ProMetic Life Sciences Inc. of Laval in Quebec on Thursday announced that it had been approved to commence the clinical trial of its orally active anti-fibrotic lead drug candidate, PBI-4050, in patients suffering from cystic fibrosis (“CF”), following the CTA clearance by Health Canada. The objectives of this 24 week randomized, double-blind, and placebo-controlled Phase 2 study includes the evaluation of the effects of PBI-4050 on pancreatic and lung function in 90 CF patients.
As a result of significantly improved standards of care over the past decades, the average life expectancy for CF patients now exceeds 40 years. However, with a longer life expectancy, the genetic condition also generates serious pancreatic and hepatic complications. In particular, 70% of CF patients will have abnormal glucose tolerance, and 35-40% of adult CF patients will develop a unique type of diabetes, a condition known as cystic fibrosis-related diabetes (“CFRD”). CFRD is associated with all the complications typical of diabetes, leading to progressive eye, kidney, peripheral nerve, and gastrointestinal tract damage. Patients with CFRD have worse lung function, poorer nutrition, more frequent hospitalization, and higher mortality rates than those affected by CF but without diabetes.
“It is well-known that CF adversely affects lung function. However there is very often co-existing pancreatic damage, leading not only to decreased production of digestive enzymes and malabsorption of food but also to impaired glucose tolerance and to frank diabetes” stated Dr John Moran, Chief Medical Officer of ProMetic. “We have already demonstrated a significant improvement in the blood glucose levels in patients with metabolic syndrome with Type 2 diabetes treated with PBI-4050, and a significant reduction of fibrosis in the lungs, pancreas and liver in multiple animal models. We believe that the unique mode of action of PBI-4050 could indeed provide much relief for the CF patients by reducing the level of fibrosis in their lungs and pancreas and by improving the production of insulin” concluded Dr. Moran.
Mr. Pierre Laurin, CEO of PLI commented: “We are very excited to commence this trial in cystic fibrosis patients who have benefited over the years from a myriad of medical interventions leading to a progressively improved life expectancy. While the primary objective in this study is to identify if we can defer or reduce the use of insulin treatment in these patients, our hope here is that the reduction of fibrosis observed in the lungs, pancreas and liver in preclinical studies translates to a further improvement in their quality of life and life expectancy”.
ProMetic has recently reported a statistically and clinically significant decrease in HbA1C levels that compares very favorably to drugs already approved and marketed for the treatment of diabetes as well as additional evidence of PBI-4050’s pharmacological and clinical activity in humans based on the analysis of new pro-inflammatory biomarkers in blood and urine samples from these patients. These results clearly indicate that the drug’s unique mode of action and related efficacy observed in diabetic animal models translate to humans. To date there have been no drug-related Serious Adverse Events and PBI-4050 has been very well tolerated by patients. – PRNewswire