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Fibrocell Science Reports Third Quarter 2015

The following excerpt is from the company's SEC filing.

Financial Results and Operational Highlights

- Company to Host Conference Call and Webcast, Today at 8:30 a.m. EST -

EXTON, PA - November 5, 2015

- Fibrocell Science, Inc. (NASDAQ: FCSC), an autologous cell and gene therapy company focused on developing transformational therapies for rare and serious skin and connective tissue diseases, today reported financial results for the quarter ended September 30, 2015 and recent operational highlights. Fibrocell will host a conference call and webcast today at 8:30 a.m. EST.

“The field of gene therapy is rapidly evolving, and we are uniquely pos itioned as the only company developing personalized cell and gene therapies to treat rare diseases of the skin and connective tissue,” said David Pernock, Chairman and Chief Executive Officer. “We are working tirelessly to complete the steps to advance FCX-007 - our lead gene-therapy product candidate for the treatment of recessive dystrophic epidermolysis bullosa (RDEB) - into human clinical trials. RDEB is a devastating disease with currently no approved treatments, so to successfully develop FCX-007 and bring it to market could be transformational for patients living with RDEB, as well as their families.”

Mr. Pernock continued, “Our other programs, azficel-T for the treatment of chronic dysphonia, and FCX-013 for the treatment of linear scleroderma, continue to progress well. We expect to report Phase II data for chronic dysphonia in the second quarter of 2016, and we are on track to complete our proof-of-concept animal study for FCX-013 in the first half of next year.”

Third Quarter 2015 and Recent Highlights

In October, data was presented at the American Society of Human Genetics (ASHG) Annual Meeting that provided additional details of positive

in vivo

preclinical data for FCX-007, Fibrocell’s orphan gene-therapy product candidate for the treatment of RDEB being developed in collaboration with Intrexon Corporation (NYSE: XON). In this

preclinical study, FCX-007 was evaluated for toxicology and biological proof-of-concept data in RDEB and normal human skin xenografts implanted onto severe combined immunodeficiency (SCID) mice. The toxicology results for FCX-007 at two- and six-weeks post-administration showed no signs of toxicity, as observed by no test-article related findings, no tumors in the skin grafts or other organs, no statistical changes in blood chemistry and no apparent systemic distribution of the vector. Additional data supporting the biological proof-of-concept of FCX-007 was also presented at ASHG. The data showed that FCX-007 cells in a human skin xenograft model expressed type VII collagen (COL7) that localized to the basement membrane zone where anchoring fibrils are formed.

In July, Fibrocell filed an Investigational New Drug application (IND) for FCX-007 for the treatment of RDEB with the U.S. Food and Drug Administration (FDA). In September, Fibrocell received feedback from the FDA on the IND related to the areas of CMC, toxicology and our proposed Phase I/II clinical trial protocol. 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 requested that Fibrocell complete an additional 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. As a result, Fibrocell expects to initiate a Phase I/II clinical trial for FCX-007 in the second quarter of 2016, subject to successful completion of the new toxicology study and...