RXi Pharmaceuticals Announces Positive Results in its First Double Blind Study in Healthy Volunteers with RXI-109

RXi Pharmaceuticals Announces Positive Results in its First Double Blind Study
                      in Healthy Volunteers with RXI-109

RXI-109 was well tolerated and produced a statistically significant and dose
dependent reduction of Connective Tissue Growth Factor (CTGF), a protein that
causes abnormal scarring when it is over-expressed in a wound

PR Newswire

WESTBOROUGH, Mass., June 6, 2013

WESTBOROUGH, Mass., June 6, 2013 /PRNewswire/ --RXi Pharmaceuticals
Corporation (OTCQB: RXII), a biotechnology company focused on discovering,
developing and commercializing innovative therapies addressing major unmet
medical needs using RNA-targeted technologies, today announced the unblinded
results of the first of their 2 placebo-controlled double blind studies in
volunteers with their anti-scarring agent RXI-109, a proprietary sd-rxRNA®
compound that has been shown in vitro and in animals to reduce mRNA for
connective tissue growth factor (CTGF). Over-expression of this protein in
wounds has been associated with abnormal scarring as seen in hypertrophic
scars and keloids.

"Based on the animal data, we were confident that RXI-109 would not cause
significant side effects or toxicities in this first study, and we are pleased
to announce that confirmation today," said Dr. Geert Cauwenbergh, President
and CEO of RXi Pharmaceuticals. He added that: "It is remarkable and a great
outcome that we have also been able to get a good read-out on a key biomarker
for scarring in this study. Indeed, almost 3 months after a single intradermal
dose of RXI-109, histological comparisons between the drug- and
placebo-treated sites in the volunteers showed that RXI-109 significantly
reduced the expression of CTGF in the wound area in a dose dependent manner,
suggesting a potent and long lasting effect on this key biomarker for abnormal
scarring. We look forward to having the unblinded results of our second Phase
1 trial, with multiple doses of RXI-109, available for our Investor and
Analyst Symposium on July 12, 2013."

About the study

In this first Phase 1 study, 15 volunteers with no known predisposition for
abnormal scarring were treated in 5 cohorts. Each subject received a single
intradermal injection of RXI-109 in two small 2-cm areas of the abdomen. Two
identical areas on the other side of the abdomen were treated with placebo
injections. The next day, 24 hours after treatment, these 4 areas received an
incision that was immediately closed again. RXI-109 was given in dose
ascending manner to 5 cohorts, with doses ranging from 0.5 mg/cm to 5 mg/cm
(or total doses of RXI-109 per volunteer from 2 to 20 mg). Subjects were
followed intensively for side effects, safety and toxicity in the 7 days post
injection, and regularly afterwards for up to 3 months, at which point they
received an abdominoplasty. Samples were collected from the portion of the
skin that was removed during the surgery for histological and biomarker
assessments on scar area and CTGF. Various parameters were monitored for
systemic side effects: ECG, blood biochemistry, blood count, urinalysis,
assessments of the incisions by physician and subjects and measurement of
RXI-109 into the systemic circulation after intradermal injection. No
significant side effects or toxicity were observed and the incisions did not
appear to slow healing on the active or the placebo side, indicating that
RXI-109 does not delay wound closure. In addition, blood level measurements
for RXI-109 indicated that the maximum systemic exposure was only about 5% of
the intradermally administered dose. The histological evaluation at 3 months
after the single intradermal injection showed that the average wound area for
all sites (30 sites treated with RXI-109 and 30 sites treated with placebo)
was not different between both treatment arms (p = 0.22), confirming no
negative effect on wound healing for the drug. Interestingly, there was a
statistically significant reduction in the average CTGF protein concentration
in the RXI-109 treated sites compared to placebo treated sites (p = 0.02). In
addition the highest reduction in CTGF protein levels in the incision areas
was observed in the 2 highest dosed treatment cohorts. The noted effect on
CTGF protein level is remarkable given the 3 month time period between the
single injection and the skin tissue harvesting.

It is concluded from this study that an intradermal injection of RXI-109 is
well tolerated, devoid of systemic toxic effects and results in minimal uptake
in the systemic circulation. In addition the study shows no negative effects
on the wound healing process in individuals with a normal wound healing
pattern. Moreover, the observed effect of RXI-109 on the reduction of CTGF
protein in the incision area 3 months after a single intradermal injection of
RXI-109, as compared to placebo, suggests that the compound has a long lasting
effect on silencing this protein, with the highest treatment doses having a
more pronounced effect than the lower doses.

About RXi Pharmaceuticals Corporation

RXi Pharmaceuticals Corporation (OTCQB: RXII) is a biotechnology company
focused on discovering, developing and commercializing innovative therapies
based on its proprietary, self-delivering RNAi platform. Therapeutics that use
RNA interference, or "RNAi," have great promise because of their ability to
down-regulate, the expression of a specific gene that may be over-expressed in
a disease condition. 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, targets connective tissue growth
factor (CTGF) to reduce dermal scarring (fibrosis), entered into human
clinical trials in June 2012. For more information, please visit

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.

RXi Pharmaceuticals Corporation
Tamara McGrillen, 508-929-3646

SOURCE RXi Pharmaceuticals Corporation

Website: http://www.rxipharma.com
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