Fibrocell Receives Guidance from FDA on Phase 3 Clinical Trial Design for FCX-007
October 25 2018 - 8:00AM
Company Plans to Submit Phase 3 Protocol to FDA in
Fourth Quarter of 2018 and Commence Trial in First Half of
2019
Fibrocell Science, Inc. (NASDAQ: FCSC), a gene therapy company
focused on transformational autologous cell-based therapies for
skin and connective tissue diseases, today announced completion of
a Type C meeting with the U.S. Food and Drug Administration (FDA)
to discuss the design of a Phase 3 clinical trial protocol of
FCX-007, the Company’s gene therapy candidate for the treatment of
recessive dystrophic epidermolysis bullosa (RDEB). The meeting was
facilitated by the current data in Fibrocell’s ongoing Phase 1/2
clinical trial of FCX-007 and the recent publication of draft
guidance from the FDA in the areas of gene therapy and
epidermolysis bullosa.
The FDA provided guidance on various design
aspects of Fibrocell’s proposed Phase 3 clinical trial. In
addition, Fibrocell received guidance on Chemistry, Manufacturing
and Control (CMC) requirements for the proposed Phase 3 clinical
trial and a potential future Biologics License Application (BLA)
for FCX-007. Based on the feedback from the meeting, Fibrocell
plans to submit the protocol in the fourth quarter of 2018 and will
provide details on the clinical trial design once it is
finalized.
“We appreciate the valuable and detailed
guidance furnished by the FDA on the design of the proposed Phase 3
clinical trial and advice on CMC requirements,” said John
Maslowski, President and CEO of Fibrocell. “We look forward to
finalizing the Phase 3 protocol design, and plan to initiate the
Phase 3 clinical trial in the first half of 2019.”
To date, FCX-007 has been evaluated in five RDEB
patients as part of an ongoing Phase 1/2 clinical trial. Fibrocell
plans to continue the Phase 2 portion of its ongoing Phase 1/2
clinical trial to collect additional data while submitting the
Phase 3 protocol to the FDA in parallel.
The FDA has granted Orphan Drug Designation,
Rare Pediatric Disease Designation and Fast Track Designation 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 manufactures clinical supply of FCX-007
and, if approved, commercial supply of FCX-007 at its cGMP
manufacturing facility located in Exton, Pennsylvania. This
multi-product, gene therapy manufacturing facility has existing
capacity to serve the U.S. RDEB market.
Fibrocell has not yet received the official FDA
meeting minutes from the Type C meeting and the information in this
release may be altered or supplemented by the information contained
in the official meeting minutes. The Company will provide further
regulatory updates on FCX-007 after receipt of the official FDA
minutes or other correspondence if there are material developments
in such minutes or correspondence.
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 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, including the Company’s plans to submit a Phase 3 protocol
to the FDA in the fourth quarter of 2018 and commence a Phase 3
clinical trial for FCX-007 in the first half of 2019; the potential
advantages of FCX-007 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: that the FDA’s
official meeting minutes may differ materially from the Company’s
understanding of the results of the Type C meeting with the FDA;
uncertainties and delays in the FDA review and approval of the
clinical trial protocol for FCX-007; 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
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