About six months after completing a Series C financing, Affymax Inc. advanced its erythropoiesis-stimulating agent, Hematide, into a placebo-controlled clinical trial.

"This is our lead product and this is the first product that Affymax has put into a clinical trial," said the company's president and CEO, Arlene Morris. "We started a Phase I trial and that will help us understand the safety and pharmacokinetics, and give us an early read on efficacy."

The move into a Phase I trial marks the beginning of Affymax's transformation from a discovery company to one also focused on drug development. In the last six months, the company has hired key people to leadership positions in the areas of clinical, medical and regulatory affairs and commercial development, and it has raised $20 million in the second tranche of a $40 million Series C financing completed in April. (See BioWorld Today, April 14, 2004.)

Hematide is being developed to treat anemia in patients with chronic kidney disease and cancer. Affymax, of Palo Alto, Calif., aims to determine Hematide's effect on reticulocytes and hemoglobin. Trial results should be available in early 2005. If positive, the company would move into Phase II trials later that year, first in end-stage renal disease and chronic kidney failure, then in cancer.

Hematide is a next-generation peptide-based erythropoiesis-stimulating agent (ESA) designed to stimulate production of red blood cells. It has been effective in animal models, including primates, and it has demonstrated an improved efficacy profile, superior stability and an extended duration of action compared to recombinant protein products. Hematide has an amino acid sequence that is unrelated to erythropoietin or any other known naturally occurring human sequence.

An ESA that is long-acting and unlikely to generate antibodies to natural erythropoietin could offer major benefits for chronic anemia patients. Advantages of peptide-based drugs can include reduced immunogenicity, improved dosing convenience, flexible storage and improved manufacturability.

"We think we have better efficacy and we have a much longer half-life with this product than those on the market," Morris told BioWorld Today.

"Another really key advantage is stability," she said. "The product has a very long shelf-life and can be stored at room temperature."

Hematide also offers a cost advantage since it is synthetic, and it has the potential to be given intranasally, Morris said. While the Phase I trial will use an intravenous form, and the Phase II trial would use both an IV form and a subcutaneous version, Affymax intends to work on developing an intranasal form of Hematide.

There are more than 3.4 million Americans with anemia. It can be brought on by chronic kidney disease, for example, and 75 percent of cancer patients experience severe fatigue associated with anemia.

ESAs on the market for anemia related to chronic kidney disease and cancer pulled in about $10 billion in worldwide sales in 2003. ESA therapy has shown ability to reduce the need for blood transfusions, to decrease anemia-associated morbidity and to lead to an improvement in quality of life. Some analysts estimate sales will rise to about $15 billion by the 2007 and 2008 time frame.

"It's a very large market," Morris said, "and what share we get of that will depend on [Hematide]."

Aside from Hematide, Affymax is advancing a peptide that is an agonist of the GCSF receptor. The company expects to select a candidate for preclinical testing by the end of this year, and file an investigational new drug application by the end of next year.

Affymax has a third preclinical program involving a TRAIL R2 agonist to treat a range of cancers. Morris said the company is in the process of optimizing the lead compounds in that program.

Privately held Affymax was founded in 1988, and then acquired by GlaxoSmithKline plc, of London, for $500 million in 1995. Six years later, GSK spun out Affymax. (See BioWorld Today, Aug. 2, 2001.)

Going forward, the company expects to partner its products following Phase II. In the near-term it will seek another financing in 2005.

"We'll probably go out in the first quarter of next year," Morris said, "and start talking to potential investors for the Series D."

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