Long-Acting Parenteral Formulation of GSK2878175 Being Developed; Co-Administration with RG-101 May Enable Single Visit Therapy for HCV Patients

regulus therapeutics logoLA JOLLA, Calif., Nov. 3, 2015 /PRNewswire/ — Regulus Therapeutics Inc. (NASDAQ:RGLS), a biopharmaceutical company leading the discovery and development of innovative medicines targeting microRNAs (miR), today announced that it has expanded development of RG-101, Regulus’ wholly-owned, GalNAc-conjugated anti-miR that targets miR-122, through a clinical trial collaboration and formulation development agreement with GlaxoSmithKline (“GSK”) (NYSE: GSK).  The companies plan to conduct a Phase II study to evaluate the combination of RG-101 and GSK2878175, an investigational non-nucleoside NS5B polymerase inhibitor, for the treatment of HCV. Concurrently, GSK will work on developing a long-acting parenteral for injection (“LAP”) formulation of GSK2878175 which could improve patient compliance through reduced dosing intervals and potentially extend opportunities for HCV therapeutic intervention. This LAP formulation of GSK2878175 may be used in additional clinical trials together with RG-101 following completion of the planned Phase II study, although any additional studies are not covered by the collaboration agreement.

“We are pleased to work with GSK to advance the scientific understanding of the potential for a combination regimen co-administered all at once to treat HCV,” said Paul Grint, M.D., President and CEO of Regulus.  “The study to be conducted [click to continue…]


Regulatory Application for RG-101 to be Filed in the Near Term: Clinical Studies in Man Expected to Commence in Early 2014

regulus therapeutics logoLA JOLLA, Calif., Nov. 4, 2013 /PRNewswire/ — Regulus Therapeutics Inc. (NASDAQ: RGLS), a biopharmaceutical company leading the discovery and development of innovative medicines targeting microRNAs, today announced that data demonstrating the positive preclinical profile of RG-101 will be presented in a late-breaking poster session at the 64th Annual Meeting of the American Association for the Study of Liver Disease (AASLD) being held in Washington, D.C. on Monday, November 4, 2013 from8:00 a.m. Eastern Standard Time (EST) to 5:30 p.m. EST. The poster is available on the Company’s website at http://www.regulusrx.com.

“RG-101 utilizes a unique mechanism of action by targeting microRNA-122, a liver-specific host factor for stability, replication and translation of HCV.  We believe that therapies that target host-encoded factors essential for HCV replication may act as attractive combination agents because they may demonstrate activity across [click to continue…]


– Nominated RG-101 in HCV as First microRNA Candidate for Clinical Development-

– Maintained Strong Financial Position with Over $90 million in Cash-

regulus therapeutics logoLA JOLLA, Calif., May 14, 2013 /PRNewswire/ — Regulus Therapeutics Inc. (NASDAQ: RGLS), a biopharmaceutical company leading the discovery and development of innovative medicines targeting microRNAs, today provided an update on its microRNA therapeutic pipeline under the company’s ‘Road to the Clinic’ strategy and reported financial results for the quarter ended March 31, 2013.

Regulus Continues to Execute on ‘Road to the Clinic’ Strategy:
Nominated RG-101 as First microRNA Candidate for Clinical Development

  • Regulus announced today that it has nominated its first microRNA candidate for clinical development, RG-101, a GalNAc-conjugated microRNA antagonist or anti-miR, which targets microRNA-122 (miR-122) for the treatment of patients with chronic hepatitis C virus (HCV) infection. Regulus is performing additional pre-clinical studies and finalizing development plans for RG-101 in HCV and expects to submit an application with regulatory authorities in early 2014.
  • Regulus plans to develop RG-101 independently of its strategic alliance with GlaxoSmithKline (GSK).  The broad strategic alliance between Regulus and GSK remains intact and GSK retains its interest in the miR-122 program in HCV, as miR-122 will remain a Collaboration Target under the alliance.   As such, the companies are in the process of amending the Product Development and Commercialization Agreement to clarify that RG-101 is fully owned by Regulus.

“The nomination of RG-101 as our first microRNA candidate for clinical development marks a tremendous milestone for Regulus and represents significant achievement of our initial goals under our ‘Road to the Clinic’ strategy,” said Kleanthis G. Xanthopoulos, Ph.D., President and CEO of Regulus. “We continue to focus [click to continue…]


Hepatitis C virus hijacks liver microRNA

by Christoph on January 3, 2012

in Publications

Stabilization of hepatitis C virus RNA by an Ago2–miR-122 complex

MicroRNAs regulate eukaryotic gene expression by binding to regions of imperfect complementarity in mRNAs, typically in the 3′ UTR, recruiting an Argonaute (Ago) protein complex that usually results in translational repression or destabilization of the target RNA. The translation and decay of mRNAs are closely linked, competing processes, and whether the miRNA-induced silencing complex (RISC) acts primarily to reduce translation or stability of the mRNA remains controversial.

miR-122 is an abundant, liver-specific miRNA that is an unusual host factor for hepatitis C virus (HCV), an important cause of liver disease in humans. Prior studies show that it binds the 5′ UTR of the messenger-sense HCV RNA genome, stimulating translation and promoting genome replication by an unknown mechanism. In this study the authors show that [click to continue…]

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from BusinessWire

miR-122 is a liver-expressed microRNA that has been shown to be a critical endogenous “host factor” for the replication of hepatitis C virus (HCV), and anti-miRs targeting miR-122 have been shown to block HCV infection (Jopling et al. (2005) Science 309, 1577-81). In earlier work, scientists at Alnylam Pharmaceuticals and Isis Pharmaceuticals demonstrated the ability to antagonize miR-122 in vivo using chemically modified single-stranded anti-miR oligonucleotides. Data from multiple preclinical studies have shown a robust HCV antiviral effect following inhibition of miR-122.

Regulus Therapeutics Inc. is developing a microRNA therapeutic targeting miR-122 for the treatment of HCV infection and plans to file an investigational new drug (IND) application in 2011.

Today, Regulus announced that the European Patent Office (EPO) and United States Patent and Trademark Office (USPTO) have recently granted claims for microRNA-122 therapy in hepatitis C viral (HCV) infections.  These two new patents will further strengthen the Regulus-controlled patent estate surrounding miR-122 compositions and methods of use, which include:

  • The ‘Sarnow’ patent claiming the use of anti-miR-122 to inhibit HCV replication (US Patent No. 7,307,067)
  • The ‘Esau’ patent claiming the use of anti-miRs targeting miR-122 as inhibitory agents (US Patent No. 7,683,036)
  • The ‘Tuschl III’ patent claiming compositions of matter for miR-122 and complementary oligonucleotides (US Patent No. 7,232,806)
  • The ‘Manoharan’ patent claiming antagomirs, including antagomirs targeting miR-122 (US Patent No. 7,582,744)
  • A recently granted Regulus-owned European application claiming the use of miR-122 antagonists for reducing cholesterol (EP Application No. 06813949.2)

(read the entire press release)

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