Laurent Pharmaceuticals Inc., a biopharmaceutical company with a clinical development program for cystic fibrosis (CF), recently announced promising data on its Phase 1b clinical trial with LAU-7b in adult CF patients.
CF is a life-threatening genetic disease in which a defective gene (CFTR) induces a salt imbalance, causing the body to form unusually thick, sticky mucus that can obstruct the airways and promote inflammation and dangerous lung infections resulting in serious respiratory and also gastrointestinal manifestations. The majority of the CF patients die due to respiratory failure. There is no cure for the disease and it is estimated that 70,000 individuals worldwide suffer from CF, including 30,000 individuals in the United States.
CF patients have an imbalance of essential fatty acids and studies have suggested that this lipid imbalance can ultimately contribute to the inflammation and lung infections that lead to respiratory failure.
LAU-7b is a new oral formulation of fenretinide, a synthetic retinoid derivative that has lipid modulating properties. Preclinical studies showed that LAU-7b is able to correct lipid anomalies in the plasma and lungs of animal CF models, leading to a reduction in lung inflammation and the severity of pulmonary infections with Pseudomonas aeruginosa.
The Phase 1b clinical trial was a randomized, double-blind, placebo controlled study on 15 adult CF patients chronically infected with Pseudomonas aeruginosa. The trial’s primary goal was to assess the safety and tolerability of oral LAU-7b in three different doses in a once a day regimen for cycles of 21 consecutive days.
Researchers found that LAU-7b was safe and well tolerated by CF patients in doses up to 300 mg in a period of three cycles of 21 consecutive days each. LAU-7b treatment was found to normalize the lipid imbalance in CF patients and decrease the oxidative stress leading to an anti-inflammatory state.
“These results showed a good safety and pharmacokinetic profile of LAU-7b in adult CF patients” said the study’s principal investigator Dr. Elias Matouk, from McGill University Health Centre (MUHC), in a news release. “We also observed a promising protective effect on certain pharmacodynamic markers, and potential association with stability of clinical parameters, especially during pulmonary exacerbations. LAU-7b is certainly a promising drug candidate for CF and we look forward to its advancement in Phase 2 clinical development.”
“We are very pleased with the results of the Phase 1b study and the collaboration with the research team from MUHC. Despite the great advances in the treatment of CF, the aberrant inflammation is still an area of high unmet needs, and is at the core of the inflammation-infection vicious cycle leading to irreversible lung damage,” said the President and CEO of Laurent Pharmaceuticals Dr. Radu Pislariu. “The few current anti-inflammatory drugs showed limited benefits in CF, thus disease-modifying concepts, such as lipid modulation, could have a better chance to address the still enigmatic link between genetic defect and the compromised host response.”