Aptose joins Beat AML research collaboration

Expansion of Collaboration Enables Identification of Optimal Patient Profiles and Combination Strategies for APTO-253

Aptose Biosciences Inc. (TSX: APS), a clinical-stage company developing new therapeutics and molecular diagnostics that target the underlying mechanisms of cancer, the Knight Cancer Institute at Oregon Health & Science University (OHSU) and The Leukemia & Lymphoma Society (LLS) today announced that Aptose has joined the Beat AML collaboration. Beat AML is a groundbreaking research initiative that includes industry and academic collaborators led by top scientists within the Knight Cancer Institute in collaboration with The Leukemia & Lymphoma Society. Its goal is to accelerate development of potential therapies for acute myeloid leukemia (AML).

Aptose's lead investigational anticancer therapeutic APTO-253 will be profiled extensively against primary cells from hundreds of AML patient samples collected by Beat AML contributors. Under the agreement, Aptose and the Knight Cancer Institute will collaborate on research related to APTO-253, which is designed to provide further insights into the optimal genetic profile of patients likely to benefit from APTO-253 therapy. The research will also aim to identify promising combinations of treatments that may further increase therapeutic efficacy. APTO-253 is a clinical-stage small molecule that acts through induction of the innate tumor suppressor gene Krüppel-like factor 4 (KLF4) and expression of the downstream cell cycle regulator, p21. At the recent American Association for Cancer Research (AACR) Annual Meeting, researchers reported that APTO-253 induces cell death, or apoptosis, in AML cell lines, and synergizes with various conventional therapies for AML and myelodysplastic syndromes (MDS). Aptose is also developing a companion diagnostic to select patients with positive genetic prognostic factors to APTO-253, offering the potential for a personalized medicine in AML.

The multi-institution Beat AML cancer research initiative - designed to leverage the expertise of functional genomic technologies and pharmaceutical collaborators − takes a next-generation personalized medicine approach to vastly accelerate research findings and ultimately improve outcomes for AML patients. AML is a particularly devastating blood cancer with less than 25 percent of newly diagnosed patients surviving beyond five years. It causes more than 10,000 deaths a year in the U.S., and treatment options largely have not changed in the past 30 years.

Brian Druker, M.D., director of the Knight Cancer Institute, serves as the lead investigator for Beat AML. Dr. Druker helped revolutionize cancer treatment from non-specific chemotherapy to highly targeted therapeutic agents with his work, in conjunction with Novartis, to develop Gleevec®.

Jeffrey Tyner, Ph.D., a top leukemia researcher with the Knight Cancer Institute and an assistant professor of Cell, Developmental & Cancer Biology at OHSU, leads the effort in assembling industry collaborators that are part of Beat AML and integral to its ability to discover new treatments.

"Through the Beat AML collaboration we are able to simultaneously test the response of patients' leukemia cells to different drugs and combinations of drugs. This dual process applied to a broad selection of patient samples better equips us to identify genetic drivers of the disease," Dr. Tyner said. "This research design also enables us to better assess the effectiveness of novel, targeted therapies based upon various genetic profiles of patients with the disease. We will be applying these insights to improve the odds of achieving long-term disease remissions for patients."

"Phase Ib/II clinical trials are planned to evaluate APTO-253 both as a single agent and as a key component of combination therapy regimens", said William G. Rice, Ph.D., Aptose's Chairman and Chief Executive Officer. "By working with Dr. Tyner at the Knight Cancer Institute and other leading researchers through the Beat AML initiative, Aptose has the opportunity to gain invaluable data that will inform the clinical development of APTO-253 and optimize its potential to improve outcomes for patients with AML."

"Through the Beat AML initiative we are hoping to do for AML patients what has been achieved with chronic myeloid leukemia - take a blood cancer that was, with few exceptions, a death sentence, and enable patients not only to survive, but to enjoy a longer, richer quality of life," Louis J. DeGennaro, Ph.D., LLS's president and chief executive officer said. "LLS is focused on finding cures and ensuring access to therapies for all blood cancer patients and our priority is to employ the best science to help us address critical unmet medical needs. We are extremely pleased to see Aptose join this collaboration, and are hopeful that their compound will prove to be an effective therapy in our quest to vastly improve outcomes for patients with AML."