A new oncology compound from AbbVie ($ABBV) and Roche's ($RHHBY) Genentech division cleared its primary hurdle in a Phase II blood cancer trial, opening the door to an FDA filing by year's end for a drug that some analysts have tapped as a likely blockbuster.
|Roche CMO Sandra Horning|
The drug, venetoclax, met its main goal of beating back cancer cells in patients with a form of chronic lymphocytic leukemia (CLL), the companies said. AbbVie and Genentech aren't yet disclosing detailed data from the 157-patient trial, adding only that its safety profile was in line with previous studies. The companies plan to present full results at a later medical meeting.
Venetoclax, formerly ABT-199, is designed to block the protein B-cell lymphoma-2, which can help cancer cells survive in the blood. The drug's first target is patients who have CLL with a 17p deletion, a tough-to-treat variant that accounts for up to 10% of all CLL cases and as many as half of all relapsed or refractory instances of the cancer, according to AbbVie.
With positive Phase II data in hand, AbbVie and Genentech plan to submit venetoclax for U.S. approval by the end of 2015. The drug's promise in CLL convinced the FDA to confer its coveted breakthrough-therapy designation in May, granting the companies access to top agency officials and a shot at an accelerated approval.
AbbVie and Roche are taking a broad approach to blood cancer with venetoclax, running late-stage studies in other forms of CLL and mid-stage trials testing the drug against non-Hodgkin's lymphoma, diffuse large B-cell lymphoma, acute myeloid leukemia and multiple myeloma.
Venetoclax's future is particularly vital to AbbVie, which paid $21 billion for Pharmacyclics this year to get a 50% share of the CLL drug Imbruvica. AbbVie believes Imbruvica can bring in billions a year with the help of a widened label, and pairing it with venetoclax could create a best-in-class combination, according to the company.
"Approximately 30 to 50 percent of people with relapsed or refractory chronic lymphocytic leukemia have the 17p deletion that makes their disease difficult to treat," said Sandra Horning, MD, Roche's chief medical officer, in a statement. "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."
- read the statement