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 (FDA) has granted Fast Track
Designation to FCX-013, the Company’s clinical stage candidate for
the treatment of moderate to severe localized scleroderma—a
chronic, debilitating genetic skin disorder with no FDA approved
therapies.
“Fast Track designation represents an important milestone in
advancing clinical development of FCX-013,” said John Maslowski,
President and Chief Executive Officer of Fibrocell. “We are pleased
the FDA has awarded this designation to FCX-013 which, we believe,
has the potential to be the first gene therapy to treat excessive
collagen accumulation in the skin and soft tissue at the site of
localized scleroderma lesions and to bring relief from the severe
pain and functional disability associated with the disorder.”
The Fast Track program is designed to facilitate development and
expedite review of new therapies that address unmet medical needs
of patients with serious conditions. The designation offers
various benefits, including more frequent meetings with the FDA to
discuss the drug's development plan, eligibility for Accelerated
Approval or Priority Review if relevant criteria are met, and
opportunity for Rolling Review, which allows the Company to submit
completed sections of its Biologics License Application (BLA) to
the FDA, rather than waiting until every section of the BLA is
completed before the entire application can be reviewed.
Previously, the FDA granted Orphan Drug Designation to FCX-013
for the treatment of localized scleroderma and Rare Pediatric
Disease Designation for the treatment of moderate to severe
localized scleroderma.
In addition, Fibrocell is developing FCX-007 for the treatment
of recessive dystrophic epidermolysis bullosa (RDEB), which is
currently under evaluation in the Phase 2 portion of a Phase 1/2
clinical trial. FDA previously granted Orphan Drug, Rare
Pediatric Disease and Fast Track Designations to FCX-007.
Fibrocell is developing FCX-007 and FCX-013 in collaboration
with Precigen, Inc., a wholly owned subsidiary of Intrexon
Corporation (NYSE:XON), a leader in synthetic biology.
Fibrocell plans to manufacture FCX-013 at its cGMP cell
manufacturing facility located in Exton, Pennsylvania.
About FCX-013
FCX-013 is Fibrocell’s clinical stage 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
Precigen’s proprietary RheoSwitch Therapeutic System®, a biologic
switch activated by Veledimex—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.
About the Phase 1/2 Clinical Trial
Fibrocell initiated the first investigator site for clinical
enrollment for an open label, single arm Phase 1/2 clinical trial
of FCX-013 in August 2018. The primary objective of the trial is to
evaluate the safety of FCX-013. Secondary analyses consist of
several fibrosis assessments including histology, skin scores,
ultrasound and additional measurements of targeted sclerotic
lesions and control sites at various time points up to 16 weeks
post-administration of FCX-013. Fibrocell is targeting ten patients
with any subtype of localized scleroderma for enrollment
(approximately 5 patients per Phase). The Phase 1 portion will
enroll adult patients, and dosing for the first three adult
patients will be staggered prior to dosing the rest of the trial’s
population. Fibrocell intends to include pediatric patients in the
Phase 2 portion of the trial after submission and approval of
safety and activity data from the adult Phase 1 patients from the
FDA and the Data Safety Monitoring Board for the trial.
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 any
subtype that affects function or produces symptoms of discomfort,
tightness and pain. Current treatments for localized scleroderma
include systemic or topical corticosteroids which target
inflammation, UVA light therapy and physical therapy. There are few
treatment options to address excessive collagen accumulation in the
skin and connective tissue. 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 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, 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-013; the potential advantages of FCX-013 and Fibrocell’s other
product candidates; 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 Mar 2024 to Apr 2024
Fibrocell Science (NASDAQ:FCSC)
Historical Stock Chart
From Apr 2023 to Apr 2024