MARLBOROUGH, Mass.,
Oct. 20, 2015 /PRNewswire/
-- RXi Pharmaceuticals Corporation (NASDAQ: RXII), a
biotechnology company focused on discovering and developing
innovative therapies primarily in the areas of dermatology and
ophthalmology today announced positive results from a collaboration
with Biogazelle NV. Biogazelle is a company with a
longstanding track record and internationally recognized expertise
in RNA gene expression analysis, with a particular focus on
non-coding RNA, such as microRNAs and long non-coding RNAs
(lncRNA).
Specifically-designed sd-rxRNAs based on RXi's proprietary,
self-delivering RNAi platform demonstrated robust and potent
reduction of the levels of lncRNAs in a target specific manner.
These findings expand the number of potential genes RXi is capable
of targeting by approximately four fold.
Dr. Craig Mello, Chairman of RXi
Pharmaceuticals' Scientific Advisory Board and co-recipient of the
2006 Nobel Prize in Medicine for RNAi, said that, "This discovery
is a significant advancement for RXi's proprietary self-delivering
RNA platform. lncRNAs represent a diverse class of promising
therapeutic targets. Currently, researchers must use multiple
oligonucleotide platforms to reduce expression of lncRNAs depending
on the subcellular localization of the target. Biogazelle's
findings suggest that RXi's technology can efficiently silence
lncRNAs regardless of their subcellular localization."
lncRNAs are a diverse class of non-protein coding RNA molecules
that are greater than 200 nucleotides in length. lncRNAs are
involved in crucial cellular processes, including the regulation of
gene expression, and have been implicated in many diseases and
disorders, including cancer, cardiovascular diseases, neurological
disorders, diabetes, and HIV.
"When we began the collaboration with RXi, we were intrigued by
their self-delivering RNAi technology, which offers a clear
advantage over conventional siRNAs. Many lncRNAs are believed to be
primarily located in the nucleus and considering siRNAs are not
expected to reduce the expression of nuclear lncRNAs, we did not
believe the platform would be competitive with other nucleic
acid-based technologies, such as antisense oligonucleotides," said
Professor Jo Vandesompele, CSO of
Biogazelle. He further added that, "Remarkably, the sd-rxRNA
platform demonstrated potent and target-specific silencing of
multiple lncRNAs, including lncRNAs that are strictly localized in
the nucleus, such as MALAT1. We're considering RXi's
self-delivering platform for therapeutic applications in the
future. "
"We greatly appreciate the thorough work that Biogazelle has
done in screening our sd-rxRNA compounds against lncRNA targets and
we look forward to working with Biogazelle in the future," said Dr.
Geert Cauwenbergh, President and CEO
of RXi Pharmaceuticals. He added that, "These results constitute a
major boost to the value of our self-delivering platform by
significantly expanding the breadth of therapeutic targets we can
now pursue. This opens up additional business development and
partnering opportunities with our sd-rxRNA platform, further
supporting RXi's initiatives to build shareholder value."
About lncRNA
Following the sequencing of the mammalian genome, ~20,000
protein-coding genes were identified. At that time 99% of the
genome was thought to contain non-functional and repetitive
sequences. More recently, researchers utilizing transcriptome
profiling approaches have discovered that ~60,000 of these
non-functional sequences of the genome are transcribed into long
non-coding RNAs (lncRNAs), many of which are functional. Long
non-coding RNAs, containing >200 nucleotides, were found to
function in various biological processes, such as cell
proliferation, differentiation and the regulation of gene
expression. Importantly, dysregulation of lncRNA expression has
been shown to be associated with the progression of many diseases
including cancer, cardiovascular diseases, neurological disorders,
diabetes and HIV. Therefore, targeting specific lncRNAs may
result in therapeutic benefit.
About sd-rxRNA®: Novel RNAi Compounds with Built-In
Drug-Like Properties
Scientists at RXi have used an alternative approach to delivery,
where drug-like as well as delivery properties are built into the
RNAi compound itself. sd-rxRNA compounds are novel RNAi compounds
with enhanced properties for therapeutic use including efficient
spontaneous cellular uptake, stability in vivo, reduced
potential for immune stimulation, and potent, long-lasting
intracellular activity. All cell types tested (primary,
neuronal and non-adherent) internalize sd-rxRNA compounds uniformly
and efficiently, resulting in effective silencing. Efficient
cellular uptake is observed in vitro and, following direct
administration in vivo, into tissues such as skin, retina,
lung, and spinal cord.
About RXi Pharmaceuticals Corporation
RXi Pharmaceuticals Corporation (NASDAQ: RXII) is a
biotechnology company focused on discovering and developing
innovative therapeutics primarily in the areas of dermatology and
ophthalmology that address high-unmet medical needs. Our discovery
and clinical development programs are based on siRNA technology as
well as immunotherapy agents. These compounds include, but are not
limited to, our proprietary, self-delivering RNAi (sd-rxRNA®)
compounds for the treatment of dermal and ocular scar formation. It
also includes an immunomodulator, Samcyprone™, a proprietary
topical formulation of diphenylcyclopropenone (DPCP), for the
treatment of disorders such as warts, alopecia areata,
non-malignant skin tumors and cutaneous metastases of melanoma.
Building on the pioneering work of RXi's Scientific Advisory
Board Chairman and Nobel Laureate Dr. Craig
Mello, the Company's first RNAi product candidate, RXI-109
(an sd-rxRNA compound) is the Company's first clinical development
candidate. RXI-109 silences Connective Tissue Growth Factor (CTGF),
which plays a key role in tissue regeneration and repair and is
initially being developed to reduce or inhibit scar formation in
the skin and in the eye. RXI-109 is currently being evaluated in
Phase 2a clinical trials in dermatology and a Phase 1/2 trial is
planned to initiate this year in ophthalmology.
RXi's robust pipeline, coupled with an extensive patent
portfolio, provides for product and business development
opportunities across a broad spectrum of therapeutic areas. We are
committed to being a partner of choice for academia, small
companies, and large multinationals. We welcome ideas and proposals
for strategic alliances, including in- and out-licensing
opportunities, to advance and further develop strategic areas of
interest. Additional information may be found on the Company's
website, www.rxipharma.com.
About Biogazelle
Biogazelle is a Ghent University spin-off founded in 2007 by
Professor Jo Vandesompele and
Dr. Jan Hellemans. It is focused on
the development of RNA-targeted therapeutics and has proprietary
knowledge to prioritize candidate therapeutic RNA targets in
specific cancer types, building on years of expertise in exploring
the human transcriptome. Therapeutic inhibition of candidate lncRNA
targets are evaluated in high-throughput in vitro RNA perturbations
and in preclinical animal models.
Biogazelle also offers expert laboratory services (with ISO
17025 accreditation) in the area of RNA, biomarker discovery and
diagnostics.
Being at the forefront of the genomics revolution, Biogazelle is
convinced that unraveling the coding and non-coding regions of the
genome will lead to RNA-targeted therapeutics and biomarkers for
better healthcare.
More information can be found on their website at
www.biogazelle.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: our ability to successfully develop RXI-109, Samcyprone™ and
our other product candidates (collectively "our product
candidates"); the future success of our clinical trials with our
product candidates; the timing for the commencement and completion
of clinical trials; our ability to enter into strategic
partnerships and the future success of these strategic
partnerships; and our ability to deploy our sd-rxRNA® technology
through partnerships, as well as the prospects of these
partnerships to provide positive returns. Forward-looking
statements about expectations and development plans of RXi's
product candidates and partnerships involve significant risks and
uncertainties, including the following: risks that we may not be
able to successfully develop and commercialize our product
candidates; risks that product development and clinical studies may
be delayed, not proceed as planned and/or be subject to significant
cost over-runs; risks related to the development and
commercialization of products by competitors; risks related to our
ability to control the timing and terms of collaborations with
third parties; and risks that other companies or organizations may
assert patent rights preventing us from developing or
commercializing our product candidates. Additional risks are
detailed in our most recent Annual Report on Form 10-K and
subsequent Quarterly Reports on Form 10-Q under the caption "Risk
Factors." Readers are urged to review these risk factors and
to not act in reliance on any forward-looking statements, as actual
results may differ from those contemplated by our 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.
Contact
RXi Pharmaceuticals Corporation
Tamara McGrillen
508-929-3646
tmcgrillen@rxipharma.com
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