Fibrocell Science receives guidance from FDA on Phase 3 FCX-007 trial

Fibrocell Science announced the completion of a Type B end-of-Phase 2 meeting with the 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, or RDEB, to support a biologics license application, or 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. 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 greater than 50% wound closure, a patient reported outcome measure and an analysis of durability out to 24 weeks. The presence of type VII collagen, or 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, or CMC, information to the investigational new drug, or 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.

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