Fibrocell Science, Inc., (NASDAQ:FCSC), an autologous cell and gene
therapy company focused on developing first-in-class treatments for
rare and serious skin and connective tissue diseases with high
unmet needs, and Intrexon Corporation (NYSE:XON), a leader in
synthetic biology, today provided an update on the status of the
previously filed Investigational New Drug (IND) application for
FCX-007, Fibrocell's orphan gene-therapy drug candidate for the
treatment of recessive dystrophic epidermolysis bullosa (RDEB),
including feedback from the U.S. Food and Drug Administration (FDA)
on the IND.
The FDA's feedback related to the proposed Phase I/II clinical
trial protocol and the areas of CMC and toxicology. 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 has now
requested that Fibrocell execute a 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.
"We appreciate the clarity received from the Agency and look
forward to completing this additional study as quickly as
possible," said David Pernock, Chairman and Chief Executive Officer
of Fibrocell. "Our goal remains the successful development of
FCX-007 for the patients and families suffering from this
devastating disease, and we expect to initiate the Phase I/II
clinical trial in the second quarter of 2016."
About FCX-007
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. 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.
About Fibrocell Science, Inc.
Fibrocell Science, Inc. (NASDAQ:FCSC) is an autologous cell
and gene therapy company primarily focused on developing
first-in-class treatments for rare and serious skin and connective
tissue diseases with high unmet medical needs. Fibrocell's
most advanced product candidate, azficel-T, uses its proprietary
autologous fibroblast technology and is in a Phase II clinical
trial for the treatment of chronic dysphonia resulting from vocal
cord scarring. In collaboration with Intrexon Corporation
(NYSE:XON), a leader in synthetic biology, Fibrocell is also
developing gene therapies for skin diseases using gene-modified
autologous fibroblasts. Fibrocell is in preclinical
development of FCX-007, its orphan gene-therapy product candidate,
for the treatment of recessive dystrophic epidermolysis bullosa
(RDEB). Fibrocell is also in preclinical development of
FCX-013, its second gene-therapy product candidate, for the
treatment of linear scleroderma. For more information, visit
fibrocell.com.
About Intrexon Corporation
Intrexon Corporation (NYSE:XON) is Powering the Bioindustrial
Revolution with Better DNA™ to create biologically-based products
that improve the quality of life and the health of the planet. The
Company's integrated technology suite provides its partners across
diverse markets with industrial-scale design and development of
complex biological systems delivering unprecedented control,
quality, function, and performance of living cells. We call our
synthetic biology approach Better DNA®, and we invite you to
discover more at www.dna.com.
Fibrocell 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: our interpretation
of the FDA's feedback relating to the IND for FCX-007 and our plans
to address such feedback; the initiation, design and timing of the
additional toxicology study requested by the FDA; the expected
timing of the submission of an amended IND for FCX-007; our plans
to initiate a Phase I/II clinical trial of FCX-007 for the
treatment of RDEB and the expected timing of such trial; the
potential advantages of our product candidates; and other
statements regarding our future operations, financial performance
and financial position, prospects, strategies, objectives and other
future events.
Forward-looking statements are based upon management's current
expectations, intentions and beliefs and are subject to a number of
risks, uncertainties, assumptions and other factors that could
cause actual results and events to differ materially and adversely
from those indicated herein including, among others: our ability to
successfully complete the additional toxicology study requested by
the FDA and whether the results of such study will validate and
support the safety of FCX-007; our ability to adequately address
the FDA's feedback relating to our IND for FCX-007; the outcome of
the FDA's review of the amended IND and approval to commence the
Phase I/II trial; varying interpretation of pre-clinical data; our
ability to maintain our collaboration with Intrexon Corporation;
and the risks, uncertainties and other factors discussed under the
caption "Item 1A. Risk Factors" in our most recent Form 10-K and
Form 10-Q filings, as well as discussions of potential risks,
uncertainties and other important factors in our subsequent filings
with the Securities and Exchange Commission. As a result, you are
cautioned not to place undue reliance on any forward-looking
statements. While we may update certain forward-looking statements
from time to time, we specifically disclaim any obligation to do
so, whether as a result of new information, future developments or
otherwise.
Intrexon Forward-Looking Statements
Some of the statements made in this press release are
forward-looking statements. These forward-looking statements
are based upon our current expectations and projections about
future events and generally relate to our plans, objectives and
expectations for the development of our business. Although
management believes that the plans and objectives reflected in or
suggested by these forward-looking statements are reasonable, all
forward-looking statements involve risks and uncertainties and
actual future results may be materially different from the plans,
objectives and expectations expressed in this press release.
CONTACT: Investor & Media Relations Contact:
Karen Casey
Fibrocell Science, Inc.
405 Eagleview Boulevard
Exton, PA 19341
+1 (484) 713-6133
kcasey@fibrocell.com
Christopher Basta
Intrexon
Vice President, Investor Relations
+1 (561) 410-7052
investors@intrexon.com
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