Positive results from pivotal Phase II study of venetoclax

Roche have announced positive results from the Phase II M13-982 study of venetoclax, an investigational medicine developed for hard-to-treat chronic lymphocytic leukaemia.

The study in partnership with AbbVie, met its primary endpoint, showing that venetoclax monotherapy resulted in a clinically meaningful reduction in the number of cancer cells (overall response rate, ORR) in a pre-defined proportion of people with previously treated (relapsed or refractory) chronic lymphocytic leukaemia (CLL) harbouring the 17p deletion. No unexpected safety signals were reported for venetoclax.

“Approximately 30 to 50 percent of people with relapsed or refractory chronic lymphocytic leukaemia have the 17p deletion that makes their disease difficult to treat,” said Sandra Horning, MD, Roche’s Chief Medical Officer and Head of Global Product Development. “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.”

Venetoclax was recently granted Breakthrough Therapy Designation (BTD) by the U.S. Food and Drug Administration (FDA) for the treatment of previously treated CLL with the 17p deletion. This designation is designed to expedite the development and review of medicines intended to treat serious diseases. People with this type of CLL typically have a median life expectancy of less than three years. This is the eighth BTD granted for a Roche medicine, and the second one in haematology.

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