Laurent Pharmaceuticals Inc. announced today that the Research Institute of the McGill University Health Centre (RI-MUHC) has started enrolling patients in an investigator-initiated clinical trial evaluating the safety, tolerability and pharmacokinetics of a novel oral formulation of Fenretinide in adult subjects with Cystic Fibrosis (CF) and Pseudomonas aeruginosa lung colonization. The study will be conducted at the Montreal Chest Institute at the MUHC in Montréal, with financial support from McGill University, MSBi Valorisation and Laurent Pharmaceuticals.
This initiation of clinical development follows years of extensive preclinical research conducted at RI-MUHC, showing that oral administration of Fenretinide, a vitamin A derivative, corrects the lipid imbalance in lungs and plasma of specific animal models of CF, resulting in reduced lung inflammation and dramatic decrease in the severity of pulmonary infections with Pseudomonas aeruginosa, a bacterium involved in perpetuating the inflammation-infection vicious cycle in CF. Pseudomonas aeruginosa is the main cause for difficult-to-treat chronic infection in CF, and is associated with lung insufficiency and reduced survival.
The Phase 1 trial is a randomized, double-blind, placebo controlled, intra-patient dose-escalation clinical trial that will enroll a minimum of 16 adult CF patients chronically infected with Pseudomonas aeruginosa. The primary objective is to establish the safety and tolerability of a once a day regimen of Fenretinide oral capsules. More information about the trial can be found at: www.clinicaltrials.gov (NCT02141958)
About Cystic Fibrosis
Cystic Fibrosis is the most common fatal hereditary disease among Caucasians, affecting an estimated 70,000 people worldwide. CF is caused by mutations in the CFTR gene and is characterized by viscous secretions in different exocrine tissues and an aberrant immuno-inflammatory response manifested by excessive but inefficient inflammation, leading to pulmonary chronic infection and loss in lung function over time. Severe pulmonary dysfunction is the usual cause of death in CF.
About Laurent Pharmaceuticals
Laurent Pharmaceuticals is a Montréal-based private biopharmaceutical company dedicated to drug development for orphan diseases. The company has a clinical stage lead program focusing on repurposing of Fenretinide as a first-in-class oral therapy for Cystic Fibrosis that received Orphan Drug Designation in 2010 from the US Food and Drug Administration.