Santaris Pharma A/S writes medical history and advances miravirsen, the first microRNA-targeted drug to enter clinical trials, into Phase 2 to treat patients infected with Hepatitis C virus
Santaris Pharma A/S Phase 2a data of miravirsen shows dose-dependent, prolonged viral reduction of 2-3 logs HCV RNA after four-week treatment in Hepatitis C patients
Hoersholm, Denmark/San Diego, California — Santaris Pharma A/S, a clinical-stage biopharmaceutical company focused on the discovery and development of RNA-targeted therapies, presents new data from the Phase 2a trial showing that miravirsen given as a four-week monotherapy treatment provided robust dose-dependent anti-viral activity with a mean reduction of 2 to 3 logs from baseline in HCV RNA (log10 IU/mL) that was maintained for more than four weeks beyond the end of therapy. These new clinical data as well as the data in the abstract are being presented in a late-breaking oral presentation on November 7 at 4p.m., at The Liver Meeting® 2011, the 62nd annual meeting of the AASLD, in San Francisco, California[1].
New clinical data from the Phase 2a study demonstrate that four out of nine patients treated at the highest dose (7 mg/kg) with miravirsen became HCV RNA undetectable with just four weeks of dosing. These data provide clinical evidence that miravirsen’s unique mechanism-of-action offers high barrier to viral resistance and the potential for cure with monotherapy. Miravirsen was also well tolerated in patients with HCV, signaling a possible advantage over standard of care treatment.
“The miravirsen Phase 2a data are very promising considering that with only four weeks of treatment, miravirsen provided robust antiviral activity and throughout the study there was no sign of viral resistance”
said Harry Janssen, M.D. Ph.D., Head of Liver Unit at the Erasmus MC University Hospital Rotterdam, The Netherlands and Lead Clinical Investigator of the study, who is presenting the data at AASLD.
“These data, taken together with the observation that four patients became HCV RNA “Due to its unique mechanism of action in targeting miR-122, miravirsen has the potential to change the way Hepatitis C is treated”
said Stefan Zeuzem, M.D., Professor of Medicine and Chief of the Department of Medicine at the JW Goethe University Hospital, Frankfurt, Germany and Lead Clinical Investigator of the trial. “Miravirsen has the potential to be used as the central backbone treatment in combination with direct acting anti-viral agents as part of an interferon-free, infrequent
About Hepatitis C
Hepatitis C infection is a viral disease caused by the Hepatitis C virus that leads to inflammation of the liver. The World Health Organization estimates about 3% of the world’s population has been infected with HCV and that some 170 million have chronic Hepatitis C and are 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[3]. In Europe, there are about 4 million carriers1. Treatment with pegylated interferon in combination with ribavirin is effective in only about 50% of patients with genotype 1 HCV2. Although the addition of one of the recently approved protease inhibitors has led to an improvement in virological response rates, not all patients can effectively be treated with triple therapy and they will continue to have an increased risk for the progression of liver disease[4]. By 2029, total annual medical costs in the United States for people with Hepatitis C are expected to more than double, from $30 billion in 2009 to approximately $85 billion[5].
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 LNA-based drug candidates against RNA targets, both mRNA and microRNA, for a range of diseases including infectious and inflammatory diseases, cardiometabolic disorders, 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 drug 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 manufacture, have manufactured and sell products that comprise LNA as active ingredient for studies performed with a view to obtaining marketing approval. 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.
About Gilde Healthcare Partners
Gilde Healthcare Partners (Utrecht, The Netherlands and Cambridge, US) is a transatlantic growth equity investor focused on private healthcare technologies and services. It has EUR 450 million (USD 625 million) under management and is actively looking to lead new investments in therapeutics, diagnostics, medical devices and services. Gilde successfully builds healthcare businesses across Europe and US, investing up to EUR 15 million in a single portfolio company. By investing in companies with clear, achievable business models, Gilde has used its financial resources and network to create significant value for both its investors and the entrepreneurs it backs. For a list of Gilde’s portfolio companies please visit the website at www.gildehealthcare.com.
Gilde Healthcare II is supported by the European Communities Growth and Employment Initiative, MAP – ETF Start up Facility.