AbbVie, Genentech Leukemia Drug Wins Breakthrough Status Tag

Published: May 11, 2015

AbbVie, Genentech Leukemia Drug Wins Breakthrough Status Tag
May 7, 2015
By Riley McDermid, BioSpace.com Breaking News Sr. Editor

A promising new blood cancer therapy from AbbVie and Genentech that snagged headlines in early December for unexpectedly high rates of response in clinical trial patients has now been granted breakthrough status from the U.S. Food and Drug Administration (FDA), the companies said Thursday.

The investigational drug, dubbed venetoclax, is an inhibitor of the B-cell lymphoma-2 (BCL-2) protein that is being developed by Abbvie in partnership with Genentech and Roche . Drugs that have received the Breakthrough Therapy Designation are deemed to be a “substantial improvement” over existing therapies and often reaps the rewards of faster approval times and extended patent protection.

"The Breakthrough Therapy Designation of venetoclax supports the continued development of this investigational medicine in CLL patients with 17p deletion," said Michael Severino, executive vice president of research and development and chief scientific officer, AbbVie. "The continuing advancement of the venetoclax development program is one example of AbbVie's focus on delivering innovative medicines that address unmet clinical needs."

Wall Street has been keeping a close eye on the drug all spring. An AbbVie exec told BioSpace exclusively in December that venetoclax could also be effective in variety of hematological malignancies, including chronic lymphocytic leukemia and non-Hodgkin's lymphoma.

Gary Gordon, vice president of oncology clinical development at AbbVie, said in an interview with BioSpace that venetoclax has multiple properties that could make it just as effective against other indications as it was when treating acute myelogenous leukemia (AML). AbbVie’s announcement at the American Society of Hematology's annual meeting certainly turned heads in December.

It presented data for its Phase II, open-label, multicenter clinical trial followed 32 patients relapsed/refractory AML or as frontline therapy for patients who are unfit for intensive therapy who took venetoclax. It found that the group showed an overall response rate (ORR) of 15.5 percent, with one patient achieving a complete response and four patients achieving a complete response with incomplete blood count recovery.

That number is significant because complete response (CR) is sometimes called complete remission, and refers to the disappearance of all signs of cancer in response to cancer treatment.

“Venetoclax is a potent, orally bioavailable B-cell lymphoma 2 (BCL-2) selective inhibitor. In order to survive, cancer cells disable their own self-destruct mechanism (apoptosis) to multiply/spread, and the BCL-2 gene prevents apoptosis of some cells, such as lymphocytes,” said Gordon.

“BCL-2 protein inhibitors play a central role in suppressing the protein and restoring apoptosis to kill cancer cells,” he said.

“Overexpression of BCL-2 is common in various hematological malignancies, including CLL and AML, and, in particular, has been associated with drug resistance and poor prognosis in AML patients. AbbVie and Genentech are pioneering BCL-2 research; no treatments that directly target the BCL-2 pathway are currently marketed.”

Venetoclax was already granted Orphan Drug Designation last March by the U.S. Food and Drug Administration for the treatment of diffuse large B-cell lymphoma (DLBCL), an aggressive type of lymphoma and the most common form of NHL. But will AbbVie attempt to push up its trial timelines in order to receive multiple indication approval?

“At this time it is too early to speculate on developmental plans, competition or changes in treatment paradigm for AML,” said Gordon, when asked about possibly leapfrogging the drug into Phase III trials, but he did say there are multiple qualities that make the drug a promising candidate.



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