Santaris Pharma A/S Advances a Second Drug From Its Cardiometabolic Program, SPC4955, Inhibiting apoB, into Phase 1 Clinical Trials for the Treatment of High Cholesterol

By Santaris Pharma As, PRNE
Tuesday, May 10, 2011

Phase 1 clinical study to assess safety and tolerability of SPC4955, a drug inhibiting synthesis of apolipoprotein B (apoB), a major protein component involved in the formation of LDL-C or "bad" cholesterol

HOERSHOLM, Denmark and SAN DIEGO, May 11, 2011 - Santaris Pharma A/S, a clinical-stage biopharmaceutical company focused
on the research and development of mRNA and microRNA targeted therapies,
today announced that it has advanced a second drug from its cardiometabolic
program, SPC4955 into Phase 1 clinical trials, for the treatment of high
cholesterol. Developed using Santaris Pharma A/S proprietary Locked Nucleic
Acid (LNA) Drug Platform, SPC4955 is a mRNA-targeted drug candidate that
inhibits apolipoprotein B (apoB), a major protein component involved in the
formation of low density lipoprotein cholesterol (LDL-C) or "bad"
cholesterol.

(Logo: photos.prnewswire.com/prnh/20100111/SPLOGO)

Cholesterol is an essential component of all cells and several important
hormones, but cholesterol levels that are out of balance or too high overall
lead to the formation of atherosclerotic plaques that cause cardiovascular
diseases such as heart attacks or strokes. According to the World Health
Organization, cardiovascular disease is the number one cause of death
globally and high cholesterol is estimated to cause 18% of strokes and 56% of
heart disease globally(1).

The randomized, dose-escalation, double-blind, placebo-controlled Phase 1
study will assess safety, tolerability, pharmacokinetics, and
pharmacodynamics of SPC4955. The study aims to enroll 30 healthy volunteers
who will be randomized to receive either subcutaneous injections of SPC4955
or placebo. In preclinical studies, SPC4955 potently and dose-dependently
inhibited the synthesis of apoB resulting in significant and durable
reductions in plasma levels of LDL-C and triglycerides(2).

Last week, Santaris Pharma A/S announced it had advanced another drug
candidate, SPC5001, a mRNA- targeted drug inhibiting the synthesis of
Proprotein Convertase Subtilisin/Kexin type 9 (PCSK9), for the treatment of
high cholesterol into Phase 1 clinical trials. PCSK9 is a protein involved in
removing low density lipoprotein cholesterol (LDL-C) or "bad" cholesterol
from the bloodstream.

"Advancing two drug candidates, SPC4955 and SPC5001, in the space of only
a week from our cardiometabolic program into the clinic is a testament to the
efficiency of our LNA Drug Platform and Drug Discovery Engine capabilities
and represents an important step for patients as multiple approaches are
needed to lower high cholesterol levels," said Arthur A. Levin, PhD, Vice
President and Chief Development Officer of Santaris Pharma A/S. "SPC4955 aims
to inhibit the production and export of LDL-C in the liver while our other
clinical candidate SPC5001, increases the clearance of LDL from the blood.
Working on two distinct compounds with different target pathways allows the
company to address the broad spectrum of patients trying to control their
high cholesterol levels."

In addition to its cardiometabolic programs, in September 2010, Santaris
Pharma A/S successfully advanced miravirsen, a lead microRNA drug candidate
targeting miR-122, into Phase 2 studies for the treatment of patients
infected with the Hepatitis C virus.

The LNA Drug Platform and Drug Discovery Engine developed by Santaris
Pharma A/S combines the Company's proprietary LNA chemistry with its highly
specialized and targeted drug development capabilities to rapidly deliver
potent single-stranded LNA-based drug candidates for a range of diseases
including metabolic disorders, infectious and inflammatory diseases, cancer
and rare genetic disorders.

With its partners, Santaris Pharma A/S also has a robust product pipeline
consisting of mRNA and microRNA drug discovery and development
collaborations. These include partnerships with Pfizer, Inc. (delivery of
lead candidates against up to 20 targets), miRagen Therapeutics
(cardiovascular diseases), Shire plc (rare genetic disorders),
GlaxoSmithKline (four viral disease drug candidates) and Enzon
Pharmaceuticals (eight cancer targets successfully delivered - three are now
in Phase 1 clinical studies).

About Locked Nucleic Acid (LNA) Drug Platform

The LNA Drug Platform and Drug Discovery Engine developed by Santaris
Pharma A/S combines the Company's proprietary LNA chemistry with its highly
specialized and targeted drug development capabilities to rapidly deliver
LNA-based drug candidates against RNA targets, both mRNA and microRNA, for a
range of diseases including cardiometabolic disorders, infectious and
inflammatory diseases, cancer and rare genetic disorders. LNA-based drugs are
a promising new class of therapeutics that are enabling scientists to develop
drug candidates to work through previously inaccessible clinical pathways.
The LNA Drug Platform overcomes the limitations of earlier antisense and
siRNA technologies to deliver potent single-stranded LNA- based drug
candidates across a multitude of disease states. The unique combination of
small size and very high affinity allows this new class of drugs candidates
to potently and specifically inhibit RNA targets in many different tissues
without the need for complex delivery vehicles. The most important features
of LNA-based drugs include excellent specificity providing optimal targeting;
increased affinity to targets providing improved potency; and favorable
pharmacokinetic and tissue-penetrating properties that allow systemic
delivery of these drugs without complex and potentially troublesome delivery
vehicles.

About Santaris Pharma A/S

Santaris Pharma A/S is a privately held clinical-stage biopharmaceutical
company focused on the discovery and development of RNA-targeted therapies.
The Locked Nucleic Acid (LNA) Drug Platform and Drug Discovery Engine
developed by Santaris Pharma A/S combine the Company's proprietary LNA
chemistry with its highly specialized and targeted drug development
capabilities to rapidly deliver potent single-stranded LNA-based drug
candidates across a multitude of disease states. The Company's research and
development activities focus on infectious diseases and cardiometabolic
disorders, while partnerships with major pharmaceutical companies include a
range of therapeutic areas including cancer, cardiovascular disease,
infectious and inflammatory diseases, and rare genetic disorders. The Company
has strategic partnerships with miRagen Therapeutics, Shire plc, Pfizer,
GlaxoSmithKline, and Enzon Pharmaceuticals. As part of its broad patent
estate, the Company holds exclusive worldwide rights to all therapeutic uses
of LNA. Santaris Pharma A/S, founded in 2003, is headquartered in Denmark
with operations in the United States. Please visit www.santaris.com for more
information.

(1) World Health Organization -
www.who.int/healthinfo/statistics/bod_cerebrovasculardiseasestroke.pdf

(2) Santaris Pharma A/S in-house data

Navjot Rai, Director, Global Communications of Santaris Pharma A/S, +1-858-764-7064, ext. 206, Cell, +1-619-723-5450, navjot.rai at santaris.com

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