Onyx Pharmaceuticals Announces Positive Top-Line Carfilzomib Data From Phase 2b Study
24 Percent Overall Response Rate and Duration of Response Exceeding Seven Months in Heavily Pretreated Advanced Multiple Myeloma Patients Onyx to Host Investor Teleconference and Webcast Today at 8:00 a.m. Eastern Time to Review Top-Line Data
EMERYVILLE, Calif., July 26 /PRNewswire-FirstCall/ -- Onyx Pharmaceuticals, Inc. (NASDAQ:ONXX) today announced positive top-line results from the Phase 2b 003-A1 study of single-agent carfilzomib, a selective, next generation proteasome inhibitor, in patients with relapsed and refractory multiple myeloma. In an independent review of the data, carfilzomib achieved an overall response rate (partial response or greater) of 24 percent and a median duration of response of 7.4 months in patients who entered the study after receiving a median of five prior lines of therapy (corresponding to a median of 13 anti-myeloma agents) and whose disease was refractory to their last therapeutic regimen. The clinical benefit rate (minimal response or greater) in the study population was 36 percent. Carfilzomib was well-tolerated and there were no new or unexpected toxicities observed. Full results of the trial will be presented at an upcoming scientific meeting. Based on these results, Onyx is continuing discussions with the U.S. Food and Drug Administration (FDA) regarding next steps in filing a new drug application (NDA) for carfilzomib, which the company expects to submit by year-end 2010 for potential accelerated approval in the U.S.
"Despite recent advances in treating multiple myeloma, all patients
eventually relapse. The unmet medical need remains great, as the
outlook for patients with relapsed and refractory disease is grim,"
said Michael G. Kauffman, M.D., Ph.D., Chief Medical Officer of
Onyx Pharmaceuticals. "According to a study from the International
Myeloma Working Group, patients, such as those enrolled in the
003-A1 study, can expect to respond to therapy only 11 percent of
the time and survive for only six to 10 months.(i) The single-agent
activity with durable disease control and favorable tolerability
observed in this study indicate that carfilzomib has the potential
to alter the natural course of this deadly disease."
"Carfilzomib has the potential to be an important therapy in
multiple myeloma and exemplifies the Onyx vision to build a leading
oncology company by developing innovative targeted therapies," said
N. Anthony Coles, M.D., President and Chief Executive Officer of
Onyx Pharmaceuticals. "We are committed to bringing this promising
treatment to patients as quickly as possible by pursuing an
accelerated approval pathway in the U.S., while simultaneously
moving forward with two Phase 3 studies. The first study, ASPIRE,
is designed to support full carfilzomib registration in the U.S. in
earlier-stage patients who have relapsed following initial lines of
therapy, and the second study is designed to support approval in
relapsed and refractory patients in Europe."
Trial Design
The 003-A1 study was an open-label, single-arm Phase 2b trial. The
trial evaluated 266 heavily-pretreated patients with relapsed and
refractory multiple myeloma whose disease was refractory to their
last treatment regimen and who had received at least two prior
therapies, including bortezomib, either thalidomide or
lenalidomide, an alkylating agent, glucocorticoids and an
anthracycline. Refractory disease was defined as < 25% response
or progression during therapy or within 60 days after completion of
therapy.(ii) Patients enrolled in the 003-A1 trial had received a
median of five prior therapeutic regimens, corresponding to a
median of 13 anti-myeloma agents. Patients received carfilzomib at
20mg/m2 for the first cycle followed by 27mg/m2 thereafter for up
to 12 cycles. Patients who completed the 12 cycles were eligible to
enter an extension study. Responses and progression were determined
according to the International Myeloma Working Group (IMWG)
criteria. The trial was conducted in collaboration with the
Multiple Myeloma Research Consortium (MMRC) and at additional sites
in the U.S. and Canada.
"The patients in the 003-A1 trial represent an advanced population
with significant unmet medical need who had received many lines of
therapy and had limited options available to them outside of a
clinical trial, strongly underscoring the need for new treatments.
We are proud to have worked with the Onyx team that is developing
carfilzomib and are encouraged by these results," said Kathy
Giusti, Founder and CEO of the MMRC and a multiple myeloma patient.
The MMRC initiated a relationship with the company (then Proteolix)
in 2006, which included the participation of 11 MMRC Member
Institutions in the 003-A1 trial, representing 36 percent of the
total trial centers and 60 percent of enrolled patients.
"There is a high unmet need for treatment options for patients with
relapsed and refractory multiple myeloma who are no longer
responding to available therapies," said Brian G.M. Durie, M.D.,
Cofounder and Chair of the Board of Directors of the International
Myeloma Foundation (IMF).
Investor Teleconference
Onyx will host a teleconference and webcast on Monday, July 26,
2010, at 8:00 a.m. Eastern Time (5:00 a.m. Pacific Time) to discuss
the top-line data from the Phase 2b 003-A1 study of
carfilzomib.
Interested parties may access a live webcast of the presentation on
the company's website at:
http://www.onyx-pharm.com/view.cfm/32/Event-Calendar
or by dialing 847-619-6547 and using the passcode 27571112. A
replay of the presentation will be available on the Onyx website or
by dialing 630-652-3044 and using the passcode 27571112#
approximately one hour after the teleconference concludes. The
replay will be available through August 9, 2010.
About the Carfilzomib Development Program
Carfilzomib is a selective, next-generation proteasome inhibitor
that has shown encouraging results in a broad clinical trial
program in multiple myeloma.
As previously reported at the 46th American Society of Clinical
Oncology (ASCO) Annual Meeting, an ongoing, companion Phase 2
study, known as the 004 study, demonstrated encouraging overall
response rates, tolerability and durable disease control when
carfilzomib was administered as a single-agent in patients with
relapsed and/or refractory multiple myeloma. In 53 evaluable
patients who had not been previously treated with bortezomib,
carfilzomib achieved an overall response rate of 55 percent and a
median duration of response of 11.5 months at 27mg/m2. Forty
percent of patients were refractory to their most recent therapy
prior to entering the trial. In the overall 004 study population,
treatment with carfilzomib was well-tolerated, and no new or
unexpected adverse events occurred. The most common Grade 3
treatment-emergent adverse events included: pneumonia (11 percent),
anemia (9.7 percent), neutropenia (9.7 percent) and
thrombocytopenia (9 percent). Peripheral neuropathy of any grade
was infrequent, and no Grade 4 adverse events were observed.
The company has also initiated 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. FDA on a Special Protocol
Assessment (SPA) and received Scientific Advice from the European
Medicines Agency (EMA) on the design and planned analysis for the
ASPIRE trial. A second Phase 3 clinical trial, known as the FOCUS
trial, is planned to evaluate carfilzomib in patients with advanced
myeloma and serve as the basis for a European registration.
Carfilzomib is also being studied in advanced solid tumors.
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.(iii) Worldwide, more than 180,000 people are
living with multiple myeloma and approximately 86,000 new cases are
diagnosed annually.(iv)
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, an alpha-folate receptor
targeted inhibitor of the 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.
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, 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, 2009,
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) Kumar S, Crowley J, Lee J-H, Lahuerta JJ, Morgan G et al.
"Outcome of Subjects with Myeloma Relapsing after IMiD and
Bortezomib Therapy: A Multicenter Study from the International
Myeloma Foundation Working Group. Poster presentation: 15th
Congress of the European Hematology Association, June 10-13, 2010,
Barcelona, Spain.
(ii) Anderson et al. Clinically relevant end points and new drug
approvals for myeloma. Leukemia. 2008. 22:231
(iii) National Cancer Institute, Surveillance Epidemiology and End
Results, 2007 Facts and Figures
(iv) International Agency for Research on Cancer, GLOBOCAN 2002
database
Source: Onyx Pharmaceuticals, Inc.
CONTACT: Investors, Julie Wood, Vice President, Corporate
Communications
& Investor Relations, +1-510-597-6505, or Media, Lori Murray,
Director,
Corporate Communications & Investor Relations, +1-510-597-6394,
both of Onyx
Pharmaceuticals, Inc.
Web Site: http://www.onyx-pharm.com/
Posted: July 2010
