West Coast Editor

Axcan Pharma Inc.’s ITAX for functional dyspepsia fizzled in Phase III, but the company said it will continue to work with the compound in other indications such as diabetic gastropathy.

The international Phase III trial with ITAX (itopride) failed to back up positive Phase II results - expected to be published in the New England Journal of Medicine today - but another Phase III study in North America is expected to report data by the middle of this year.

Frank Verwiel, president and CEO of Mont St.-Hilaire, Quebec-based Axcan, said during a brief conference call before the company’s annual meeting that he was "very surprised and disappointed, certainly in light of what we had seen, and what you will be able to see" in the published Phase II report.

Wall Street was upset, too, and took away $4.57 from the specialty pharma company’s shares (NASDAQ:AXCA), which closed Wednesday at $11.76, down 28 percent.

"Until we complete the analysis, we are not in a position to provide more detailed statistical information about placebo rates, endpoints or other data points," Verwiel said, adding that the study missed both primary endpoints, which involved a questionnaire administered by the physician and a self-assessment done by the patient.

One of the criteria for the Phase III trial was diabetes, so the results likely will provide "limited insight" on the way forward in diabetic gastropathy, and no decision will be made on the regulatory path with regard to functional dyspepsia until further analysis is done and the other Phase III trial is finished, he said.

Axcan’s scientists will examine closely some pre-specified subgroups to see if a benefit profile is discoverable there, Verwiel said.

"You look at things like age, gender, demographics, pre-medication, and of course an important one is patients with heartburn during and before the study," he said.

Regarding details of the latest trial results, Verwiel said they will make "an important discussion also for the scientific community later. Meanwhile, Axcan is "working on one pilot study [with ITAX in the diabetic indication], and hopefully later this year, we’ll be able to share some details around that study."

Verwiel called Axcan a "solid, stable and prosperous company," with 15 products on the market and record sales in the last two quarters.

Functional, or non-ulcer, dyspepsia is characterized by upper abdominal pain with no apparent organic cause. Another company working in the area is Gastrotech Pharma A/S, of Copenhagen, Denmark, which is developing GTP-010, an analogue of glucagon-like peptide-1, with Indianapolis-based Eli Lilly and Co. in a Phase II trial against irritable bowel syndrome, and the drug may have use in functional dyspepsia. Late last year, DOR BioPharma Inc., of Miami, agreed to buy Gastrotech for $9 million in shares and up to $30 million in milestone payments. (See BioWorld Today, Nov. 3, 2005.)

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