PDL BioPharma/Orphan Therapeutics will further analyze results of a failed Phase III trial evaluating the vasoactive peptide terlipressin for the treatment of type 1 hepatorenal syndrome and discuss the outcome with FDA before determining future development plans for the drug, PDL announced Aug. 3.

"Until discussions with the FDA occur, we cannot determine what further development steps would be required to move this program forward, or whether PDL would elect to move forward," the firm told "The Pink Sheet" DAILY Aug. 7.

The primary endpoint for the double-blind, placebo-controlled study was the "percentage of patients alive at day 14 who demonstrated reversal of type 1 HRS, based upon two measurements of serum creatinine levels less than or equal to 1.5 mg/dL without dialysis or recurrence of disease."

Type 1 HRS is a life-threatening complication of advanced liver disease. The study, conducted by Orphan, enrolled 112 patients.

Though it did not meet the primary endpoint, PDL said "the data showed a positive trend toward treatment success."

PDL obtained U.S. rights to terlipressin through its acquisition of ESP Pharma in March 2005; ESP had already established an agreement with Orphan, and the Phase III trial was underway (¹ Also see "ESP Picks Up J&J's Retavase On Heels Of Protein Design Labs Merger Deal" - Pink Sheet, 1 February, 2005.).

There currently is no FDA-approved treatment indicated for type 1 HRS. Terlipressin was the latest-stage compound in development in PDL's pipeline, the company said.

Two other late-stage drugs are Nuvion (visilizumab), in Phase II/III for the treatment of IV steroid-refractive ulcerative colitis, and ularitide for treatment of acute decompensated heart failure, which will enter Phase III in Europe later this year.

-Daniel Fowler

PDL’s Terlipressin Fails Phase III Trial; Company To Meet With FDA

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