Arisaph Pharmaceuticals Reports Positive Results From Two Safety Clinical Trials For Its Niacin Analog (ARI-3037MO)

  Arisaph Pharmaceuticals Reports Positive Results From Two Safety Clinical
                  Trials For Its Niacin Analog (ARI-3037MO)

PR Newswire

BOSTON, Nov. 7, 2012

BOSTON, Nov. 7, 2012 /PRNewswire/ --

  oARI-3037MO was well tolerated in single and repeat ascending dose trials
    in healthy volunteers
  oARI-3037MO did not provoke flushing or any other treatment-related adverse
    effects in 58 healthy male and female volunteers
  oARI-3037MO was well absorbed orally and plasma levels demonstrated good
    dose proportionality
  oARI-3037MO showed encouraging lipid changes in both clinical trials in
    healthy subjects

Arisaph Pharmaceuticals, Inc. announced today during an oral presentation at
the American Heart Association (AHA) meeting in Los Angeles, CA that its
niacin analog, ARI-3037MO, was extremely well tolerated in a single-ascending
dose (SAD) and a multiple-ascending dose (MAD) trial in healthy male and
female volunteers. The results showed that ARI-3037MO did not provoke
flushing or any other adverse skin changes, nor did it cause increases in
liver enzymes or blood glucose at doses up to 6 grams per day. In both
clinical trials, ARI-3037MO also demonstrated encouraging lipid effects. Based
on these promising results, ARI-3037MO potentially represents a
transformational, new niacin-like option for treatment of patients with
dyslipidemia, especially in the setting of metabolic syndrome, a disorder that
afflicts as many as 70 million Americans^1.

(Logo: http://photos.prnewswire.com/prnh/20110321/NE69091LOGO )

"The absence of flushing and other niacin induced adverse events highlights a
differentiated safety profile for our novel niacin analog," said Christopher
P. Kiritsy, Co-Founder, President and Chief Executive Officer of Arisaph
Pharmaceuticals. "Moreover, the evidence of lipid changes in such short
duration trials in healthy volunteers shows that our preclinical efficacy data
are translating and breeds optimism for the development of our best-in-class
niacin analog."

In two placebo controlled, safety and pharmacokinetic trials (SAD and MAD), 58
healthy male and female volunteers were given either single escalating doses
of ARI-3037MO (up to 6 grams per day) or repeat escalating doses of ARI-3037MO
(up to 3.5 grams per day) for 14 days. In both trials, ARI-3037MO was given
once daily without a titration scheme. The results showed that ARI-3037MO was
extremely well tolerated with no treatment-related adverse events.
Specifically, treatment with ARI-3037MO did not show any evidence of flushing
or adverse skin changes, such as itching or rash, using a validated
visual-analog scale. Additionally, no deleterious changes in liver enzymes or
blood glucose were observed with ARI-3037MO, even when given at high doses in
a repeat-dose setting.

The pharmacokinetics of ARI-3037MO revealed that the drug was well absorbed
orally and showed good dose proportionality. In both clinical trials,
ARI-3037MO produced encouraging lipid signals. For example, a single dose of 6
grams of ARI-3037MO produced a 15% increase in HDL and attenuated the
postprandial increase in serum triglycerides (TGs). In the MAD trial, the 2g
and 3.5g doses of ARI-3037MO decreased LDL-cholesterol (LDL-C) from baseline
and improved post-prandial triglycerides by more than 30% compared with
placebo. Such lipid trends are encouraging considering that the trials were
short-duration, safety and pharmacokinetic studies in healthy volunteers and
would not be expected to show pharmacodynamic effects.

"Niacin's efficacy across a broad range of lipid risk factors has long been
recognized but flushing and increases inblood glucose have limited its use,
particularly in high risk metabolic syndrome patients," commented Dr. Ernst
Schaefer, Professor of Medicine, Tufts University School of Medicine. "The
results from these two safety trials show that ARI-3037MO is devoid of such
dose-limiting side effects, resulting in a significantly improved safety and
tolerability profile compared with other niacin-based therapies."

In preclinical studies presented at the AHA in 2011, ARI-3037MO showed robust
changes in lipids, including LDL-C, HDL-C and triglycerides. For example,
once-daily treatments of ARI-3037MO lowered LDL-C in excess of 50% and lowered
TGs by more than 70% compared with placebo in high-fat-fed hamster models.
Additionally, non-clinical toxicology studies conducted in dogs and rats with
ARI-3037MO showed that high exposure multiples could be achieved without
serious adverse events, even at doses greater than 1000 mg per kilogram. The
favorable preclinical safety and efficacy data reveal that ARI-3037MO has an
extremely wide therapeutic index, indicating that ARI-3037MO will not be dose
limited by adverse events. Consequently, it is expected that patients will be
able to achieve improved efficacy observed with higher doses of niacin. 

About Niacin Analog Program:
The natural B vitamin niacin is a well-known lipid lowering agent, which has
been used to treat mixed lipid disorders for over 50 years. Niacin is a
first-line drug for the treatment of hyperlipidemia, and it is used in
combination with statins to further reduce LDL cholesterol and triglycerides
or to increase HDL cholesterol in patients with depressed HDL cholesterol
levels. Niacin can produce changes in HDL cholesterol up to 35%, lower LDL
cholesterol up to 25% and lower triglycerides up to 50%. Despite niacin's
broad lipid altering profile, the use of niacin-based therapies is diminished
because of unpleasant side effects, particularly flushing. Flushing,
representative of tingling and/or redness of the skin, is the principal side
effect of niacin, and in some patients it can be extremely uncomfortable.
Arisaph has developed a novel class of structural analogs of niacin that are
expected to demonstrate a dramatically improved safety and tolerability
profile while retaining the beneficial lipid effects associated with niacin.

About Arisaph
Arisaph Pharmaceuticals Inc, a drug discovery, biopharmaceutical company
located in Boston, Massachusetts, was founded by Dr. William Bachovchin
(Professor of Biochemistry, Tufts University School of Medicine), Christopher
Kiritsy (Former EVP Corporate Development and CFO, Kos Pharmaceuticals, Inc.)
and Michael Jaharis (Founder, Chairman Emeritus, Kos Pharmaceuticals, Inc.) to
develop differentiated therapies for cardiometabolic diseases and cancer.
Arisaph has developed a rich pipeline of products at various stages of
development, including a niacin analog (ARI-3037MO), in phase 2 clinical
development, and a small molecule immune modulator for the treatment of
cancer. The Company's vision is to create a fully integrated pharmaceutical
company, leveraging its drug discovery expertise to develop transformational,
patent protected, medicines that offer distinct safety, efficacy and/or
tolerability benefits compared with existing therapies for large markets,
whose needs are not being fully met by current therapeutics.

Certain statements in this press release, including statements regarding the
Company's research and development effort, the Company's expectation to
initiate or complete human clinical studies, the Company's ability to finance
its development programs into human clinical testing, and the Company's
ability to successfully capitalize on the early stage research are subject to
risks and uncertainties. These risks and uncertainties include risks and
uncertainties related to: our ability to discover and develop new compounds
and products using a novel approach to drug discovery; the early stage of all
of our discovery and development efforts; our ability to complete preclinical
and clinical development of our products; our ability to obtain and maintain
regulatory approvals for our products; competition from other technologies and
technologies similar to ours; obtaining, maintaining and protecting
intellectual property utilized by our products; changes in legislation and
regulations affecting our products and potential product candidates; our need
to obtain additional funding to support our business activities; our
dependence on collaborators and other third parties for development,
manufacture, marketing, sales and distribution of products; the ability of our
licensees to achieve developmental, regulatory and other milestones and to
commercialize their products; the effect of conditions in the pharmaceutical
industry and the economy in general, as well as certain other risks and
uncertainties.

References:

1. NHANES Data, National Health Statistics Report, May 2009

  Contact: Arisaph Pharmaceuticals, Inc.
           Christopher Kiritsy
           President and CEO
           (617) 986-4500

SOURCE Arisaph Pharmaceuticals, Inc.

Website: http://www.arisaph.com