- Single ESS Treatment Provided Successful Skin Replacement on
Infant Patient, Supporting Natural Skin Growth That Required no
Additional Grafts or Reconstructive Surgery
Amarantus Bioscience Holdings, Inc. (OTCQX:AMBS), a
biotechnology company developing therapeutic and diagnostic product
candidates in orphan indications and neurology, reported on
preliminary 13-year longitudinal follow-up data presented on
Engineered Skin Substitute (ESS) for the treatment of pediatric
severe burns and Congenital Giant Hair Nevus at the Tissue Products
for Emergency Preparedness Symposium at the National Archives in
Washington, DC in May 2015.
Amarantus completed the acquisition of ESS in
July 2015, and has focused the Company’s near-term execution
strategy on the advancement of ESS in several therapeutic areas
including Congenital Giant Hairy Nevus and pediatric severe burns,
two rare pediatric diseases that the Company believes would be
eligible for a Rare Pediatric Disease Designation (RPDD) by the US
Food and Drug Administration (FDA). The Sponsor of a treatment
approved under the RPDD pathway is eligible to receive a PRV that
may be sold to other companies. Two PRVs have been sold in the last
6 months for an aggregate of $595 million to major pharmaceutical
companies. The proceeds from the sale of a PRV are in addition to
any potential revenues that could be generated from product sales.
The Company intends to apply to the FDA for both orphan drug
designation (ODD) and RPDD for ESS treatment of Congenital Giant
Hairy Nevus in the fourth quarter of 2015.
Congenital Giant Hairy Nevus is a rare pediatric
disease defined by one or more, large, darkly pigmented and
sometimes hairy patches with a projected adult diameter of over 40
cm with hypertrichosis. The condition is sometimes known as giant
congenital melanocytic nevus. The estimated prevalence for the
largest forms is 0.002% of births, affecting approximately 1 in
50,000 to 1 in 500,000 births annually. Melanocytic Nevi often grow
proportionally to the body size as the child matures. As they
mature, they often develop thickness, and become elevated, although
these features can also be present from birth. Prominent terminal
hairs often form, especially after puberty. With maturity, the
nevus can have variation in color, and the surface might be
textured with proliferative growths. Neurocutaneous melanosis is
associated with the presence of either giant congenital melanocytic
nevi or non-giant nevi of the skin. It is estimated that
neurocutaneous melanosis is present in 2% to 45% of patients with
giant congenital melanocytic nevi. Neurocutaneous melanosis is
characterized by the presence of congenital melanocytic nevi on the
skin and melanocytic tumors in the leptomeninges of the central
nervous system.1
CONGENITAL GIANT HAIRY NEVUS PUBLISHED DATA FOR
ESS
In the scientific publication Plastic and
Reconstructive Surgery. 2004;114(6):1523-1528 entitled “Autologous
cultured skin substitutes conserve donor autograft in elective
treatment of congenital giant melanocytic nevus,” the authors
reported on the treatment of two (2) cases of Congenital Giant
Hairy Nevus. ESS was previously known as Cultured Skin Subtitute
(CSS) or Permaderm™, a trademark Amarantus continues to own for the
product.
ABSTRACT
The above report presents two patients
having giant congenital nevi (400 to 650 cm 2) treated by excision
and grafting with autologous cultured skin substitutes prepared
from less than 20 cm2 of donor skin autograft. Nevi were excised to
fascia and grafted with cadaveric allograft for 1 week, followed by
removal of the allograft and grafting with cultured skin
substitutes. The mean ratio of closed to donor areas was 26. In
comparison to sheet split-thickness skin grafting, cultured skin
substitutes exhibited comparable cosmesis, pliability, and
durability while reducing the donor-site area by approximately one
order of magnitude.
PRELIMINARY 13-YEAR LONG-TERM FOLLOW-UP DATA
A copy of the public presentation made at the
Tissue Products for Emergency Preparedness Symposium at the
National Archives in Washington, DC in May 2015, was disclosed in a
regulatory filing under form 8-K by Amarantus on October 8th, 2015.
The presentation discloses pictures from an up-to 13-year
follow-up evaluation from one of the patients originally reported
on in the scientific publication noted above. The patient received
no reconstructive surgery after the initial ESS treatment. A link
to the presentation can be found at
http://www.sec.gov/Archives/edgar/data/1424812/000114420415058546/v421839_ex99-1.htm.
“The preliminary long-term follow-up data on the
engraftment of ESS for the treatment of Congenital Giant Hairy
Nevus provide a compelling rationale for the further evaluation of
ESS as potential revolutionary treatment across a broad swath of
pediatric dermatologic indications,” said Gerald E. Commissiong,
President & CEO of Amarantus. “Congenital Giant Hairy Nevus can
be a devastating condition affecting not only the physical, but
also the psychosocial maturation of newborns as they grow into
childhood. Any clinical benefit that could be provided in terms of
cosmesis outcomes will be heralded both by children and their
families. ESS represents a potential paradigm shift in this
indication, as not only is there potential initial cosmesis
benefit, but also a potential long-term cosmesis improvement, as
well as the potential reduction or elimination in reconstructive
procedures due to the unique properties of ESS growing with
pediatric patients – which distinguishes ESS from all other
available treatment options in the market or in development that we
are aware of. This property makes ESS potentially especially
beneficial for the pediatric dermatologic population which is in
dire need of a permanent full thickness skin replacement
options.”
Amarantus is also preparing to initiate a
multi-center, controlled clinical study of ESS for the treatment of
severe burns in the adult population, and has recently executed a
Collaborative Research And Development Agreement (CRADA) with the
U.S. Army to support the advancement of ESS for the treatment of
the military population that is being partially supported by a
grant from the Armed Forces Institute of Regenerative Medicine
(AFIRM). With the integration of the Cutanogen Corporation now
complete, Amarantus intends to vigorously pursue additional
non-dilutive funding from the Department of Health and Human
Services (HHS) and Biomedical Advanced Research and Development
Authority (BARDA) for disaster readiness for the further
development of ESS primarily for the treatment of thermal burns for
routine burn care and mass casualties.
The Company believes, the development of ESS for
the treatment of adult severe burns will provide additional proof
of concept for the commercial manufacture of ESS for the treatment
of pediatric severe burns and Congenital Giant Hairy Nevus. The
Company intends to achieve GMP manufacturing status for ESS late in
the fourth quarter of 2015. The Company also expects to initiate
the clinical study with the U.S. Army shortly thereafter in the
first quarter of 2016.
About Engineered Skin Substitute
(ESS)
Engineered Skin Substitute (ESS) is a
tissue-engineered skin prepared from autologous (patient's own)
skin cells. It is a combination of cultured epithelium with a
collagen-fibroblast implant that produces a skin substitute that
contains both epidermal and dermal components. This model has been
shown in preclinical studies to generate a functional skin barrier.
Most importantly, self-to-self skin grafts for autologous skin
tissue are less likely to be rejected by the immune system of the
patient, unlike with porcine or cadaver grafts in which immune
system rejection is a possibility. ESS has been used in an
investigator initiated clinical setting in over 130 human subjects,
primarily pediatric patients, for the treatment of severe burns up
to 95% total body surface area.
About Amarantus BioScience Holdings,
Inc.
Amarantus BioScience Holdings (OTCQX:AMBS) is a
biotechnology company developing treatments and diagnostics for
diseases in the areas of neurology and orphan diseases. The Company
has an exclusive worldwide license to intellectual property rights
associated to Engineered Skin Substitute (ESS), an orphan drug
designated autologous full thickness skin replacement product in
development for the treatment of adult severe burns currently
preparing to enter Phase 2 clinical studies. The Company is
currently evaluating human clinical data from previously conducted
studies in pediatric severe burns and Congenital Giant Hairy Nevus
to support clinical development expansion into those areas.
AMBS also has development rights to eltoprazine, a small molecule
currently in a Phase 2b clinical program for Parkinson's disease
levodopa-induced dyskinesia with the potential to expand into adult
ADHD and Alzheimer's aggression. AMBS owns the intellectual
property rights to a therapeutic protein known as
mesencephalic-astrocyte-derived neurotrophic factor (MANF) and is
developing MANF as a treatment for orphan ophthalmic disorders,
initially in retinitis pigmentosa (RP) and retinal artery occlusion
(RAO). AMBS also owns the discovery of neurotrophic factors
(PhenoGuard™) that led to MANF's discovery.
AMBS' Diagnostics division owns the rights to
MSPrecise®, a proprietary next-generation DNA sequencing (NGS)
assay for the identification of patients with relapsing-remitting
multiple sclerosis (RRMS) at first clinical presentation, has an
exclusive worldwide license to the Lymphocyte Proliferation test
(LymPro Test®) for Alzheimer's disease, which was developed by
Prof. Thomas Arendt, Ph.D., from the University of Leipzig, and
owns intellectual property for the diagnosis of Parkinson's disease
(NuroPro).
For further information please visit
www.Amarantus.com, or connect with the Company on Facebook,
LinkedIn, Twitter and Google+.
Forward-Looking Statements
Certain statements, other than purely historical
information, including estimates, projections, statements relating
to our business plans, objectives, and expected operating results,
and the assumptions upon which those statements are based, are
forward-looking statements. These forward-looking statements
generally are identified by the words "believes," "project,"
"expects," "anticipates," "estimates," "intends," "strategy,"
"plan," "may," "will," "would," "will be," "will continue," "will
likely result," and similar expressions. Forward-looking statements
are based on current expectations and assumptions that are subject
to risks and uncertainties which may cause actual results to differ
materially from the forward-looking statements. Our ability to
predict results or the actual effect of future plans or strategies
is inherently uncertain. Factors which could have a material
adverse effect on our operations and future prospects on a
consolidated basis include, but are not limited to: changes in
economic conditions, legislative/regulatory changes, availability
of capital, interest rates, competition, and generally accepted
accounting principles. These risks and uncertainties should also be
considered in evaluating forward-looking statements and undue
reliance should not be placed on such statements.
1 https://en.wikipedia.org/wiki/Congenital_melanocytic_nevus
Investor and Media Contact:
Ascendant Partners, LLC
Fred Sommer
732- 410-9810
fred@ascendantpartnersllc.com
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