EMERYVILLE, Calif.,
March 28, 2011 /PRNewswire/ -- Onyx
Pharmaceuticals, Inc. (Nasdaq: ONXX) today announced its plans to
expand the Phase 3 European clinical trial FOCUS, which is
evaluating the efficacy and tolerability of carfilzomib, a
selective next-generation proteasome inhibitor. The FOCUS trial is
designed to support the registrational filing with the European
Medicines Agency (EMA) in patients with relapsed and refractory
myeloma.
The FOCUS (CarFilzOmib for AdvanCed
Refractory MUltiple Myeloma European Study) trial
modification includes two key enhancements to the study: changing
the primary end point to overall survival (OS) from
progression-free survival (PFS) and correspondingly increasing
patient enrollment to 300 from 84. These modifications are
supported by overall survival data from the Phase 2b 003-A1 study
evaluating single-agent carfilzomib in patients with relapsed and
refractory multiple myeloma announced at the American Society of
Hematology (ASH) meeting in December
2010. The company expects to review these data with
the EMA and is moving forward with plans to do so.
"Given the encouraging carfilzomib data reported to date,
including the 003-A1 survival data, Onyx decided to make this
change to demonstrate a potential mortality benefit for
carfilzomib," said Ted Love, M.D.,
executive vice president, research and development and technical
operations at Onyx. "We plan to upsize the trial at the
currently active sites by leveraging the momentum we are seeing in
enrollment as well as by adding additional sites, as
appropriate."
Focus Trial Design
As modified, the Phase 3 FOCUS study will be a randomized
300-patient trial evaluating carfilzomib versus best supportive
care of low dose steroids plus cytoxan (optional), in patients with
relapsed and refractory multiple myeloma following treatment with
at least three prior therapies. Patients are being randomized
to receive carfilzomib (20mg/m2 on days 1 and 2 of cycle 1 only,
then 27mg/m2 subsequently). The primary endpoint is overall
survival with secondary endpoints including PFS, overall response
rate (ORR), clinical benefit rate (CBR) and duration of response
(DOR), as well as safety. The study design incorporates
planned interim analyses on the primary endpoint.
Onyx on Track for Submission of NDA in the U.S.
In January, Onyx announced that the U.S. Food and Drug
Administration (FDA) had granted fast track designation for
carfilzomib. Onyx has initiated a rolling submission of a New
Drug Application (NDA) for potential accelerated approval of
carfilzomib in the U.S. Through the Fast Track designation, Onyx is
eligible to submit the carfilzomib NDA on a rolling basis, allowing
Onyx to begin the NDA filing process immediately and giving the FDA
an opportunity to review the completed sections of the registration
application. Onyx has now submitted the non-clinical section of the
carfilzomib NDA with the FDA and commenced its rolling NDA process.
Onyx intends to complete its submission of the NDA for potential
accelerated approval of carfilzomib in the U.S. as early as
mid-2011.
About the Carfilzomib Development Program
The carfilzomib development program includes a large, randomized
international Phase 3 clinical trial, known as the ASPIRE trial,
studying the combination of lenalidomide and low dose dexamethasone
with or without carfilzomib in patients with relapsed multiple
myeloma. The company has an agreement with the U.S. Food and Drug
Administration (FDA) on a Special Protocol Assessment (SPA) and
received Scientific Advice from the European Medicines Agency (EMA)
on the design and planned analysis of the ASPIRE trial.
Carfilzomib is also being evaluated in a broad investigator
sponsored trial program including 1st line multiple myeloma,
combination studies, lymphoma and other malignancies.
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.
Onyx Pharmaceuticals, Inc. is a biopharmaceutical company
committed to improving the lives of people with cancer. The
company, in collaboration with Bayer HealthCare Pharmaceuticals
Inc., is developing and marketing Nexavar® (sorafenib) tablets, a
small molecule drug that is currently approved for the treatment of
liver cancer and advanced kidney cancer. Additionally, Nexavar is
being investigated in several ongoing trials in a variety of tumor
types. Beyond Nexavar, Onyx has established a development pipeline
of anticancer compounds at various stages of clinical testing,
including carfilzomib, a selective proteasome inhibitor, that is
currently being evaluated in multiple clinical trials for the
treatment of patients with relapsed or relapsed/refractory multiple
myeloma in various settings and solid tumors. ONX 0801, an
alpha-folate receptor targeted inhibitor of thymidylate synthase,
and ONX 0912, an oral proteasome inhibitor, are currently in Phase
1 testing. For more information about Onyx, visit the company's
website at www.onyx-pharm.com.
Forward Looking Statements
This press 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 clinical development and regulatory processes related
to carfilzomib, and potential benefits of the modification of the
FOCUS Phase 3 study. These statements are subject to risks
and uncertainties that could cause actual results and events to
differ materially from those anticipated, including, but not
limited to, risks and uncertainties related to: Onyx may
never receive marketing approval for carfilzomib; failures or
delays in Onyx's clinical trials; if approved, Onyx may be
unsuccessful in launching, maintaining adequate supply of or
obtaining reimbursement for carfilzomib; serious adverse side
effects, if they are associated with carfilzomib; competition;
government regulation; and protection of Onyx's intellectual
property. Any statements contained in this report 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" 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
SOURCE Onyx Pharmaceuticals, Inc.