- Phase 3 clinical trial, named
DEFI-RDEB, is expected to commence in the second quarter 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 the completion
of a Type B end-of-Phase 2 meeting with the U.S. Food and Drug
Administration (FDA) to discuss the design of a Phase 3 clinical
trial for FCX-007, the Company’s gene therapy candidate for the
treatment of recessive dystrophic epidermolysis bullosa (RDEB), to
support a Biologics License Application (BLA) filing. In
addition, the Company reported additional positive safety and wound
healing data from its ongoing Phase 1/2 clinical trial of FCX-007.
FCX-007 Phase 3 Trial Design and FDA
Type B Meeting Feedback
In the Type B face-to-face meeting, the FDA
provided guidance on various design aspects of Fibrocell’s proposed
Phase 3 clinical trial, named DEFI-RDEB (dermal
fibroblasts-RDEB). The
trial is designed as an open label, multi-centered, intra-patient
controlled trial expected to enroll 15-20 patients. Selected
wounds will be monitored prior to dosing to confirm they are
non-healing. For each patient, up to three pairs of wounds
will be identified at baseline and randomized, with one wound
receiving FCX-007 and the other wound left as the untreated
control. Two doses of FCX-007 will be administered four weeks
apart to the treated wounds. Both treated and untreated
wounds will also receive standard of care, including routine skin
care and bandaging.
The proposed primary outcome measure for the
DEFI-RDEB trial is a comparison of the proportion of FCX-007
treated wounds and untreated matched wounds with complete closure
in a prospectively defined wound pair at 12 weeks
post-administration of the first dose. Secondary endpoints
include evaluation of the proportion of wounds achieving >50%
wound closure, a patient reported outcome measure and an analysis
of durability out to 24 weeks. The presence of type VII
collagen (COL7) will be assessed from biopsy samples in a
subpopulation of patients as an exploratory endpoint.Fibrocell
plans to submit a revised clinical trial protocol and statistical
analysis plan based upon the FDA’s feedback and requested
Chemistry, Manufacturing and Controls (CMC) information to the
Investigational New Drug (IND) application. “The FDA has
provided us with invaluable advice on the design of the DEFI-RDEB
trial, and we are grateful for their comprehensive feedback,” said
John Maslowski, President and Chief Executive Officer of
Fibrocell. “RDEB remains an urgent unmet medical need, and we
believe the initiation of a Phase 3 clinical trial of FCX-007 for
this devastating disease is a significant milestone for both
patients and Fibrocell. We are working diligently to incorporate
the FDA’s comments into the IND, with the expectation of initiating
the Phase 3 trial in the second quarter of 2019.”FCX-007
Phase 1/2 Trial UpdateFibrocell also reported today
updated data from its ongoing Phase 1/2 clinical trial for
FCX-007. To date, FCX-007 has been evaluated in eight wounds
across five adult RDEB patients in the trial. Consistent with
previously reported results, no product-related serious adverse
events or circulating autoantibodies to COL7 have been reported.The
proportion of wounds healing at increasing closure percentages
12-week post-administration of a single injection session of
FCX-007 are as follows:
|
Wound Closure Percentage |
|
>50% |
>75% |
Complete |
FCX-007 Treated Wounds |
88% (7/8) |
75% (6/8) |
63% (5/8) |
Untreated Control Wounds |
29% (2/7) |
14% (1/7) |
0% (0/7) |
The complete wound closure result (63% treated
vs. 0% untreated) was the basis for the primary endpoint design in
the DEFI-RDEB trial.The proportion of wounds with >50% closure
at various post-administration visits are as follows:
|
Study Visit |
|
4 Weeks |
12 Weeks |
25 Weeks |
52 Weeks |
FCX-007 Treated Wounds |
100% (8/8) |
88% (7/8) |
67% (2/3) |
66% (2/3) |
Untreated Control Wounds |
13% (1/8) |
29% (2/7) |
0% (0/2) |
33% (1/3) |
“We continue to be encouraged by the positive
safety and efficacy trends for FCX-007”, stated Alfred Lane, MD,
Chief Medical Advisor of Fibrocell and Professor of Dermatology and
Pediatrics (Emeritus) at the Stanford University School of
Medicine, “As the FDA has provided the necessary feedback to design
a Phase 3 trial for FCX-007, we look forward to determining the
potential benefits to patients suffering with RDEB.”Fibrocell
recently completed dosing of a sixth patient—the first pediatric
patient dosed with FCX-007—in the current Phase 1/2 clinical trial.
Remaining Phase 2 patients who have not received dosing will
be contacted to determine if they would agree to reconsent into the
Phase 3 trial.As a reminder, the FDA has granted Orphan Drug
Designation, Rare Pediatric Disease Designation and Fast Track
Designation to FCX-007. Fibrocell is also developing FCX-013
for the treatment of moderate to severe localized
scleroderma. Fibrocell reported FCX-013 received Fast Track
Designation from the FDA in the third quarter of 2018, and its IND
for this gene therapy candidate was granted allowance by the FDA
earlier in the year. Fibrocell is currently enrolling the
Phase 1 portion of a Phase 1/2 clinical trial for FCX-013, and
expects to complete enrollment of Phase 1 adult patients in the
third quarter of 2019. In addition to the Fast Track
Designation, FCX-013 has also been granted Orphan Drug and Rare
Pediatric Disease Designations by the FDA. Financial
Results for the Twelve Months Ended December 31, 2018
For the year ended December 31, 2018, Fibrocell
reported a diluted net loss of $1.45 per share, compared to a
diluted net loss of $6.67 per share for the same period in
2017.
Research and development expenses decreased 51%
to approximately $6.0 million for the year ended December 31, 2018,
as compared to approximately $12.2 million for the same period in
2017. The decrease in FCX-007 spending was due primarily to
decreased costs from our clinical partner Intrexon Corporation, as
the Phase 1 portion of the FCX-007 Phase 1/2 clinical trial was
substantially completed at the end of 2017; movement in-house of
the manufacturing of the drug product used in the Phase 1/2
clinical trial of FCX-007 previously contracted to a third party
manufacturer and a decrease of approximately $0.5 million in an
estimate of costs to settle a dispute with one of Intrexon’s
vendors, for which a settlement was agreed to and was paid by us in
August 2018. The decrease in FCX-013 spending was related primarily
to decreased costs from Intrexon of approximately $2.3 million, as
substantially all of the costs of the pre-clinical phase of the
FCX-013 program were incurred at the end of 2017, while 2018 has
been used for clinical trial start-up activities.
Fibrocell used approximately $12.7 million in
cash for operations during the year ended December 31, 2018, and
used approximately $17.0 million in cash for operations during the
year ended December 31, 2017.
As of December 31, 2018, the Company had cash
and cash equivalents of approximately $14.4 million and working
capital of approximately $12.4 million. The Company believes that
its cash and cash equivalents will be sufficient to fund operations
into the fourth quarter of 2019.Conference Call and
Webcast
To participate on the live call, please dial
888-394-8218 (domestic) or +1-323-794-2588 (international), and
provide the conference code 8315981 five to ten minutes before
the start of the call. The conference call will also be webcast
live under the investor relations section of Fibrocell's website at
www.fibrocell.com/investors/events and will be archived there for
30 days following the call. 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 COL7. FCX-007 is a genetically-modified autologous
fibroblast that encodes the gene for COL7. 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.
Fibrocell is developing FCX-007 in collaboration
with Intrexon Corporation (Nasdaq: XON), a leader in synthetic
biology. Fibrocell manufactures clinical supply of FCX-007
and, if approved, plans to conduct future commercial manufacture of
FCX-007 at its cGMP manufacturing facility located in Exton,
Pennsylvania. Fibrocell’s multi-product gene therapy manufacturing
facility in Exton has sufficient vector supply to complete the
Company’s clinical trials and existing manufacturing capacity to
serve the U.S. market for RDEB. 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 Intrexon’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 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 for the treatment of
moderate to severe localized scleroderma, and is currently
enrolling the Phase 1 portion of a Phase 1/2 clinical trial.
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 StatementsThis 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
initiate a Phase 3 clinical trial for FCX-007 in the second quarter
of 2019; the expected trial design of DEFI-RDEB, and expectation to
enroll 15-20 patients therein; the timing of our Phase 1/2 clinical
trial of FCX-013, including our expectation to complete enrollment
of Phase 1 adult patients in the third quarter 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; the
Company’s belief that its cash and cash equivalents will be
sufficient to fund operations into the fourth quarter of 2019 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: Fibrocell has
not yet received the FDA’s official meeting minutes, and they may
differ materially from the Company’s understanding of the results
of the Type B 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 Intrexon; 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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