– FCX-007 well-tolerated up to 52 weeks
post-administration –
Fibrocell Science, Inc. (NASDAQ:FCSC), a gene therapy company
focused on transformational autologous cell-based therapies for
skin and connective tissue diseases, today provided an update on
the interim results and progress of its Phase 1/2 clinical trial of
FCX-007 for the treatment of recessive dystrophic epidermolysis
bullosa (RDEB). The results were presented by M. Peter
Marinkovich, MD, the trial’s Study Director at the Stanford
University site and Associate Professor of Dermatology at the
Stanford University School of Medicine, at the 7th International
Investigative Dermatology meeting on May 19, 2018.
Four adult patients (n=7 wounds) aged 20 to 37
have been dosed with FCX-007 in the margins of and across targeted
wounds, as well as in separate intact skin sites. Three
patients received a single intradermal injection session at
baseline. One patient received a second injection session in
the remaining unhealed areas of wounds at 25 weeks
post-administration, as allowed by the clinical trial
protocol.
Safety data from these patients show FCX-007 was
well-tolerated up to 52 weeks post-administration. There were no
serious adverse events and no product related adverse events
reported. No type VII collagen (COL7) autoantibody response
was noted.
Various COL7 expression signals were detected
throughout the data set using either immunofluorescence (IF) or
immunoelectron microscopy (IEM) up to 52 weeks
post-administration. Anchoring fibril structures have also
been observed using IEM.
Wounds were evaluated during a monitoring period
prior to dosing and they were observed to be open for up to eight
months. Compared to the baseline measurement collected at Day 0
before the administration of FCX-007, the percentage of dosed
wounds healing > 50% when compared to baseline were observed as
follows:
- 100% (7/7) at 4 weeks post-administration- 86%
(6/7) at 12 weeks- 67% (2/3) at 25/32 weeks- 100% (1/1) at 52
weeks
A similar trend was also observed for treated
wounds healing > 75% when compared to baseline. Untreated
wounds of similar size to the treated wounds were selected and
monitored as controls on each patient. The percentage of
untreated control wounds healing > 50% when compared to baseline
were observed as follows:
- 14% (1/7) at 4 weeks post-administration- 17%
(1/6) at 12 weeks - 0% (0/2) at 25/32 weeks - 0% (0/1) at 52
weeks
“The Phase 1 portion of the trial of FCX-007
continues to be encouraging and reinforces the potential for
treating RDEB patients,” said Alfred Lane, MD, Chief Medical
Advisor of Fibrocell and Professor of Dermatology and Pediatrics
(Emeritus) at the Stanford University School of Medicine. “As
we move into Phase 2 of the trial, I am looking forward to
incorporating these learnings into the trial and determining the
impact on patient outcomes.”
There is one patient enrolled in the Phase 2
portion of the trial, with three additional screening visits
scheduled prior to the end of June 2018. Enrollment of six
patients is expected to be completed in the third quarter of
2018.
“We are pleased with the continued progress of
our FCX-007 program that offers promise to be transformative for
RDEB patients,” said John Maslowski, President and Chief Executive
Officer of Fibrocell. “Based on safety, pharmacology and wound
healing data, we plan to continue exploring dose range and
administration for future patients. We are looking forward to
moving ahead with this protocol that advances the clinical trial of
FCX-007 and positions us to achieve future milestones for the
program.”
Fibrocell is developing FCX-007 in collaboration
with Precigen, Inc., a wholly owned subsidiary of Intrexon
Corporation (NYSE:XON), a leader in synthetic biology.
Fibrocell Conference Call &
Webcast
On Monday, May 21, 2018 at 8:30 a.m. EDT,
Fibrocell will host a conference call and webcast to discuss the
interim results and progress of the Phase 1/2 clinical trial of
FCX-007. A slide presentation summarizing the data will be
referenced during the call and is posted in the Investors section
of the Company’s website www.fibrocell.com/investors/events under
the event, “Fibrocell FCX-007 Conference Call & Webcast.”
Following the presentation, there will be a
question-and-answer session with John Maslowski, President and CEO
of Fibrocell, and Alfred Lane, MD, Chief Medical Advisor of
Fibrocell and Professor of Dermatology and Pediatrics (Emeritus) at
the Stanford University School of Medicine.
To participate on the live call, please dial
866-548-4713 (domestic) or +1-323-794-2093 (international) and
provide the conference code 5629866. The conference call will also
be webcast live from the Investors section of Fibrocell's website
at www.fibrocell.com/investors/events under the event,
“Fibrocell FCX-007 Conference Call & Webcast,” and will be
archived there for 30 days.
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., a wholly owned subsidiary of 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. FCX-007
has been granted Orphan Drug, Rare Pediatric Disease and Fast Track
Designations by the U.S. Food and Drug Administration.
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 product
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 (NYSE: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 exploration of strategic alternatives;
the timing of dosing and reporting of interim data and trial
updates for its Phase 1/2 clinical trial of FCX-007; the completion
of enrollment in the Phase 2 portion of its Phase 1/2 clinical
trial of FCX-007; the potential for FCX-007 to receive Priority
Review Vouchers upon market authorization; 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: 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; that interim
clinical trial results are not necessarily indicative of final
clinical results and final clinical trial results may not be
positive with regard to safety or efficacy of FCX-007;
uncertainties and delays relating to the initiation, enrollment and
completion of pre-clinical studies and clinical trials; whether
pre-clinical study and 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;
Fibrocell’s ability to maintain its collaboration with Precigen,
Inc., a wholly owned subsidiary of 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 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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