2011 Press Releases

Onyx Pharmaceuticals Announces Data Presentations Highlighting Carfilzomib at 53rd American Society of Hematology Annual Meeting

Final Results from Carfilzomib Phase 2 004 Study to be Presented

South San Francisco, CA. — Nov. 07, 2011

Onyx Pharmaceuticals, Inc. (Nasdaq: ONXX) today announced the presentation of several studies evaluating carfilzomib, a selective, next-generation proteasome inhibitor, at the American Society of Hematology (ASH) Annual Meeting, December 10-13, 2011, at the San Diego Convention Center in San Diego, CA.

“The results being presented at ASH demonstrate carfilzomib’s potential activity along all lines of multiple myeloma, and we are encouraged by this clinical dataranging from frontline to relapsed and refractory disease,” said Ted W. Love, M.D., Executive Vice President and Head of Research and Development and Technical Operations at Onyx Pharmaceuticals. “We are committed to bringing this promising therapy to patients as quickly as possible. We have submitted a new drug application for carfilzomib for the potential treatment of patients with relapsed and refractory multiple myeloma under the accelerated approval pathway, and also have a Phase 3 development program well underway.”

Carfilzomib data highlights include:

Final Results from the Bortezomib-Naïve Group of PX-171-004, a Phase 2 Study of Single-Agent Carfilzomib in Patients with Relapsed and/or Refractory Multiple Myeloma

  • Dr. Ravi Vij, Washington University School of Medicine
  • Monday, December 12, 5:00 – 5:15 p.m., Ballroom 20D
  • Oral Session: Myeloma – Therapy, excluding Transplantation: Novel drugs in relapsed/refractory myeloma patients
  • Abstract #813

Final Results of a Frontline Phase 1/2 Study of Carfilzomib, Lenalidomide, and Low-dose Dexamethasone in Multiple Myeloma

  • Dr. Andrzej Jakubowiak, University of Chicago
  • Monday, December 12, 2:45 – 3:00 p.m., Ballroom 20D
  • Oral Session: Myeloma – Therapy, excluding Transplantation: Combinations Therapy for Myeloma
  • Abstract #631

Carfilzomib Combined with Thalidomide and Dexamethasone (CARTHADEX) as Induction Treatment Prior to High-Dose Melphalan in Newly Diagnosed Patients with Multiple Myeloma, a Trial of the European Myeloma Network

  • Dr. Pieter Sonneveld, Erasmus Medical Center, Rotterdam, Netherlands
  • Monday, December 12, 3:15 – 3:30 p.m., Ballroom 20D
  • Oral Session: Myeloma – Therapy, excluding Transplantation: Combinations Therapy for Myeloma
  • Abstract #633

Unfavorable Cytogenetic Characteristics Do Not Adversely Impact Response Rates in Patients with Relapsed and/or Refractory Multiple Myeloma Treated with Single-Agent Carfilzomib on the 003-A1 Study

  • Dr. Andrzej Jakubowiak, University of Chicago
  • Saturday, December 10, 2011, 5:30 – 7:30 p.m., Hall GH
  • Poster session: Myeloma – Therapy, excluding Transplantation: Poster I
  • Abstract #1875

Integrated Safety Data from Phase 2 Studies of Monotherapy Carfilzomib in Patients with Relapsed and Refractory Multiple Myeloma: An Updated Analysis

  • Dr. Seema Singhal, Northwestern University Feinberg School of Medicine
  • Saturday, December 10, 2011, 5:30 – 7:30 p.m., Hall GH
  • Poster session: Myeloma – Therapy, excluding Transplantation: Poster I
  • Abstract #1876

Multivariate Modelling Reveals Evidence of a Dose-Response Relationship in Phase 2 Studies of Single-Agent Carfilzomib

  • Dr. Pierre Squifflet, International Drug Development Institute, Louvain-la-Neuve, Belgium
  • Saturday, December 10, 2011, 5:30 – 7:30 p.m., Hall GH
  • Poster session: Myeloma – Therapy, excluding Transplantation: Poster I
  • Abstract #1877

A Phase 1b/2 Study of Prolonged Infusion Carfilzomib in Patients with Relapsed and/or Refractory Multiple Myeloma: Updated Efficacy and Tolerability from the Completed 20/56 mg/m2 Expansion Cohort of PX-171-007

  • Dr. Kyriakos Papadopoulos, South Texas Accelerated Research Therapeutics
  • Sunday, December 11, 2011, 6:00 – 8:00 p.m., Hall GH
  • Poster session: Myeloma – Therapy, excluding Transplantation: Poster II
  • Abstract #2930

Phase 2 Study of Carfilzomib Combined with Other Anti-Myeloma Agents in Relapsed-Refractory Multiple Myeloma – Updates on the UARK Compassionate Use Protocol

  • Dr. Saad Usmani, University of Arkansas for Medical Sciences
  • Sunday, December 11, 2011, 6:00 – 8:00 p.m., Hall GH
  • Poster session: Myeloma – Therapy, excluding Transplantation: Poster II
  • Abstract #2947

The Speed of Response to Single-Agent Carfilzomib in Patients with Relapsed and/or Refractory Multiple Myeloma: An Exploratory Analysis of Results from Two Multicenter Phase 2 Clinical Trials

  • Dr. Michael Wang, The University of Texas MD Anderson Cancer Center
  • Monday, December 12, 2011, 6:00 – 8:00 p.m., Hall GH
  • Poster session: Myeloma – Therapy, excluding Transplantation: Poster III
  • Abstract #3969

Carfilzomib Development Program
Carfilzomib is being studied in several clinical trials either as a single-agent or in combination with other therapies, including:

  • A Phase 3 clinical trial, known as the ASPIRE trial, is evaluating the combination of lenalidomide and low dose dexamethasone with or without carfilzomib in patients with relapsed multiple myeloma who have received one to three prior therapies. The company has an agreement with the FDA on a Special Protocol Assessment (SPA) on the design and planned analysis of the ASPIRE trial.
  • A Phase 3 clinical trial, called the FOCUS trial to support registration in Europe, is evaluating single-agent carfilzomib in patients with relapsed and refractory myeloma who have received three or more prior therapies.
  • A Phase 2 clinical trial, known as the 004 study, is evaluating single-agent carfilzomib in patients with relapsed and/or refractory multiple myeloma who have received one to three prior therapies.
  • A Phase 1b/2 study, known as the 006 study, is evaluating carfilzomib in combination with lenalidomide and low dose dexamethasone in patients with relapsed and/or refractory myeloma.
  • A Multiple Myeloma Research Consortium (MMRC) Phase 1/2 study is evaluating carfilzomib in combination with lenalidomide and low dose dexamethasone in newly diagnosed patients. This study is supported by Onyx Pharmaceuticals, MMRC, and Celgene Corporation.
  • A Phase 1/2 study being conducted by Onyx’s partner Ono Pharmaceutical Co., Ltd is evaluating carfilzomib in Japanese patients with relapsed/refractory multiple myeloma.
  • An expanded access program is underway in partnership with the Multiple Myeloma Research Foundation for eligible patients in the U.S., with relapsed and refractory multiple myeloma for whom no satisfactory treatment alternatives are available.

About Multiple Myeloma
Multiple myeloma is the second most common hematologic cancer and results from an abnormality of plasma cells, usually in the bone marrow. In the United States, more than 50,000 people are living with multiple myeloma and approximately 20,000 new cases are diagnosed annually.i Worldwide, more than 180,000 people are living with multiple myeloma and approximately 86,000 new cases are diagnosed annually.ii

About Onyx Pharmaceuticals, Inc.
Based in South San Francisco, California, Onyx Pharmaceuticals, Inc. is a global biopharmaceutical company engaged in the development and commercialization of innovative therapies for improving the lives of people with cancer and other serious diseases. The company is focused on developing novel medicines that target key molecular pathways. For more information about Onyx, visit the company's website at www.onyx-pharm.com.

Forward-Looking Statements
This news release contains “forward-looking statements” of Onyx within the meaning of the federal securities laws. These forward-looking statements include without limitation, statements regarding the progress and results of the clinical development, the expanded access program, safety, regulatory processes, commercialization efforts or commercial potential of carfilzomib. These statements are subject to risks and uncertainties that could cause actual results and events to differ materially from those anticipated, including risks related to the submission, review, potential approval of the NDA, development and commercialization of pharmaceutical products. Any statements contained in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Reference should be made to Onyx’s Annual Report on Form 10-K for the year ended December 31, 2010, filed with the Securities and Exchange Commission under the heading “Risk Factors” and Onyx’s Quarterly Reports on Form 10-Q for a more detailed description of such factors. Readers are cautioned not to place undue reliance on these forward-looking statements that speak only as of the date of this release. Onyx undertakes no obligation to update publicly any forward-looking statements to reflect new information, events, or circumstances after the date of this release except as required by law.

i National Cancer Institute, Surveillance Epidemiology and End Results, 2007 Facts and Figures
ii International Agency for Research on Cancer, GLOBOCAN 2002 database