Regulus Completes RG-101 Enrollment in Phase II Combination Study

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 Europe and 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.

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.

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