Canadian pharmaceutical company, Prometic Life Sciences Inc provided an update on their ongoing Phase 2 clinical trial involving the use of PBI-4500 to treat patients with Alström syndrome.

Alström syndrome is a rare genetic condition due to mutations in the ALMS1 gene. Symptoms vary greatly among patients but most commonly start to appear in childhood and can include vision abnormalities, hearing loss, obesity, cardiomyopathy, and hyperlipidemia, and risk of liver disease.  There is currently no treatment for Alström syndrome except to manage the multitude of symptoms.

PBI-4050 is a drug developed by Prometic with anti-fibrotic activities and is being tested in a number of diseases, including Alström syndrome.

Interim analysis of their Phase 2 clinical trial (n=12) indicates the drug is effective in reducing cardiac fibrosis and liver flexibility in the Alström syndrome patients (see figures below).

In a press release, John Moran, M.D., chief M=medical officer of Prometic said, “Progressive cardiac fibrosis is the most serious feature of Alström syndrome, and the resultant heart failure is the most common cause of death,” adding, “Therefore, the results observed with the cardiac MRI are very promising and exceeded our expectations.”

Moving forward, the company plans to meet with European and the United States regulatory authorities to determine the next steps. The current phase 2 clinical trial by the Canadian company is currently being conducted in England.