The Phase 2 door was opened when the U.S. Food and Drug Administration (FDA) and Health Canada approved the company’s investigational new drug (IND) application for LAU-7b. The randomized, double-blind, placebo-controlled Phase 2 study, which will start later this year, is called APPLAUD. It will evaluate the effectiveness and safety of LAU-7b in both countries.
Researchers want to see if LAU-7b can preserve lung function in up to 136 adult CF patients by reducing persistent unresolved inflammation in patients’ lungs. Patients will take the therapy for six months.
“There are no drugs approved for treating lung inflammation in CF and, despite the emergence of important new therapies, pulmonary insufficiency continues to be the primary cause of mortality in patients with CF,” Radu Pislariu, the president of Laurent Pharmaceuticals, said in a press release. “Both the therapeutic concept and the design of the Phase 2 are ground-breaking, and we are pleased to have achieved these important regulatory milestones. We believe LAU-7b has the potential to make a life-changing impact for patients with CF.”
LAU-7b is an oral form of the retinoid fenretinide, administered once a day. Retinoids are a group of compounds related to vitamin A.
Fenretinide may help reduce inflammation in CF patients’ lungs. LAU-7b is designed to correct the faulty metabolism and chronic inflammation stemming from the genetic defect that causes cystic fibrosis.
“Current anti-inflammatory therapies have limited potential for chronic use, either because their questionable long-term benefits in patients with CF or due to concerns over safety,” said Larry Lands, principal investigator of the Canadian portion of the trial.
“LAU-7b proposes an interesting treatment approach that uses the body’s own ability to resolve inflammation, which is a more natural way to modulate inflammatory response without directly interfering with defense mechanisms,” added Lands, who is director of Pediatric Respiratory Medicine at McGill University Health Center in Montreal.
The Canadian part of the Phase 2 clinical trial is being funded by Cystic Fibrosis Canada. The U.S. part is being financed by Cystic Fibrosis Foundation Therapeutics, which gave Laurent $3 million to help develop the drug.
“New anti-inflammatory treatments are needed to stop lung destruction in CF, as well as dedicated approaches toward trial design for these therapies,” said Michael Konstan, principal investigator of the U.S. portion of the trial.
“The design of this Phase 2 follows the recommendations of the US Cystic Fibrosis Foundation’s Anti-inflammatory Therapy Working Group, and is well informed by the LAU-7b Phase 1b trial and a natural history study conducted previously in adult patients with CF,” added Konstan, the vice dean of translational research at Case Western Reserve University School of Medicine in Cleveland.