Biotech Values

[poorgradstudent]

Apparently Mr. Market likes the carfilzomib data from ONXX.

I think the major concern going in was how well it would do with patients who failed Velcade. My view is that it did well in this regard. The significant reduction in peripheral neuropathy is also a plus.

Onyx Pharmaceuticals Announces Positive Complete Results from Carfilzomib Phase 2b Study
24 Percent Overall Response Rate and Duration of Response Exceeding Eight Months in Heavily Pretreated Advanced Multiple Myeloma Patients; Median Overall Survival 15.5 months

Investor Teleconference with Carfilzomib Trial Investigators Today at 10:00 a.m. ET

Emeryville, CA. — Dec. 07, 2010

Onyx Pharmaceuticals, Inc. (Nasdaq: ONXX) today announced positive complete results from the Phase 2b 003-A1 study of single-agent carfilzomib, a next generation proteasome inhibitor, in patients with relapsed and refractory multiple myeloma. Carfilzomib achieved an overall response rate (ORR) (partial response or greater) of 24.1 percent and a median duration of response (DOR) of 8.3 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. In addition, patients enrolled in the study had progressive disease upon entering the trial. The clinical benefit rate (CBR) (minimal response or greater) in the study population was 34.2 percent. The median overall survival (OS) was 15.5 months. Overall survival for responding patients (= minimal response) has not yet been reached; however, based on current data is expected to exceed 19 months.

Seventy-seven percent of patients had grade 1/2 peripheral neuropathy (PN) upon entry into the study. New or worsening of PN was uncommon and Grade 3 PN occurred in less than 1 percent of patients. There were no Grade 4 PN events. A subset analysis of patients with PN (n= 202) on study was also presented at ASH. Efficacy responses in patients with baseline PN were comparable to those seen in the full study population with an ORR of 24 percent and CBR of 34 percent. Based on the full 003-A1 results, Onyx plans to submit a New Drug Application (NDA) filing as early as mid-2011 for potential accelerated approval in the U.S.

These data are being presented today at the 52nd American Society of Hematology Annual Meeting in Orlando by David Siegel, M.D., Ph.D., Division Chief for Myeloma at John Theurer Cancer Center at Hackensack University Medical Center. The results will also be included in the 2011 Highlights of ASH meetings, taking place January through April.

“There is a significant need for new treatment options for these patients with refractory multiple myeloma who have exhausted all other options,” said Dr. Siegel. “This heavily pretreated patient population has received all classes of approved and commonly used myeloma therapies, and the durable responses and tolerability demonstrate carfilzomib’s potential as a promising treatment.”

In the overall study population, no new or unexpected toxicities were observed. The most common Grade 3/4 adverse events were thrombocytopenia (27 percent), anemia (22 percent), lymphopenia (18 percent), and neutropenia (10 percent).

In a subset analysis of patients who were refractory to bortezomib in their last line of therapy (n=128), carfilzomib achieved an ORR of 19 percent and CBR of 31 percent. In patients who had received only one prior bortezomib regimen (n=122), the ORR was 30 percent and the CBR was 40 percent.

“We believe that carfilzomib has the potential to be an important new treatment option for patients with relapsed and refractory myeloma,” said Michael G. Kauffman, M.D., Ph.D., Chief Medical Officer of Onyx Pharmaceuticals. “We are particularly encouraged by the activity and very low rates of peripheral neuropathy observed in this study.”

Two hundred and sixty-six patients with relapsed and refractory multiple myeloma were enrolled in the study, and 257 patients were evaluable for response. The primary endpoint was ORR. Secondary endpoints included CBR, DOR, OS, time to progression (TTP), progression free survival (PFS), and safety.

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 percent response or progression during therapy, or progression within 60 days after completion of therapy.i 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.

ASH Investor Teleconference
Investigators will discuss data presentations surrounding carfilzomib in relapsed and/or refractory multiple myeloma, as featured at the 52nd American Society of Hematology (ASH) Annual Meeting and Exposition in Orlando, Florida. The teleconference will begin at 10:00 a.m. ET on December 7, 2010. The live webcast will be available at:

http://www.onyx-pharm.com/view.cfm/32/Event-Calendar