Regulus Earns $10.0 Million Milestone Payment from AstraZeneca and Achieves Key Goal for 2015

regulus therapeutics logoLA JOLLA, Calif., Dec. 18, 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-125(AZD4076), by its collaboration partner AstraZeneca. RG-125(AZD4076) is a GalNAc-conjugated anti-miR-103/107 oligonucleotide that has been shown to improve insulin sensitivity and glucose tolerance in animal models. RG-125(AZD4076) was jointly identified and selected as a clinical candidate in April 2015 by AstraZeneca under the companies’ strategic alliance to discover, develop and commercialize microRNA therapeutics. AstraZeneca will pay Regulus $10.0 million and will assume further development of RG-125(AZD4076).

Previous research has demonstrated a causative role of microRNAs in pathophysiological processes of metabolic diseases, and increased expression of miR-103/107 in the liver has been associated with insulin resistance in people with non-alcoholic steatopheatitis (NASH), or fatty liver disease.  In mechanistic studies, RG-125(AZD4076) showed effects on biological pathways implicated in NASH progression.

“RG-125(AZD4076) acts as a novel insulin sensitizer which represents a potential new mechanism to treat patients with metabolic diseases such as type 2 diabetes and NASH,” said Paul Grint, MD, President and Chief Executive Officer of Regulus. “Regulus is pleased to [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-

regulus therapeutics logoLA 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…]

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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

Regulus Therapeutics LogoLA 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…]


from Epigenie blog – May 5, 2010 

Up to now, all miRNAs have been assumed to work the same way when it comes to finding their mRNA targets, but a new paper shows that miRNAs behave more like rugged individualists in how they function.

Researchers from the University of Kentucky broke out the RIP-Chip method (Argonaute, aka AGO, co-IP assays coupled with downstream microarray analysis) in H4 glioneuronal cells transfected with certain miRNAs to get an idea of what each miRNA is targeting and how. The RIP-Chip technique showed hard evidence of what mRNAs are targeted, and even the proteins involved in the microribonucleoparticles (miRNPs) for each miRNA tested (miR-107, miR-124, miR-128, and miR-320). After looking over targets of each miRNA, here’s what they found:

  • miR-124 displayed a standard targeting profile: using a 5’ seed sequence to target a 3’ mRNA UTR.
  • miR-107, however, targeted the open reading frame (ORF) regions, and not the 3’UTRs, of its targets.
  • miR-128 and miR-320 were shown to utilize more of a mixture of 5’ and 3’ seed regions to bind targets.

Sure, the authors only looked at one cell-line, and RIP-Chip assays can only asses non-degraded targets, but the take-home message is the same: miRNAs are like snowflakes, every one is unique. So, the only way to really know what your favorite miRNA is targeting, is to go test it out.

Get zeroed in on all the miRNA targeting details in RNA Biology, May 2010.

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