MARLBOROUGH, Mass.,
May 2, 2014 /PRNewswire/ -- RXi
Pharmaceuticals Corporation (NASDAQ: RXII), a biotechnology company
focused on discovering, developing and commercializing innovative
therapies addressing major unmet medical needs using RNA-targeted
technologies, today announced results from the assessment of
connective tissue growth factor (CTGF) protein levels following
intravitreal injection, of RXI‑109 in the eyes of cynomolgus
monkeys, as part of a dose-range finding study to build the
Company's ophthalmology franchise.
CTGF protein levels were determined by immunohistochemistry
methods seven days following injection and quantified by digital
image analysis of stained slides. Intravitreal administration of
RXI‑109 resulted in a reduction of CTGF protein levels in a
dose-dependent manner in the retina. Whole eye sections were
collected in this study and the Company was also able to determine
CTGF protein levels in the cornea, and noted that these protein
levels were also reduced in a dose-dependent manner in the cornea
tissue.
"These remarkable results constitute a major boost to the value
of our newly formed ophthalmology franchise," said Dr. Geert Cauwenbergh, President and CEO of RXi
Pharmaceuticals. He added that, "Not only do these data confirm
that RXI‑109 lowers CTGF protein levels in a dose dependent manner
in the retina of non-human primates, but it also demonstrates that,
with an intravitreal injection of RXI‑109, sufficient compound
migrates to the cornea to lower CTGF protein levels in that eye
tissue as well. This finding opens up an avenue to possibly develop
topical forms of RXI‑109 to combat corneal scarring which often
occurs secondary to trauma or infection and can lead to visual
impairment, including blindness."
About Retinal Scarring
Retinal scarring is the development of scar tissue on the retina
as a result of injury or as a consequence of disease. During injury
many critical cellular pathways become active including
proliferation, adhesion, migration, angiogenesis and scar tissue
remodeling. CTGF modulates signals from a wide range of factors in
the cell and in this way impacts some of these critical cellular
pathways. Overproduction of CTGF is implicated in fibrotic disease
and scarring in many tissues including the retina. Specifically,
CTGF has been found to be upregulated in proliferative
vitreoretinopathy membranes of both human and rabbit (He, Chen et
al., 2008) and has been shown to be expressed by Müller cells and
RPE cells, two cell types that undergo proliferation in the
presence of injury and contribute to retinal scarring.
About Corneal Scarring
The cornea is a transparent tissue at the front of the eye that
plays two key roles in the health of the eye. First, it provides a
physical barrier to protect the inner eye from environmental
factors. Second, the cornea is instrumental in directing incoming
light to the lens and retina. Scarring of the cornea resulting from
injury, disease, or vision correction surgery can have a dramatic
impact on vision. In some cases, a corneal transplant may be
needed. Importantly, CTGF expression levels have been found to be
increased during corneal wound healing in rat corneas and in human
corneal fibroblasts and it has been proposed that a reduction of
CTGF may be an important step towards reducing corneal scarring
(Blalock et al., 2003).
About RXI-109
RXI-109 is an sd-rxRNA® that targets the mRNA of connective
tissue growth factor (CTGF), a gene known to modulate fibrosis and
scar formation. RXI-109 is initially being developed to reduce or
inhibit scar formation in the skin following surgery. The
first clinical trials with RXI‑109 (RXI‑109‑1201 and RXI‑109‑1202)
showed excellent safety and tolerability, with ascending single and
multiple doses respectively, as well as dose dependent effects on
the CTGF protein and on the mRNA that controls production of this
protein.
Two Phase 2 clinical trials are currently underway to evaluate
the effectiveness and safety of RXI‑109 on the outcome of scar
revision surgeries performed on hypertrophic scars resulting from
lower abdominal surgeries (RXI‑109‑1301) and to evaluate the
effectiveness and safety RXI‑109 in healthy subjects who undergo an
elective surgical excision of two similarly sized and placed
keloids (RXI‑109‑1401).
About RXi Pharmaceuticals Corporation
RXi Pharmaceuticals Corporation (NASDAQ: RXII) is a
biotechnology company focused on discovering, developing and
commercializing innovative therapies based on its proprietary,
self-delivering RNAi (sd-rxRNA®) platform. Therapeutics that use
RNA interference, or "RNAi," have great promise because of
their ability to down-regulate the expression of specific genes
that may be over-expressed in disease conditions. Building on the
pioneering work of scientific founder and Nobel Laureate Dr.
Craig Mello, a member of the RXi
Scientific Advisory Board, RXi's first RNAi product candidate,
RXI‑109, entered into human clinical trials in June 2012 and is currently conducting Phase 2
clinical trials. RXI‑109 targets connective tissue growth
factor (CTGF) to reduce dermal scarring (fibrosis). RXi's
sd‑rxRNA oligonucleotides are designed for therapeutic use and have
drug-like properties, such as high potency, target specificity,
serum stability, reduced immune response activation, and efficient
cellular uptake. These hybrid oligonucleotide molecules combine the
beneficial properties of conventional RNAi and antisense
technologies. This allows sd‑rxRNAs to achieve efficient
cellular uptake and potent, long-lasting intracellular activity.
For more information, please visit www.rxipharma.com.
Forward-Looking Statements
This press release contains forward-looking statements within
the meaning of the Private Securities Litigation Reform Act of
1995. Such statements include, but are not limited to, statements
about future expectations, planned and future development of RXi
Pharmaceuticals Corporation's products and technologies.
Forward-looking statements about expectations and development plans
of RXi's products involve significant risks, and uncertainties:
risks that RXi may not be able to successfully develop its
candidates, or that development of RNAi-based therapeutics may be
delayed or not proceed as planned, or that we may not develop any
RNAi-based product; risks that the development process for our
product candidates may be delayed, risks related to development and
commercialization of products by our competitors, risks related to
our ability to control timing and terms of collaborations with
third parties, and the possibility that other companies or
organizations may assert patent rights preventing us from
developing our products. Actual results may differ from those
contemplated by these forward-looking statements. RXi does not
undertake to update forward-looking statements to reflect a change
in its views, events or circumstances that occur after the date of
this release.
References
He, S., Y. Chen, et al. "Connective tissue growth factor as a
mediator of intraocular fibrosis." Invest Ophthalmol Vis Sci
2008; (9): 4078-88.
Blalock, T.D., M. R. Duncan, et
al. "Connective tissue growth factor expression and action in human
corneal fibroblast cultures and rat corneas after photorefractive
keratectomy." Invest Ophthalmol Vis Sci 2003; 44 (5):
1879-87.
Contacts
RXi Pharmaceuticals Corporation
Tamara McGrillen, 508-929-3646
tmcgrillen@rxipharma.com
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SOURCE RXi Pharmaceuticals Corporation