Genome firm plans trials on humans Human Genome hopes to turn research into therapies

1st test expected this year

Proteins could help cancer, arthritis, neurological patients

March 20, 1997|By Mark Guidera | Mark Guidera,SUN STAFF

Human Genome Sciences Inc., the Rockville company mapping genes and researching their role in regulating body functions and diseases, expects to launch a human clinical trial late this year on its first potential medical therapy to spin out of its research.

"We have some very promising candidates for a human trial late this year," Dr. William Haseltine, the company's chief executive officer, said yesterday.

Industry analysts said the trial, if launched as planned, would be a major step forward for the company, which is attempting to expand beyond strictly a genomics information company into a pharmaceuticals developer.

While the company has not decided which therapy it will seek regulatory approval for to test in humans, it has six possible candidates, said Haseltine. The company plans to choose two of those for human trials.

Human Genome expects the first trial to be launched late this year, followed by another human trial on a different therapy early next year, Haseltine said.

The company also plans to license the rights to develop and commercialize proteins it decides not to develop for commercialization on its own, said Haseltine, as a way to raise money to pay for the clinical trials.

The candidates Human Genome says show promise for development as medical treatments are based on human proteins, the organic compounds contained in genes that regulate cell behavior.

The company has determined that these proteins -- called "therapeutic proteins" -- generate medically useful activities in the body.

The candidates Human Genome is considering for its first human trial include:

Therapies based on two proteins that inhibit the growth of bone marrow cells, a characteristic that may have promise as a treatment to help cancer patients' blood cell counts recover quickly from aggressive chemotherapy. Chemotherapy can knock out the bone marrow's ability to produce blood cells.

Therapies based on two proteins that assist with tissue repair, a trait that may have applications for treating skin ulcers, burns and wounds.

A therapy based on a protein that reduces inflammation and bone and cartilage destruction. These characteristics might have applications for treating arthritis and its complications.

A therapy based on a protein that protects motor neurons. It may have applications in treating neurological diseases.

By developing and testing a potential new therapy on its own, said Haseltine, the company gains "the independent right to control its licensing and marketing."

Elizabeth Silverman, a New York-based genomics analyst with Punk, Ziegel & Knoell, an investment bank, agrees that Human Genome gains a bargaining edge by shepherding therapeutic protein candidates through early human trials itself.

"The more data you have to offer about a drug's potential, the more control you have over marketing agreements and the more rights you are likely to retain," she said.

The company has royalty and licensing agreements with five pharmaceutical companies that have paid millions to gain access to its gene database. But the Rockville biotechnology firm has retained the right to develop and commercialize as many as six therapeutic proteins annually itself.

Human Genome's decision to shoulder the high potential cost of human trials has made some investors wary of the stock, Silverman said.

Still, she said, research on human proteins for new drug and medical therapies has grown steadily in recent years, and many experts are bullish on their potential for powerful new drugs and other medical therapies.

At least six biotechnology companies, from powerhouse Amgen Inc., based in California, to fledgling Cel-Sci Inc., which has research and development offices in Baltimore, and now Human Genome, are attempting to develop new treatments using naturally occurring proteins.

"Some of these proteins could result in blockbuster drugs," said Silverman.

Products based on therapeutic proteins already on the market include insulin, interferon and human growth hormones.

Human Genome said it expects to pay for the trials mostly with money the company raised from a public offering last week. The company raised $105 million in Friday's offering of 3 million shares, priced at $37 each.

Haseltine said the company also expects to use some of the secondary offering proceeds to cover costs associated with filing for patent protections on more of its gene discoveries.

As of February, the company had been issued five U.S. patents covering full-length gene sequences, or codes. Human Genome has 230 other patent applications concerning gene codes pending.

Pub Date: 3/20/97

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