Onyx Pharmaceuticals Completes Enrollment in ASPIRE Phase 3 Carfilzomib Combination Trial for the Potential Treatment of Relapsed Multiple Myeloma Re

the European Medicines Agency (EMA) on the design and planned analysis of the ASPIRE trial."The ASPIRE trial, which is designed to support full approval of carfilzomib for patients with relapsed multiple myeloma, is an important component of our overall regulatory strategy to bring carfilzomib to the broadest number of patients in need of new treatment options," said Ted W. Love, M.D., Executive Vice President and Head of Research and Development and Technical Operations at Onyx Pharmaceuticals. "We are pleased to have completed enrollment in the ASPIRE study ahead of schedule, and could have interim results as early as the first half of 2013."The FDA is currently reviewing a New Drug Application (NDA) for potential accelerated approval of carfilzomib in the U.S. for the treatment of patients with relapsed and refractory multiple myeloma. The Prescription Drug User Fee Act (PDUFA) date for completion of the NDA review by the FDA is July 27, 2012.Phase 3 Trial DesignThe ASPIRE (CArfilzomib, Lenalidomide, and DexamethaSone versus Lenalidomide and Dexamethasone for the treatment of PatIents with Relapsed Multiple MyEloma) trial is evaluating carfilzomib in combination with lenalidomide and low-dose dexamethasone, versus lenalidomide and low dose dexamethasone alone, in patients with relapsed multiple myeloma following treatment with one to three prior regimens. The primary endpoint of the trial is progression-free survival. Secondary endpoints include overall survival, overall response rate, duration of response, disease control rate, safety, time-to-progression and time-to-next treatment. Patients were randomized to receive carfilzomib (20mg/m2 on days 1 and 2 of cycle 1 only, then 27mg/m2 subsequently), in addition to a standard dosing schedule of lenalidomide (25mg per day for 21 days on, 7 days off) and low-dose dexamethasone (40mg per week in 4 week cycles), versus lenalidomide and low-dose dexamethasone alone.The study targeted enrollment of 780 patients and is being conducted at approximately 200 sites in North America, Europe, and Israel. For information about this study, please visitwww.clinicaltrials.gov. Phase 1/2 Study ResultsAs previously reported at the 47thAmerican Society of Clinical Oncology (ASCO) Annual Meeting, a Phase 1/2 dose-escalation trial, known as the 006 study, evaluated carfilzomib in combination with lenalidomide and low dose dexamethasone in patients with relapsed and/or refractory myeloma. The overall response rate in the cohorts of patients receiving full doses of the combination was 78 percent in 51 evaluable patients. The combination was well tolerated and demonstrated activity, including in patients who had received prior bortezomib treatment and an immunomodulatory drug (IMiD). No dose-limiting toxicities were observed, and the maximum tolerated dose was not reached. The maximum per-protocol doses of 27mg/m2 carfilzomib, 25mg lenalidomide, and low dose (40mg) dexamethasone were administered. The most common Grade 3/4 adverse events included neutropenia (23%), thrombocytopenia (15%), anemia (15%), hypophosphatemia (13%), fatigue (12%), hyperglycemia (8%), lymphopenia (8%), hyponatremia (6%), diarrhea (6%) and pneumonia (6%). Hematologic adverse events were reversible and manageable.

Read more here: http://www.sacbee.com/2012/02/22/4283523/onyx-pharmaceuticals-completes.html#storylink=cpy

Onyx Pharmaceuticals Announces Data Presentations Highlighting Carfilzomib Development Program at 52nd American Society of Hematology Annual Meeting

Onyx Pharmaceuticals, Inc. today announced the presentation of several studies evaluating carfilzomib, a selective, next-generation proteasome inhibitor, at the American Society of Hematology (ASH) Annual Meeting, December 4-7, 2010, at the Orange County Convention Center in Orlando, FL.

"The accumulating carfilzomib data support its potential as a new treatment option for patients with multiple myeloma, and we are committed to bringing this promising compound to patients as quickly as possible," said Ted W. Love, M.D., Executive Vice President and Head of Research and Development of Onyx Pharmaceuticals. "Based on the full results of the Phase 2b 003-A1 carfilzomib study, which will be presented at ASH, we plan to submit a New Drug Application (NDA) filing for accelerated approval as early as mid-2011."

Onyx Signs Deal with Ono Pharma

Onyx Pharmaceuticals Inc. recently announced that it has entered into an agreement with a Japanese company, Ono Pharmaceutical Co. Ltd., for the development and commercialization of two of Onyx Pharma’s compounds, carfilzomib and ONX 0912, in Japan.

Carfilzomib is currently in multiple trials for the treatment of patients with multiple myeloma and other cancers, and Onyx Pharma plans to file a New Drug Application with the US Food and Drug Administration (FDA) by the end of 2010 for accelerated approval of the candidate. ONX 0912, which is a follow-on drug of carfilzomib, is currently in phase I testing.

As per the terms of the agreement, Ono Pharma will have full rights to develop and market both the compounds for all oncology indications in Japan, while Onyx Pharma retains the marketing rights for the rest of the world.

For the deal, Ono Pharma will make an upfront payment of ¥5 billion ($59 million) to Onyx Pharma and the company will also be entitled to receive up to $280 million on the achievement of certain development and sales milestones. Onyx Pharma will also receive double-digit royalty payments on the sales of these drugs, when approved and marketed in Japan.

Japan-based Ono Pharmaceutical Co. Ltd. is primarily engaged in the manufacture and sale of pharmaceutical products. The company’s products include oral medications for the treatment of bronchial asthma, disturbances of peripheral circulation, chronic pancreatitis and overactive bladder, as well as injectable drugs for the treatment of acute lung injury, generalized intravascular coagulation syndrome, acute phase cerebral thrombosis and blood pressure regulators used during surgical operations.

We currently have a Neutral recommendation on Onyx Pharma, which is supported by a Zacks #3 Rank (short-term Hold rating). We view this deal as a positive for Onyx Pharma, as this partnership allows the company to benefit from Ono Pharma's drug development experience in Japan.

Moreover, the agreement has brought in cash, which should come in good use as Onyx Pharma conducts pre-launch activities for carfilzomib. Carfilzomib could hit the US market as early as 2011 if it is granted accelerated approval by the FDA.

Onyx Pharmaceuticals Inc. Q1 2009 Earnings Call Transcript

Onyx Pharmaceuticals Inc.

Q1 2009 Earnings Call

May 6, 2009; 5:00 pm ET

Executives

Dr. Tony Coles - President & Chief Executive Officer

Laura Brege - Chief Operating Officer

Matt Fust - Chief Financial Officer

Todd Yancey - Vice President of Clinical Development

Julie Wood - Vice President, Investor Relations and Corporate Communications

Analysts

Mona Ashiya - JP Morgan

Jim Birchenough - Barclays Capital

Jessica Li - Goldman Sachs

Howard Liang - Leerink Swann

Jason Zhang - BMO Capital Markets

Stephen Willey - Thomas Weisel Partners

David Moskowitz - Caris & Company

Philip Nadeau - Cowen and Company

Presentation

Operator

Good afternoon ladies and gentleman and welcome to the first quarter financial results conference call. At this time all participants are in a listen-only mode. Later we will conduct a question-and-answer session. Please note that this conference is being recorded.

I’ll now turn the call over to Onyx Pharmaceutical. Onyx Pharmaceuticals you may begin.

Julie Wood

Thank you. Hello and welcome. I’m Julie Wood, Vice President of Investor Relations and Corporate Communications at Onyx Pharmaceuticals. We thank you for joining us today for our first quarter 2009 financial results conference call.

Leading our call is Onyx President and Chief Executive Officer, Dr. Tony Coles. Also providing updates on the teleconference are Laura Brege, our Chief Operating Officer; and Matt Fust, our Chief Financial Officer. Joining us during the Q-and-A period will be Dr. Todd Yancey, Vice President of Clinical Development

Please note that we will be making forward-looking statements during this teleconference that could include financial, clinical or commercial projections. Statements that are not historical facts are forward-looking. References to what we expect, believe, intend to do, plan, estimate or other statements referring to future events or results are intended to identify these statements as forward-looking.

Forward-looking statements are inherently subject to risks and uncertainties. For a discussion of these risks and uncertainties, we refer you to our 10-K for the year ended December 31, 2008, as well as to our 10-Q for the first quarter of 2009.

In addition, we will be presenting and discussing non-GAAP financial measures in this presentation. For reconciliation of these non-GAAP financial measures to corresponding GAAP measures, please see today’s press release which is posted on our website.

I would now like to turn the call over to Tony Coles, who will begin the discussion with an overview of our business. After Tony’s remarks, the management team will review commercial, clinical and financial highlights before we open the call for questions-and-answers. Tony

Dr. Tony Coles

Thanks Julie. There is little doubt that we are living in interesting times and that the rules as we knew them are changing. This is even apparent in our industry, once thought of as recession proof, where we are now seeing the impact of the global economic recession even a month that will weather companies. We’d like to offer that there are some things that will continue to be indicators of success, despite the current economic situation.

Onyx continues to generate revenue from Nexavar, as it expands the large untapped market in liver cancer and drives positive cash flow. We are also increasing our development opportunities, maintaining our profitability and growing already strong cash reserves. These are positive and fundamental indicators of our current and expected success. Our goal, short and long term, is to continually strengthen top line performance and manage Nexavar as a business. So, let’s get started.

We are pleased to report our progress for the first quarter of 2009. Global net sales of Nexavar grew 17% in the first quarter, compared to the same period last year. This growth was driven largely by a string of successful liver cancer launches and the first stages of a focused market expansion for liver cancer.

While the current economic conditions caused some headwinds for Nexavar in the first quarter, we believe this effect will be temporary and that product and reimbursement approvals throughout the year will complement our ongoing market development efforts to achieve strong growth for the brand in 2009. With our clinical program for Nexavar and our recently expanded product portfolio, we remain positioned for continued success. With this in mind I want to take a few minutes to highlight the strengths that differentiate the company today.

First, in kidney cancer, Nexavar remains an important therapy for treatment. With more physicians using targeted therapies sequentially to extend patient lives, we expect Nexavar will remain an important choice for our physicians.

In liver cancer, which is the third leading cause of cancer related deaths, Nexavar is the only approved oral targeted therapy. We don’t expect any competition for sometime, giving us a leadership advantage in this market. We’ve continued unmet medical need in these two tumors around the world. We believe we are a long way from realizing the full value of Nexavar.

Second, it’s our expectation that Nexavar will have broad application across a number of other tumors. Nexavar’s dual mechanism of action, proven efficacy and too tough to treat diseases and established safety profile in tens of thousands of patients worldwide, has spurred tremendous interest from clinicians wanting to explore its use across a range of cancers.

Bayer Halts Skin-Cancer-Drug Test

FRANKFURT--Bayer AG said Monday that it had stopped its Phase III trial of Nexavar as a skin-cancer treatment because it wasn't as effective as hoped, but the pharmaceutical company said it was keeping to its sales guidance for the drug, which is used to treat other cancers.

"We're disappointed with the results of the study and that the therapy did not bring benefit to patients with melanoma, a historically difficult tumor to treat," said Dimitris Voliotis, vice president in global clinical oncology at Bayer HealthCare.

Bayer is jointly developing Nexavar with Onyx Pharmaceuticals Inc.

A spokesman for Bayer, based in Leverkusen, Germany, said it will keep concentrating on treating lung, breast, liver and kidney cancer with its fastest-growing drug. He added that the annual sales guidance of 750 million euros ($993 million) for Nexavar didn't include the drug's possible use in skin cancer.

The trial aimed to improve overall survival of patients receiving Nexavar in combination with chemotherapy, against those receiving a placebo with chemotherapy.

One analyst said the news was slightly disappointing but said he wouldn't change his overall positive stance toward the drug. He added that skin cancer is difficult to treat, and said it is positive that there weren't any distinct side effects from the treatment.

Revenue for Nexavar came in at 464 million euros in 2008.

UPDATE: Bayer Stops Phase III Nexavar Trial For Skin Cancer

FRANKFURT - Pharmaceutical giant Bayer AG (BAY.XE) said Monday that it had stopped its phase III trial of Nexavar as a skin-cancer treatment as it wasn't as effective as hoped, but said it was keeping to its sales guidance for the drug, which is used to treat other cancers. 

"We're disappointed with the results of the study and that the therapy did not bring benefit to patients with melanoma, a historically difficult tumor to treat," said Dimitris Voliotis, Vice President in Global Clinical Oncology at Bayer HealthCare. 

Bayer is jointly developing Nexavar with Onyx Pharmaceuticals Inc. (ONXX). 

A spokesman for the Leverkusen, Germany-based company said it will keep concentrating on treating lung, breast, liver and kidney cancer with its fastest-growing drug. He added that the EUR750 million annual sales guidance for Nexavar didn't include the drug's possible use in skin cancer. 

The trial, sponsored by the National Cancer Institute, aimed to improve overall survival of patients receiving Nexavar in combination with chemotherapy, against those receiving a placebo with chemotherapy. 

One analyst, who asked not to be named, said the news was slightly disappointing but said he wouldn't change his overall positive stance toward the drug. He added that skin cancer is difficult to treat, and said it's positive that there weren't any distinct side effects from the treatment.