The U.S. Food and Drug Administration (FDA) has agreed to allow Fibrocell Science to begin clinical trials testing its lead candidate FCX-013 for the treatment of moderate to severe localized scleroderma.
This news comes on the heels of an investigational new drug (IND) application that was filed by Fibrocell earlier this year. An IND is a request for FDA authorization to administer an investigational therapy to humans. It is the route through which a company can advance an investigational therapy to clinical trials.
“We are pleased the FDA has granted allowance of our IND for FCX-013 to begin clinical trials for the treatment of moderate to severe localized scleroderma, offering patients the potential for relief from this chronic, painful and debilitating disorder,” John Maslowski, president and chief executive officer of Fibrocell, said in a press release.
“With no FDA-approved therapies available, we look forward to advancing FCX-013 into the clinic,” Maslowski added.
Scleroderma is a chronic skin disorder that is characterized by excessive collagen production. FCX-013 is a gene therapy that uses a cell called a fibroblast that has been genetically engineered to produce a protein called matrix metalloproteinase 1 (MMP-1), which is involved in breaking down collagen.
FCX-013 is administered under the skin at the location of fibrotic lesions. It is designed in conjunction with Intrexon’s proprietary RheoSwitch Therapeutic System, which allows MMP-1 to only be produced when a person orally takes a compound called veledimex. This allows the person to have tight control over when MMP-1 is produced and to stop its production once the fibrosis has been resolved.
Fibrocell plans to initiate enrollment for its first open-label, single-arm Phase 1/2 clinical trial in the third quarter of 2018. It plans to recruit 10 patients with any subtype of localized scleroderma, with a goal of approximately five patients per phase.
The trial will be conducted to primarily assess the safety of FCX-013, with secondary outcomes including histology, skin scores, ultrasound and additional measurements of targeted sclerotic lesions and control sites. The trial will assess these objectives at various time points up to 16 weeks after administration of FCX-013.
The Phase 1 portion of the trial will include adult patients. The first three will be dosed prior to the dosing of the rest of the patients. The Phase 2 portion will include pediatric patients, and will be conducted after the data on the safety and efficacy from the Phase 1 patients has been approved by the FDA and a Data Safety Monitoring Board.
“There are no approved therapies for localized scleroderma. Current treatments are aimed at impacting inflammation, but few options exist to treat the excessive collagen deposition in the skin and soft tissue, which may produce pain and limitation in motion and growth. With FCX-013, the goal is to bring relief to patients by targeting the abnormal collagen metabolism to improve skin function,” said Alfred Lane, MD, chief medical advisor of Fibrocell and professor of Dermatology and Pediatrics (Emeritus) at the Stanford University School of Medicine.
In 2016, FCX-013 received an Orphan Drug Designation for the treatment of localized scleroderma from the FDA. The therapy has also been granted Rare Pediatric Disease Designation for the same indication.