Phase 2 Trial for Oral Cystic Fibrosis Treatment Gets Canada’s Green Light

Phase 2 Trial for Oral Cystic Fibrosis Treatment Gets Canada’s Green Light

The Canadian biopharmeceutical corporation ProMetic Life Sciences has been authorized by Health Canada to begin a study on PBI-4050, its orally active anti-fibrotic investigational drug for the treatment of cystic fibrosis (CF).

PBI-4050 has shown excellent safety and efficacy profiles, confirmed through several experiments in living subjects, that target fibrosis. Fibrosis is a complex process by which continued inflammation causes vital organs to lose proper function as normal tissue is replaced by fibrotic scar tissue.

The double-blind, randomized, and placebo-controlled 24-week Phase 2 clinical trial will investigate the effects of PBI-4050 on lung and pancreatic function in 90 patients with CF.

CF is a chronic disease affecting the lungs and, but less frequently, the pancreas, liver, kidneys, and gut. The life threatening condition results from a defective gene that causes a buildup of thick mucus in affected organs.

Improvements in care have increased life expectancy for many CF patients, leading to new medical challenges. Around 70% of patients have abnormal glucose tolerance. Between 35% and 40% of adult CF patients develop cystic fibrosis-related diabetes (CFRD), which in turn can lead to progressive eye, kidney, gastrointestinal tract, and peripheral nerve damage seen typically in many diabetics.

“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 (pre-diabetes),” said Dr. John Moran, Chief Medical Officer of ProMetic, in a press release.

Moran said the company has already demonstrated significant improvement in blood glucose levels in patients with metabolic syndrome with Type 2 diabetes treated with PBI-4050. A significant reduction of fibrosis in the lungs, pancreas and liver has also been found in multiple animal models.

“We believe that the unique mode of action of PBI-4050 could indeed provide much relief for CF patients by reducing the level of fibrosis in their lungs and pancreas and by improving the production of insulin,” Moran said.

Pierre Laurin, CEO of ProMetic said the company is excited to commence the trial in cystic fibrosis patients who benefit with longer 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,” Lauren said.

The company also revealed a significant decrease in HbA1C levels similar to approved and marketed medications for diabetes, which further confirms PBI-4050’s clinical and pharmacological activity in humans. The results were based on the study of pro-inflammatory biomarkers in blood and urine samples.

In conclusion, study results indicated that PBI-4050’s exclusive mechanism of action and related efficacy observed in animal models against diabetes can translate to positive results in humans.

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