Ups and Downs in Clinical Trial Results

Several kidney-related drug trials have recently yielded results. ProMetic Life Sciences (Laval, Quebec), announced that it had successfully completed its phase 1b clinical trial of PBI-4050 in patients with chronic kidney disease (CKD).

The randomized double-blind, placebo-controlled, multidose trial was designed to demonstrate the safety and tolerability of PBI-4050, an orally active antifibrotic drug candidate. The trial also determined the pharmacokinetic profile of PBI-4050 while monitoring multiple oral doses during 10 days in patients with stage 3b or 4 stable renal impairment. The trial was performed in a group of eight patients: six patients received PBI-4050, and two received a placebo.

“We are pleased to see that the safety and pharmacokinetic profiles of our lead drug candidate remain unaffected by the severely impaired renal function in the patients tested,” said chief medical officer John Moran. “Since fibrosis is the pathological pathway leading to organ failure and death in many diseases of differing etiologies … we plan to test the efficacy of this drug in several fibrosis-related conditions.” Phase II trials in patients with metabolic syndrome and resulting in type 2 diabetes were expected to begin patient enrollment in April 2015.

Pharmalink AB, a specialty pharmaceutical company based in Sweden, has announced that a phase 2b trial of Nefecon for the treatment of primary IgA nephropathy has met its primary efficacy endpoint at a planned interim analysis. The trial was stopped early with respect to statistical analysis of the endpoint.

The randomized double-blind, placebo-controlled clinical trial assessed the safety and efficacy of two different doses of Nefecon, a new oral modified-release capsule of the corticosteroid budesonide. The corticosteroid was administered daily during a 9-month treatment period to patients with primary IgA nephropathy having persistent proteinuria despite optimized standard-of-care therapy. The trial was conducted in 62 centers in 10 European countries and was originally intended to recruit 90 patients, but 150 eventually were included.

Bengt Fellström, MD, PhD, professor of nephrology at Uppsala University Hospital, and principal investigator of the Nefecon trial, said, “IgA nephropathy is the most common inflammatory renal disease and in real need of new treatment options. Existing options are insufficient to prevent a significant proportion of patients from progressing to renal failure, with a devastating impact on patients’ quality of life.”

Zacks Equity reported the results of a phase 2a trial that failed to meet its primary endpoint of progression-free survival in patients with advanced renal cell carcinoma. Shares of Lpath, based in San Diego, “plunged in after-market trading” after the company’s announcement of results from a phase 2a study of oncology candidate Asonep, Zacks wrote. The company will decide on the future of the candidate upon completion of the renal cell carcinoma trial.