Onyx Pharmaceuticals Announces Agreement with the FDA on a Special Protocol Assessment for Planned Phase 3 Carfilzomib Combinati
February 02 2010 - 7:00AM
PR Newswire (US)
EMERYVILLE, Calif., Feb. 2 /PRNewswire-FirstCall/ -- Onyx
Pharmaceuticals, Inc. (NASDAQ:ONXX) today announced that the
company reached agreement with the U.S. Food and Drug
Administration (FDA) on a Special Protocol Assessment (SPA) for the
Phase 3 international randomized trial. This pivotal trial, which
is expected to begin in the first half of 2010, will enroll
patients with relapsed multiple myeloma following treatment with
one to three prior regimens. It is designed to evaluate the
efficacy of carfilzomib in combination with lenalidomide and low
dose dexamethasone, versus lenalidomide and low dose dexamethasone
alone. "The SPA enables us to initiate this carfilzomib Phase 3
combination trial in the first half of this year with increased
clarity on the full approval pathway," said Michael Kauffman, M.D.,
Ph.D., chief medical officer at Onyx. "In the Phase 2b data (from
the "006" trial) reported at the American Society of Hematology
meeting last December, carfilzomib showed promising response rates
and good tolerability with this three-drug combination in patients
with relapsed or refractory myeloma." Dr. Kauffman continued, "This
planned Phase 3 study complements and builds upon the encouraging
single-agent carfilzomib data reported earlier in heavily
pretreated patients with relapsed and refractory multiple myeloma.
Despite the established use of existing therapies, over time
virtually all multiple myeloma patients relapse and succumb to
their disease, meaning new innovative agents are needed. As a
medical community, we have made important strides in extending the
life of myeloma patients and need to continue to identify new ways
we can benefit those who have this disease." Carfilzomib
Development Strategy Based on carfilzomib's efficacy and
tolerability profile demonstrated to-date, Onyx is pursuing a broad
approach to advancing this therapy for multiple myeloma patients.
In the advanced relapsed refractory setting, carfilzomib is
currently being evaluated in a Phase 2b study that could support a
new drug application filing by the end of 2010. In addition to this
potential accelerated approval pathway and for patients earlier in
the course of their disease, Onyx is evaluating the combination of
carfilzomib, Revlimid® and low-dose dexamethasone in a Phase 3
study, as covered by this SPA, which is expected to begin in the
first half of 2010. About Special Protocol Assessments A Special
Protocol Assessment is a written agreement with the FDA on the
design and planned analysis for a clinical trial. It is intended to
form the basis for a marketing application and may only be changed
through a written agreement between the sponsor and the FDA, or if
the FDA becomes aware of new public health concerns. About the
Phase 3 Trial The Phase 3 ("009") trial is a 700 patient,
randomized, open-label, global multi-center study comparing two
treatment regimens for patients with relapsed multiple myeloma and
will be conducted in approximately 200 centers worldwide. This
study is designed to demonstrate the clinical benefit of
carfilzomib in combination with lenalidomide and dexamethasone
based on a primary endpoint of progression-free survival. Patients
will be randomized to receive carfilzomib (20mg/m2 on days 1 and 2
of cycle 1 only, then 27mg/m2 subsequently) or matching placebo, in
addition to a standard dosing schedule of lenalidomide (25mg qd 21
days on, 7 days off) and low-dose dexamethasone (40mg q week).
About Carfilzomib Carfilzomib is a selective, next-generation
proteasome inhibitor that has shown encouraging results in a broad
clinical trial program in multiple myeloma. Carfilzomib is
currently undergoing evaluation as a single agent in multiple Phase
2 and Phase 1 clinical trials in relapsed or refractory multiple
myeloma. These trials include a Phase 2b monotherapy study (known
as "003-A1") in patients with relapsed, refractory multiple
myeloma, which could support a new drug application (NDA) filing by
the end of 2010. Carfilzomib is also being evaluated in advanced
solid tumors. About Multiple Myeloma Multiple myeloma (MM) 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 MM and
approximately 20,000 new cases are diagnosed annually(i).
Worldwide, more than 180,000 people are living with MM 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
next-generation proteasome inhibitor, that is currently being
evaluated in multiple clinical trials for the treatment of patients
with relapsed or relapsed/refractory multiple myeloma and solid
tumors. ONX 0801, a targeted alpha-folate inhibitor, is currently
in Phase 1 testing. For more information about Onyx, visit the
company's website at http://www.onyx-pharm.com/. Nexavar®
(sorafenib) tablets is a registered trademark of Bayer HealthCare
Pharmaceuticals. 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 timing,
progress and results of the clinical development, safety,
regulatory processes and approval, 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 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, 2008,
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 DATASOURCE: Onyx Pharmaceuticals, Inc. CONTACT: Investors,
Julie Wood, Vice President, Investor Relations, +1-510-597-6505, or
Media, Lori Murray, Director, Corporate Communications,
+1-510-597-6394, both of Onyx Pharmaceuticals, Inc. Web Site:
http://www.onyx-pharm.com/
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