Fibrocell, Intrexon Offer Update on Regulation for FCX-007 for Treatment of RDEB

Fibrocell Science, Inc., FCSC, an autologous cell and gene therapy company focused on developing first-in-class treatments for rare and serious skin and connective tissue diseases with high unmet needs, and Intrexon Corporation XON, a leader in synthetic biology, today provided an update on the status of the previously filed Investigational New Drug (IND) application for FCX-007, Fibrocell's orphan gene-therapy drug candidate for the treatment of recessive dystrophic epidermolysis bullosa (RDEB), including feedback from the U.S. Food and Drug Administration (FDA) on the IND. The FDA's feedback related to the proposed Phase I/II clinical trial protocol and the areas of CMC and toxicology. With respect to toxicology, a hybrid pharmacology/toxicology study based on the injection of FCX-007 in RDEB human skin that was xenografted onto SCID (severe combined immunodeficiency) mice was included in the IND and showed no signs of toxicity. However, the FDA has now requested that Fibrocell execute a toxicology-specific study in which FCX-007 will be injected in non-grafted SCID mice. The new toxicology study is targeted to initiate in the fourth quarter of 2015. Fibrocell expects to amend the IND in response to the FDA's feedback and to include data from the new toxicology study in the first quarter of 2016. "We appreciate the clarity received from the Agency and look forward to completing this additional study as quickly as possible," said David Pernock, Chairman and Chief Executive Officer of Fibrocell. "Our goal remains the successful development of FCX-007 for the patients and families suffering from this devastating disease, and we expect to initiate the Phase I/II clinical trial in the second quarter of 2016."