Randall Osborne

Describing its second genomics-derived drug candidate, keratinocyte growth factor-2 (KGF-2), as a potential "blockbuster" treatment for wound healing, Human Genome Sciences Inc. (HGS) took the compound into Phase I trials.

In preclinical studies, KGF-2 not only promoted healing but regenerated dermal tissue, said William Haseltine, chairman and CEO of Rockville, Md.-based HGS.

"As far as I know, this is unique," he said.

KGF-2 is being evaluated in one safety trial with a topical formulation, and one with a systemic version.

Because the drug works on surface, or epithelial, cells as well as deeper cells, and repairs damage to many tissues lining internal organs, KGF-2 may have applications in conditions that involve injury to endothelial tissues as well.

Earlier studies showed KGF-2 healed wounds by creating new skin in a short period of time; lessened severity of mucositis (a side effect of cancer therapy) in the mouth and intestines; and restored almost normal architecture to cells lining the gastrointestinal tract, damaged by inflammatory bowel disease.

To assign a dollar value to the potential market for KGF-2, Haseltine said, would be premature. "It depends how effectively it works in each area, and what competing products are on the market," he said.

KGF-2 is one of six new members of the fibroblast growth factor family discovered and tested by the company, which was expected to disclose today the start of new clinical studies.

Launching trials with KGF-2, Haseltine said, proves an important scientific point about expressed sequence tag (EST) technology. ESTs are partial cDNA clones, which can be used to identify expressed genes.

"When we started with ESTs, there was a tremendous amount of skepticism," Haseltine said. "Everybody was touting what they called functional genomics or positional cloning. They doubted that we could even take drugs into the clinic ourselves."

First Protein Drug Targets White Cell Recovery

Late last year, HGS launched clinical trials of its first genomics-derived therapeutic protein, myeloid progenitor inhibitor factor (MPIF-1), which accelerates the recovery of white blood cells in chemotherapy patients and helps regain normal platelet levels. (See BioWorld Today, Dec. 18, 1997, p. 1.)

MPIF-1 is one of 23 new members of the chemokine-interleukin family discovered and tested by HGS.

"We proved it's something that can be done again," Haseltine said.

One of HGS's collaborators, Schering-Plough Corp., of Madison, N.J., has the first option, if HGS completes Phase IIa trials of KGF-2, to pay 50 percent of post-Phase II costs and share half the profits, Haseltine said.

"Following that, SmithKline [Beecham plc] has the option," he said. "If they both turn it down, then HGS has it free of any obligation."

If the safety trials show satisfactory results, Haseltine said, HGS will begin advanced studies in more specific indications which have been determined but which have not been disclosed.

HGS initially made its gene sequence databases available to SmithKline, of London, in 1993. Two years ago they expanded access to rival pharmaceutical firms, in deals valued at more than $140 million over five years. (See BioWorld Today, July 3, 1996, p. 1.)

HGS' shares (NASDAQ:HGSI) closed Monday at $41.0625, down $0.0625.