Date: 2015-04-25
Type of information: Presentation of results at a congress
phase: 1
Announcement: presentation of results at The International Liver Congress™ 2015 of the European Association for the Study of the Liver (EASL) taking place in Vienna from April 22-26
Company: Regulus Therapeutics (USA - CA)
Product: RG-101
Action
mechanism: oligonucleotide/microRNA inhibitor. RG-101 is a GalNAc-conjugated anti-miR targeting miR-122 for the treatment of HCV.
Disease: hepatitis C
Therapeutic area: Infectious diseases
Country:
Trial
details: Regulus has evaluated RG-101 in a completed clinical study conducted in The Netherlands. 58 healthy volunteers and 32 HCV patients with multiple genotypes, liver fibrosis status and treatment history were enrolled in the four part study: (i) a single ascending-dose study in which healthy volunteer subjects received a single subcutaneous dose of RG-101, 0.5 mg/kg, 1 mg/kg, 2 mg/kg, 4 mg/kg and 8 mg/kg or placebo; (ii) a multiple-ascending dose study in which healthy volunteer subjects received a monthly single subcutaneous dose for four months of RG-101 or placebo; (iii) a single-dose drug-drug interaction study in which healthy volunteer subjects received a single subcutaneous dose of RG-101 in combination with simeprevir (OLYSIO™), an approved direct acting antiviral; and (iv) a single-dose study in which HCV patients received either a single subcutaneous dose of RG-101 or placebo at two doses, 2 mg/kg of RG-101 (the first dose cohort) or 4 mg/kg of RG-101 (the second dose cohort), to assess the safety and viral load reduction. The primary objective is to evaluate safety and tolerability and the secondary objectives are to evaluate pharmacokinetics, viral load reduction and any impact an oral direct acting antiviral, such as simeprevir (OLYSIO™), may have on the pharmacokinetics of RG-101.
Latest
news: * On April 25, 2015, Regulus Therapeutics, a biopharmaceutical company leading the discovery and development of innovative medicines targeting microRNAs, presented new data strengthening the profile of RG-101, a wholly-owned, GalNAc-conjugated anti-miR targeting microRNA-122 (\"miR-122\") for the treatment of HCV, during an oral late-breaking session at ILC 2015 in Vienna, Austria. Extended follow-up results evaluating a single subcutaneous administration of either 2 mg/kg or 4 mg/kg of RG-101 as monotherapy in HCV patients with varied genotypes, liver fibrosis status and treatment history showed that 10/22 patients had HCV RNA levels below the limit of quantification ("BLOQ") at 12 weeks and 70 percent of those patients remained BLOQ at 20 weeks (7/10). In addition, the positive results that were previously reported from the completed clinical study of RG-101 were reviewed during the oral late-breaker. Regulus also made several poster presentations highlighting the pharmacokinetics and pharmacodynamics of RG-101 in healthy volunteers, the preclinical pharmacokinetics, pharmacodynamics, and toxicity of RG-101, and the efficacy of RG-101 in a preclinical model of HCV: - A Single Subcutaneous Dose of 2 mg/kg or 4 mg/kg of RG-101, a Galnac-Conjugated Oligonucleotide with Antagonist Activity against miR-122, Results in Significant Viral Load Reductions in Chronic Hepatitis C Patients - Pharmacokinetics and pharmacology of RG-101, a novel GalNAC-conjugated oligonucleotide targeting microRNA-122 in healthy volunteers - Conjugated oligonucleotide targeting microRNA-122 in rodents and cynomologus monkeys - RG-101, a novel GalNac-conjugated inhibitor of microRNA-122 demonstrates significant viral load reduction and reduces liver steatosisin human hepatocyte chimeric mice infected with genotype 1A or hard-to treat genotype 3A hepatitis C virus. Regulus\' \'Clinical Map Initiative\' outlines certain corporate goals to advance its microRNA therapeutics pipeline over the next several years. Regulus plans to investigate RG-101 in combination with oral agents to potentially shorten treatment durations, optimize clinical outcomes and potentially improve responses in certain underserved HCV patient populations, and further as a single agent (multiple doses of RG-101 in specific populations). In the near term, Regulus expects to initiate the above described studies in the second quarter of 2015 and to report interim data by the end of 2015.
