetoclax may help restore the natural process that allows these leukemic cells to self destruct, representing a potential new way of helping people with this form of CLL who typically have a poor prognosis and limited treatment options."

The open label, single arm, multicenter Phase II trial (M13-982) included 107 patients and about 50 patients will be enrolled in the safety expansion cohort.

The trial’s primary endpoint is overall response rate (ORR) as determined by an independent review committee, and secondary endpoints include complete response (CR), partial response (PR) and progression-free survival (PFS).

Data from the M13-982 trial will be submitted at an upcoming medical meeting and the company plans to submit these data to the FDA, European Medicines Agency (EMA) and other health authorities around the world for approval consideration.