Cellegy Pharmaceuticals Inc. is enrolling a confirmatory 150-patient Phase III trial for Cellegesic, a drug for treating pain associated with chronic anal fissures.
When complete, the confirmatory study is expected to support statistically significant pain-reduction data noted in two earlier Phase III trials. The earlier trials were included in a new drug application that Cellegy withdrew in April 2002 after the FDA said the company had not adequately prespecified the statistical method used to analyze the trial results. (See BioWorld Today, April 29, 2002.)
Cellegy's stock tanked 50 percent upon pulling the application. Nevertheless, the company went back to the drawing board, opening up discussions with the FDA about how best to proceed. Ironically, the South San Francisco-based company is in a similar position today with another drug, Fortigel (testosterone gel), for hypogonadism. Cellegy and partner PDI Inc., of Upper Saddle River, N.J., received a non-approvable letter in early July. (See BioWorld Today, July 8, 2003.)
To get Cellegesic (nitroglycerin ointment) to market, Cellegy and the FDA settled on a "special protocol assessment," meant to provide assurance that if prespecified trial results were achieved, the product would be approved. The protocol includes a statistical methodology for analysis of the results.
The trial will evaluate one active dose vs. placebo, compared with two active doses and placebo in the earlier Phase III. The primary endpoint is reduction in pain over a 21-day period, compared with 56 days in the previous study. According to the company, in the previous study, pain reduction was noted during the first three weeks of the study.
Other changes include a new team of clinical research associates and outside advisers hired by David Karlin, Cellegy's vice president, clinical research, who began working with the company late last year. His team will monitor the 20 domestic clinical sites while a new contract research organization has been commissioned to handle the other 20 sites.
Already, 25 of 150 patients have enrolled at seven sites. To the company, that is positive news.
"We're making good progress, so the study is well under way," Richard Juelis, Cellegy's chief financial officer, told BioWorld Today. "Based on the initial accrual, we are pretty optimistic we can get it done quickly, but it is a bit early to predict when we will get it done."
Previously, the company had estimated completion around the end of 2003. (See BioWorld Today, Sept. 10, 2002.)
Meanwhile, Karlin released a prepared statement saying patients selected for the confirmatory trial are those with the greatest need for pain relief, who are most likely to benefit from Cellegesic. "Based on initial recruitment, we are optimistic that the study can be conducted expeditiously and provide the confirmatory data required by the FDA for approval."
Juelis said anal fissures are a fairly prevalent condition that patients are reluctant to talk about. There are no specific drugs approved for the condition. Instead, physicians often prescribe painkillers or recommend surgery when the condition is chronic. "There's a high side effect profile on surgeries, so clearly patients will use nitroglycerin ointment to avoid surgery and to relieve what is in most cases very severe pain," he said.
The company's current plan is to launch and market Cellegesic on its own in the U.S., and to seek a partner in Europe.
Cellegy's stock (NASDAQ:CLGY) fell 5 cents Wednesday to close at $2.60.