Santaris Pharma A/S provides update on Miravirsen for treatment of HCV...

Santaris Pharma A/S gives a preview update on miravirsen for treatment of HCV that will be presented at the 7th Annual Meeting of the Oligonucleotide Therapeutics Society (OTS) held September 8-10 in Copenhagen, Denmark. Miravirsen is the first microRNA-targeted drug inhibiting miR-122 to make it to clinic (it's currently in phase II trials) and to specifically target HCV. Miravirsen is the result of Santaris Pharma A/S propritary Locked Nucleic Acid Technology (LNA)drug platform, which has the potential to deliver drugs 'naked' without the complex delivery systems of RNA-based drugs. LNA-based compounds have the potential to inhibit entire microRNA families and is a new approach that opens up possibilities of successfully treating complex diseases such as cancer, viral infections, cardiovascular and muscle diseases

Santaris Pharma A/S Showcases microRNA and mRNA Research Advancements Utilizing its Proprietary Locked Nucleic Acid Technology at Oligonucleotide Therapeutics Society Meeting

HOERSHOLM, Denmark and SAN DIEGO, September 7, 2011 /PRNewswire/ --

Santaris Pharma A/S provides update on the development of miravirsen, a microRNA-targeted drug inhibiting miR-122, which is currently in Phase 2 clinical trials to treat patients infected with the Hepatitis C virus (HCV)
Scientists at Santaris Pharma A/S demonstrate specificity of "tiny" 8-mer LNA-based compounds, which have shown to successfully inhibit entire microRNA families providing potential new approach for treating cancer, viral infections, cardiovascular and muscle diseases

Data presented by scientists at Santaris Pharma A/S demonstrate successful "naked" delivery uptake of LNA-based compounds, a key advantage over other RNA-based technologies that require complex delivery vehicles
Versatility of the LNA Drug Platform allows Santaris Pharma A/S to develop both microRNA and mRNA-targeted drugs with excellent specificity and increased affinity to drug target, providing improved potency and favorable tissue-penetrating properties


Santaris Pharma A/S, a clinical-stage biopharmaceutical company focused on the research and development of mRNA and microRNA targeted therapies, today announced advancements from the company's RNA-targeted research programs utilizing its proprietary Locked Nucleic Acid (LNA) Drug Platform. In addition to a presentation on the Company's microRNA-targeted drug miravirsen, a total of six abstracts/posters are being showcased at the 7th Annual Meeting of the Oligonucleotide Therapeutics Society (OTS) held September 8-10 in Copenhagen, Denmark.

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 that can selectively inhibit mRNA and microRNA targets for a range of diseases including metabolic disorders, infectious and inflammatory diseases, cancer and rare genetic disorders.

"Santaris Pharma A/S is pleased to have the OTS hold its annual meeting here in Denmark this year as it recognizes the groundbreaking RNA-targeted research and development being conducted in Denmark from companies such as Santaris Pharma A/S as well as public institutions and research centers," said Henrik Oerum, Ph.D., Vice President and Chief Scientific Officer of Santaris Pharma A/S.

"Santaris Pharma A/S is excited to showcase its microRNA and mRNA-targeted research advancements utilizing its proprietary Locked Nucleic Acid Drug Platform," Dr. Oerum added. "The Company is providing an update on the progress of its lead microRNA-targeted drug candidate, miravirsen, for the treatment of Hepatitis C; presenting data on the use of Santaris Pharma A/S proprietary 'tiny' LNA-based drugs to inhibit entire microRNA-targeted families and highlighting data demonstrating 'naked' delivery uptake of LNA-based drugs, a key advantage over other RNA based technologies. This collection of data attests to the versatility of the LNA Drug Platform and clearly supports Santaris Pharma A/S leadership position in discovering and developing RNA-targeted medicines."

In a talk titled, "Targeting miRNA-122 for the Treatment of HCV," Dr. Oerum will provide an update on the development of miravirsen, the first microRNA-targeted drug to enter clinical trials, which is now in Phase 2 studies to assess the safety and tolerability of the drug in treatment-naïve patients infected with HCV. Data from Phase 1 clinical studies with miravirsen in healthy volunteers show that the drug is well tolerated.

Previously published data in Science demonstrated that miravirsen successfully inhibited miR-122 and dramatically reduced Hepatitis C virus in the liver and in the bloodstream in chimpanzees chronically infected with the Hepatitis C virus(1). Because of its unique mechanism of action and tolerability profile, miravirsen has the potential to be an effective treatment option for patients with HCV.

In addition, scientists at Santaris Pharma A/S will present data using proprietary bioinformatics techniques to demonstrate the specificity of "tiny" 8-mer LNA-based compounds, which have shown to successfully inhibit entire microRNA families providing potential new approach for treating a range of diseases including cancer, viral infections, cardiovascular and muscle diseases. Recent data published in Nature Genetics showed that the high affinity and target specificity of tiny LNA-based compounds enabled functional inhibition of entire microRNA families in a range of tissues without off-target effects(2).

MicroRNAs have emerged as an important class of small regulatory RNAs encoded in the genome. They act to control the expression of sets of genes and entire pathways and are thus thought of as master regulators of gene expression associated with many diseases. Because they dictate the expression of fundamental regulatory pathways, microRNAs represent potential drug targets in the treatment of many disease processes.

Santaris Pharma A/S also will present data demonstrating successful "naked" delivery uptake of LNA-based compounds, an advantage over other RNA-based technologies that require complex delivery vehicles. Data showed that LNA-based compounds readily enter cells in an active form allowing in vitro prediction of potent therapeutically active compounds as well as in vivo distribution to manifold cell types and tissues. Without the need for using complex delivery vehicles, the versatility of the LNA Drug Platform is the key for scientists to develop both microRNA and mRNA-targeted drugs with excellent specificity and increased affinity to drug target, providing improved potency and favorable tissue-penetrating properties.

The Santaris Pharma A/S LNA Drug Platform is the only RNA technology with both mRNA and microRNA targeted drugs in clinical trials, demonstrating the broad utility of the proprietary platform. 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. In addition, Santaris Pharma A/S also advanced two mRNA-targeted drugs, SPC5001 targeting PCSK9 and SPC4955 targeting apoB, for the treatment of high cholesterol into Phase 1 in May 2011.

With its partners, Santaris Pharma A/S 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 target pathways previously considered inaccessible. 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 http://www.santaris.com for more information.

(1) Lanford et al, Therapeutic silencing of microRNA-122 in primates with chronic hepatitis C virus infection. Science 327 (5962): 198-201

(2) Obad, et al. Silencing of microRNA families by seed-targeting tiny LNAs. Nature Genetics 10.1038/ng.786.

First micro-RNA-Targeted Drug to treat HCV enters Phase II Clinical Trials...

HOERSHOLM, Denmark and SAN DIEGO, September 22, 2010 /PRNewswire/ --

- Santaris Pharma A/S initiates Phase 2a clinical trial with miravirsen (SPC3649) to assess safety and tolerability in treatment-naive patients with chronic Hepatitis C

- miravirsen is the first microRNA-targeted drug to receive Investigational New Drug (IND) acceptance from FDA, paving the way to conduct Phase 2 trials for treatment of Hepatitis C in the United States

- Developed using Santaris Pharma A/S Locked Nucleic Acid (LNA) Drug Platform, miravirsen inhibits miR-122, a microRNA important for Hepatitis C viral replication, thereby significantly reducing the levels of Hepatitis C virus

- Due to unique mechanism-of-action, miravirsen holds promise as new treatment option for Hepatitis C patients, including the 50% of patients not responsive to current standard of care(1)

Santaris Pharma A/S, a clinical-stage biopharmaceutical company focused on the discovery and development of RNA-targeted therapies, today announced that it has advanced miravirsen (SPC3649), the first microRNA-targeted drug to enter clinical trials, into Phase 2 studies to assess the safety and tolerability of the drug in treatment-naive patients infected with the Hepatitis C virus (HCV).

Paving the way to conduct the first clinical trials of a microRNA-targeted drug in the United States, Santaris Pharma A/S also received acceptance of its Investigational New Drug (IND) application from the U.S. Food and Drug Administration (FDA). In addition to the United States, the Phase 2a clinical trials will be conducted in the Netherlands, Germany, Poland, Romania, and Slovakia.

The World Health Organization estimates about 3% of the world's population has been infected with HCV and that some 170 million are chronic carriers at risk of developing liver cirrhosis and/or liver cancer(2). Approximately 3-4 million Americans are chronically infected with an estimated 40,000 new infections per year(1). In Europe, there are about 4 million carriers(2). The current standard of care, pegylated interferon in combination with ribavirin, is effective in only about 50% of those treated(1).

Developed using Santaris Pharma A/S proprietary Locked Nucleic Acid (LNA) Drug Platform, miravirsen is a specific inhibitor of miR-122, a liver specific microRNA that the Hepatitis C virus requires for replication. Miravirsen is designed to recognize and sequester miR-122, making it unavailable to the Hepatitis C virus. As a result, the replication of the virus is effectively inhibited and the level of Hepatitis C virus is reduced.

"Advancing miravirsen, the first microRNA-targeted drug to enter clinical trials, into Phase 2 studies in patients with Hepatitis C demonstrates Santaris Pharma A/S leadership in developing RNA-targeted medicines," said Arthur A. Levin, Ph.D., Vice President, Chief Development Officer and President, US Operations. "Receiving IND acceptance from the FDA to conduct the first clinical trials with a microRNA-targeted drug in the United States brings Santaris Pharma A/S one step closer to potentially providing a growing number of patients chronically infected with HCV with a more effective and better tolerated treatment option."

The LNA Drug Platform is the only technology with both mRNA and microRNA targeted drugs in clinical trials, reinforcing the broad utility of the platform. The unique combination of small size and very high affinity, which is only achievable with LNA-based drugs, allows this new class of drugs to potently and specifically inhibit RNA targets in many different tissues without the need for complex delivery vehicles. LNA-based drugs are a promising new type of therapy that enables scientists to develop drugs to attack previously inaccessible pathways.

"Using our LNA Drug Platform to advance the first microRNA-targeted therapy into human clinical trials was certainly a scientific breakthrough," said Henrik Oerum, Ph.D., Vice President and Chief Scientific Officer of Santaris Pharma A/S. "We are extremely pleased with the results of the Phase I trials and excited to progress miravirsen into Phase 2 clinical trials. Because of its unique mechanism of action and tolerability profile, miravirsen has the potential to be an effective treatment option for patients with HCV."

The randomized, double-blind, placebo-controlled, ascending multiple-dose Phase 2a study will assess the safety and tolerability of miravirsen and is designed to enroll up to 55 treatment-naive patients with chronic Hepatitis C virus genotype 1 infection. Secondary endpoints include pharmacokinetics of miravirsen and its effect on viral load. Miravirsen will be given as subcutaneous injections weekly or every other week for four weeks.

Data from Phase 1 clinical studies with miravirsen in healthy volunteers show that the drug is well tolerated. A recent study published in Science demonstrated that miravirsen successfully inhibited miR-122 and dramatically reduced Hepatitis C virus in the liver and in the bloodstream in chimpanzees chronically infected with the Hepatitis C virus(3). Miravirsen provided continued efficacy in the animals up to several months after the treatment period with no adverse events and no evidence of viral rebound or resistance.

In addition to miravirsen, Santaris Pharma A/S has a robust product pipeline targeting mRNAs and microRNAs both internally as well as in partnerships and collaborations with miRagen Therapeutics (cardiovascular diseases), Shire plc (rare genetic disorders), Pfizer (undisclosed therapeutic areas), GlaxoSmithKline (viral disease) and Enzon Pharmaceuticals (oncology).

About microRNAs

MicroRNAs have emerged as an important class of small RNAs encoded in the genome. They act to control the expression of sets of genes and entire pathways and are thus thought of as master regulators of gene expression. Recent studies have demonstrated that microRNAs are associated with many disease processes. Because they are single molecular entities that dictate the expression of fundamental regulatory pathways, microRNAs represent potential drug targets for controlling many biologic and disease processes.

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 potent single-stranded LNA-based drug candidates against RNA targets, both mRNA and microRNA, for a range of diseases including metabolic disorders, infectious and inflammatory diseases, cancer and rare genetic disorders. 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, which is only achievable with LNA-based drugs, allows this new class of drugs to potently and specifically inhibit RNA targets in many different tissues without the need for complex delivery vehicles. LNA-based drugs are a promising new type of therapy that enables scientists to develop drugs to attack previously inaccessible clinical pathways. The most important features of LNA-based drugs include excellent specificity, providing optimal targeting; increased affinity to targets providing improved potency; and strong pharmacology upon systemic delivery without complicated 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 metabolic 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 http://www.santaris.com for more information.