Achieves Key ‘Clinical Map Initiative’ Goal for 2015 and Advances Orphan Disease Efforts
LA JOLLA, Calif., June 4, 2015 /PRNewswire/ – Regulus Therapeutics Inc. (NASDAQ: RGLS), a biopharmaceutical company leading the discovery and development of innovative medicines targeting microRNAs, announced today that dosing has begun in a first-in-human Phase I clinical study of RG-012, a single stranded, chemically modified oligonucleotide that binds to and inhibits the function of microRNA-21 (“miR-21″). RG-012 is being developed by Regulus in a strategic alliance with Genzyme, a Sanofi company, for the treatment of Alport syndrome, a life-threatening genetic kidney disease with no approved therapy. The Phase I clinical study is being conducted in the United States as a randomized, double-blind, placebo-controlled, single ascending dose study to evaluate the safety, tolerability and pharmacokinetics of subcutaneous dosing of RG-012 in healthy volunteers.
“Advancement of RG-012 into the clinic represents an important achievement under our ‘Clinical Map Initiative’ and further underscores our focus on discovering and developing novel microRNA therapies for orphan and rare diseases such as Alport syndrome,” said Paul Grint, M.D., President and Chief Executive Officer of Regulus. “We expect that the results from this first-in-human clinical study [click to continue…]
-Regulus Earns $2.5 Million Milestone Payment from AstraZeneca-
-RG-125 (AZD4076) is Regulus’ 3rd Clinical Candidate, Achieving Key ‘Clinical Map Initiative’ Goal for 2015-
LA JOLLA, Calif., April 7, 2015 /PRNewswire/ – Regulus Therapeutics Inc. (NASDAQ:RGLS), a biopharmaceutical company leading the discovery and development of innovative medicines targeting microRNAs, announced today the selection of RG-125 (AZD4076), a GalNAc-conjugated anti-miR targeting microRNA-103/107 (“miR-103/107″) for the treatment of Non Alcoholic Steatohepatitis (“NASH) in patients with type 2 diabetes/pre-diabetes, as a clinical candidate by AstraZeneca under the companies’ strategic alliance to discover, develop and commercialize microRNA therapeutics. RG-125 (AZD4076) is the first compound from the alliance to be selected for clinical development by AstraZeneca. In connection with the candidate selection, AstraZeneca will pay Regulus $2.5 million and will assume development of the program following acceptance of an Investigational New Drug application. In the near term, Regulus and AstraZeneca plan to submit key preclinical data on the RG-125 (AZD4076) program to be presented at a scientific meeting later this year and expect to initiate a Phase I study of RG-125 (AZD4076) in humans by the end of 2015.
“Regulus is very pleased that AstraZeneca has chosen to advance a microRNA therapeutic candidate from this exciting program toward the clinic. RG-125 acts as a novel insulin sensitizer which we believe may inform a differentiated development path to treat patients with complicated metabolic disorders,” said Neil W. Gibson, Ph.D., Chief Scientific Officer of Regulus. “RG-125 represents our third clinical development candidate to arise from our novel technologies in less than two years, which confirms the productivity of our platform, achieves a key goal under our ‘Clinical Map Initiative’, and underscores our leadership in the microRNA therapeutics field.”
Marcus Schindler, Head of Cardiovascular and Metabolic Diseases, Innovative Medicines and Early Development, AstraZeneca, said: “This is a tremendous achievement for our collaboration and an exciting step for AstraZeneca to be [click to continue…]
Regulus Receives Orphan Medicinal Product Designation from the European Commission for RG-012, a microRNA Therapeutic in Development for the Treatment of Alport Syndrome
LA JOLLA, Calif., March 25, 2015 /PRNewswire/ – Regulus Therapeutics Inc. (NASDAQ:RGLS), a biopharmaceutical company leading the discovery and development of innovative medicines targeting microRNAs, announced today that the European Commission has granted orphan medicinal product designation for RG-012, a single stranded, chemically modified oligonucleotide that binds to and inhibits the function of microRNA-21 (“miR-21″) for the treatment of Alport syndrome, a life-threatening genetic kidney disease with no approved therapy. In July 2014, the U.S. Food & Drug Administration granted orphan drug designation to RG-012 for the treatment of Alport syndrome.
“We are pleased to have received orphan medicinal product designation in the European Union for RG-012, a key microRNA therapeutic program under our ‘Clinical Map Initiative’,” said Paul Grint, M.D., Chief Medical Officer of Regulus. “Alport syndrome is a life threatening disease and patients have very limited treatment options because there is currently no approved therapy. We believe that RG-012 represents an opportunity to make a significant impact in the lives of patients with Alport syndrome and we look forward to advancing this program into the clinic.”
Regulus is currently enrolling patients in a natural history of disease study called ATHENA to gather information about the changes in renal function over time in patients with Alport syndrome. Data from [click to continue…]
Regulus Announces Key Goals Under its ‘Clinical Map Initiative’ for 2015
Accelerates RG-101 for HCV with Dual-Track Clinical Development Strategy; Top-Line, Single Dose, 4 mg/kg Results as Well as 2mg/kg
Extended Follow Up Results from Ongoing Study to be Reported in Early February 2015
LA JOLLA, Calif., Jan. 8, 2015 /PRNewswire/ – Regulus Therapeutics Inc. (NASDAQ:RGLS), a biopharmaceutical company leading the discovery and development of innovative medicines targeting microRNAs, today announced key goals for 2015 under its ‘Clinical Map Initiative’ to advance its microRNA therapeutics portfolio and biomarkers platform.
“Regulus enters 2015 with the scientific and financial strength to realize the transformative potential of microRNAs. As such, we’ve set aggressive goals for the year focused on creating a clear path to value for what we believe to be our greatest opportunities,” said Kleanthis G. Xanthopoulos, Ph.D., President and CEO of Regulus. “Under our ‘Clinical Map Initiative’, we are focusing our near term efforts on accelerating RG-101 for HCV with a Phase II dual-track clinical development strategy, while advancing our overall therapeutics pipeline and aligning our biomarker efforts to streamline our clinical development decisions.”
Key Goals Under Regulus’ ‘Clinical Map Initiative’ for 2015
- ‘Clinical Map’ of RG-101 for HCV Defined: Dual-Track Strategy Accelerates Phase II Development; Multiple Data Read-Outs in 2015. Following the favorable interim results reported inOctober 2014 from its ongoing clinical study, Regulus has accelerated development of RG-101, a wholly-owned, GalNAc-conjugated anti-miR targeting microRNA-122 (“miR-122″) for the treatment of HCV. Regulus is pursuing a Phase II dual-track development strategy (i) 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 (ii) to investigate RG-101 further as a single agent to determine [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
LA 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…]