MARLBOROUGH, Mass.,
Sept. 11, 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 that the Company's President
& CEO, Dr. Geert Cauwenbergh,
presented at the Rodman & Renshaw 16th Annual Global
Investment Conference on Wednesday,
September 10, 2014. Dr. Cauwenbergh provided early
interim data from their first Phase 2a clinical trial
(RXI-109-1301) as well as other advancements in the Company's
dermatology and ophthalmology franchises.
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Clinical Trial Update
In the Company's first Phase 2a trial (RXI-109-1301), eight
patients in each of two cohorts were enrolled and have reached the
one month post-surgery observation point. Because this trial
was designed with an adaptive protocol, it provides the ability to
review early data and optimize future cohorts and/or trials.
While one month is an early time point to evaluate scar
formation, assessments of 1-month post surgery photographs by 11
blinded evaluators suggest that delayed treatment with RXI-109
(Cohort 2) is better than immediate treatment (Cohort 1). In
Cohort 2 only, there was a statistical preference for RXI-109
treated scars by both comparative observations (RXI-109 treated-
vs. placebo-treated scars) and by evaluation of the scars using a
visual analog scale. Based on these early observations, the
dosing regimen in this study can be fine-tuned and we can more
rapidly move forward to an optimized treatment for prevention of
scarring after surgery. Additional results to date show that
RXI-109 is well tolerated with no systemic side effects.
Local effects are mild and similar to those seen in the Company's
prior Phase 1 clinical trial (erythema, occasional burning or
stinging sensation). Evaluation of the 3, 6 and 9 month time
points in this and the other ongoing Phase 2a trials will provide
continued insight into dosing regimen, and give further guidance
for future study design.
"Our progress in the past few months has been remarkable, both
in advancing our RXI-109 clinical program and in expanding the
development potential within our dermatology and ophthalmology
franchises," said Dr. Geert
Cauwenbergh, President and CEO. He added that, "The
interim findings in the first Phase 2a trial with RXI-109 are of
significant help in accelerating the evaluation of the best
treatment regimen for patients with hypertrophic scars, allowing
for additional patients to be included in cohorts with a more
effective dosing regimen and saving time and costs by eliminating
less optimal treatment schedules."
New Targets for sd-rxRNA® in Dermatology
The Company also announced that collagenase and tyrosinase were
selected as new discovery stage targets for their self-delivering
platform. For each of these two targets, we have identified potent
sd-rxRNA compounds for further evaluation. These two targets are
important for various diseases in skin. e.g., photo aging, wound
healing, hyperpigmentation as well as in other disease areas
(including osteo- and rheumatoid arthritis, acne scarring,
blistering skin disorders, corneal erosions and endometriosis and
certain neurological cancers).
Ophthalmology Franchise Update
As part of the ongoing ophthalmology program, the Company filed
a request for a pre-IND meeting with the FDA in advance of filing
an IND for RXI-109 for diseases in the eye. IND-supporting
toxicology studies are in progress to support RXI-109 being
developed as a treatment for ocular scarring.
In addition, the Company announced that a potent anti-VEGF
sd-rxRNA has been identified, which may be considered for
development in age-related macular degeneration (AMD) alone or in
combination with RXI-109 to block both the neovascular and the
scarring component of AMD. The VEGF compound is based in
part on the target sequence of Bevasiranib, the siRNA compound
acquired along with additional RNAi assets from OPKO Health,
Inc.
About RXI-109 Clinical Trials
RXi Pharmaceuticals' first clinical program involves RXI‑109, an
sd-rxRNA compound, developed for the reduction of dermal scar
formation. RXI‑109 is designed to reduce the expression of
connective tissue growth factor (CTGF), a critical regulator of
biological pathways involved in fibrosis, including scar formation
in the skin. 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, as well as dose-dependent
effects on the CTGF protein and on the mRNA that controls
production of this protein.
In November 2013, the Company
started its first Phase 2a study (RXI-109-1301) in patients who had
pre-existing hypertrophic scars present on their lower abdomen for
at least one year. In that study, the patients undergo scar
revision surgery, after which they are treated with RXI-109 on one
end of the scar and placebo on the opposite end of the scar.
In April of this year, the Company began its second Phase 2a study
(RXI-109-1401) for RXI-109 treatment to prevent recurrence of
keloids in patients undergoing keloidectomy (removal of keloid).
Patients with two keloids of similar size and location are eligible
for the study. After keloidectomy, the lesions are closed and one
is treated with RXI-109, and the other is treated with placebo. As
is the case for the study in hypertrophic scars, patients will be
followed for several months (clinically and with photographs) after
the end of treatment.
The Company's third Phase 2a study (RXI-109-1402) was initiated
in July 2014 for RXI-109 for the
reduction of recurrence of hypertrophic scars following elective
scar revision surgery. In this study, patients with either one long
hypertrophic scar, or two scars comparable in length, anatomical
location and characteristics, are eligible to receive scar revision
surgery. For a single scar, a portion of the revised scar
segment will be treated with RXI-109 and a comparably sized length
on the opposite end of the excised scar segment will be left
untreated. If two scars are revised, one revised scar segment will
be treated with RXI-109 and one scar will be left untreated after
revision surgery. This third Phase 2a study will follow patients
for nine months. Investigator and independent reviewer assessments
will be used to evaluate the effectiveness of RXI-109 in preventing
scar formation. Reviewers will evaluate and compare the appearance
of the revised areas after treatment with RXI-109 or when left
untreated.
All three Phase 2a trials incorporate a within-subject
comparison of revised sites treated with RXI-109 vs. control
sites. This is a powerful study design because it decreases
the potential impact of variability due to patient-to-patient
healing characteristics.
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, a self-delivering RNAi compound (sd-rxRNA®), entered into
human clinical trials in June 2012
and is currently being evaluated in Phase 2 clinical trials to
reduce the formation of dermal scars (fibrosis). RXI-109 is
designed to reduce the expression of connective tissue growth
factor (CTGF), a critical regulator of biological pathways involved
in fibrosis, including scar formation in the skin. 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: the risk that we may not be able to
successfully develop our 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 products; risks that the
development process for our product candidates may be delayed,
risks related to the development and commercialization of products
by our competitors, the risk related to our ability to control the
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.
Contact
RXi Pharmaceuticals Corporation
Tamara McGrillen
508-929-3646
tmcgrillen@rxipharma.com
SOURCE RXi Pharmaceuticals Corporation