Human Genome Sciences Inc., the Rockville genomics firm, said yesterday that it has filed for regulatory approval to launch a human clinical trial on its first drug candidate, a treatment to prevent the toxic effects of chemotherapy on bone marrow.
If approved by the U.S. Food and Drug Administration, the trial would mark a turning point not only for HGS but also for the young but fast-growing genomics industry, analysts said. Analysts expect the trial to get the green light from the FDA.
"It is a milestone for genomics," said Elizabeth Silverman, a genomics analyst with BancAmerica Robertson Stevens. "It's the first [genomics] company to answer critics who have said, 'Interesting technology, but where are the products?' "
Genomics involves, in part, the study of human genes and the role they play in triggering or preventing disease and regulating other body functions.
Drugmakers are increasingly becoming interested in genomics to guide them in designing powerful new drugs that are highly specific in treating disease or regulating key body functions.
Silverman cautioned that, while Human Genome is the first company to seek approval to test a genomics-based drug on humans, the effectiveness of the treatment is unknown.
Also, as with any new treatment entering early human testing, it could be several years before the product comes before the FDA for marketing approval.
Human Genome hopes to show in the trial that its drug can suppress production of the progenitor cells responsible for production of bone marrow, which is vital to platelet and other blood cell production.
A vexing problem of chemotherapy is bone marrow damage, which can lead to blood disorders such as low platelet production.
By temporarily suppressing production of the precursor cells during chemotherapy, Human Gemone believes, the cells will be spared destruction by the toxic chemicals and the risk of blood disorders avoided.
Dr. Craig Rosen, senior vice president for research and development at Human Genome, said it was too early to estimate how much the treatment -- known in medical terms as a myeloid progenitor inhibitory factor -- might cost if it is ever approved for marketing.
Rosen said the potential market would be those cancer patients at risk of developing blood disorders from chemotherapy.
An estimated 400,000 people in the United States will be treated with chemotherapy this year, according to the American College of Surgeons' National Cancer Database.
Should a highly effective treatment land on the market, it could have the potential to dramatically affect the bottom line at HGS, which reported a net loss for the first nine months of the year of $12.9 million on revenue of $22.6 million.
For example, Amgen Inc.'s Neupogen, a biotechnology product given after chemotherapy treatments to stimulate white blood production, generates about $500 million in revenue annually.
Thus, data from HGS' human clinical trials will be closely watched by industry analysts, Silverman predicted.
Shares in Human Genome closed yesterday at $40.87, up $1.37.
Rosen said HGS developed the its myeloid progenitor inhibitory factor treatment by using its vast data base of genetic information to pinpoint a protein responsible for specifically regulating the progenitor, or "stem" cells, for bone marrow.
In animal studies, said Rosen, the protein halted production of the precursor cells without any toxic side effects.
Rosen said the company was excited about the drug's potential.
"By protecting progenitor cells early, we may be able to protect other cells along the pathway," said Rosen.
Rosen said the first trial will test the therapy on a small group of healthy volunteers to ensure there are no toxic side effects. That trial is expected to last several months.
HGS did not disclose where the first trial would be held or who the principal investigator would be.
If the early trial is successful, HGS would seek FDA approval to begin testing the drug on cancer patients.
Pharmaceutical giant Schering Plough, which is among a group of pharmaceutical companies that have paid Human Genome for access to its genomics database, has the right to co-develop and market the drug with HGS.
Schering Plough does not have to make a decision on a joint venture until after data are available from studies on cancer patients, said Kate de Santis, a spokeswoman for HGS.
Human Genome has targeted five other proteins for potential drug development and expects to seek FDA approval early next year to begin a clinical trial on another protein-based treatment.
That protein, the company has said, has shown promise in speeding the healing of open wounds.
Pub Date: 12/18/97