ProMetic Life Sciences Inc., a biopharmaceutical company focused on the development of small-molecule drugs and plasma-derived therapeutics, recently announced that it has ended the patient enrollment phase on its Phase 2 open-label clinical trial assessing the drug candidate PBI-4050 in patients with metabolic syndrome and type 2 diabetes. The company will now initiate a pivotal, placebo-controlled Phase 2 study in type 2 diabetes patients.
PBI-4050 is an oral drug candidate that targets fibrosis, or the replacement of normal tissue by fibrotic scar tissue. Fibrosis usually occurs as the result of a chronic inflammation and can impair the function of vital organs. PBI-4050 has been shown to be safe and well-tolerated, and to have anti-fibrotic activity in several key organs, including the liver, lungs, heart, and kidneys.
The company’s decision was based on the finding of a statistically and clinically significant reduction in HbA1C (glycated hemoglobin, a measure of the average blood glucose over time) and the improvement of blood glucose control in 10 out of the first 11 patients enrolled in the trial after 12 weeks of treatment. This decrease in blood glucose was comparable to other already approved anti-diabetic drugs.
“The primary objectives of this open label study were to confirm the safety and tolerability of PBI-4050 in this patient population, and to look for early evidence of pharmacological activity in humans,” said Dr. John Moran, chief medical officer of ProMetic, in a company press release. “We now have clear evidence that the drug’s unique mode of action and related efficacy observed in diabetic animal models translate to humans. It is important to note that these patients still had elevated HbA1c values despite treatment with standard oral antidiabetic medications, making this improvement in HbA1C very impressive indeed.
“To date there have been no drug-related Serious Adverse Events and PBI-4050 has been very well tolerated by patients. Moreover, we also observed that the PBI-4050 treatment reduced waist circumference, body weight and body mass index (BMI), that is, there is a striking improvement in the physical parameters related to metabolic syndrome,” Dr. Moran added.
“This drug may be of benefit for people with type 2 diabetes because it has a different mode of action and avoids some undesired side effects seen with many of the drugs currently used, such as weight gain or hypoglycemia. I am excited that ProMetic is proceeding to a randomized placebo-controlled study,” said the principal investigator for the clinical trial, Dr. Peter Senior, professor of Medicine and director of the Department of Endocrinology at the University of Alberta, Canada.
Prometic will file an application for PBI-4050 as an Investigational New Drug (IND) with the U.S. Food and Drug Administration (FDA) for the treatment of type 2 diabetes and chronic kidney disease (CKD), and also for idiopathic pulmonary fibrosis (IPF).
“With these positive results in diabetic patients, we also intend to pursue an additional orphan indication with PBI-4050 to be announced shortly,” said Pierre Laurin, chief executive officer of ProMetic. “We expect PBI-4050 to perform especially well in patients affected by both diabetes and progressive fibrotic diseases.”