Astellas and Ambit Announce Data Presentations Highlighting Quizartinib at the American Society of Hematology 53rd Annual Meeting

TOKYO and SAN DIEGO, Dec. 1, 2011 /PRNewswire/ -- Astellas Pharma Inc. (Tokyo: 4503, Astellas) and Ambit Biosciences Corporation (Ambit) today announced that four poster presentations and four oral presentations highlighting the findings from multiple preclinical and clinical studies evaluating quizartinib (formerly known as AC220), a potent and selective FLT3 inhibitor intended for the treatment of acute myeloid leukemia (AML), will be presented at the American Society of Hematology (ASH) 53rd Annual Meeting, December 10-13, 2011, at the San Diego Convention Center in San Diego, Calif. 

Poster Presentations

• AC220, a Potent and Specific FLT3 Inhibitor, Enhances the Activity of Combined Cytarabine and Daunorubicin Chemotherapy in a FLT3-ITD Model of AML (abstract #1538) will be presented by Ambit's Barbara Belli, Ph.D., from 5:30 to 7:30 p.m. Pacific Standard Time (PST) on Saturday, December 10, in Hall GH. The poster will be displayed during the Acute Myeloid Leukemia – Therapy, excluding Transplantation: Poster I session.
• A Phase II Open-Label, AC220 Monotherapy Efficacy Study In Patients With Refractory/Relapsed FLT3-ITD Positive Acute Myeloid Leukemia: Updated Interim Results (abstract #2576) will be presented by MD Anderson Cancer Center's Jorge Cortes, M.D., from 6 to 8 p.m. PST on Sunday, December 11, in Hall GH. This poster will be displayed during the Acute Lymphoblastic Leukemia - Therapy, excluding Transplantation: Poster II session.
• Detection of Phosphorylated and Total FLT3 and STAT5 in Whole Blood: Modulation by AC220 from Phase I and II Trials in AML (abstract #2607) will be presented by Ambit's Ron Nepomuceno, Ph.D., from 6 to 8 p.m. PST on Sunday, December 11, in Hall GH. This poster will be displayed during the Acute Myeloid Leukemia – Therapy, excluding Transplantation: Poster II session.
• Phospho-specific Flow Cytometry of Fixed Whole Blood Demonstrates In Vivo FLT3 Inhibition in Circulating Leukemic Blasts During AC220 Therapy and Accurately Detects the Development of Therapeutic Resistance (abstract #3502) will be presented by the University of Pennsylvania's  Alexander Perl, M.D., from 6 to 8 p.m. PST on Monday, December 12, in Hall GH. This poster will be displayed during the Molecular Pharmacology, Drug Resistance: Poster III session.

Oral Presentations

• FLT3 Inhibitor AC220 Is a Potent Therapy for Myeloproliferative Disease in c-Cbl RING Finger Mutant Mice (abstract #642) will be presented by the University of Western Australia's Wallace Langdon, Ph.D., at 4 p.m. PST on Monday, December 12, in Room 25. The presentation will take place during the Malignant Stem and Progenitor Cells: Characterization and Targeting of Pre-Leukemic and Leukemic Stem Cells oral session.
• Persistent ERK Activation in Bone Marrow Blasts May Account for the Difference in Bone Marrow Versus Peripheral Blood Response to FLT3 Inhibition in FLT3/ITD AML (abstract #736) will be presented by John Hopkins University's Xiaochuan Yang, Ph.D., at 5:15 p.m. PST on Monday, December 12, in Room 5AB. The presentation will take place during the Molecular Pharmacology, Drug Resistance: Molecular Mechanisms and Drug Testing oral session.
• The Response of FLT3/ITD AML to FLT3 Inhibition: Apoptosis of Peripheral Blood Blasts and Differentiation of Bone Marrow Blasts (abstract #943) will be presented by John Hopkins University's Mark Levis, M.D., at 7:30 a.m. PST on Tuesday, December 13, in Room 30. This presentation will take place during the Acute Myeloid Leukemia – Therapy, excluding Transplantation: Phase I Trials and Biologic Correlates oral session.
• Validation of FLT3-ITD as a Therapeutic Target in Human Acute Myeloid Leukemia (abstract #937) will be presented by the University of California at San Francisco's Neil Shah, M.D., at 7:30 a.m. PST on Tuesday, December 13, in Room 31. This presentation will take place during the Acute Myeloid Leukemia - Biology and Pathophysiology: Clinical and Epidemiologic Studies of AML and APL oral session.

About Quizartinib

Quizartinib, formerly known as AC220, is being developed in collaboration between Ambit Biosciences and Astellas Pharma Inc. and is a novel, potent, highly selective, orally bioavailable FMS-like tyrosine kinase-3 (FLT3) inhibitor.  Quizartinib is currently under evaluation in a Phase 2 clinical trial as monotherapy treatment for adult and elderly patients with relapsed/refractory AML that have an internal tandem duplication (ITD) mutation in the FLT3 gene.

About Acute Myeloid Leukemia

Acute myeloid leukemia (AML) is a form of blood cancer.  According to the American Cancer Society, approximately 13,000 adults were newly diagnosed with AML in 2009 in the United States with approximately 9,000 expected to die of the disease in that year.  AML is generally a disease of older people and is uncommon before the age of 40.  The average age of a patient with AML is 67 and median survival for these patients is less than six months.  The five-year survival rate for all AML patients is less than 15 percent. According to a report from Decision Resources, the U.S. AML market is expected to more than double by 2015.

About the Ambit Biosciences/Astellas Pharma Inc. Collaboration

In December 2009, Ambit Biosciences and Astellas Pharma Inc. entered into a global strategic partnership agreement to jointly research, develop and commercialize FLT3 kinase inhibitors in multiple indications, including the lead investigational compound, quizartinib.  The companies are presently evaluating quizartinib in a Phase 2 clinical trial in relapsed and refractory AML patients that have the internal tandem duplication (ITD) mutation in the FLT3 gene.  The companies are also collaborating on a comprehensive development program to explore the utility of quizartinib in other AML patient subpopulations.  Additionally, the companies are collaborating on a research and development program for additional FLT3 inhibitors for a variety of oncology and non-oncology indications.  The companies share equal responsibility and expenses for the development of products in the U.S. and Europe, while Astellas has sole responsibility in the rest of the world.  Astellas will be responsible for implementation of commercialization activities worldwide.  Ambit received a $40 million up-front payment upon entering into the collaboration agreement, and is eligible to receive up to $350 million in development milestone payments, undisclosed sales milestones, and tiered, double-digit royalties on global revenues.  Ambit also has an option to co-promote products in the U.S. where Astellas and Ambit share equally all profits and losses generated from U.S. sales.

About Ambit Biosciences

Ambit Biosciences is a privately held biopharmaceutical company engaged in the development of a robust pipeline of small molecule kinase inhibitors for the treatment of cancer, inflammatory disease and other indications.  Ambit's lead compound, quizartinib (AC220), is a novel, potent, highly selective, orally bioavailable FMS-like tyrosine kinase-3 (FLT3) inhibitor, and is currently under clinical investigation in patients with relapsed or refractory AML.  Ambit is developing quizartinib in collaboration with Astellas Pharma Inc. as part of a worldwide agreement to jointly develop and commercialize FLT3 kinase inhibitors in oncology and non-oncology indications.  In addition to quizartinib, Ambit's clinical pipeline includes AC430, an oral JAK2 inhibitor, and AC480, a pan-HER inhibitor. Ambit also has a preclinical candidate, CEP-32496, a BRAF inhibitor licensed to Cephalon. For more information, visit www.ambitbio.com.

About Astellas

Astellas Pharma Inc., located in Tokyo, Japan, is a pharmaceutical company dedicated to improving the health of people around the world through provision of innovative and reliable pharmaceuticals. Astellas has approximately 16,800 employees worldwide. The organization is committed to becoming a global category leader in Urology, Immunology including Transplantation and Infectious Diseases, Oncology, Neuroscience, and DM complications and Metabolic Diseases. For more information on Astellas Pharma Inc., please visit our website at www.astellas.com/en.

Contacts:
Ambit Biosciences:
Alan Fuhrman
Chief Financial Officer
(858) 334-2133

Ian Stone or David Schull (Media)
Russo Partners
(619) 308-6541
(212) 845-4271
[email protected]
[email protected]

Cindy McGee (Investors)
Russo Partners
(619) 308-6538
[email protected]

Astellas Pharma Inc.:
Corporate Communications
+81-3-3244-3201

SOURCE Ambit Biosciences Inc.