LAU-7b is a novel and improved solid dosage form of fenretinide, requiring. once-a-day oral administration. LAU-7b was recently tested in adult patients with CF in a dose-ascending Phase 1b study, showing a good safety and tolerability, and promising pharmacokinetic and pharmacodynamic results.
The study population involved in the Phase 1b trial was previously characterized in a non- interventional, natural history study, which explored the predictive value for pulmonary exacerbation and response to antibiotic treatment during exacerbations of systemic lipidomic, inflammation and oxidation biomarkers. Data collected from the non-interventional study was used to compare the response to exacerbation antibiotic treatment of these markers to plasma levels obtained in Phase 1b.
The Phase 1b study was a single center, randomized, double-blind, placebo-controlled Phase 1b clinical study to evaluate the safety and tolerability of three increasing oral doses of fenretinide compared to placebo, and to evaluate the pharmacokinetics of fenretinide in adult CF patients.
The administration of up to 300 mg of LAU-7b for 21 consecutive days was safe and well tolerated by adult CF patients, and achieved the proposed target plasma concentration at steady state (Css) in all patients, with the 300 mg dose. The study drug normalized the plasma levels of AA and DHA in the vast majority of patients, causing a shift towards an anti-inflammatory and pro-resolving pattern, particularly during pulmonary exacerbation. The 300 mg dose was therefore recommended for the upcoming Phase 2 clinical study. More information about the Phase 1b trial can be found at: www.clinicaltrials.gov (NCT02141958).
A Phase 2 clinical trial in adult patients with CF is currently in preparation in US and Canada, from which a portion will be conducted in the US Cystic Fibrosis Foundation’s clinical trial network, the largest CF network of its kind in the world.