Fibrocell Announces First Subjects Enrolled in Phase I/II Clinical Trial of FCX-007 for Treatment of Recessive Dystrophic Epi...
July 27 2016 - 4:01PM
Fibrocell Science, Inc. (NASDAQ:FCSC) today announced that the
first two adult subjects were enrolled in the Phase I/II clinical
trial of FCX-007 for the treatment of Recessive Dystrophic
Epidermolysis Bullosa (RDEB). FCX-007 is Fibrocell’s orphan
gene-therapy product candidate for the treatment of RDEB, a
devastating, painful, blistering skin disease with a high mortality
rate.
“Enrollment of the first patients is an
important milestone for Fibrocell and the RDEB community in the
advancement of FCX-007, potentially the first therapy to treat the
underlying cause of RDEB,” said David Pernock, Chairman and Chief
Executive Officer of Fibrocell.
Fibrocell now expects 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 for the adult subjects in
the Phase I portion of this trial are expected in the second half
of 2017, as well as six-month data for a cohort of this group.
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.
About FCX-007
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
type VII collagen (COL7). FCX-007 is a genetically-modified
autologous fibroblast that encodes the gene for COL7 and is being
developed in collaboration with Intrexon Corporation. 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 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.
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 orphan gene-therapy product
candidate, FCX-007, is entering a Phase I/II trial for the
treatment of recessive dystrophic epidermolysis bullosa (RDEB).
Fibrocell is in pre-clinical development of FCX-013, its orphan
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 and related conditions. For more information, visit
www.fibrocell.com or follow us on Twitter at @Fibrocell.
Trademarks
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.
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 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: Fibrocell’s ability to obtain additional capital to
fund its operations; 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;
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.
Investor Relations Contact:
John Woolford
Westwicke Partners
443.213.0506
john.woolford@westwicke.com
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