Fibrocell Announces FDA Fast Track Designation of FCX-013

Fibrocell Science announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation to FCX-013, the Company’s clinical stage candidate for the treatment of moderate to severe localized scleroderma, a chronic, debilitating genetic skin disorder with no FDA approved therapies.

"Fast Track designation represents an important milestone in advancing clinical development of FCX-013," said John Maslowski, President and Chief Executive Officer of Fibrocell. "We are pleased the FDA has awarded this designation to FCX-013 which, we believe, has the potential to be the first gene therapy to treat excessive collagen accumulation in the skin and soft tissue at the site of localized scleroderma lesions and to bring relief from the severe pain and functional disability associated with the disorder."

The Fast Track program is designed to facilitate development and expedite review of new therapies that address unmet medical needs of patients with serious conditions. The designation offers various benefits, including more frequent meetings with the FDA to discuss the drug's development plan, eligibility for Accelerated Approval or Priority Review if relevant criteria are met, and opportunity for Rolling Review, which allows the Company to submit completed sections of its Biologics License Application (BLA) to the FDA, rather than waiting until every section of the BLA is completed before the entire application can be reviewed.

Previously, the FDA granted Orphan Drug Designation to FCX-013 for the treatment of localized scleroderma and Rare Pediatric Disease Designation for the treatment of moderate to severe localized scleroderma.

FCX-013 is Fibrocell’s clinical stage gene therapy candidate for the treatment of moderate to severe localized scleroderma. FCX-013 is an autologous fibroblast genetically modified using lentivirus and encoded for matrix metalloproteinase 1 (MMP-1), a protein responsible for breaking down collagen. FCX-013 incorporates Precigen’s proprietary RheoSwitch Therapeutic System, a biologic switch activated by Veledimex—an orally administered compound—to control protein expression at the site of the localized scleroderma lesions. FCX 013 is designed to be injected under the skin at the location of the fibrotic lesions where the genetically-modified fibroblast cells will produce MMP-1 to break down excess collagen accumulation.

Fibrocell initiated the first investigator site for clinical enrollment for an open label, single arm Phase 1/2 clinical trial of FCX-013 in August 2018. The primary objective of the trial is to evaluate the safety of FCX-013. Secondary analyses consist of several fibrosis assessments including histology, skin scores, ultrasound and additional measurements of targeted sclerotic lesions and control sites at various time points up to 16 weeks post-administration of FCX-013. Fibrocell is targeting ten patients with any subtype of localized scleroderma for enrollment (approximately 5 patients per Phase). The Phase 1 portion will enroll adult patients, and dosing for the first three adult patients will be staggered prior to dosing the rest of the trial’s population. Fibrocell intends to include pediatric patients in the Phase 2 portion of the trial after submission and approval of safety and activity data from the adult Phase 1 patients from the FDA and the Data Safety Monitoring Board for the trial.

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