PBI-4050 Phase 2 Trial Demonstrates Efficacy for Alstr


Positive data was anounced regarding PBI-4050, a possible treatment for Alström syndrome.

Results from the ongoing phase 2 open label clinical trial evaluating PBI-4050 for the treatment of Alström syndrome were announced at the International Liver Congress 2018, showing PBI-4050 decreased insulin resistance in the liver and PBI-4050 clinical activity as measured in fat biopsies and the heart, liver, kidney, and fat tissue in patients treated with PBI-4050 over an average of 52 weeks.

“The clinical activity of PBI-4050 has now been confirmed in the liver, the heart, the kidney, and in the fat tissue in the Alström syndrome patients, stated Pierre Laurin, president and CEO of Prometic, in a recent statement. “We look forward to presenting these detailed trial results this summer at meetings scheduled with European and US regulatory authorities to determine the clinical-regulatory pathway for PBI-4050 in Alström syndrome.”

In the 12 patients enrolled in the phase 2 trial, improved liver function and reduced fibrosis in fat tissues were demonstrated as shown from fat biopsies in patients treated with PBI-4050. With prolonged treatment, clinical activity and tolerability of PBI-4050 were sustained. At the time of measurement, the average treatment duration of PBI-4050 had reached 52 weeks. In addition, with longer treatment exposure, further anti-fibrotic clinical activity in the heart, liver, and fat tissue was observed. Previously, on March 28th, Prometic reported positive heart and liver data from this PBI-4050 phase 2 study.

A significant clinical improvement was shown in an analysis of data comparing fat biopsies taken at baseline and after 24 weeks of PBI-4050 treatment. Over this extended period of time, PBI-4050’s clinical activity and tolerability were also confirmed with no drug-related serious adverse events. Specifically, after 24 weeks of PBI-4050 treatment, adipocytes were more distinct and smaller in size, and no coalescence was observed. The insulin clamp technique was used to measure the improvement of the liver function in Alström syndrome patients and confirmed a significant reduction of endogenous glucose production (EGP) and reduction of hepatic liver resistance after 24 weeks of PBI-4050 treatment.

“In addition to improved FibroS can and MRI measurements in the liver, we now have direct evidence of a significant improvement of liver physiology in these patients,” added John Moran, MD, FRACP, FACP, Prometic’s Chief Medical Officer. “By using a demanding and complex technique called “insulin clamp” we were able to demonstrate that PBI-4050 significantly reduced the liver resistance to insulin.”

According to Promeitcs, “PBI-4050 is an orally active lead drug candidate with excellent safety and efficacy profiles demonstrated in a large number of animal models of fibrosis affecting different organs, including the lung, liver, heart, kidney, and pancreas.”

Alström syndrome is a genetic disease in which a rare inherited autosomal recessive syndrome is characterized by the onset of obesity in childhood or adolescence, Type 2 diabetes, and often with severe insulin resistance, dyslipidemia, hypertension and severe multi-organ fibrosis involving the liver, kidney, and heart. The effects of PBI-4050, which have been demonstrated in animal models, have been replicated in phase 2 studies in idiopathic pulmonary fibrosis (IPF) in metabolic syndrome with type 2 diabetes and in Alström syndrome.

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