Laurent Pharmaceuticals is looking to expand adult cystic fibrosis therapeutic options with the initiation of a Phase 1 trial for their novel drug Fenretinide. The trial is currently recruiting participants at Montreal Chest Institute and is sponsored by McGill University Health Center in Quebec.
Approximately 16 adult cystic fibrosis patients with P. aeruginosa lung colonization will be enrolled in the trial, with 12 receiving Fenretinide and 4 receiving a placebo. Since it is a phase 1 safety trial, it is relatively short and will consist of a 21-day treatment cycles with 7-day drug-free periods between cycles. Once a day, participants will receive an oral dose of Fenretinide and be monitored for vital signs, pulmonary function, adverse effects, and other safety measures. Doses will be escalated up to three times for three 21-day treatment cycles. As secondary measures, pharmacokinetics and pharmacodynamics will be assessed using plasma concentrations and markers of lipids and inflammation. Of interest is also forced expiratory volume in one second, the usual readout of clinical trials evaluating cystic fibrosis drugs. Results are expected in September 2014.
In 2010, the Food and Drug Administration granted Orphan Drug Designation for the use of Fenretinide to treat Pseudomonas aeruginosa infections in cystic fibrosis patients.
The designation was granted due to a number of effective research initiatives at McHill, including a study that demonstrated a reduction in lung inflammation in mice infected with P. aeruginosa, the most prevalent infection in cystic fibrosis patients.
It is thought that Fenretinide, a derivative of vitamin A, acts in the lungs and plasma to correct lipid imbalance. “I am really excited to continue the development of this extremely interesting technology in the context of the newly created Laurent Pharmaceuticals,” said the leader of the study Dr. Danuta Radzioch, in a news story.
According to Dr. Radu Pislariu, MD, President and CEO of Laurent, “The concept of using Fenretinide in cystic fibrosis has the potential to become an important disease-modifying oral treatment in a market dominated by symptomatic inhalation therapies. I’m very pleased with the support received from MSBiV and its academic partners in the creation of this start-up as the commercialization vehicle for the technology developed by Dr. Radzioch and her team.”
McGill University, Research Institute of the McGill University Health Centre, and MSBi Valorisation launched the biopharmaceuticals start-up Laurent Pharmaceuticals just over a year ago. Laurent specializes in drug development for orphan diseases, and their main focus is the orphan disease cystic fibrosis.