Merck and Pfizer receive FDA Breakthrough Therapy designation for avelumab in metastatic Merkel cell carcinoma

MerckMerck and Pfizer today announced that the US Food and Drug Administration (FDA) has granted avelumab*, an investigational fully human anti-PD-L1 IgG1 monoclonal antibody, Breakthrough Therapy designation for the treatment of patients with metastatic Merkel cell carcinoma (MCC) who have progressed after at least one previous chemotherapy regimen. Breakthrough Therapy designation is designed to accelerate the development and review of medicines that are intended to treat a serious condition, and preliminary clinical evidence indicates that the therapy may demonstrate a substantial improvement over current available therapies. MCC is a rare and aggressive type of skin cancer.(1,2) Each year, there are approximately 1,500 new cases of MCC diagnosed in the US.(3) There is currently no therapy approved specifically for the treatment of metastatic MCC.(4)

The Breakthrough Therapy designation is based on the preliminary evaluation of clinical data from the global Phase II study, JAVELIN Merkel 200, which is assessing the safety and efficacy of avelumab in patients with metastatic MCC whose disease has progressed after at least one prior chemotherapy regimen. Results from this Phase II study are planned for presentation at upcoming scientific congresses in 2016. The designation represents a significant milestone and has the potential to speed the development of avelumab for metastatic MCC patients.

JAVELIN Merkel 200 is a multicenter, single-arm, open-label Phase II study with a primary objective of overall response rate. Secondary endpoints include duration of response, progression-free survival, overall survival and safety. The study, which enrolled 88 patients, is being conducted in sites across Asia Pacific, Australia, Europe and North America.

"Metastatic Merkel cell carcinoma is a devastating disease with limited treatment options currently available for patients," said Dr. Luciano Rossetti, Head of Global Research & Development at Merck’s biopharma business. "With this Breakthrough Therapy designation, we are one step closer to our goal of making a significant difference to patients living with difficult-to-treat cancers, such as metastatic Merkel cell carcinoma, by researching and developing potential new treatment options."

"In less than two months, the alliance between Merck and Pfizer has achieved its third regulatory milestone for avelumab, including Orphan Drug designation and Fast Track designation granted in September and October," said Dr. Mace Rothenberg, Senior Vice President of Clinical Development and Medical Affairs and Chief Medical Officer for Pfizer Oncology. "We are very pleased with the progress of the JAVELIN clinical development program and we are looking forward to presenting additional data on the potential of this investigational compound in Merkel cell carcinoma and other tumor types in 2016."

The clinical development program for avelumab now includes more than 1,400 patients who have been treated across more than 15 tumor types, including breast cancer, gastric/gastro-esophageal junction cancers, head and neck cancer, MCC, mesothelioma, melanoma, non-small cell lung cancer, ovarian cancer, renal cell carcinoma and urothelial (e.g., bladder) cancer.

About the FDA Designations
Breakthrough Therapy designation is designed to expedite the development and review of drugs which are intended to treat a serious condition, and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over available therapy on a clinically significant endpoint(s). The FDA also recently granted avelumab Fast Track and Orphan Drug designations for the treatment of metastatic MCC. The FDA's granting of the Breakthrough Therapy, Fast Track and Orphan Drug designations for metastatic MCC does not alter the standard regulatory requirement to establish the safety and effectiveness of a drug through adequate and well-controlled studies to support approval.

About Merkel Cell Carcinoma (MCC)
MCC is a rare and aggressive disease in which cancer cells form in the top layer of the skin, close to nerve endings. MCC, which is also known as neuroendocrine carcinoma of the skin or trabecular cancer, often starts in those areas of skin that are most often exposed to the sun, including the head and neck, arms, legs, and trunk. Risk factors for MCC include sun exposure and having a weak immune system (i.e., solid organ transplant recipients, people with HIV/AIDS and people with other cancers, such as chronic lymphocytic leukemia, are at higher risk). Caucasian males over age 50 are at increased risk. MCC tends to metastasize at an early stage, spreading initially to nearby lymph nodes, and then potentially to more distant areas in the body, including other lymph nodes or areas of skin, lungs, brain, bones or other organs. Current treatment options for MCC include surgery, radiation and chemotherapy. Treatment for metastatic or Stage IV MCC is generally palliative.

About Avelumab
Avelumab (also known as MSB0010718C) is an investigational fully human anti-PD-L1 IgG1 monoclonal antibody. By inhibiting PD-L1 interactions, avelumab is thought to potentially enable the activation of T-cells and the adaptive immune system. By retaining a native Fc-region, avelumab is thought to engage the innate immune system and induce antibody-dependent cell-mediated cytotoxicity (ADCC). In November 2014, Merck and Pfizer announced a strategic alliance to co-develop and co-commercialize avelumab.

About Merck-Pfizer Alliance
Immuno-oncology is a top priority for Merck and Pfizer. The global strategic alliance between Merck and Pfizer enables the companies to benefit from each other's strengths and capabilities and further explore the therapeutic potential of avelumab, an investigational anti-PD-L1 antibody initially discovered and developed by Merck. The immuno-oncology alliance will jointly develop and commercialize avelumab and advance Pfizer's PD-1 antibody. The alliance will collaborate on up to 20 high priority immuno-oncology clinical development programs, including combination trials, many of which are expected to commence in 2015.

Pfizer Inc.: Working together for a healthier world®
At Pfizer, we apply science and our global resources to bring therapies to people that extend and significantly improve their lives. We strive to set the standard for quality, safety and value in the discovery, development and manufacture of healthcare products. Our global portfolio includes medicines and vaccines, as well as many of the world's best-known consumer healthcare products. Every day, Pfizer colleagues work across developed and emerging markets to advance wellness, prevention, treatments and cures that challenge the most feared diseases of our time. Consistent with our responsibility as one of the world's premier innovative biopharmaceutical companies, we collaborate with health care providers, governments and local communities to support and expand access to reliable, affordable health care around the world. For more than 150 years, Pfizer has worked to make a difference for all who rely on us.

About Merck
Merck is a leading science and technology company in healthcare, life science and performance materials. Around 40,000 employees work to further develop technologies that improve and enhance life - from biopharmaceutical therapies to treat cancer or multiple sclerosis, cutting-edge systems for scientific research and production, to liquid crystals for smartphones and LCD televisions. In 2014, Merck generated sales of € 11.3 billion in 66 countries.

Founded in 1668, Merck is the world's oldest pharmaceutical and chemical company. The founding family remains the majority owner of the publicly listed corporate group. Merck, Darmstadt, Germany holds the global rights to the Merck name and brand. The only exceptions are the United States and Canada, where the company operates as EMD Serono, EMD Millipore and EMD Performance Materials.

* Avelumab is the proposed International Nonproprietary Name for the anti-PD-L1 monoclonal antibody (MSB0010718C). Avelumab is under clinical investigation and has not been proven to be safe and effective. There is no guarantee any product will be approved in the sought-after indication by any health authority worldwide.

1. Hughes MP et al. Merkel cell carcinoma: epidemiology, target, and therapy. Curr Dermatol Rep 2014 Jan 22(3): 46-53
2. Kaae J et al. Merkel cell carcinoma: incidence, mortality, and risk of other cancers. J Natl Cancer Inst 2010 102(11): 793-01
3. Lemos BD et al. Pathologic nodal evaluation improves prognostic accuracy in Merkel cell carcinoma: analysis of 5823 cases as the basis of the first consensus staging system. J Am Acad Dermatol 2010 63(5): 751-61
4. Miller NJ et al. Emerging and mechanism-based therapies for recurrent or metastatic Merkel cell carcinoma. Curr Treat Options Oncol 2013 14(2): 249-63

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