ProMetic Life Sciences Inc. announced that the Drug Safety Monitoring Board (DSMB) recommended that patient enrolment should continue in ProMetic's ongoing Alström syndrome phase 2 clinical trial. This recommendation follows the DSMB's review of the safety data accumulated in the first eight Alström syndrome patients that had received treatment with PBI-4050. The DSMB determined that no safety or tolerability issues had been observed in these patients.

The early efficacy results in this phase 2, open-label study demonstrate that the first five patients (100%) who completed 12 weeks of treatment with PBI-4050 had a significant reduction of liver fibrosis, as measured by transient elastography (FibroScan). This reduction was also sustained for the first patient, who continued to show reduced fibrosis after having completed 24 weeks of treatment. The initial efficacy results also demonstrate that those patients with the most elevated liver enzymes at baseline (aspartate aminotransferase [AST], alanine aminotransferase [ALT], and alkaline phosphatase) [ALP], had a significant reduction in all of these enzymes, to within normal ranges, after completing 4, 8 and 12 weeks of treatment with PBI-4050.

Dr. Tarekegn Geberhiwot, principal investigator of the clinical trial from the Department of Endocrinology and Metabolism, Queen Elizabeth Hospital, Birmingham, UK commented, "PBI-4050 has shown encouraging findings in patients with Alström Syndrome, being well tolerated and safe for up to 24 weeks. There have been beneficial effects observed in glycaemic control and early potential anti-fibrotic effects on the liver. The rapidity of the effect is remarkable, with a reduction in the elevated levels of liver enzymes apparent within the first 4 weeks, and significant reductions in FibroScan scores within only 12 weeks in patients with established severe fibrosis"

"To me, patients with Alström syndrome display the most extreme features of Type 2 diabetes and liver fibrosis and a trial of PBI-4050 in this population is as challenging a test of the efficacy of PBI-4050 as I can imagine" stated Dr John Moran, ProMetic's chief medical officer. "The progression of liver fibrosis is much more aggressive in patients with Alström syndrome than in "typical" metabolic syndrome patients with obesity, diabetes and fatty liver, and so the major improvement in FibroScan score in all 5 patients in only 12 weeks is way beyond our expectations. Non-alcoholic fatty liver disease (NAFLD) is the manifestation of metabolic syndrome in the liver. Due to the worldwide obesity epidemic, NAFLD now affects 2030% of the general population and thus has become by far the most common cause of chronic liver disease and we firmly believe that PBI-4050 will also be effective in that population", added Dr. Moran.

The ongoing Alström syndrome phase 2 clinical trial is an open label, single arm and single center study investigating the safety, tolerability and efficacy of ProMetic's small molecule lead compound PBI-4050 in a total of 20 patients. The trial is being performed at the specialty center for the care of UK patients with Alström syndrome at the Queen Elizabeth Hospital, Birmingham, UK. This center has recently published data showing that many of these patients show evidence of non-alcoholic fatty liver disease and advanced liver fibrosis at an early age, confirming previous publications showing a very high incidence of progression of NAFLD into liver cirrhosis with associated mortality in Alström patients.

"We are absolutely thrilled to see the beneficial effects that PBI-4050 has in patients with Alstrm syndrome", stated Mr. Pierre Laurin, president and chief executive officer of ProMetic. "These early results have motivated us to expand our program to Alström patients elsewhere in Europe and in North America. Furthermore, these results provide us with valuable clinical information as ProMetic pursues other unmet medical conditions where fibrosis plays a key role", added Laurin.

Alström syndrome is a rare inherited autosomal recessive syndrome characterized by the onset of obesity in childhood or adolescence, Type 2 diabetes with severe insulin resistance, dyslipidemia, hypertension and severe multi-organ fibrosis, involving the liver, kidney and heart.

Alström syndrome is also characterized by a progressive loss of vision and hearing, a form of heart disease that enlarges and weakens the heart muscle (dilated cardiomyopathy), and short stature. This disorder can also cause serious or life-threatening medical problems involving the liver, kidneys, bladder, and lungs. Some individuals with Alström syndrome have a skin condition called acanthosis nigricans, which causes the skin in body folds and creases to become thick, dark, and velvety. The signs and symptoms of Alström syndrome vary in severity, and not all affected individuals have all of the characteristic features of the disorder.