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EX-99.2 - EXHIBIT 99.2 - Fibrocell Science, Inc. | a020518finalfcsccorporat.htm |
8-K - 8-K - Fibrocell Science, Inc. | fcsc20518form8-k.htm |
Fibrocell Announces Submission of Investigational New Drug Application for FCX-013
for the Treatment of Moderate to Severe Localized Scleroderma
EXTON, PA — February 5, 2018 — Fibrocell Science, Inc. (NASDAQ: FCSC), a gene therapy
company focused on transformational autologous cell-based therapies for skin and connective
tissue diseases, today announced the submission of an Investigational New Drug (IND)
Application with the U.S. Food and Drug Administration (FDA) for FCX-013, the Company’s gene
therapy candidate for the treatment of moderate to severe localized scleroderma.
Localized scleroderma is a chronic autoimmune skin disorder that manifests as excess production
of collagen resulting in thickening, or fibrosis, of the skin and connective tissue. Moderate to
severe forms of localized scleroderma, including the linear subtype, can result in significant
morbidity, including pain, restricted motion, disfigurement and developmental issues. FCX-013 is
an autologous fibroblast genetically modified to express matrix metalloproteinase 1 (MMP-1), a
protein responsible for breaking down collagen. FCX-013 incorporates Intrexon Corporation’s
proprietary RheoSwitch Therapeutic System®, a biologic switch activated by an orally
administered compound to control protein expression once the initial fibrosis has been resolved.
“We are pleased to announce the submission of the IND as it represents a significant milestone
in advancing development of FCX-013 for the treatment of moderate to severe localized
scleroderma,” said John Maslowski, President and Chief Executive Officer of Fibrocell. “Adequate
treatment options do not exist for this vulnerable patient population. We are committed to
developing our novel gene therapy candidate to address this important unmet medical need of
patients suffering from this chronic, painful and debilitating disorder.”
The FDA has granted Orphan Drug Designation to FCX-013 for the treatment of localized
scleroderma. In addition, FCX-013 has been granted Rare Pediatric Disease Designation for the
treatment of moderate to severe localized scleroderma.
About FCX-013
Fibrocell is in pre-clinical development of FCX-013, its gene therapy candidate for the treatment
of moderate to severe localized scleroderma. FCX-013 is an autologous fibroblast genetically
modified using lentivirus and encoded for matrix metalloproteinase 1 (MMP-1), a protein
responsible for breaking down collagen. FCX-013 incorporates Intrexon’s proprietary RheoSwitch
Therapeutic System®, a biologic switch activated by an orally administered compound to control
protein expression at the site of the localized scleroderma lesions. FCX-013 is designed to be
injected under the skin at the location of the fibrotic lesions where the genetically-modified
fibroblast cells will produce MMP-1 to break down excess collagen accumulation. With the FCX-
013 therapy, the patient will take an oral compound to facilitate protein expression. Once the
fibrosis is resolved, the patient will stop taking the oral compound which will halt further MMP-1
production.
About Localized Scleroderma
Localized scleroderma is a chronic autoimmune skin disorder that manifests as excess production
of extracellular matrix, specifically collagen, resulting in thickening of the skin and connective
tissue. Localized scleroderma encompasses several subtypes which are classified based on the
depth and pattern of the lesion(s). The moderate to severe forms of the disorder include linear,
generalized, deep, pansclerotic and mixed morphea subtypes. Linear scleroderma is the most
common subtype in juvenile localized scleroderma and is associated with high morbidity and
lifelong disability. Linear lesions of the limbs may cause limb length discrepancy due to impaired
growth, muscle atrophy and joint contractures—orthopedic complications are reported in 30% to
50% of patients. Current treatments for localized scleroderma, which include systemic or topical
corticosteroids, UVA light therapy and physical therapy, only address the symptoms of the
disorder. We estimate that there are approximately 90,000 patients in the U.S. considered to have
moderate to severe localized scleroderma.
About Fibrocell
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, is the subject of a Phase 1/2 clinical 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 moderate to severe localized
scleroderma. 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 Fibrocell on Twitter at @Fibrocell.
Trademarks
Fibrocell, the Fibrocell logo, and Fibrocell Science are trademarks of Fibrocell Science, Inc. and/or
its affiliates. RheoSwitch Therapeutic System is a registered trademark of Intrexon Corporation.
All other names may be trademarks of their respective owners.
Forward-Looking Statements
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 and development of
FCX-013; the potential advantages of Fibrocell’s product candidates, including FCX-013, 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 studies and clinical trials; whether pre-clinical study and clinical trial results will validate
and support the safety and efficacy of Fibrocell’s product candidates; unanticipated or excess
costs relating to the development of Fibrocell’s gene therapy product candidates; Fibrocell’s ability
to obtain additional capital to continue to fund operations; 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 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.
# # #
Investor & Media Relations Contacts:
Karen Casey
484.713.6133
kcasey@fibrocell.com