ProMetic Life Sciences Inc. announced plans to initiate a Phase 2 clinical trial to test its anti-fibrotic lead drug candidate PBI-4050 in cystic fibrosis (CF) patients with related diabetes as well as liver steatosis.
Cystic fibrosis is a chronic disease of the lungs and, less frequently, of the pancreas, liver, kidneys, and gut, and can be life-threatening. It results from a defective gene that causes a buildup of thick mucus in affected organs.
“Much progress has been accomplished in recent years in treating cystic fibrosis, yet, cystic fibrosis remains a devastating medical condition seriously affecting various key organs and quality of life for those who suffer from it,” said Norma Beauchamp, chief executive officer of Cystic Fibrosis Canada, in a press release. “Innovative therapies such as the one put forward by ProMetic … could play a key role in alleviating some of the associated medical complications while further improving quality of life of people living with cystic fibrosis in Canada and around the world.”
Improvements in care have increased life expectancy for many CF patients, leading to new medical challenges. Some 70% of these patients have an abnormal glucose tolerance, and between 35% and 40% of adult CF patients develop a condition known as cystic fibrosis-related diabetes (CFRD), which in turn can lead to the progressive eye, kidney, peripheral nerve, and gastrointestinal tract damage seen in many diabetics. PBI-4050, an orally active drug intended to prevent organ scarring and failure, may be a possible treatment.
“CFRD is a devastating complication of CF which adds to the already heavy burden of treatment and leads to a more rapid decline in lung function and a higher mortality. This double-blind placebo-controlled phase II clinical trial will investigate whether PBI-4050 can reverse fibrosis in the pancreas and help manage the glucose intolerance and related diabetic complications experienced by the CF patients,” said Dr. John Moran, chief medical officer of ProMetic. “The clinical trial will also allow us to monitor the effects of PBI-4050 on other important parameters in CF patients, especially lung and liver function.”
Added Dr. Lyne Gagnon, vice president of R&D preclinical at ProMetic, “PBI-4050 has been shown to significantly reduce fibrosis in several key organs in preclinical models … For this reason and because of the positive effects recently observed in type 2 diabetic patients, we believe that PBI-4050 could provide significant clinical benefits to patients affected by this medical condition.”
The company will study pancreatic fibrosis, glucose intolerance, and diabetes in the trial.
ProMetic Life Sciences Inc. is a biopharmaceutical company based in Laval, Canada, with expertise in bioseparations, plasma-derived therapeutics and small-molecule drug development.