Fibrocell Science, Inc. (NASDAQ:FCSC), an autologous cell and gene therapy company translating personalized biologics into medical breakthroughs for diseases affecting the skin, connective tissue and joints, today announced the initiation of adult patient recruitment in its Phase I/II clinical trial of FCX-007 for the treatment of recessive dystrophic epidermolysis bullosa (RDEB).
“Progressing FCX-007—our first genetically-modified drug candidate developed in collaboration with Intrexon Corporation—into human clinical trials is a significant accomplishment for Fibrocell, and represents a meaningful step forward in the development of a treatment for RDEB,” said David Pernock, Chairman and Chief Executive Officer of Fibrocell. “RDEB is a devastating, painful disease with high mortality. The opportunity to develop the first disease-modifying therapy for RDEB and potentially provide it to the patients and families that so desperately need it is amazing.”
With the recent discontinuation of its non-genetically modified program for vocal cord scarring, Fibrocell will now focus its efforts and resources on its gene therapy portfolio of drug candidates developed in conjunction with its collaborator Intrexon Corporation (NYSE:XON) including FCX-007 for RDEB, FCX-013 for the treatment of linear scleroderma, and genetically-modified fibroblasts to treat chronic inflammatory and degenerative diseases of the joint, including arthritis and related conditions.
Mr. Pernock continued, “We are excited to begin a new era for Fibrocell with our efforts and focus dedicated to our gene therapy portfolio of drug candidates.”
About the Phase I/II Clinical Trial
The primary objective of this open-label, Phase I/II clinical trial is to evaluate the safety of FCX-007 in RDEB subjects. Additionally, the trial will evaluate type VII collagen expression and the presence of anchoring fibrils resulting from FCX-007, as well as evidence of wound healing. Six adult subjects are expected to be treated with FCX-007 in the Phase I portion of the trial and six pediatric subjects in the Phase II portion of the trial. Prior to conducting studies on pediatric subjects, Fibrocell is required to obtain allowance from the U.S. Food and Drug Administration and submit evidence of FCX-007 activity in adult subjects and final data from its ongoing toxicology study.
FCX-007 is Fibrocell’s novel gene-therapy product candidate for the treatment of recessive dystrophic epidermolysis bullosa (RDEB), a congenital and progressive orphan skin disease caused by the deficiency of the protein type VII collagen (COL7). FCX-007 is a genetically-modified autologous fibroblast that encodes COL7 and is being developed in collaboration with Intrexon Corporation (NYSE:XON). By genetically modifying autologous fibroblasts, ex vivo, to produce COL7, culturing them and then treating blisters and wounds locally via injection, FCX-007 offers the potential to address the underlying cause of the disease by providing high levels of COL7 directly to the affected areas, thereby avoiding systemic treatment.
About Recessive Dystrophic Epidermolysis Bullosa (RDEB)
Recessive dystrophic epidermolysis bullosa (RDEB) is the most severe form of dystrophic epidermolysis bullosa (DEB), a congenital, progressive, devastatingly painful and debilitating genetic disorder that often leads to death. RDEB is caused by a mutation of the COL7A1 gene, the gene which encodes for type VII collagen, a protein that forms anchoring fibrils. Anchoring fibrils hold together the layers of skin, and without them, skin layers separate causing severe blistering, open wounds and scarring in response to any kind of friction, including normal daily activities like rubbing or scratching. Children who inherit the condition are often called “butterfly children” because their skin is as fragile as a butterfly’s wings. We estimate there are approximately 1,100 – 2,500 RDEB patients in the U.S. Currently, treatments for RDEB address only the sequelae, including daily bandaging, hydrogel dressings, antibiotics, feeding tubes and surgeries.
Fibrocell is an autologous cell and gene therapy company translating personalized biologics into medical breakthroughs for diseases affecting the skin, connective tissue and joints using genetically-modified autologous fibroblasts. Fibrocell’s most advanced gene-therapy product candidate, FCX-007, is entering a Phase I trial for the treatment of recessive dystrophic epidermolysis bullosa (RDEB). Fibrocell is in pre-clinical development of FCX-013, its gene-therapy product candidate for the treatment of linear scleroderma. Both FCX-007 and FCX-013 are being developed in collaboration with Intrexon Corporation (NYSE:XON), a leader in synthetic biology. In addition, Fibrocell and Intrexon are in collaboration to develop a gene therapy for the treatment of arthritis. For more information, visit www.fibrocell.com or follow us on Twitter at @Fibrocell.
Fibrocell, the Fibrocell logo, Fibrocell Science and LAVIV® are trademarks of Fibrocell Science, Inc. and/or its affiliates. All other names may be trademarks of their respective owners.
This press release contains, and our officers and representatives may from time to time make, statements that are “forward-looking statements” within the meaning of the safe harbor provisions of the U.S. Private Securities Litigation Reform Act of 1995. All statements that are not historical facts are hereby identified as forward-looking statements for this purpose and include, among others, statements relating to: Fibrocell’s focus on the advancement of its gene therapy programs; the potential advantages of Fibrocell’s product candidates; and other statements regarding Fibrocell’s future operations, financial performance and financial position, prospects, strategies, objectives and other future events.
Forward-looking statements are based upon management’s current expectations and assumptions and are subject to a number of risks, uncertainties and other factors that could cause actual results and events to differ materially and adversely from those indicated herein including, among others: Fibrocell’s ability to obtain additional capital to fund its operations; FDA allowance to treat pediatric subjects in the Phase II portion of Fibrocell’s Phase I/II clinical trial of FCX-007; uncertainties relating to the initiation and completion of pre-clinical and clinical trials; whether pre-clinical and clinical trial results will validate and support the safety and efficacy of Fibrocell’s product candidates; Fibrocell’s ability to maintain its collaboration with Intrexon Corporation; and the risks, uncertainties and other factors discussed under the caption “Item 1A. Risk Factors” in Fibrocell’s most recent Form 10-K filing. As a result, you are cautioned not to place undue reliance on any forward-looking statements. While Fibrocell may update certain forward-looking statements from time to time, Fibrocell specifically disclaims any obligation to do so, whether as a result of new information, future developments or otherwise.
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