EXTON, Pa., Sept. 19, 2016 (GLOBE NEWSWIRE) — Fibrocell Science, Inc. (NASDAQ:FCSC) today announced that two additional adult subjects were enrolled in the Phase I/II clinical trial of FCX-007 for the treatment of Recessive Dystrophic Epidermolysis Bullosa (RDEB), satisfying enrollment in one of two cohorts in the Phase I portion of the trial.
“We are excited about the steady progress of the trial, as four of the six adult RDEB patients required for the Phase I portion of the study have been enrolled since late July,” said David Pernock, Chairman and Chief Executive Officer of Fibrocell. “This advancement is an important milestone toward potentially offering patients the first gene therapy to treat the underlying cause of this devastating disease.”
Six adult subjects are targeted for enrollment in the Phase I portion of the trial. The subjects are divided into two cohorts in order to evaluate the safety of FCX-007 in each population type. One cohort is comprised of those who have positive expression of the non-collagenous portion of the type VII collagen (COL7) protein (NC1+). The second cohort includes those who do not express the non-collagenous portion of the protein (NC1-).
Enrollment of the latest subjects fulfills the NC1+ cohort and also provides the first subject for the NC1- cohort. Two more subjects are required for the NC1- cohort to complete enrollment in the Phase I portion of the trial.
Fibrocell continues to expect to dose the first subject in this trial at the end of 2016. Additional adult subjects will be dosed after a required 90-day waiting period is complete to ensure there are no safety concerns for the first dose of the new gene-therapy product. Three-month post-treatment follow-up data for safety, mechanism of action and efficacy in the Phase I portion of the trial are expected in the second half of 2017, as well as six-month data for multiple subjects.
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 enrolling 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 clinical-stage, orphan gene-therapy product candidate for the treatment of recessive dystrophic epidermolysis bullosa (RDEB), a congenital and progressive skin disease caused by the deficiency of the protein COL7. FCX-007 is a genetically-modified autologous fibroblast that encodes the gene for 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 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 while avoiding systemic distribution. To learn more about the FCX-007 Phase I/II clinical trial, please visit www.clinicaltrials.gov and search the identifier NCT02810951.
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 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 and connective tissue. Fibrocell’s most advanced product candidate, FCX-007, has begun a Phase I/II trial for the treatment of recessive dystrophic epidermolysis bullosa (RDEB). Fibrocell is in pre-clinical development of FCX-013, its product candidate for the treatment of linear scleroderma. In addition, Fibrocell has a third program in the research phase for the treatment of arthritis and related conditions. Fibrocell’s gene-therapy portfolio is being developed in collaboration with Intrexon Corporation (NYSE: XON), a leader in synthetic biology. For more information, visit www.fibrocell.com or follow us on Twitter at @Fibrocell.
Fibrocell, the Fibrocell logo and Fibrocell Science 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 expectations regarding the timing of the completion of adult subject enrollment, dosing and reporting of results for the Phase I portion of the Phase I/II clinical trial of FCX-007; 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: uncertainties and delays relating to the initiation, enrollment 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; FDA allowance to enroll pediatric subjects in the Phase II portion of the Phase I/II clinical trial of FCX-007; Fibrocell’s ability to maintain its collaboration with Intrexon Corporation; Fibrocell’s ability to obtain additional capital to fund its operations; and the risks, uncertainties and other factors discussed under the caption “Item 1A. Risk Factors” in Fibrocell’s most recent Form 10-K filing and Form 10-Q filings. 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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