About RG-101

Regulus’ wholly-owned candidate is RG-101, a GalNAc-conjugated anti-miR targeting miR-122 for the treatment of hepatitis C virus (HCV) infection.  miR-122 is the most abundant miR in the liver and is essential for HCV stability, replication and translation in hepatocytes. Regulus believes that its miR-122 antagonist, RG-101, has the potential to become a powerful new agent in the treatment of HCV infection.

RG-101 has several important properties that may differentiate it from other therapies currently on the market for the treatment of HCV.

  • A novel mechanism of action
  • Activity against all genotypes
  • Activity against HCV strains with mutations that cause resistance to oral direct-acting antivirals (DAAs)
  • Ability to be safely combined with and potentially enhance the efficacy and/or shorten the required treatment period of existing oral DAA therapies.

Ongoing RG-101 Clinical Trials

Regulus’ Phase II program and development strategy for RG-101 includes evaluating RG-101 in combination studies with different approved direct-acting anti-HCV agents (DAAs);  in combination with investigational oral DAAs that can be formulated into long acting injections providing the potential for a single-visit therapy; and in certain underserved HCV patient populations.

About Hepatitis C Infection

Hepatitis C is the result of a hepatocyte-specific infection induced by the virus known as HCV. Chronic HCV may lead to significant liver disease, including chronic active hepatitis, cirrhosis, and hepatocellular carcinoma. Approximately 185 million people are chronically infected with HCV worldwide. An estimated 10 million people in the United States, European Union, and Japan have chronic HCV infection. According to the World Health Organization, more than 500,000 people die from HCV annually. The CDC estimates that there are currently approximately 3.5 million persons infected with HCV in the United States.

The field of HCV treatments is changing rapidly with the introduction of new direct acting antiviral (DAA) drugs in several classes – protease inhibitors, polymerase inhibitors, NS5A inhibitors, and combinations of these – with superior efficacy and tolerability compared to interferon-based or prior generation protease inhibitor-based regimens. However, despite the advances made in recent years, there remain underserved populations of infected individuals who do not respond to current treatments, who relapse following successful treatment, who are not compliant over up to 12 weeks of daily treatment, or who poorly tolerate one or more drugs used in the new standard-of-care cocktails. Resistance to antiviral therapy is a major problem associated with the high mutation rate of HCV and is seen even with combinations of drugs working through multiple mechanisms. Thus, new therapeutic strategies based on novel mechanisms of action, with an aim towards achieving higher efficacy, pan-genotypic activity, shortened treatment durations, and simplified administration leading to improved tolerability and patient adherence, are areas of high unmet medical need in HCV.