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FDA Grants Orphan Drug Designations to OncoMed's Tarextumab for the Treatment of Pancreatic and Small Cell Lung Cancer

REDWOOD CITY, Calif., Jan. 29, 2015 (GLOBE NEWSWIRE) -- OncoMed Pharmaceuticals, Inc. (Nasdaq:OMED), a clinical-stage company developing novel therapeutics that target cancer stem cells (CSCs), or tumor-initiating cells, today announced that the U.S. Food and Drug Administration's Office of Orphan Products Development (OOPD) has granted orphan drug designation to tarextumab (anti-Notch 2/3, OMP-59R5) for the treatment of both pancreatic cancer and small cell lung cancer.

"We are excited to receive two separate orphan drug designations for tarextumab for the treatment of pancreatic and small cell lung cancer," said Paul J. Hastings, OncoMed's Chairman and Chief Executive Officer. "OncoMed is enrolling patients in two randomized Phase 2 clinical trials of tarextumab in pancreatic and small cell lung cancer and we recently reported promising safety and early efficacy data from our Phase 1b studies in these indications."

OncoMed is currently enrolling patients in a randomized Phase 2 clinical trial of tarextumab with gemcitabine plus Abraxane® (paclitaxel protein-bound particles for injectable suspension) (albumin bound) in patients with first-line advanced pancreatic cancer. In January 2015, OncoMed announced positive final Phase 1b clinical and biomarker data from its study of tarextumab in combination with standard of care in pancreatic cancer. Tarextumab was well tolerated with manageable side effects and the three drug combination achieved an overall clinical benefit rate (defined as partial responses and stable disease) of 73 percent (21 of 29 patients). Biomarker analyses showed that among patients whose tumor samples had elevated levels of Notch3 gene expression suggestions of higher response rates and longer survival were noted. For patients with high Notch3 expression, median progression-free and overall survival were 6.6 months and 14.6 months, respectively.

In December 2014, OncoMed began enrolling patients in a randomized Phase 2 clinical study of tarextumab in combination with etoposide and platinum-based chemotherapy in first-line extensive stage small cell lung cancer patients. In a Phase 1b study of tarextumab in combination with etoposide and platinum chemotherapy, the combination was well tolerated. Of sixteen patients evaluable for efficacy, thirteen achieved partial responses (81.3%) and the remaining three patients achieved stable disease for a clinical benefit rate of 100 percent. OncoMed plans to present final data from this Phase 1b clinical study, including safety, biomarker, tumor response and survival data, at a medical meeting later this year.

The FDA Orphan Drug Designation program provides incentives for sponsors to develop medicines that address rare diseases and disorders that might otherwise be overlooked. Such incentives include federal grants, tax credits, waiver of PDUFA filing fees and a seven-year marketing exclusivity period.

According to the American Cancer Society, there are approximately 46,000 new cases of pancreatic cancer each year in the United States. The majority of patients with pancreatic cancer are diagnosed after their cancer has spread locally and/or metastasized to distant organs. Rates of pancreatic cancer have been increasing over the past ten years and it is now the fourth-leading cause of cancer-related deaths. The average life expectancy after the diagnosis of metastatic pancreatic cancer is less than one year.

Small cell lung cancer (SCLC) is expected to make up about ten to fifteen percent of the 224,210 newly diagnosed lung cancer cases and the 159,260 deaths estimated to occur in the U.S. in 2014. SCLC tends to grow and spread quickly, and is typically not discovered until it has metastasized to other parts of the body (extensive stage). In spite of a high sensitivity to chemotherapy and remission rates of up to 80 percent following initial treatment, the median overall survival is less than one year for patients with extensive stage disease.

About Tarextumab (anti-Notch2/3, OMP-59R5)

Tarextumab (anti-Notch2/3, OMP-59R5) is a fully human monoclonal antibody that targets the Notch2 and Notch3 receptors. Preclinical studies have suggested that tarextumab exhibits two mechanisms of action: (1) by downregulating Notch pathway signaling, tarextumab has anti-cancer stem cell activity , and (2) tarextumab affects pericytes, impacting the stromal and tumor microenvironment. Tarextumab is currently being studied in two randomized Phase 2 clinical trials. The "ALPINE" study (Antibody therapy in first-Line Pancreatic cancer Investigating anti-Notch Efficacy and safety) is assessing tarextumab with Abraxane® (paclitaxel protein-bound particles for injectable suspension) (albumin bound) plus gemcitabine in first-line advanced pancreatic cancer patients. The "PINNACLE" study (A Phase 1b/2 Study of OMP-59R5 in Combination with Etoposide and Platinum Therapy in Subjects with Untreated Extensive Stage Small Cell Lung Cancer) is testing tarextumab in combination with etoposide and cisplatin and etoposide and carboplatin in first-line extensive stage small cell lung cancer patients. Tarextumab is part of OncoMed's collaboration with GlaxoSmithKline (GSK). GSK has an option to obtain an exclusive license to tarextumab through completion of the proof-of-concept Phase 2 trials.

About OncoMed Pharmaceuticals

OncoMed Pharmaceuticals is a clinical-stage company focused on discovering and developing novel therapeutics targeting cancer stem cells (CSCs). OncoMed has six anti-cancer product candidates in clinical development, including demcizumab (anti-DLL4, OMP-21M18), tarextumab (anti-Notch2/3, OMP-59R5), anti-Notch1 (OMP-52M51), anti-DLL4/VEGF bispecific antibody (OMP-305B83), vantictumab (anti-FZD7, OMP-18R5), and ipafricept (FZD8-Fc, OMP-54F28), which each target key cancer stem cell signaling pathways including Notch and Wnt. OncoMed plans to file an Investigational New Drug application in early 2015 for anti-RSPO3 (OMP-131R10), an antibody targeting a third key cancer stem cell signaling pathway called R-spondin-LGR. OncoMed is also pursuing discovery of additional novel anti-CSC and cancer immunotherapy product candidates. OncoMed has formed strategic alliances with Celgene Corporation, Bayer Pharma AG and GlaxoSmithKline (GSK). Additional information can be found at the company's website: www.oncomed.com.

Forward-Looking Statements

To the extent that statements contained in this press release are not descriptions of historical facts regarding OncoMed Pharmaceuticals, they are forward-looking statements reflecting the current beliefs and expectations of management made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995, including OncoMed's expectations regarding the safety and potential efficacy of tarextumab; the mechanisms of action of tarextumab; the timing of the availability and presentation of data for the Phase 1b clinical study of tarextumab in small cell lung cancer; and the timing of an Investigational New Drug application filing for OncoMed's anti-RSPO3 antibody. Such forward-looking statements involve substantial risks and uncertainties that could cause OncoMed's clinical development programs, future results, performance or achievements to differ significantly from those expressed or implied by the forward-looking statements. Such risks and uncertainties include, among others, the uncertainties inherent in the preclinical and clinical development process; the risks and uncertainties of the regulatory approval process; OncoMed's dependence on its collaboration partners, including Celgene, GSK and Bayer, for the funding of its partnered programs; OncoMed's ability to raise additional capital to support the development of its unpartnered programs; OncoMed's dependence on the development and marketing efforts of its partners for the commercial success of its partnered product candidates; OncoMed's reliance on third parties to conduct certain preclinical studies and all of its clinical trials; OncoMed's reliance on single source third-party contract manufacturing organizations to manufacture and supply its product candidates; OncoMed's ability to validate, develop and obtain regulatory approval for companion diagnostics; OncoMed's ability to achieve market acceptance and commercial success of its product candidates once regulatory approval is achieved; OncoMed's ability to discover, develop and commercialize additional product candidates; the ability of competitors to discover, develop or commercialize competing products more quickly or more successfully; OncoMed's dependence on its Chairman and Chief Executive Officer, its Chief Scientific Officer, its Chief Medical Officer and other key executives; risk of third party claims alleging infringement of patents and proprietary rights or seeking to invalidate OncoMed's patents or proprietary rights; and the ability of OncoMed's proprietary rights to protect its technologies and product candidates. OncoMed undertakes no obligation to update or revise any forward-looking statements. For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to OncoMed's business in general, see OncoMed's Annual Report on Form 10-K for the fiscal year ended December 31, 2013, filed with the Securities and Exchange Commission (SEC) on March 18, 2014, OncoMed's Quarterly Report on Form 10-Q for the fiscal quarter ended March 31, 2014, filed with the SEC on May 8, 2014, OncoMed's Quarterly Report on Form 10-Q for the fiscal quarter ended June 30, 2014, filed with the SEC on August 7, 2014, and OncoMed's Quarterly Report on Form 10-Q for the fiscal quarter ended September 30, 2014, filed with the SEC on November 4, 2014.

CONTACT: Media & Investors OncoMed Pharmaceuticals Michelle Corral Investor Relations and Corporate Communications michelle.corral@oncomed.com (650) 995-8373 Investors Shari Annes Annes Associates shari.annes@oncomed.com (650) 888-0902

Source:OncoMed Pharmaceuticals, Inc.