Carfilzomib Safety Data From Ongoing Phase 2b Pivotal Trial in Relapsed and Refractory Multiple Myeloma Show Promising Safety an
December 06 2009 - 10:30AM
PR Newswire (US)
Pivotal Trial Fully Enrolled - Full Data Anticipated in 2010 NEW
ORLEANS, Dec. 6 /PRNewswire-FirstCall/ -- Onyx Pharmaceuticals,
Inc. (NASDAQ:ONXX) today announced updated safety data from the
pivotal Phase 2b 003-A1 study, known as the 003 trial,
demonstrating that carfilzomib is well-tolerated in heavily
pre-treated relapsed and refractory multiple myeloma patients.
These data were presented today at the ASH/ASCO Joint Symposium at
the 51st annual meeting of the American Society of Hematology (ASH)
in New Orleans. Enrollment in this trial is complete (n=269), and
full results, expected in 2010, could support a potential new drug
application (NDA) filing by year-end 2010. "These results show that
carfilzomib is well-tolerated and can be administered at a full
dose over a long period of time even in a very sick patient
population for whom all available treatment options have been
exhausted and who have multiple comorbidities," said lead
investigator Sundar Jagannath, M.D., chief of the Multiple Myeloma
Program, Bone Marrow and Blood Stem Cell Transplantation at St.
Vincent's Comprehensive Cancer Center in New York. "We look forward
to the full data from this trial next year." Dr. Jagannath also
presented results from the Phase 2 003-A0 study, which recruited
patients whose myeloma had relapsed from two or more prior
therapies and was refractory to their previous therapy. This study
is the lead-in study for the current Phase 2b registrational trial
in the same population. Data from this study was initially
presented at the American Society for Clinical Oncology (ASCO)
annual meeting in June 2009. Patients in the study had a median of
five prior therapies. Previously presented efficacy data from 39
evaluable patients in the 003-A0 pilot study included an 18 percent
overall response rate (partial response or better) and a 26 percent
clinical benefit rate (minor response or better), median time to
tumor progression of 5.1 months and a median of 7.4 months response
duration. Based on the emerging safety profile, the 003-A0 protocol
was amended in 2008 to permit increased dosing of up to 27 mg/m(2).
The protocol was also expanded into the 003-A1 Phase 2b trial,
enrolling 269 patients with relapsed and refractory myeloma and a
dose escalation from 20 mg/m(2) to 27 mg/m(2) after one cycle. The
primary endpoint for the Phase 2b 003-A1 pivotal study is overall
response rate, and secondary endpoints include clinical benefit
response, duration of response, progression-free survival,
time-to-progression, overall survival and safety. Dr. Jagannath
presented new safety data on 141 patients in the 003-A1 trial,
showing that carfilzomib was well tolerated at the 27 mg/m(2) dose.
Grade 3/4 hematologic events included anemia (13.5 percent),
thrombocytopenia (16.3 percent), neutropenia (3.5 percent), and
febrile neutropenia (0.7 percent). The rate of grade 3/4 peripheral
neuropathy was 0.7 percent. The regimen was well tolerated with
prolonged administration of more than 12 cycles (48 weeks) in
approximately 10% of patients, and no clinically significant
cumulative toxicities have been noted to date. Both portions of the
trial are being conducted in collaboration with the Multiple
Myeloma Research Consortium. Investor Teleconference Principal
investigators will discuss data presentations surrounding
carfilzomib in relapsed or refractory multiple myeloma, as featured
at ASH. The webcast event will begin at 9:00 a.m. CT/10:00 a.m. ET
on December 7, 2009. The live webcast will be available at:
http://www.onyx-pharm.com/view.cfm/32/Event-Calendar or by dialing
847-413-3362 and using the passcode 25914947. A replay of the
presentation will be available on the Onyx website or by dialing
630-652-3044 and using the passcode 25914947 later in the day. The
replay will be available on the Onyx website through January 7,
2010. 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 in
patients with relapsed, refractory multiple myeloma, the pivotal
trial that 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 anticipated benefits of the acquisition of
Proteolix and the timing, progress and results of the clinical
development, 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 the risk that Proteolix's operations will not be
integrated successfully into Onyx's, the risk that Onyx may not
realize the anticipated benefits of the acquisition and 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, Associate Director, Corporate Communications,
+1-510-597-6394 Web Site: http://www.onyx-pharm.com/
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