Fibrocell Science, Inc. announced the completion of a Type B end-of-Phase 2 meeting with the U.S. Food and Drug Administration (FDA) to discuss the design of a Phase 3 clinical trial for FCX-007, the Company’s gene therapy candidate for the treatment of recessive dystrophic epidermolysis bullosa (RDEB), to support a Biologics License Application (BLA) filing. In addition, the Company reported additional positive safety and wound healing data from its ongoing Phase 1/2 clinical trial of FCX-007. In the Type B face-to-face meeting, the FDA provided guidance on various design aspects of Fibrocell’s proposed Phase 3 clinical trial, named DEFI-RDEB (dermal fibroblasts-RDEB). The trial is designed as an open label, multi-centered, intra-patient controlled trial expected to enroll 15-20 patients. Selected wounds will be monitored prior to dosing to confirm they are non-healing. For each patient, up to three pairs of wounds will be identified at baseline and randomized, with one wound receiving FCX-007 and the other wound left as the untreated control. Two doses of FCX-007 will be administered four weeks apart to the treated wounds. Both treated and untreated wounds will also receive standard of care, including routine skin care and bandaging. The proposed primary outcome measure for the DEFI-RDEB trial is a comparison of the proportion of FCX-007 treated wounds and untreated matched wounds with complete closure in a prospectively defined wound pair at 12 weeks post-administration of the first dose. Secondary endpoints include evaluation of the proportion of wounds achieving >50% wound closure, a patient reported outcome measure and an analysis of durability out to 24 weeks. The presence of type VII collagen (COL7) will be assessed from biopsy samples in a subpopulation of patients as an exploratory endpoint. Fibrocell plans to submit a revised clinical trial protocol and statistical analysis plan based upon the FDA’s feedback and requested Chemistry, Manufacturing and Controls (CMC) information to the Investigational New Drug (IND) application. Fibrocell also reported updated data from its ongoing Phase 1/2 clinical trial for FCX-007. To date, FCX-007 has been evaluated in eight wounds across five adult RDEB patients in the trial. Consistent with previously reported results, no product-related serious adverse events or circulating autoantibodies to COL7 have been reported. The complete wound closure result (63% treated vs. 0% untreated) was the basis for the primary endpoint design in the DEFI-RDEB trial. Fibrocell recently completed dosing of a sixth patient—the first pediatric patient dosed with FCX-007—in the current Phase 1/2 clinical trial. Remaining Phase 2 patients who have not received dosing will be contacted to determine if they would agree to reconsent into the Phase 3 trial. As a reminder, the FDA has granted Orphan Drug Designation, Rare Pediatric Disease Designation and Fast Track Designation to FCX-007. Fibrocell is also developing FCX-013 for the treatment of moderate to severe localized scleroderma. Fibrocell reported FCX-013 received Fast Track Designation from the FDA in the third quarter of 2018, and its IND for this gene therapy candidate was granted allowance by the FDA earlier in the year. Fibrocell is currently enrolling the Phase 1 portion of a Phase 1/2 clinical trial for FCX-013, and expects to complete enrollment of Phase 1 adult patients in the third quarter of 2019. In addition to the Fast Track Designation, FCX-013 has also been granted Orphan Drug and Rare Pediatric Disease Designations by the FDA.