ProMetic Life Sciences Inc., a biopharmaceutical company developing bioseparations, plasma-derived therapeutics and small-molecule therapies, announced this week that it is planning to commence a Phase 2 double-blind, placebo-controlled clinical trial of its lead drug candidate PBI-4050 in patients with cystic fibrosis (CF) and related diabetes as well as liver steatosis.
Cystic fibrosis is a life-threatening, genetic disease that causes persistent lung infections and progressively limits the ability to breathe. CF causes a thick buildup of mucus in the lungs, pancreas, and other organs. In the lungs, the mucus clogs the airways and traps bacteria, leading to infections, damage, and respiratory failure. In the pancreas, the mucus prevents the release of digestive enzymes that allow the body to break down food and absorb nutrients.
As an effect of improved standards of care over the past years, the average life expectancy for CF patients now exceeds 40 years. However, estimates indicate that 70% of adult patients will have an abnormal glucose tolerance, and 35% to 40% will develop a certain form of diabetes, a condition called cystic fibrosis-related diabetes (CFRD). CFRD is associated with all the complications typical of diabetes, leading to progressive damage in the eye, kidney, peripheral nerve, and gastrointestinal tract.
The trial will evaluate PBI-4050, an orally active lead drug candidate with excellent safety and efficacy profiles in several in vivo experiments targeting fibrosis. PBI-4050 is designed to regulate inflammation and fibrosis by reducing the amount of pro-fibrotic cytokines, fibrocyte differentiation, and microfibroblast activation, thereby improving organ function.
Dr. John Moran, chief medical officer of ProMetic, said in a press release: “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. 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.”
“PBI-4050 has been shown to significantly reduce fibrosis in several key organs in preclinical models and this, irrespective of how the injuries were induced or whether they were acute or chronic in nature,” added Dr. Lyne Gagnon, vice president of R&D preclinical at ProMetic. “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.”
According to Ms. Norma Beauchamp, chief executive officer of Cystic Fibrosis Canada: “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. Innovative therapies such as the one put forward by ProMetic and its lead anti-fibrotic compound 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.”
CF affects about 70,000 individuals in North America.