Fibrocell Awarded $1.4 Million FDA Orphan Grant for FCX-007 for Treatment of Recessive Dystrophic Epidermolysis Bullosa
25 September 2018 - 10:00PM
Fibrocell Science, Inc. (NASDAQ: FCSC), a gene therapy company
focused on transformational autologous cell-based therapies for
skin and connective tissue diseases, today announced that the U.S.
Food and Drug Administration’s (FDA) Office of Orphan Products
Development (OOPD) has awarded a $1.4 million clinical trial
research grant for Fibrocell’s continued clinical development of
FCX-007, the Company’s gene therapy candidate for the treatment of
recessive dystrophic epidermolysis bullosa (RDEB), a devastating,
rare skin blistering disease with high mortality.
“We are delighted with the FDA’s recognition to
support the ongoing progress of our clinical trials of FCX-007,”
said John Maslowski, President and Chief Executive Officer of
Fibrocell. “With no FDA approved therapies available, this grant
further validates the significant opportunity of FCX-007’s
potential to relieve the pain and suffering from the debilitating,
chronic blisters and open wounds of RDEB and offer hope to patients
and their families.”
Fibrocell’s $1.4 million grant, which will be
distributed over the next four years, was awarded by the FDA
through the OOPD’s Orphan Products Clinical Trials Grants Program.
This program supports the clinical development of products for use
in rare diseases or conditions for which “no current therapy exists
or where the proposed product will be superior to the existing
therapy.” FDA stated in a press release for these awards that
“[g]rant applications were reviewed and evaluated for scientific
and technical merit by more than 100 rare disease experts, which
included representatives from academia, the National Institutes of
Health and the FDA.”
FCX-007 is currently being evaluated in the
Phase 2 portion of a Phase 1/2 clinical trial for the treatment of
RDEB. Six patients ages seven and older are targeted to be
treated with FCX-007 in the Phase 2 portion of the clinical trial.
Fibrocell expects to report an interim data analysis for FCX-007
and provide a clinical trial update from Phase 1 patients and
available data from Phase 2 patients in the first quarter of
2019.
The FDA has granted Orphan Drug Designation for
the treatment of dystrophic epidermolysis bullosa, including RDEB,
Rare Pediatric Disease Designation for the treatment of RDEB and
Fast Track Designation for the treatment of RDEB to FCX-007.
Fibrocell is developing FCX-007 in collaboration
with Precigen, Inc., a wholly owned subsidiary of Intrexon
Corporation (NASDAQ: XON), a leader in synthetic biology.
Fibrocell plans to manufacture FCX-007 at its cGMP cell
manufacturing facility located in Exton, Pennsylvania.
About FCX-007
FCX-007 is Fibrocell's clinical stage, gene
therapy product candidate for the treatment of 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 the gene
for COL7 and is being developed in collaboration with Precigen,
Inc. 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.
About the Phase 1/2 Clinical
Trial
The primary objective of this open-label
clinical trial is to evaluate the safety of FCX-007 in RDEB
patients. Additionally, the trial is assessing wound healing
and pharmacology at 4, 12, 25 and 52 weeks
post-administration. Six patients ages seven and older are
targeted to be treated with FCX-007 in the Phase 2 portion of the
trial. To learn more about the clinical trial, please visit
www.clinicaltrials.gov and search the identifier NCT02810951.
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
RDEB. Fibrocell is also developing FCX-013, the Company's clinical
stage candidate for the treatment of moderate to severe localized
scleroderma. Fibrocell's gene therapy portfolio is being
developed in collaboration with Precigen, Inc., a wholly owned
subsidiary of Intrexon Corporation (NASDAQ: 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. 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 clinical development of
FCX-007; the potential advantages of FCX-007 and Fibrocell’s other
product candidates; the potential benefits of the clinical trial
research grant from the OOPD; the potential benefits of Fast Track
Designation, Orphan Drug Designation and Rare Pediatric Disease
Designation; 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 clinical trials; whether 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; uncertainties associated with being able to
identify, evaluate and complete any strategic transaction or
alternative; the impact of the announcement of the Board of
Directors’ review of strategic alternatives, as well as any
strategic transaction or alternative that may be pursued, on the
Company's business, including its financial and operating results
and its employees; Fibrocell’s ability to maintain its
collaboration with Precigen, Inc.; 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
Contact:Karen Casey484.713.6133kcasey@fibrocell.com
Fibrocell Science (NASDAQ:FCSC)
Historical Stock Chart
From Apr 2024 to May 2024
Fibrocell Science (NASDAQ:FCSC)
Historical Stock Chart
From May 2023 to May 2024