ProMetic to Begin Phase 2 Clinical Trial of Anti-Fibrotic Oral Drug in CF Patients

ProMetic Life Sciences Inc. recently announced that Health Canada has approved the initiation of a clinical trial assessing the company’s anti-fibrotic lead drug candidate, PBI-4050, in patients with cystic fibrosis (CF). The Phase 2 study, which is expected to begin in shortly, aims to evaluate the drug’s effects on pancreatic and lung function in CF patients.

Abnormal glucose tolerance is extremely common in CF patients, and a specific type of diabetes, called cystic fibrosis-related diabetes (CFRD), affects up to 40%–50% of adult CF patients. CFRD shares features common to both type 1 and type 2 diabetes, especially insufficient insulin production due to scarring of the pancreas, and delayed and abnormal insulin response (insulin resistance). Patients with CFRD have poorer lung function, more frequent hospitalizations, and higher mortality rates than those 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,” Dr. John Moran, chief medical officer of ProMetic, said in a press release. “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,” Dr. Moran added.

PBI-4050 is an orally active drug candidate whose efficiency and safety were shown in several in vivo experiments targeting fibrosis. ProMetic has also demonstrated PBI-4050’s pharmacological and clinical activity in humans, and its ability to significantly decrease, in measures equivalent to already approved diabetic drugs, levels of HbA1C, a form of hemoglobin that indicates the concentration of plasma glucose.

“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,” said Pierre Laurin, CEO of ProMetic.