LA JOLLA, Calif., March 16, 2016 /PRNewswire/ — Regulus Therapeutics Inc. (NASDAQ: RGLS), a biopharmaceutical company leading the discovery and development of innovative medicines targeting microRNAs, today announced that new data on RG-101, the company’s anti-miR-122 in Phase II development for the treatment of Hepatitis C Virus infection (HCV), will be presented as an oral presentation during the general session at The International Liver Congress™ (ILC) 2016, April 13-17 in Barcelona, Spain. Three poster presentations outlining additional RG-101 clinical and preclinical research will also be included as part of the meeting’s poster sessions.
“Regulus and our clinical investigators are pleased to be making several presentations at this year’s International Liver Congress, including an oral presentation during the general session elaborating on recent interim results from an ongoing Phase II combination study of RG-101 for the treatment of HCV,” said Paul Grint, M.D., President and Chief Executive Officer of Regulus. “We believe the work presented further supports our novel approach to treating HCV as well as the clinical utility of RG-101 within the HCV landscape.”
- Friday, April 15, 8:45am CET (General Session): RG-101 in Combination with 4 Weeks of Oral Direct Acting Antiviral Therapy Achieves High Virologic Response Rates in Treatment Naïve Genotype 1 and 4 Chronic Hepatitis C Patients: Interim Results from a Randomised, Multi-Center, Phase 2 Study (G. Horvath, Hungary).
- Abstract THU-254: A Single Dose of Anti-miR-122 Oligonucleotide RG-101 Results in a Less Activated Phenotype of NK Cells in Patients with Chronic Hepatitis C (F. Stelma, Netherlands).
- Abstract THU-232: Sequence Analysis for Resistance Monitoring Following A Single Dose of RG-101, an anti-miR Targeting microRNA-122, in Chronic Hepatitis C Patients (M. van der Ree, Netherlands).
- Abstract THU-239: RG-101 Demonstrates Favorable in Vitro Antiviral Activity and Cross Resistance Profile to Support Clinical Combination in HCV Patients (S. Neben, United States).
When available, the abstracts related to the presentations can be accessed through the ILC/EASL website at ilc-congress.eu.
About RG-101 for HCV
RG-101 is Regulus’ wholly-owned, GalNAc-conjugated anti-miR targeting miR-122 for the treatment of HCV. In a completed Phase I human proof-of-concept study, Regulus demonstrated that treatment with a single subcutaneous dose of RG-101 as monotherapy resulted in significant and sustained viral load reductions in all treated HCV patients, including patients with difficult to treat genotypes, various liver fibrosis status and those who have experienced viral relapse after a prior IFN-containing regimen.
Recently, Regulus presented favorable interim data from an ongoing Phase II study evaluating the combination of RG-101 with multiple approved DAAs positioning RG-101 for both front-line and second-line commercial opportunities. Patients received a single subcutaneous injection of 2 mg/kg of RG-101 on Day 1, followed by 28 days of a once daily oral DAA (Harvoni®, Olysio®, or Daklinza™), followed by an additional subcutaneous injection of 2 mg/kg of RG-101 on Day 29. Regulus is planning to report primary endpoint results at 12 weeks following conclusion of treatment in late Q2 2016.
In collaboration with GSK, Regulus recently initiated a Phase II study evaluating the combination of RG-101 and GSK2878175, a non-nucleoside NS5B polymerase inhibitor, in treatment-naïve patients chronically infected with HCV genotypes 1 and 3. Additionally, enrollment is nearly complete in a multi-center, open label, non-randomized Phase I study to compare the safety, tolerability, pharmacokinetics, and pharmacodynamics of 2 mg/kg of RG-101 in subjects with severe renal insufficiency or end-stage renal disease (ESRD) to healthy control subjects, and further explore RG-101 in hepatitis C infected subjects with severe renal insufficiency or ESRD. Regulus anticipates reporting safety and efficacy data from the HCV/severe renal impairment or ESRD arm in the second half of 2016.
The discovery of microRNAs in humans during the last decade is one of the most exciting scientific breakthroughs in recent history. microRNAs are small RNA molecules, typically 20 to 25 nucleotides in length, that do not encode proteins but instead regulate gene expression. More than 800 microRNAs have been identified in the human genome, and over two-thirds of all human genes are believed to be regulated by microRNAs. A single microRNA can regulate entire networks of genes. As such, these molecules are considered master regulators of the human genome. microRNA expression, or function, has been shown to be significantly altered or dysregulated in many disease states, including oncology, fibrosis, metabolic diseases, immune-inflammatory diseases and HCV. Targeting microRNAs with anti-miRs, chemically modified, single-stranded oligonucleotides, offers a unique approach to treating disease by modulating entire biological pathways and may become a new and major class of drugs with broad therapeutic application.