Physicians' Financial News November 2007

By Matthew Mahady
Published: Tuesday, Jun 29, 2010
Oncology-Centered Focus Fuels Continued Pharmion Growth

Unlike some biotechnology companies, whose product lines and research and development efforts are intended to address a variety of disease states, Pharmion, headquartered in Boulder, Colorado, built its name by exclusively restricting its research and development efforts to the realms of hematology and oncology. Although, as will be demonstrated, some Pharmion products have attained broader indications, the overwhelming focus of company development efforts, as well as the continued thrust of the overall company mission, is to combat cancer.

Company Basics

Pharmion was founded in 2000, becoming a publicly traded company (PHRM on the Nasdaq exchange) in November 2003. Currently, the company is comprised of over 450 employees who are distributed throughout the world in 13 different locations. Pharmion follows a dual-tiered strategy in order to facilitate the expansion of its product line. Compounds are either developed in-house or purchased from other drug manufacturers via collaborative agreements and/or outright acquisitions. Alliances, collaborative relationships and partnerships have been forged with a range of industry players, ranging from major traditional pharmaceutical companies such as Pfizer and Schering to cutting-edge biotech concerns such as MethylGene and GPC-Biotech.

The company has two FDA-approved products, Vidaza and Innohep, as well as four product candidates (one of which is an alternative formulation of Vidaza) in various stages of clinical development (Figure). In addition, the company is developing and marketing two products outside the United States in various international venues.

Within the company, Innohep and Vidaza, Pharmion’s two flagship agents, are viewed as very important. A company spokesperson characterized the two agents as “the products that really started up this company.”

Injectable Innohep is designed to treat acute symptomatic deep vein thrombosis (DVT) with or without pulmonary embolism when administered in conjunction with warfarin sodium.

Vidaza is an injectable therapy that has been indicated to treat all myelodysplastic syndrome (MDS) subtypes including refractory anemia, refractory anemia with ringed sideroblasts if accompanied by neutropenia or thrombocytopenia or requiring transfusions, refractory anemia with excess blasts, refractory anemia with excess blasts in transformation, and chronic myelomonocytic leukemia. The DNA methyltransferase inhibitor is currently the market leader and generally acknowledged as the treatment standard for MDS.

Vidaza’s MDS treatment credentials were further bolstered with the late summer 2007 release of Phase III survival data drawn from what the company claims was the largest randomized study ever conducted of higher-risk MDS patients. A spokesperson called the results “monumental,” speculating that the 74% increase in survival over two years that was demonstrated has likely been a “key driver” in the dramatic run-up of Pharmion’s stock price over the past several months (a later section of this article covers Pharmion’s Wall Street fortunes in more detail).

Commenting on the significance of the research, Patrick J. Mahaffy, Chief Executive Officer and President, Pharmion, stated, “As the only therapy to have ever demonstrated a survival advantage in MDS, and especially to have demonstrated an improvement of this magnitude, Vidaza is unique in the treatment for this disease. We are extremely gratified with the results from the Vidaza Survival Study, which for the first time bring the hope of prolonged survival for patients with higher-risk MDS.”

Vidaza is also a significant drug because, upon obtaining FDA approval, the DNA demethylating agent became the world’s first epigenetic product. According to a company spokesperson, Epigenetic therapies represent “an emerging area of therapeutic approach,” not to mention a completely new class of anticancer compounds.

In the Pipeline

Agents in clinical trials include amrubicin, a third-generation synthetic anthracycline intended to treat small-cell lung cancer that has been on the Japanese market since 2002; MGCD0103, an oral, isotype-selective, small molecule histone deacetylase (HDAC) inhibitor being developed in partnership with MethylGene that has the potential to combat a variety of cancers including an array of solid- tumor malignancies, MDS, acute myeloid leukemia (AML), relapsed or refractory non-Hodgkin’s lymphoma, and relapsed or refractory Hodgkin’s lymphoma and is the subject of several concurrent Phase II trials, both as a monotherapy and in combination with other agents; oral azacitidine (an oral version of injectable Vidaza), an internally developed and formulated agent that has demonstrated the potential to treat MDS, AML, and malignant solid tumors; and Orplatna, a hormone-refractory prostate cancer (HRPC) therapy, which is being touted as “the only orally bioavailable platinum-based compound in advanced clinical development,” according to information published on the Pharmion website.

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