Washington Editor

Human Genome Sciences Inc. said it would terminate development of repifermin in chronic venous ulcers following a disappointing Phase II trial.

The company's 352-patient Phase II trial of topically administered repifermin (keratinocyte growth factor-2) in chronic venous ulcers failed to hit its primary endpoint of complete wound closure within 20 weeks of initiating treatment.

"As we've looked at the data, clearly it is an overall no-effect result," David Stump, senior vice president, drug development, of Rockville, Md.-based HGS, told BioWorld Today. "There's no subgroup analysis or secondary endpoint analysis which would suggest that there is a positive result buried somewhere in the overall data."

Patients who were randomized in the multicenter, double-blind, placebo-controlled trial had chronic venous ulcers from 3 to 25 square centimeters in size that were unhealed for three to 36 months prior to enrollment. There were no significant differences between repifermin and placebo on secondary endpoints of time to complete wound closure or complete wound closure by 16 weeks to 26 weeks of treatment.

"We had good biology for the molecule, but we were in a difficult indication," Stump said. "When you do an adequately powered study, often in Phase II you get negative answers - that's part of drug development."

Repifermin is a human protein discovered by HGS. In clinical trials, it has been well tolerated.

Despite missing its mark in venous ulcers, HGS and partner GlaxoSmithKline plc, of London, are now looking toward analyzing data from a Phase II trial of repifermin in cancer therapy-induced mucositis. Repifermin in that form is a daily intravenous injection. Stump said the 90-patient Phase II is expected to be completed by the end of the year.

As for the future of repifermin outside that pending indication, Stump said HGS currently isn't studying the molecule in other diseases, but "we're always open to interesting indications. We will look into others, each on their own merits."

Meanwhile, Stump said HGS is studying other interesting products. "We're very optimistic looking forward. We're always disappointed when any trial fails, but we are very quickly refocusing on products for future development," he said.

One such product is LymphoStat-B. HGS also said Friday it activated clinical sites and started to screen patients for randomization and treatment in a Phase II trial of LymphoStat-B in systemic lupus erythematosus. The product is a human monoclonal antibody to B-lymphocyte stimulator that has been granted fast-track designation in the lupus indication.

The company's double-blind, placebo-controlled, multicenter Phase II trial is designed to evaluate the safety, optimal dosing and preliminary efficacy of LymphoStat-B in patients with active systemic lupus erythematosus. The 52-week trial is expected to enroll 300 patients.

Stump said the company has completed the protocol for a Phase II study of LymphoStat-B, which is un-partnered, in rheumatoid arthritis and currently is activating sites for the trial.

HGS's stock (NASDAQ:HGSI) closed Friday at $13.17, down 89 cents.