Onyx Pharmaceuticals, Inc. (Nasdaq: ONXX) reviewed 2012 accomplishments and announced several 2013 business updates, which the company discussed during its presentation at the 31st Annual J.P. Morgan Healthcare Conference on Monday, January 7, 2013 at 9:30 a.m. Pacific Time.
“In 2012, we executed successful launches for Kyprolis” (carfilzomib) for Injection and Stivarga (regorafenib) tablets, a Bayer compound, transforming Onyx from a single-product company into a company with three approved therapies for patients with some of the most difficult-to-treat cancers,” said N. Anthony Coles M.D., Chairman and Chief Executive Officer of Onyx. “Since the launch of Kyprolis in late July of last year, we have seen strong initial adoption trends and rapid account penetration, with net sales exceeding $62 million in 2012. We continue to strategically invest in the development of Kyprolis to unlock additional value across all lines of therapy. We look forward to continuing our business momentum in 2013, with many near-term clinical, regulatory, and commercial milestones across the proteasome and kinase inhibitor franchises.”
2012 Achievements and 2013 Milestones
Kyprolis” (carfilzomib) for Injection
* In July 2012, the U.S. Food and Drug Administration (FDA) granted accelerated approval of Kyprolis” (carfilzomib) for Injection for the treatment of patients with multiple myeloma who have received at least two prior therapies, including bortezomib and an immunomodulatory agent, and have demonstrated disease progression on or within 60 days of completion of the last therapy. Approval is based on response rate. Clinical benefit, such as improvement in survival or symptoms, has not been verified.
* Since the launch of Kyprolis in late July 2012, net sales have exceeded $62M for 2012. Through October 2012, approximately 25% of the estimated 10,000 to 15,000 patients living with third-line or later multiple myeloma in the U.S. annually have received Kyprolis.
* In October 2012, Onyx completed enrollment in the FOCUS trial, an international Phase 3 clinical trial evaluating single-agent carfilzomib in patients with relapsed and refractory myeloma. An interim analysis is planned in the second half of 2013 and, if results are positive, could support regulatory filings in Europe in patients with relapsed/refractory myeloma.
* As part of the global commercialization strategy for Kyprolis, Onyx is exploring opportunities in countries outside of the U.S. that consider marketing authorization based on U.S. approval. Onyx expects to pursue agreements with established distributors for regulatory and commercialization activities in those markets.
* In February 2012, Onyx completed enrollment in the ASPIRE trial, an international Phase 3 trial evaluating carfilzomib plus lenalidomide (Revlimid) and low-dose dexamethasone when compared to lenalidomide and low-dose dexamethasone alone in 780 patients with relapsed multiple myeloma. An interim analysis is planned in the fourth quarter 2013 or later.
* In July 2012, Onyx initiated the ENDEAVOR trial, a global Phase 3 trial evaluating carfilzomib in combination with dexamethasone versus bortezomib (Velcade) with dexamethasone in 888 patients with relapsed multiple myeloma.
* In 2013, Onyx expects to initiate a Phase 3 trial evaluating carfilzomib in patients with newly diagnosed multiple myeloma.
Oprozomib
* A new extended release tablet of oprozomib, an oral proteasome inhibitor, is being assessed in an ongoing Phase 1b/2 trial in hematologic malignancies including multiple myeloma. Preliminary data are expected to be submitted to a medical conference in the first half of 2013.
Stivarga (regorafenib) tablets
* In September 2012, the U.S. FDA approved Bayer’s Stivarga (regorafenib) tablets for the treatment of patients with mCRC who have been previously treated with currently available therapies (including fluoropyrimidine-, oxaliplatin- and irinotecan-based chemotherapy, an anti-VEGF therapy, and, if KRAS wild type, an anti-EGFR therapy). The approval of Stivarga is based on results from the pivotal Phase 3 CORRECT study that demonstrated improvement in overall survival (OS) and progression-free survival (PFS) compared to placebo in patients with mCRC whose disease had progressed after approved standard therapies.
* In May 2012, Bayer submitted an application for European marketing authorization for regorafenib for the treatment of patients with mCRC.
* In September 2012, the Japanese Ministry of Health, Labor and Welfare (MHLW) granted priority review of Bayer’s New Drug Application (NDA) for regorafenib for the treatment of patients with mCRC.
* In the fourth quarter of 2012, Bayer submitted a supplemental NDA for priority review to the FDA and a NDA to the Japanese MHLW for regorafenib in a second potential indication for metastatic and/or unresectable gastrointestinal stromal tumors.
* As part of a broad development program, Bayer has communicated plans to initiate two additional Phase 3 trials in 2013, in second-line hepatocellular carcinoma (HCC) and in CRC following resection of liver metastases.
Stivarga is a Bayer compound developed by Bayer and jointly promoted by Bayer and Onyx in the U.S. In 2011, Bayer entered into an agreement with Onyx, under which Onyx receives a royalty on all global net sales of Stivarga in oncology.
Nexavar (sorafenib) tablets
* Last week, Onyx and Bayer announced top-line results from the DECISION trial in patients with locally advanced or metastatic radioactive iodine (RAI)-refractory differentiated thyroid cancer. The study met its primary endpoint of improving progression-free survival. Full results are expected to be presented at an upcoming medical meeting. The companies anticipate that this data will form the basis for regulatory submission of Nexavar in the treatment of RAI-refractory differentiated thyroid cancer.
* In the first half of 2013, enrollment is expected to complete in the RESILIENCE trial, a Phase 3 study comparing capecitabine (Xeloda) in combination with sorafenib or placebo for treatment of locally advanced or metastatic HER2-negative breast cancer.
Nexavar is co-developed by Onyx and Bayer, except in Japan where Bayer manages all development. The companies co-promote Nexavar in the U.S. Outside of the U.S. Bayer has exclusive marketing rights, and Bayer and Onyx share profits globally, excluding Japan.
Pipeline
* Pfizer Inc. has announced plans to begin a Phase 3 study in 2013 of PD-0332991 (PD-991), a CDK 4/6 inhibitor, for patients with estrogen receptor-positive, HER2-negative advanced breast cancer. Based on a collaboration agreement between the two companies, Onyx would receive a single-digit royalty on any future net sales worldwide if commercialized, as well as milestone payments.
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