Regulus Discontinuing clinical development of RG-101 for HCV

Regulus Announces Pipeline Updates and Advancements

Phase II RG-012 HERA and Renal Biopsy studies for Alport syndrome move forward
Discontinuing clinical development of RG-101
Pre-clinical pipeline progresses

LA JOLLA, Calif., June 12, 2017 /PRNewswire/ -- Regulus Therapeutics Inc. (NASDAQ: RGLS), a biopharmaceutical company leading the discovery and development of innovative medicines targeting microRNAs, today announced updates to its pre-clinical and clinical development programs.

"We are squarely focused on taking the steps necessary to advance our pipeline and continue building shareholder value. To that end, we recognize that we must be disciplined in our investment choices and focus our resources and capital on our most promising discovery and development programs, including the application of important development, regulatory and commercial considerations," said Jay Hagan, President and Chief Executive Officer of Regulus. "MicroRNA therapeutics have the potential to become an important new class of drugs with broad therapeutic application. Regulus' focus will be in diseases with significant unmet medical need in organs to which we have been able to preferentially deliver oligonucleotide therapeutics effectively, such as the liver and kidney."

RG-012 for Alport syndrome: Initiation of the Phase II clinical programs for RG-012 for the treatment of Alport syndrome is on track as planned. Important changes to the Phase II study design have been incorporated with the goal to accelerate patient enrollment, improve statistical power, and potentially achieve proof of mechanism data by the end of 2017. HERA, the Phase II randomized (1:1), double-blinded, placebo-controlled study evaluating the safety and efficacy of RG-012 in Alport syndrome patients, has been modified to increase enrollment to 40 patients to improve its statistical power. Dose frequency has also been adjusted to once every other week. The separate renal biopsy study will evaluate RG-012 renal tissue pharmacokinetics, target engagement and downstream effects on genomic disease biomarkers. Data from the renal biopsy study is anticipated by year-end and interim data from HERA is anticipated mid-2018. 

RG-101 (anti-miR122) for HCV: The Company announced today that it plans to discontinue clinical development of RG-101 upon completion of the one remaining clinical study, which is expected to occur in July 2017. Comprehensive pre-clinical investigation and thorough evaluation of the clinical data from RG-101 has led to the identification of a bilirubin transport mechanism as the likely cause for the cases of hyperbilirubinemia in the RG-101 program. We believe that a combination of factors including inhibition of conjugated bilirubin transport by RG-101, impaired baseline bilirubin transport in HCV patients and the preferential uptake of RG-101 by hepatocytes contributed to this mechanism. Additional patient specific contributing factors cannot be excluded. Applying the learnings from the RG-101 program, alternative compounds targeting miR-122 have been identified that maintain potent HCV antiviral activity while lacking inhibition of the bilirubin transporter. These compounds have the potential for rapid clinical proof-of-concept of a novel, markedly shortened treatment regimen for HCV and will be considered for further development pending an updated global commercial market assessment for HCV.

RGLS4326 (anti-miR-17) for autosomal dominant polycystic kidney disease (ADPKD): The IND for RGLS4326 is on track for filing by year end 2017. IND enabling toxicology, repeat pharmacology and manufacturing work have been completed as scheduled to support regulatory submissions. Data from the pre-clinical program have been recently published in Nature Communications and support the rationale for targeting miR-17 for the treatment of ADPKD, an orphan indication with no treatment options affecting approximately 600,000 people in the United States. 

RGLS5040 (anti-miR-27) for cholestatic disease: RGLS5040, an unconjugated inhibitor of microRNA27, has been discontinued based on a positioning of the compound with respect to the competitive landscape coupled with the results from repeat pharmacology studies as part of IND-enabling work. The Company continues to work on developing highly effective therapeutics for genetic forms of cholestatic disease as part of its overall research activities targeting unmet diseases of the liver and kidney.
"With the focus of our efforts on the most promising programs in our portfolio and the opportunity to explore additional novel clinical applications of miRNA targeted therapeutics, I am more excited than ever about the opportunity to deliver game-changing therapeutics for patients with great unmet medical need," said Dr. Timothy Wright, Regulus' Chief R&D Officer.
Separately, AstraZeneca informed the Company that it intends to terminate the clinical development program for AZD4076(RG-125) for the treatment of NASH in Type 2 Diabetes/Pre-diabetes. Pursuant to the terms of the licensing agreement, AstraZeneca's rights with respect to AZD4076(RG-125) will revert to Regulus when the termination becomes effective in twelve months.  AZD4076(RG-125) 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.
http://ir.regulusrx.com/releasedetail.cfm?ReleaseID=1029833

Regulus Announces Continuation of RG-101 Clinical Hold - FDA requests longer-term follow-up data from ongoing studies

Regulus Announces Continuation of RG-101 Clinical Hold

FDA requests longer-term follow-up data from ongoing studies

LA JOLLA, Calif., Jan. 27, 2017 /PRNewswire/ -- Regulus Therapeutics Inc. (Nasdaq: RGLS), a biopharmaceutical company leading the discovery and development of innovative medicines targeting microRNAs, today announced that it received written communication from the U.S. Food and Drug Administration (FDA) that the clinical development program for RG-101 remains on clinical hold. In June 2016, RG-101 was placed on clinical hold following the Company's submission of a second serious adverse event (SAE) of jaundice.

Late last year, Regulus submitted a complete response to the FDA's initial request for information, which included identification of a potential mechanism of hyperbilirubinemia. The Company also submitted a proposal to mitigate this risk. Subsequently, the FDA has requested the final safety and efficacy data from on-going RG-101 clinical and pre-clinical studies before reconsidering the clinical hold. These data will be available once the current study protocols are complete through 48 weeks of follow up, which is anticipated in the fourth quarter. The FDA also requested additional expert review of liver safety data in light of the proposed mechanism of hyperbilirubinemia.

"While we are disappointed that the clinical hold was not lifted at this time, we plan to continue to work with the FDA to address their additional requests as we seek the removal of the clinical hold," said Dr. Timothy Wright, Chief R&D Officer of Regulus.

About Regulus
Regulus Therapeutics Inc. (Nasdaq:RGLS) is a biopharmaceutical company leading the discovery and development of innovative medicines targeting microRNAs. Regulus has leveraged its oligonucleotide drug discovery and development expertise to develop a well-balanced microRNA therapeutics pipeline complemented by a rich intellectual property estate to retain its leadership in the microRNA field. Regulus is advancing several programs in renal, hepatic and central nervous systems diseases, both independently and with our strategic alliance partners, Sanofi and AstraZeneca. Regulus maintains its corporate headquarters in La Jolla, CA. For more information, please visit http://www.regulusrx.com.

About microRNAs
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.

http://ir.regulusrx.com/releasedetail.cfm?ReleaseID=1009493

Safety, tolerability, and antiviral effect of RG-101 in patients with chronic hepatitis C: a phase 1B, double-blind, randomised controlled trial

Of Interest - Update
January 31, 2017 Regulus Announces Continuation of RG-101 Clinical Hold - FDA requests longer-term follow-up data from ongoing studies

Collection of headlines and research:

Safety, tolerability, and antiviral effect of RG-101 in patients with chronic hepatitis C: a phase 1B, double-blind, randomised controlled trial - (01/11/16) 

Long-term safety and efficacy of [RG-101] microRNA-targeted therapy in chronic hepatitis C patients - (01/11/16)

Source - NATAP

The Lancet
Safety, tolerability, and antiviral effect of RG-101 in patients with chronic hepatitis C: a phase 1B, double-blind, randomised controlled trial

Meike H van der Ree, MD, J Marleen de Vree, MD, Femke Stelma, MD, Sophie Willemse, MD, Marc van der Valk, MD, Svend Rietdijk, MD, Richard Molenkamp, PhD, Janke Schinkel, MD, Ad C van Nuenen, BSc, Prof Ulrich Beuers, MD, Salah Hadi, MD, Marten Harbers, PhD, Eva van der Veer, MSc, Kai Liu, PhD, John Grundy, PhD, Amy K Patick, PhD, Adam Pavlicek, PhD, Jacqueline Blem, BSc, Michael Huang, MD, Paul Grint, MD, Steven Neben, PhD, Neil W Gibson, PhD, Neeltje A Kootstra, PhD, Dr Hendrik W Reesink, MD

Published: 10 January 2017
DOI: http://dx.doi.org/10.1016/S0140-6736(16)31715-9

Summary
Background
miR-122 is an important host factor for hepatitis C virus (HCV) replication. The aim of this study was to assess the safety and tolerability, pharmacokinetics, and antiviral effect of a single dose of RG-101, a hepatocyte targeted N-acetylgalactosamine conjugated oligonucleotide that antagonises miR-122, in patients with chronic HCV infection with various genotypes.

Methods
In this randomised, double-blind, placebo-controlled, multicentre, phase 1B study, patients were randomly assigned to RG-101 or placebo (7:1). We enrolled men and postmenopausal or hysterectomised women (aged 18–65 years) with chronic HCV genotype 1, 3, or 4 infection diagnosed at least 24 weeks before screening who were either treatment naive to or relapsed after interferon-α based therapy. Patients with co-infection (hepatitis B virus or HIV infection), evidence of decompensated liver disease, or a history of hepatocellular carcinoma were excluded. Randomisation was done by an independent, unblinded, statistician using the SAS procedure Proc Plan. The first cohort received one subcutaneous injection of 2 mg/kg RG-101 or placebo; the second cohort received one subcutaneous injection of 4 mg/kg or placebo. Patients were followed up for 8 weeks (all patients) and up to 76 weeks (patients with no viral rebound and excluding those who were randomised to the placebo group) after randomisation. The primary objective was safety and tolerability of RG-101. This trial was registered with EudraCT, number 2013-002978-49.

Findings
Between June 4, 2014, and Oct 27, 2014, we enrolled 32 patients with chronic HCV genotype 1 (n=16), 3 (n=10), or 4 (n=6) infections. In the first cohort, 14 patients were randomly assigned to receive 2 mg/kg RG-101 and two patients were randomly assigned to receive placebo, and in the second cohort, 14 patients were randomly assigned to receive 4 mg/kg RG-101 and two patients were randomly assigned to receive placebo. Overall, 26 of the 28 patients dosed with RG-101 reported at least one treatment-related adverse event. At week 4, the median viral load reduction from baseline was 4·42 (IQR 3·23–5·00) and 5·07 (4·19–5·35) log10 IU/mL in patients dosed with 2 mg/kg RG-101 or 4 mg/kg RG-101. Three patients had undetectable HCV RNA levels 76 weeks after a single dose of RG-101. Viral rebound at or before week 12 was associated with the appearance of resistance associated substitutions in miR-122 binding regions in the 5′ UTR of the HCV genome.

Interpretation
This study showed that one administration of 2 mg/kg or 4 mg/kg RG-101, a hepatocyte targeted N-acetylgalactosamine conjugated anti-miR-122 oligonucleotide, was well tolerated and resulted in substantial viral load reduction in all treated patients within 4 weeks, and sustained virological response in three patients for 76 weeks.

Funding
Regulus Therapeutics, Inc.
http://www.thelancet.com/journals/lancet/onlineFirst

AASLD 2016 - New Combination Therapy Could Cure Hepatitis C With Only 4 Weeks of Treatment

News provided by
American Association for the Study of Liver Diseases (AASLD)

New Combination Therapy Could Cure Hepatitis C With Only 4 Weeks of Treatment

Nov 11, 2016, 08:00 ET

BOSTON, Nov. 11, 2016 /PRNewswire/ -- Two injections of RG-101, in combination with a four-week course of direct-acting antiviral (DAA) therapy, has been shown to clear hepatitis C in patients at both 12 and 24 weeks after treatment in the majority of patients, according to research presented this week at The Liver Meeting® — held by the American Association for the Study of Liver Diseases.

Hepatitis C (HCV) is a liver disease commonly spread through blood. In recent years, DAA treatment has changed the prognosis of this disease from chronic to curable, and the current standard of care for HCV consists of eight to 24 weeks of oral DAA therapy.

RG-101 is a newly developed therapy, delivered through injection that specifically targets the microNRA in the liver that is essential for HCV to continue to replicate (or stay active) in a person's body. Researchers recently assessed the safety and effectiveness of just a four-week treatment of combining RG-101 with oral DAA therapy in people with HCV genotypes one and four.

"Research today focuses on shorter therapies with high efficacy and good safety, explains Mihály Makara, MD; consultant, Hepatology Center of Buda in Budapest, Hungary, and an investigator in the study. "In previous studies, eight weeks seemed to be a limit. RG101 has a pharmacokinetic profile that provides a unique opportunity for shorter treatment."

Dr. Makara's team enrolled 79 participants in their study. The participant's average age was around 45 years, and 54 percent of participants were women. Each participant was placed into one of three DAA treatment groups (with consideration for ensuring people with various disease characteristics were evenly distributed): ledipasvir/sofobuvivr (group one), simeprevir (group two) or daclatasivr (group three), and received these medications along with a 2 mg/kg injection of RG-101 at days one and 29 in the study.

At the beginning of the study, the average viral load (a measurement of HCV in the body) was 638,000 IU/Ml. Seventy-seven percent of participants had genotype one, and the majority (86 percent) had stage zero-to-one fibrosis (scarring of the liver).

Dr. Makara's team noted the combination therapy was well tolerated, and most side effects of the treatment were mild-to-moderate. Additionally, no one stopped taking the combination therapy due to side effects.

One hundred percent of participants in the group one, 96 percent in group two, and 92 percent in group three showed undetectable levels of HCV after 12 weeks post treatment. After 24 weeks post treatment 29 patients were available for follow up. Among these patients, 100 percent in group one, 80 percent in group two, and 89 percent in group three were free from HCV.

"This study showed promising results that curing chronic hepatitis C infection is possible with a very short course of treatment," says Dr. Makara of the findings.

Dr. Makara will present these findings at AASLD's press conference in Room 313 at John B. Hynes Veterans Memorial Convention Center in Boston on Saturday, November 12 at 4pm. The study entitled "RG-101 in Combination with 4 Weeks of Oral Direct Acting Antiviral Therapy Achieves High SVR Rates in Treatment Naïve Genotype 1 and 4 Chronic Hepatitis C Patients" will be presented by Gabor Horvath, MD on Sunday, November 13 at 5:15pm in the Auditorium and Balcony. The corresponding abstract (number 111) can be found in the journal, Hepatology – Special Issue: The 67th Annual Meeting of the American Association for the Study of Liver Diseases: The Liver Meeting 2016.

About the AASLD
AASLD is a medical subspecialty society representing clinicians and researchers in liver disease. The work of our members has laid the foundation for the development of drugs used to treat patients with viral hepatitis. Access to care and support of liver disease research are at the center of AASLD's advocacy efforts.

AASLD is the leading organization of scientists and healthcare professionals committed to preventing and curing liver disease. AASLD was founded in 1950 by a small group of leading liver specialists and has grown to an international society responsible for all aspects of hepatology.

Press releases and additional information about AASLD are available online at www.aasld.org.

Regulus Reports Clinical Hold of RG-101

Update
Tuesday, January 31, 2017

Regulus Announces Continuation of RG-101 Clinical Hold - FDA requests longer-term follow-up data from ongoing studies

Regulus Reports Clinical Hold of RG-101

Timelines of On-going Studies are not Expected to be Impacted
Conference Call Today at 5:00 p.m. ET
16:10 ET from Regulus Therapeutics Inc.

LA JOLLA, Calif., June 27, 2016 /PRNewswire/ -- Regulus Therapeutics Inc. (Nasdaq: RGLS), a biopharmaceutical company leading the discovery and development of innovative medicines targeting microRNAs, today announced it received verbal notice from the U.S. Food and Drug Administration (FDA) that its Investigational New Drug (IND) for RG-101 for the treatment of chronic hepatitis C virus (HCV) infection has been placed on clinical hold. Regulus anticipates it will receive a formal clinical hold letter from the FDA within 30 days and plans to work diligently with the agency to seek the release of the clinical hold.

The FDA initiated the clinical hold after Regulus reported a second serious adverse event (SAE) of jaundice. The SAE occurred in a HCV patient with end-stage renal disease on dialysis enrolled in its on-going Phase I US study 117 days after receiving a single dose of RG-101.

Timelines for Regulus' three on-going studies of RG-101 are not expected to be impacted as all patients have been enrolled and completed their dosing of RG-101 and will continue with protocol scheduled visits. Regulus remains on track to deliver follow-up results from these studies at upcoming relevant scientific meetings.

Conference Call Details

Regulus will host a conference call and webcast today at 5:00 p.m. Eastern Time to discuss today's announcement. A live webcast of the call will be available online at www.regulusrx.com. To access the call, please dial (877) 257-8599 (domestic) or (970) 315-0459 (international) and refer to conference ID 41706266. To access the telephone replay of the call, dial (855) 859-2056 (domestic) or (404) 537-3406 (international), passcode 41706266. The webcast and telephone replay will be archived on the company's website following the call.

About Regulus

Regulus Therapeutics Inc. (Nasdaq: RGLS) is a biopharmaceutical company leading the discovery and development of innovative medicines targeting microRNAs. Regulus has leveraged its oligonucleotide drug discovery and development expertise to develop a well-balanced microRNA therapeutics pipeline complemented by a maturing microMarkersSM biomarkers platform and a rich intellectual property estate to retain its leadership in the microRNA field. Regulus is developing RG-101, a GalNAc-conjugated anti-miR targeting microRNA-122 for the treatment of chronic hepatitis C virus infection, and RG-012, an anti-miR targeting microRNA-21 for the treatment of Alport syndrome, a life-threatening kidney disease driven by genetic mutations with no approved therapy. In addition, RG-125, a GalNAc-conjugated anti-miR targeting microRNA-103/107 for the treatment of NASH in patients with type 2 diabetes/pre-diabetes, has entered Phase I clinical development through its strategic alliance with AstraZeneca. Regulus is also advancing several programs toward clinical development in renal, hepatic and central nervous systems diseases, both independently and with our strategic alliance partners, Sanofi and AstraZeneca. Regulus' commitment to innovation has resulted in multiple peer-reviewed publications in notable scientific journals and has resulted in the formation of strategic alliances with AstraZeneca and Sanofi. Regulus maintains its corporate headquarters in La Jolla, CA. For more information, please visit http://www.regulusrx.com.

About microRNAs

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.

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.

Forward-Looking Statements

Statements contained in this press release regarding matters that are not historical facts are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, including statements associated with the expected ability of Regulus to undertake certain activities and accomplish certain goals (including with respect to development and other activities related to RG-101), the projected timeline of clinical development activities, and expectations regarding future therapeutic and commercial potential of Regulus' business plans, technologies and intellectual property related to microRNA therapeutics and biomarkers being discovered and developed by Regulus. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Words such as "believes," "anticipates," "plans," "expects," "intends," "will," "goal," "potential" and similar expressions are intended to identify forward-looking statements. These forward-looking statements are based upon Regulus' current expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, which include, without limitation, risks associated with the process of discovering, developing and commercializing drugs that are safe and effective for use as human therapeutics, and in the endeavor of building a business around such drugs. These and other risks concerning Regulus' financial position and programs are described in additional detail in Regulus filings with the Securities and Exchange Commission. All forward-looking statements contained in this press release speak only as of the date on which they were made. Regulus undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.

Logo - http://photos.prnewswire.com/prnh/20160519/370141LOGO

Regulus Reports Positive Data/Phase II studies of RG-101 for the treatment of Hepatitis C

Regulus Reports Positive Top-line Data

- Results Demonstrate First Successful Shortened 4-week Treatment Regimen to Date -
- Conference Call Today at 8:30 AM EST -

LA JOLLA, Calif., June 7, 2016 /PRNewswire/ -- Regulus Therapeutics Inc. (NASDAQ:RGLS), a biopharmaceutical company leading the discovery and development of innovative medicines targeting microRNAs, today announced  top-line results from the primary endpoint analysis of one of the company's ongoing Phase II studies of RG-101 for the treatment of Hepatitis C Virus infection (HCV). The study was designed to evaluate a shortened, four-week treatment regimen containing a subcutaneous administration of 2 mg/kg of RG-101 at Day 1 and Day 29, in combination with 4 weeks of once/daily approved anti-viral agents Harvoni®, Olysio®, or Daklinza™.  The study enrolled 79 treatment naïve genotype 1 and 4 HCV patients (Harvoni® arm, n=27, Olysio® arm, n=27, Daklinza™ arm, n=25).  The primary endpoint of the study is virologic response 12 weeks following conclusion of treatment.

Time Since Treatment  Completion	RG-101 + Harvoni	RG-101 + Olysio	RG-101 + Daklinza
Week 12	27/27 (100%)	26/27 (96.3%)	22/24 (91.7%)*
Week 16	21/21 (100%)	19/20 (95.0%)	20/22 (90.9%)
Week 20	14/14 (100%)	13/15 (86.7%)	13/13 (100%)
Week 24	10/10 (100%)	8/10 (80.0%)	8/9 (88.9%)

*One patient missed the Week 12 visit. Viral load results for this patient at Week 8 and 16 were collected and indicate that the patient was a responder at both time points.

The results from this interim analysis demonstrate significant virologic response through 24 weeks of follow-up.  RG-101 plus Harvoni continues to demonstrate 100% response rates.  As previously reported, the combination of RG-101 plus either Olysio or Daklinza monotherapies have seen small numbers of viral relapse.  The results reported today include four new relapses: two in the Olysio arm (weeks 20 and 32) and two in the Daklinza arm (weeks 12 and 24).  RG-101 in combination with four weeks of oral DAA therapy has been generally well tolerated with the majority of adverse events considered mild or moderate, and with no study discontinuations.  Commonly reported adverse events (AEs) included fatigue, headache, and injection site reactions. 

"These data strengthen our conviction in the clinical utility of RG-101 to shorten oral HCV treatment regimens to four weeks or less. We are very encouraged by the consistent trend in safety and efficacy, which positions RG-101 to play an important role in advancing the current treatment options for HCV patients worldwide," said Paul Grint, M.D., President and CEO of Regulus.

Conference Call & Webcast Information
Today at 8:30 a.m. EST, Regulus will host a conference call and webcast to discuss the topline results. A live webcast of the call will be available online at www.regulusrx.com. To access the call, please dial (877) 257-8599 (domestic) or (970) 315-0459 (international) and refer to conference ID 25993262. To access the telephone replay of the call, dial (855) 859-2056 (domestic) or (404) 537-3406 (international), passcode 25993262. The webcast and telephone replay will be archived on the company's website following the call.

About Hepatitis C Virus Infection (HCV)
Hepatitis C is a result of a hepatocyte specific infection induced by the virus known as HCV.  Chronic HCV may lead to significant liver disease, including chronic active hepatitis, cirrhosis, and hepatocellular carcinoma. Up to 185 million people are chronically infected with HCV worldwide, and more than 500,000 people die from HCV annually.  The CDC estimates that there are currently approximately 3.5 million persons infected with HCV in the United States.  HCV shows significant genetic variation in worldwide populations due to its frequent rates of mutation and rapid evolution. There are six genotypes of HCV, with several subtypes within each genotype, which vary in prevalence across the different regions of the world. The response to treatment varies from individual to individual underscoring the inadequacy of existing therapies and highlights the need for combination therapies that not only target the virus but endogenous host factors as well, such as microRNA-122 (miR-122). Regulus believes that its miR-122 antagonist, RG-101, may be a useful agent in emerging combination regimens to address difficult-to-treat genotypes and to potentially expand upon the current therapies available to clinicians treating HCV patients.

About RG-101 for HCV
RG-101 is Regulus' wholly-owned, GalNAc-conjugated anti-miR targeting miR-122, which the HCV virus uses to replicate. Therapies that interfere with miR-122 could inhibit viral replication, acting earlier in the viral life cycle than currently approved oral agents.  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. 

Earlier this year, Regulus began enrolling patients in an open-label Phase II clinical trial combining RG-101 and GSK2878175 for the treatment of HCV to evaluate the potential to achieve sustained viral responses post treatment with a single subcutaneous administration of 4 mg/kg of RG-101 in combination with daily oral administrations of 20 mg of GSK2878175 for up to 12 weeks in treatment-naïve patients chronically infected with HCV genotypes 1 and 3.  Regulus and GSK anticipate reporting interim results from this study by year-end.  In an expanded collaboration with GSK, the companies plan to conduct a multi-centered, randomized, dose-ranging Phase II study evaluating the combination of RG-101 and GSK's long-acting parenteral ("LAP") formulation of GSK2878175 as a potential single-visit cure in patients chronically infected with HCV.  This study will be conducted outside the United States and is planned to begin in the fourth quarter of 2016.  Based on predicted enrollment rates, interim results from this expanded collaboration should be available in the second half of 2017, enabling a potential initiation of a pivotal study in late 2017. 

About microRNAs
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.

About Regulus
Regulus Therapeutics Inc. (NASDAQ:RGLS) is a biopharmaceutical company leading the discovery and development of innovative medicines targeting microRNAs.  Regulus has leveraged its oligonucleotide drug discovery and development expertise to develop a well-balanced microRNA therapeutics pipeline complemented by a maturing microMarkers^SM biomarkers platform and a rich intellectual property estate to retain its leadership in the microRNA field.  Regulus is developing RG-101, a GalNAc-conjugated anti-miR targeting microRNA-122 for the treatment of chronic hepatitis C virus infection, and RG-012, an anti-miR targeting microRNA-21 for the treatment of Alport syndrome, a life-threatening kidney disease driven by genetic mutations with no approved therapy.  In addition, RG-125, a GalNAc-conjugated anti-miR targeting microRNA-103/107 for the treatment of NASH in patients with type 2 diabetes/pre-diabetes, has entered Phase I clinical development through its strategic alliance with AstraZeneca.  Regulus is also advancing several programs toward clinical development in renal, hepatic and central nervous systems diseases, both independently and with our strategic alliance partners, Sanofi and AstraZeneca. Regulus' commitment to innovation has resulted in multiple peer-reviewed publications in notable scientific journals and has resulted in the formation of strategic alliances with AstraZeneca and Sanofi.  Regulus maintains its corporate headquarters in La Jolla, CA.  For more information, please visit http://www.regulusrx.com.

Forward-Looking Statements
Statements contained in this press release regarding matters that are not historical facts are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, including statements associated with the expected ability of Regulus to undertake certain activities and accomplish certain goals (including with respect to development and other activities related to RG-101), the projected timeline of clinical development activities, and expectations regarding future therapeutic and commercial potential of Regulus' business plans, technologies and intellectual property related to microRNA therapeutics and biomarkers being discovered and developed by Regulus.  Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Words such as "believes," "anticipates," "plans," "expects," "intends," "will," "goal," "potential" and similar expressions are intended to identify forward-looking statements. These forward-looking statements are based upon Regulus' current expectations and involve assumptions that may never materialize or may prove to be incorrect.  Actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, which include, without limitation, risks associated with the process of discovering, developing and commercializing drugs that are safe and effective for use as human therapeutics, and in the endeavor of building a business around such drugs.  These and other risks concerning Regulus' financial position and programs are described in additional detail in Regulus filings with the Securities and Exchange Commission.  All forward-looking statements contained in this press release speak only as of the date on which they were made. Regulus undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.

Interim Data on RG-101 at International Liver Congress™ (ILC 2016)

International Liver Congress 2016 Press Release:

Shorter treatment course potentially on the horizon for Hepatitis C patients
April 15, 2016
Press Releases

Investigational treatment, RG-101 combined with direct-acting antivirals shows potential to be effective in Hepatitis C with a ‘shorter than standard’ four week course

April 15, 2016, Barcelona, Spain: Data from a Phase 2 clinical trial show that an investigational injectable treatment known as RG-101 in combination with a four week course of oral direct-acting antiviral (DAA) treatment was well tolerated and resulted in high virologic response rates post-treatment among Hepatitis C (HCV) infected patients with genotypes 1 and 4, who had not been treated previously. The findings, presented today at The International Liver Congress™ 2016 in Barcelona, Spain are an interim analysis, with ongoing research to assess virologic response over a 48 week follow-up period to further assess the safety and efficacy of a four week treatment course.

The investigational treatment, RG-101 works on microRNA-122, which the virus uses to replicate. Drugs that interfere with miR-122 could inhibit HCV replication, acting earlier in the viral lifecycle than currently approved HCV protease, polymerase or NS5A inhibitors.1 The current standard of care treatment in HCV consists of eight-12 weeks of DAA oral agents.2

“These early results indicate the potential for RG-101 with oral DAA combination therapy to provide an effective Hepatitis C regimen for patients with a short treatment course of just four weeks,” said Dr Mihaly Makara from the Buda Hepatology Centre, Budapest, Hungary, and lead study author. “We very much hope the long-term, 48-week follow-up data follows the same trend.”

This international study enrolled 79 patients with chronic HCV, genotype 1 or 4 who had not previously received treatment. Each patient received a 2mg/kg injection of RG-101 on Day one, with a four week course of oral DAAs (either ledipasvir/sofosbuvir, simeprevir, or daclatasvir), following by a second 2mg/kg injection of RG-101 on Day 29. The mean baseline viral load among patients was 5.805 (log10) IU/mL.

Interim analysis showed that 97.4% (37/38) and 100% (14/14) of patients at eight and 12 weeks respectiviely had a high virologic response. This was determined by assessing HCV levels ‘below the lower limit of quantification’ (<12 IU/mL), using the Abbot RealTimeHCV Assay, a consolidated HCV viral load and HCV genotype testing method.3 

The combination therapy was generally well tolerated, with the majority of side effects reported being mild in nature, including headache and fatigue, reported in 11.4% of patients.

“It is encouraging to see a potential treatment combination on the horizon that could limit treatment duration for patients,” said Professor Tom Hemming Karlsen, EASL Vice Secretary. “We will be eagerly awaiting the 48 week follow-up data.”

Regulus Press Release

April 15, 2016

Regulus Presents Additional Interim Data on RG-101 at International Liver Congress™ (ILC 2016)
- Continued High Virologic Response Rates across All Treatment Arms Out through 24 weeks of Follow Up -
- Data Supports RG-101's Potential to Reduce Treatment Regimen to 4 Weeks -
- Conference Call at 8AM EDT (2PM CEST) Today -

LA JOLLA, Calif., April 15, 2016 /PRNewswire/ -- Regulus Therapeutics Inc. (NASDAQ:RGLS), a biopharmaceutical company leading the discovery and development of innovative medicines targeting microRNAs, today announced additional interim results from one of the company's ongoing Phase II studies of RG-101 for the treatment of Hepatitis C Virus infection (HCV) during an oral presentation at the International Liver Congress 2016 (ILC 2016) taking place April 13-17 in Barcelona, Spain. The study was designed to evaluate a shortened, four-week treatment regimen containing a subcutaneous administration of 2 mg/kg of RG-101 at Day 1 and Day 29, in combination with 4 weeks of once/daily approved anti-viral agents Harvoni®, Olysio®, or Daklinza™.  The study enrolled 79 treatment naïve genotype 1 and 4 HCV patients (Harvoni® arm, n=27, Olysio® arm, n=27, Daklinza™ arm, n=25). 

Today, Regulus reported longer-term follow up results across all arms of the ongoing study:

 

Follow-Up After 2nd Dose of RG-101	RG-101 + Harvoni®	RG-101 + Olysio®	RG-101 + Daklinza™
Week 8	21/21 (100%)	21/21 (100%)	20/22* (90.9%)
Week 12	14/14 (100%)	14/15* (93.3%)	12/12 (100%)
Week 16	9/9 (100%)	8/9* (88.9%)	9/9 (100%)
Week 20	2/2 (100%)	2/2 (100%)	2/2 (100%)
Week 24	1/1 (100%)	2/2 (100%)	-- (--)

 

Response defined as HCV RNA viral load below LLOQ using RealTime HCV Assay (Abbott) with LLOQ = 12

 

 *Includes one relapse patient in the Olysio arm (week 12) and one relapse patient in the Daklinza arm (week 8)

 

"We believe the data reported at the ILC meeting further demonstrate the clinical utility of RG-101 to shorten oral HCV treatment regimens to just four weeks," said Paul Grint, M.D., President and CEO of Regulus.  "With interim data now through 24 weeks of follow-up, the consistent trend in efficacy and safety is encouraging, which supports the potential of RG-101 to become a backbone agent in combination with all classes of oral therapies. Throughout the year, we look forward to obtaining more data from multiple studies across our broad Phase 2 development program."

 

To date, RG-101 has been generally well tolerated with the majority of adverse events considered mild or moderate, and with no study discontinuations.  Changes in pharmacodynamic markers are indicative of effective target engagement and consistent with the company's prior experience with miR-122 inhibition.  The primary endpoint analysis (12 week follow up) for all 79 patients in the study is anticipated to be reported in late Q2 2016.

 

The investigator slides presented at the ILC 2016 meeting are available on the investor relations page of Regulus' website at www.regulusrx.com.

 

Conference Call & Webcast Information

Today at 8:00 a.m. EDT, Regulus will host a conference call and webcast to discuss the ILC 2016 interim results. A live webcast of the call will be available online at www.regulusrx.com.  To access the call, please dial (877) 257-8599 (domestic) or (970) 315-0459 (international) and refer to conference ID 89822720.  To access the telephone replay of the call, dial (855) 859-2056 (domestic) or (404) 537-3406 (international), passcode 89822720. The webcast and telephone replay will be archived on the company's website following the call. 

 

About Hepatitis C Virus Infection (HCV)

Hepatitis C is a result of a hepatocyte specific infection induced by the virus known as HCV.  Chronic HCV may lead to significant liver disease, including chronic active hepatitis, cirrhosis, and hepatocellular carcinoma. Up to 185 million people are chronically infected with HCV worldwide, and more than 500,000 people die from HCV annually.  The CDC estimates that there are currently approximately 3.5 million persons infected with HCV in the United States.  HCV shows significant genetic variation in worldwide populations due to its frequent rates of mutation and rapid evolution. There are six genotypes of HCV, with several subtypes within each genotype, which vary in prevalence across the different regions of the world. The response to treatment varies from individual to individual underscoring the inadequacy of existing therapies and highlights the need for combination therapies that not only target the virus but endogenous host factors as well, such as microRNA-122 (miR-122). Regulus believes that its miR-122 antagonist, RG-101, may be a useful agent in emerging combination regimens to address difficult-to-treat genotypes and to potentially expand upon the current therapies available to clinicians treating HCV patients.

 

About RG-101 for HCV

RG-101 is Regulus' wholly-owned, GalNAc-conjugated anti-miR targeting miR-122, which the HCV virus uses to replicate. Therapies that interfere with miR-122 could inhibit viral replication, acting earlier in the viral life cycle than currently approved oral agents.  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. 

Regulus has reported 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.

 

About microRNAs

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.

 

About Regulus

Regulus Therapeutics Inc. (NASDAQ: RGLS) is a biopharmaceutical company leading the discovery and development of innovative medicines targeting microRNAs.  Regulus has leveraged its oligonucleotide drug discovery and development expertise to develop a well-balanced microRNA therapeutics pipeline complemented by a maturing microMarkers^SM biomarkers platform and a rich intellectual property estate to retain its leadership in the microRNA field.  Regulus is developing RG-101, a GalNAc-conjugated anti-miR targeting microRNA-122 for the treatment of chronic hepatitis C virus infection, and RG-012, an anti-miR targeting microRNA-21 for the treatment of Alport syndrome, a life-threatening kidney disease driven by genetic mutations with no approved therapy.  In addition, RG-125, a GalNAc-conjugated anti-miR targeting microRNA-103/107 for the treatment of NASH in patients with type 2 diabetes/pre-diabetes, has entered Phase I clinical development through its strategic alliance with AstraZeneca.  Regulus is also advancing several programs toward clinical development in orphan disease indications, oncology and fibrosis.  Regulus' commitment to innovation has resulted in multiple peer-reviewed publications in notable scientific journals and has resulted in the formation of strategic alliances with AstraZeneca and Sanofi.  Regulus maintains its corporate headquarters in La Jolla, CA.  For more information, please visit http://www.regulusrx.com.

 

Forward-Looking Statements

Statements contained in this press release regarding matters that are not historical facts are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, including statements associated with the expected ability of Regulus to undertake certain activities and accomplish certain goals (including with respect to development and other activities related to RG-101), the projected timeline of clinical development activities, and expectations regarding future therapeutic and commercial potential of Regulus' business plans, technologies and intellectual property related to microRNA therapeutics and biomarkers being discovered and developed by Regulus.  Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Words such as "believes," "anticipates," "plans," "expects," "intends," "will," "goal," "potential" and similar expressions are intended to identify forward-looking statements. These forward-looking statements are based upon Regulus' current expectations and involve assumptions that may never materialize or may prove to be incorrect.  Actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, which include, without limitation, risks associated with the process of discovering, developing and commercializing drugs that are safe and effective for use as human therapeutics, and in the endeavor of building a business around such drugs.  These and other risks concerning Regulus' financial position and programs are described in additional detail in Regulus filings with the Securities and Exchange Commission.  All forward-looking statements contained in this press release speak only as of the date on which they were made. Regulus undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.

RG-101 Interim Analysis Shows 97% Response at 8 Week Follow-Up- RG-101 Containing Regimen

RG-101 Interim Analysis Shows 97% Response at 8 Week Follow-Up- RG-101 Containing Regimen 

Has Potential to Reduce Harvoni®, Olysio®, or Daklinza™ Rx to 4 Week Duration -

- Regulus to Accelerate Development of RG-101 as Backbone Therapy in HCV -

- Conference Call at 8:30am EST Today -
LA JOLLA, Calif., Feb. 17, 2016 /PRNewswire/ -- Regulus Therapeutics Inc. (NASDAQ: RGLS), a biopharmaceutical company leading the discovery and development of innovative medicines targeting microRNAs, today announced interim results from one of the company's ongoing Phase II studies of RG-101 for the treatment of Hepatitis C Virus infection (HCV). The study was designed to evaluate a shortened, four-week treatment regimen containing a subcutaneous administration of 2 mg/kg of RG-101 at Day 1 and Day 29, in combination with 4 weeks of once/daily approved anti-viral agents Harvoni®, Olysio®, or Daklinza™. The study enrolled 79 treatment naïve genotype 1 and 4 HCV patients (Harvoni® arm, n=27, Olysio® arm, n=27, Daklinza™ arm, n=25). Thirty-eight patients have been evaluated through 8 weeks of follow up. Ninety-seven percent of those patients (37/38) had HCV RNA viral load measurements below the limit of quantification. To date, RG-101 has been generally well tolerated with the majority of adverse events considered mild or moderate, and with no study discontinuations. For those patients through 12 weeks of follow-up, 100% remained below the limit of quantification (14/14). The primary endpoint analysis (12 week follow up) for all 79 patients in the study are anticipated to be reported in late Q2 2016.

"These sustained virologic responses demonstrate the potential ability of RG-101 to successfully reduce currently marketed oral treatment regimens to just four weeks, a major clinical breakthrough that the HCV field has not been able to achieve until today and I look forward to future results," said Eric Lawitz, M.D., Vice President, Scientific and Research Development, The Texas Liver Institute, and Clinical Professor of Medicine, University of Texas Health Science Center in San Antonio. "In addition, I believe this novel approach might allow treating physicians to overcome compliance issues in a wide variety of patient populations."

"The potent antiviral activity and sustained, durable responses observed from this interim analysis, provide evidence that RG-101 may have clinical utility as a potential backbone agent in combination with oral therapies to treat a wide range of HCV patients," said Paul Grint, M.D., President and CEO of Regulus. "Based on the results announced today, Regulus intends to accelerate development of RG-101 given its promising potential to shorten treatment regimens."

Conference Call & Webcast Information

Today at 8:30 a.m. EST, Regulus will host a conference call and webcast to discuss these interim results. A live webcast of the call will be available online at www.regulusrx.com. To access the call, please dial (877) 257-8599 (domestic) or (970) 315-0459 (international) and refer to conference ID 54426274. To access the telephone replay of the call, dial (855) 859-2056 (domestic) or (404) 537-3406 (international), passcode 54426274. The webcast and telephone replay will be archived on the company's website following the call.

About Hepatitis C Virus Infection (HCV)

Hepatitis C is a result of a hepatocyte specific infection induced by the virus known as HCV. Chronic HCV may lead to significant liver disease, including chronic active hepatitis, cirrhosis, and hepatocellular carcinoma. Up to 185 million people are chronically infected with HCV worldwide, and more than 500,000 people die from HCV annually. The CDC estimates that there are currently approximately 3.5 million persons infected with HCV in the United States. HCV shows significant genetic variation in worldwide populations due to its frequent rates of mutation and rapid evolution. There are six genotypes of HCV, with several subtypes within each genotype, which vary in prevalence across the different regions of the world. The response to treatment varies from individual to individual underscoring the inadequacy of existing therapies and highlights the need for combination therapies that not only target the virus but endogenous host factors as well, such as microRNA-122. Regulus believes that its miR-122 antagonist, RG-101, may be a useful agent in emerging combination regimens to address difficult-to-treat genotypes and to potentially expand upon the current therapies available to clinicians treating HCV patients.

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, followed by 28 days of once daily DAAs 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 analysis at 12 weeks following conclusion of treatment in late Q2 2016.

In the first-quarter of 2016, Regulus, in collaboration with GSK, plans to initiate 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 expected to complete in the first half of 2016 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.

About microRNAs

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.

About Regulus

Regulus Therapeutics Inc. (NASDAQ: RGLS) is a biopharmaceutical company leading the discovery and development of innovative medicines targeting microRNAs. Regulus has leveraged its oligonucleotide drug discovery and development expertise to develop a well-balanced microRNA therapeutics pipeline complemented by a maturing microMarkersSM biomarkers platform and a rich intellectual property estate to retain its leadership in the microRNA field. Regulus is developing RG-101, a GalNAc-conjugated anti-miR targeting microRNA-122 for the treatment of chronic hepatitis C virus infection, and RG-012, an anti-miR targeting microRNA-21 for the treatment of Alport syndrome, a life-threatening kidney disease driven by genetic mutations with no approved therapy. In addition, RG-125, a GalNAc-conjugated anti-miR targeting microRNA-103/107 for the treatment of NASH in patients with type 2 diabetes/pre-diabetes, has entered Phase I clinical development through its strategic alliance with AstraZeneca. Regulus is also advancing several programs toward clinical development in orphan disease indications, oncology and fibrosis. Regulus' commitment to innovation has resulted in multiple peer-reviewed publications in notable scientific journals and has resulted in the formation of strategic alliances with AstraZeneca and Sanofi. Regulus maintains its corporate headquarters in La Jolla, CA. For more information, please visit http://www.regulusrx.com.

Forward-Looking Statements

Statements contained in this press release regarding matters that are not historical facts are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, including statements associated with the expected ability of Regulus to undertake certain activities and accomplish certain goals (including with respect to development and other activities related to RG-101), the projected timeline of clinical development activities, and expectations regarding future therapeutic and commercial potential of Regulus' business plans, technologies and intellectual property related to microRNA therapeutics and biomarkers being discovered and developed by Regulus. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Words such as "believes," "anticipates," "plans," "expects," "intends," "will," "goal," "potential" and similar expressions are intended to identify forward-looking statements. These forward-looking statements are based upon Regulus' current expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, which include, without limitation, risks associated with the process of discovering, developing and commercializing drugs that are safe and effective for use as human therapeutics, and in the endeavor of building a business around such drugs. These and other risks concerning Regulus' financial position and programs are described in additional detail in Regulus filings with the Securities and Exchange Commission. All forward-looking statements contained in this press release speak only as of the date on which they were made. Regulus undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.

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Regulus Completes RG-101 Enrollment in Phase II Combination Study For HCV

Regulus Completes RG-101 Enrollment in Phase II Combination Study

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SOURCE Regulus Therapeutics Inc.

- Additional studies on track for enrollment in H12016 with multiple data read-outs expected throughout 2016 -

LA JOLLA, Calif., Jan. 21, 2016 /PRNewswire/ -- Regulus Therapeutics Inc. (NASDAQ: RGLS), a biopharmaceutical company leading the discovery and development of innovative medicines targeting microRNAs, today announced key 2016 programmatic goals to advance RG-101, Regulus' wholly-owned therapy for the treatment of HCV. RG-101 is a GalNAc-conjugated anti-miR targeting miR-122, a host factor for HCV infection. Regulus' Phase II program and development strategy for RG-101 includes evaluating RG-101 in combination studies with different approved direct-acting anti-HCV agents (DAAs), in combination with an investigational oral DAA that can be formulated into a Long Acting Parenteral formulation for injection (LAP) providing the potential for a single-visit therapy, and in certain underserved HCV patient populations.

"Regulus begins 2016 with a multi-faceted clinical development plan for RG-101 in both Europeand the United States which we believe, if successful, will position our lead microRNA therapeutic well against the backdrop of the rapidly evolving HCV landscape," said Paul Grint, M.D., President and CEO of Regulus. "Regulus aims to enhance the value of RG-101 by maturing its profile in combination with oral agents and in certain underserved patient populations and we look forward to reporting results from multiple studies throughout 2016."

Phase II DAA Combination Studies:
Enrollment Complete in Phase II Combination Study; Interim Results in mid-Feb. Regulus announced today that patient enrollment is now complete in an ongoing Phase II study evaluating the combination of RG-101 with multiple approved DAAs. Treatment-naïve patients chronically infected with genotypes 1 or 4 were randomized to one of three treatment arms (n=78). Patients receive a single subcutaneous injection of 2 mg/kg of RG-101, followed by 28 days of once/daily DAAs 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 interim results from this study in mid-February 2016 in time for submission for potential publication at the European Association for the Study of the Liver (EASL) annual meeting. Primary endpoint results for sustained viral response data 12 weeks following conclusion of treatment (SVR12) are anticipated to be disclosed late in Q2 2016.
GSK Combination Study on Track; Data by Year-End 2016. In March 2016, Regulus plans to initiate a multi-center, open-label Phase II study evaluating the combination of a single subcutaneous injection of 4 mg/kg of RG-101 and daily oral administrations of 20 mg of GSK2878175, an investigational non-nucleoside NS5B polymerase inhibitor, for up to 12 weeks in treatment-naïve patients chronically infected with HCV genotypes 1 and 3. Concurrently, GSK will work on developing a "LAP" formulation of GSK2878175 as a single intra-muscular injection, providing the potential for a single-visit therapeutic treatment for HCV that 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 current collaboration agreement. Regulus expects to report safety and efficacy data from the GSK Phase II study before the end of 2016.

IND-Opening Study for Underserved HCV Population(s):
Clearance Received from US FDA to Initiate First Study in US; Enrollment Ongoing, Data by YE 2016. Regulus announced today that enrollment has commenced 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. The Phase I study is designed to have three treatment arms (n=24): (i) healthy volunteers (n=8); (ii) patients with severe renal impairment or ESRD (n=8); and (iii) HCV patients with severe renal impairment or ESRD (n=8). Enrollment is expected to be complete in the first half of 2016 with efficacy data from the HCV/severe renal impairment or ESRD arm anticipated in the second half of 2016.

About microRNAs
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.

Regulus Therapeutics (RGLS) Announces Multiple RG-101 Presentations at ILC 2015

Regulus Therapeutics (RGLS) Announces Multiple RG-101 Presentations at ILC 2015

Regulus Therapeutics (Nasdaq: RGLS) announced that multiple presentations related to RG-101, a GalNAc-conjugated anti-miR targeting microRNA-122 ("miR-122") for the treatment of chronic Hepatitis C Virus (HCV) infection, are scheduled for presentation at The International Liver Congress™ (ILC) 2015, April 22-26 in Vienna, Austria.

Investigators will present new clinical data on RG-101 in a late-breaking oral presentation titled "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" on April 25, 2015 from 17:30-17:45 pm CET (abstracts relating to late-breaking oral presentations are embargoed until presentation). In addition, in multiple poster presentations, clinical and preclinical results will be presented on RG-101, including pharmacokinetics and pharmacodynamics in healthy volunteers, pharmacokinetics, pharmacodynamics, and toxicity in rodents and non-human primates, and efficacy in a human hepatocyte chimeric mouse model of HCV.

"Regulus and our clinical investigators are extremely pleased to be making several presentations on RG-101 for the treatment of HCV during the International Liver Congress that will further demonstrate the compound's novel mechanism and potential," said Neil W. Gibson, Ph.D., Chief Scientific Officer of Regulus. "We also look forward to showcasing the significant progress we are making in advancing microRNA therapeutics to patients with an expanded examination of RG-101's preclinical profile."

"Pharmacokinetics and Pharmacology of RG-101, a Novel GalNAc-Conjugated Oligonucleotide Targeting microRNA-122, in Healthy Volunteers" (Abstract: P1352)

Regulus will present clinical data from the healthy volunteer portion of the completed clinical study of RG-101. These data demonstrate that RG-101, tested at single doses from 0.5 mg/kg to 8 mg/kg, shows dose responsive pharmacodynamics with maximal changes at 2 mg/kg in healthy volunteers and no drug-drug interactions observed when administered in combination with the direct acting anti-viral drug simeprevir (OLYSIO™). In addition, RG-101 was well tolerated in healthy volunteers at all doses tested.

"Pharmacokinetics, Pharmacodynamics, and Toxicity Profile of RG-101, a Novel GalNAc-conjugated Hepatocyte-Targeting Inhibitor of microRNA-122, in Rodents and Cynomolgus Monkeys" (Abstract: P0907)

Regulus will present preclinical data that supports the evaluation of RG-101 in human clinical trials. RG-101 was administered subcutaneously at doses from 0.1 to 450 mg/kg in mice and 1.5 to 150 mg/kg in monkeys. Following subcutaneous administration, rapid absorption and clearance of RG-101 was observed in plasma with rapid uptake and efficient conversion to unconjugated oligonucleotide in the liver and reduced concentrations in kidney. No Observed Adverse Effect Levels (NOAELs) of 450 mg/kg and 45 mg/kg in mouse and monkey, respectively, were declared for RG-101. Taken together, the pharmacokinetic, pharamacodynamic, and toxicology profile of RG-101 suggests that conjugation of oligonucleotides to GalNAc produces significant advantages that are likely to translate to more effective and safer clinical outcomes.

"RG-101, a Novel GalNAc-conjugated Inhibitor of microRNA-122, Demonstrates Significant Viral Load Reduction and Reduces Liver Steatosis in Human Hepatocyte Chimeric Mice Infected with Genotype 1A or Hard-to-Treat Genotype 3A Hepatitis C Virus (HCV)" (Abstract: P0904)

Regulus will present preclinical data demonstrating the effects of RG-101 on HCV infection using human hepatocyte chimeric mice (PXB-Mice) infected with genotype 1A and genotype 3A HCV isolates. In this model, RG-101 was effective against multiple HCV genotypes in vivo and fatty liver (steatosis) in PXB-Mice was dramatically reduced in line with the known role of miR-122 in hepatic lipid metabolism. These results suggest that RG-101 may have additional benefits beyond reduction in HCV viral titer.

The abstracts related to the poster presentations can be accessed through the ILC/EASL website at https://events.easl.eu/EventProgramme/ILC2015/POSTER.aspx

RG-101 - Decreased viral load in patients with various HCV genotypes

Commentary @ Healio

Oct 23

Potential drug decreased viral load in patients with various HCV genotypes
Interim results from an ongoing clinical trial revealed that a microRNA therapeutic was well-tolerated and safe for the treatment of hepatitis C virus infection, according to a company release from Regulus Therapeutics.

In the trial, currently taking place in the Netherlands, RG-101 (Regulus Therapeutics), a GalNac-conjugated anti-miR targeting microRNA-122, was dosed at 2 mg/kg and given to 14 patients with HCV and various genotypes. This treatment resulted in significant and sustained reductions in HCV RNA among the patients, including those with difficult to treat genotypes and experienced viral relapse after a prior interferon-based regimen, according to the release. 

Continue reading @ Healio

Investment Commentary
Regulus Gets Seat at Hep C Table With Today's Impressive Drug Results 

By Adam Feuerstein
Oct 22
But you're getting way ahead of the facts if you think Regulus has a game-changing hepatitis C therapy in its grasp already. There's a lot more questions to be answered.
Continue reading @ TheStreet.com

Press Release

A Single Subcutaneous Dose of 2mg/kg of RG-101, Regulus' Wholly-Owned, GalNac-Conjugated anti-miR Targeting microRNA-122, Demonstrates 4.1 log10 Mean Viral Load Reduction as Monotherapy at Day 29 in Patients with Varied HCV Genotypes and Treatment History

- Interim Results from Ongoing Study Demonstrate Human Proof-of-Concept and Underscore the Value of Regulus' microRNA Platform -
- Conference Call Today at 8:30 a.m. EDT to Discuss Results -

LA JOLLA, Calif., Oct. 22, 2014 /PRNewswire/ -- Regulus Therapeutics Inc. (NASDAQ:RGLS), a biopharmaceutical company leading the discovery and development of innovative medicines targeting microRNAs, today announced that it has demonstrated human proof-of-concept with a microRNA therapeutic from an ongoing clinical study evaluating RG-101, a wholly-owned, GalNac-conjugated anti-miR targeting microRNA-122 ("miR-122"), for the treatment of hepatitis C virus infection ("HCV"). Interim results from the ongoing clinical study demonstrate that treatment with a single subcutaneous dose of 2 mg/kg of RG-101 as monotherapy resulted in significant and sustained reductions in HCV RNA in a varied group of patients, including difficult to treat genotypes and patients who experienced viral relapse after a prior IFN-containing regimen. Additionally, RG-101 was safe and well tolerated and has demonstrated a very favorable pharmacokinetic profile to date, which may allow for combination with oral direct-acting antiviral ("DAA") agents to treat HCV.

Interim results from the ongoing clinical study are summarized below:

In the first dose cohort of part IV of the ongoing study, 16 HCV patients were enrolled with multiple genotypes, 10 GT1s, 5 GT3s, and 1 GT4. 14 patients, 8 naïve and 6 patients who experienced viral relapse after a prior IFN-containing regimen, received a single subcutaneous dose of 2 mg/kg of RG-101 as monotherapy while 2 patients received placebo.

In the 14 HCV treated patients, there was a mean viral load reduction of 4.1 log10 at day 29 (range -5.8 log10 to -2.3 log10).

6 out of 14 patients had HCV RNA levels below the limit of quantification at day 29 and the 3 patients from this group who have reached day 57 still have HCV RNA levels below the limit of quantification.
Viral load reduction occurs within the first 96 hours and virologic response is not influenced by IL-28 genotype.
Due to the long-lasting and sustained virologic effect seen, the ongoing study protocol has been amended to follow patients for up to six months after dosing to evaluate the possibility for certain patients to achieve viral cure after a single dose of RG-101.

There were no drug-drug interactions from part III of the ongoing study in which RG-101 was combined with simeprevir (OLYSIO™), an approved oral DAA (protease inhibitor), and the combination had no effect on the pharmacokinetic profile of RG-101 or simeprevir (OLYSIO™).
RG-101 is safe and well tolerated with no serious adverse events reported to date.

"We are very excited to have demonstrated our first human proof-of-concept results with a microRNA therapeutic from the ongoing study of RG-101, which is a significant milestone in Regulus' history, and represents a key achievement under our 'Clinical Map Initiative'," said Kleanthis G. Xanthopoulos, Ph.D., President and Chief Executive Officer of Regulus. "We believe these interim data are exceptional and provide strong evidence to support the rapid advancement of RG-101 into future clinical studies, while presenting a clear opportunity for a potentially disruptive therapy to the current HCV treatment paradigm."

"RG-101 is the first microRNA therapeutic in clinical development to combine the most advanced RNA technologies from three leading RNA therapeutics companies; chemistry 2.5 from Isis, GalNAc conjugate from Alnylam, and Regulus' unique and proprietary chemistry including the novel linker that facilitates the release of the parent oligonucleotide after hepatocyte uptake," said Neil W. Gibson, Ph.D., Chief Scientific Officer. "We believe the innovative design of RG-101 has led to achievement of our first human proof-of-concept results, and hope these findings will advance the growth of our microRNA therapeutics pipeline."

"We are very pleased and encouraged with the interim results and believe these findings strongly support the rapid advancement of RG-101 into Phase II development," said Paul Grint, M.D., Regulus' Chief Medical Officer. "Currently, we plan to file an Investigational New Drug Application with the U.S. Food and Drug Administration in the first quarter of 2015 and plan to initiate a Phase II DAA combination study of RG-101 in HCV patients in the second quarter of 2015. In addition, we look forward to reporting additional data from the ongoing study in the first half of next year."

"The efficacy and sustained viral response seen with a single dose of RG-101 is very promising and it was encouraging to see response across a diversity of genotypes and treatment experience in this clinical trial. Additionally, all patients in the first cohort on active therapy demonstrated a viral response to RG-101, which is also very encouraging," said Dr. Eric Lawitz, M.D., Vice President, Scientific and Research Development, The Texas Liver Institute, and Clinical Professor of Medicine, University of Texas Health Science Center in San Antonio. "These findings suggest the clinical benefit of utilizing a unique mechanism of action to potentially treat difficult patient populations. There may be an opportunity to improve upon the current real world compliance issues with therapies such as RG-101 that may be administered subcutaneously by a clinician. I look forward to seeing RG-101 advance into future clinical trials."

Conference Call & Webcast Information

Regulus will host a conference call and webcast at 8:30 a.m. Eastern Daylight Time today to discuss its interim results from the ongoing clinical study of RG-101 for the treatment of HCV. A live webcast of the call will be available online at www.regulusrx.com. To access the call, please dial (877) 257-8599 (domestic) or (970) 315-0459 (international) and refer to conference ID 24374685. To access the telephone replay of the call, dial (855) 859-2056 (domestic) or (404) 537-3406 (international), passcode ID 24374685. The webcast and telephone replay will be archived on the company's website for ninety days following the call.

About RG-101 for HCV

RG-101 is a wholly-owned, GalNAc-conjugated anti-miR targeting miR-122 for the treatment of HCV. Regulus is currently evaluating RG-101 in an ongoing study being conducted in the Netherlands. The study has the following four parts: (I) a single ascending-dose study in which healthy volunteer subjects receive 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 receive 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 receive a single subcutaneous dose of RG-101 in combination with simeprevir, an approved DAA; and (IV) a single-dose study in which HCV patients receive 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 DAA, such as simeprevir, may have on the pharmacokinetics of RG-101. Up to 100 healthy volunteer subjects and HCV patients with multiple HCV genotypes and treatment history are planned to be enrolled.

Today, Regulus reported interim results from the above study and plans to report additional results from the ongoing study in 2015.

About microRNAs

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.

About Hepatitis C Virus Infection (HCV)

Hepatitis C is a result of a hepatocyte specific infection induced by the virus known as HCV. Chronic HCV may lead to significant liver disease, including chronic active hepatitis, cirrhosis, and hepatocellular carcinoma. Up to 170 million people are chronically infected with HCV worldwide, and more than 350,000 people die from HCV annually. The CDC estimates that there are currently approximately 3.2 million persons infected with HCV in the United States. HCV shows significant genetic variation in worldwide populations due to its frequent rates of mutation and rapid evolution. There are six genotypes of HCV, with several subtypes within each genotype, which vary in prevalence across the different regions of the world. The response to treatment varies from individual to individual underscoring the inadequacy of existing therapies and highlights the need for combination therapies that not only target the virus but endogenous host factors as well, such as microRNA-122.

Regulus believes that its' miR-122 antagonist, RG-101, may be a useful agent in emerging combination regimens to address difficult-to-treat genotypes and to potentially expand upon the current therapies available to clinicians treating HCV patients.

Update to the 'Clinical Map Initiative'

Launched in February 2014, Regulus' 'Clinical Map Initiative' outlines certain corporate goals to advance its microRNA therapeutics pipeline over the next several years. In October 2014, Regulus demonstrated human proof-of-concept with a microRNA therapeutic, RG-101, a wholly-owned, GalNac-conjugated anti-miR targeting microRNA-122 for the treatment of HCV. The company plans to rapidly advance RG-101 in clinical development and expects to initiate a Phase II DAA combination study in HCV patients in the second quarter of 2015. In addition, Regulus expects to initiate a Phase I clinical study of RG-012 for the treatment of Alport syndrome, nominate a third microRNA candidate for clinical development by the end of 2014, and maintain a strong financial position and end 2014 with at least $75.0 million in cash, cash equivalents and short-term investments.

About Regulus

Regulus Therapeutics Inc. (NASDAQ:RGLS) is a biopharmaceutical company leading the discovery and development of innovative medicines targeting microRNAs. Regulus is uniquely positioned to leverage a mature therapeutic platform that harnesses the oligonucleotide drug discovery and development expertise of Alnylam Pharmaceuticals, Inc. and Isis Pharmaceuticals, Inc., which founded the company. Regulus has a well-balanced microRNA therapeutics pipeline entering clinical development, an emerging microRNA biomarkers platform to support its therapeutic programs, and a rich intellectual property estate to retain its leadership in the microRNA field. Regulus intends to focus its proprietary efforts on developing microRNA therapeutics for oncology indications and orphan diseases and is currently advancing several programs toward clinical development in oncology, fibrosis and metabolic diseases. Specifically, Regulus is developing RG-012, an anti-miR targeting microRNA-21 for the treatment of Alport syndrome, a life-threatening kidney disease driven by genetic mutations with no approved therapy, and RG-101, a GalNAc-conjugated anti-miR targeting microRNA-122 for the treatment of chronic hepatitis C virus infection. Regulus' commitment to innovation and its leadership in the microRNA field have enabled the formation of strategic alliances with AstraZeneca and Sanofi and a research collaboration with Biogen Idec focused on microRNA biomarkers. In addition, the Company has established Regulus microMarkers™, a division focused on identifying microRNAs as biomarkers of human disease, which is designed to support its therapeutic pipeline, collaborators and strategic partners.

For more information, please visit http://www.regulusrx.com.

Forward-Looking Statements

Statements contained in this presentation regarding matters that are not historical facts are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, including the expected ability of Regulus to undertake certain activities and accomplish certain goals with respect to RG-101, the projected timeline of clinical development activities related to RG-101, and expectations regarding future therapeutic and commercial potential with respect to RG-101. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Words such as "believes," "anticipates," "plans," "expects," "intends," "will," "goal," "potential" and similar expressions are intended to identify forward-looking statements. These forward-looking statements are based upon Regulus' current expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, which include, without limitation, risks associated with the process of discovering, developing and commercializing drugs that are safe and effective for use as human therapeutics, and in the endeavor of building a business around such drugs. These and other risks concerning Regulus' are described in additional detail in Regulus filings with the Securities and Exchange Commission. All forward-looking statements contained in this presentation speak only as of the date on which they were made. Regulus undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.

OLYSIO™ is a registered trademark of Janssen Therapeutics.

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