Fibrocell Science, Inc.’s Data Safety Monitoring Board (DSMB) has recommended continuation of the Phase 1/2 clinical trial of FCX-007 for the treatment of Recessive Dystrophic Epidermolysis Bullosa (RDEB), following a review of safety data from the first patient treated. No product-related adverse events were reported. FCX-007 has been granted Orphan Drug, Rare Pediatric Disease and Fast Track Designations by the FDA. Fibrocell is developing FCX-007 in collaboration with Intrexon Corporation (NYSE: XON), a leader in synthetic biology.

As specified in the protocol of this Phase 1/2 trial, additional adult patients can begin to be dosed with this recommendation from the DSMB. Six adult patients are targeted for the Phase 1 portion of the trial. Twelve-week post-treatment data for safety, mechanism of action and efficacy for multiple patients in the Phase 1 portion of this trial are expected in the third quarter of 2017.

The primary objective of this open-label clinical trial is to evaluate the safety of FCX-007 in RDEB patients. Additionally, the trial will assess the mechanism of action of FCX-007 through evaluation of COL7 expression and the presence of anchoring fibrils, as well as the efficacy of FCX-007 through evidence of wound healing. Assessments are performed at 4-, 12-, 25- and 52-weeks post-administration of FCX-007. Six adult patients are targeted to be treated with FCX-007 in the Phase 1 portion of the trial and six pediatric patients in the Phase 2 portion of the trial. Prior to conducting clinical trials on pediatric patients, Fibrocell is required to obtain allowance from the FDA by submitting evidence of FCX-007 activity in adult patients and data from its completed pre-clinical toxicology study.

“We appreciate the DSMB’s support to continue the evaluation of our leading gene therapy candidate, FCX-007,” said John Maslowski, Chief Executive Officer of Fibrocell. “We look forward to dosing additional adult RDEB patients in the Phase 1 portion of the trial with this novel therapy that has the potential to be transformative for patients suffering from this devastating disease.”