LA JOLLA, Calif., April 25, 2015 /PRNewswire/ -- Regulus
Therapeutics Inc. (NASDAQ: RGLS), a biopharmaceutical company
leading the discovery and development of innovative medicines
targeting microRNAs, today presented new data strengthening the
profile of RG-101, a wholly-owned, GalNAc-conjugated anti-miR
targeting microRNA-122 ("miR-122") for the treatment of
HCV, during an oral late-breaking session at ILC 2015 in
Vienna, Austria. Extended
follow-up results evaluating a single subcutaneous administration
of either 2 mg/kg or 4 mg/kg of RG-101 as monotherapy in HCV
patients with varied genotypes, liver fibrosis status and treatment
history showed that 10/22 patients had HCV RNA levels below the
limit of quantification ("BLOQ") at 12 weeks and 70 percent of
those patients remained BLOQ at 20 weeks (7/10). In addition,
the positive results that were previously reported from the
completed clinical study of RG-101 were reviewed during the oral
late-breaker.
Regulus also made several poster presentations highlighting the
pharmacokinetics and pharmacodynamics of RG-101 in healthy
volunteers, the preclinical pharmacokinetics, pharmacodynamics, and
toxicity of RG-101, and the efficacy of RG-101 in a preclinical
model of HCV. The ILC 2015 oral presentation slides and
posters related to RG-101 will be accessible at
www.regulusrx.com.
"With multiple presentations at this year's ILC meeting, Regulus
is pleased to tell the complete development story of RG-101, our
unique microRNA therapeutic for the treatment of HCV, with positive
preclinical results through impressive data in our first-in-human
clinical trial," said Paul Grint,
MD, Chief Medical Officer of Regulus. "All told, we believe that
RG-101 is the most potent, durable and versatile compound in
development to treat HCV. In the near term, we look forward
to testing the ability of RG-101 to shorten the duration of
treatment to just four weeks in combination with oral agents, which
is a key goal to advance the program under our 'Clinical Map
Initiative' strategy."
"ILC 2015 is an extremely important meeting to advance the
treatment of HCV and we are pleased to present such significant
data on RG-101 in the oral late-breaking session," said
Hendrik W. Reesink, M.D., Ph.D.,
Associate Professor in the Department of Gastroenterology and
Hepatology at the Academic Medical Center in The Netherlands.
"Having so many patients with undetectable levels of virus after
just a single administration so many months out is truly remarkable
and these findings continue to suggest the clinical benefit of
RG-101's ability to improve upon current therapies in a wide range
of HCV patients. I look forward to seeing RG-101 progress
into future clinical studies."
Click here to watch Regulus' President and CEO,
Kleanthis G. Xanthopoulos, Ph.D.,
provide additional detail on the upcoming combination studies of
RG-101 for the treatment of HCV.
Completed RG-101 Study Design
Regulus has evaluated RG-101 in a completed clinical study
conducted in The Netherlands. 58
healthy volunteers and 32 HCV patients with multiple genotypes,
liver fibrosis status and treatment history were enrolled in the
four part study: (i) a single ascending-dose study in which healthy
volunteer subjects received 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
received 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 received a single subcutaneous
dose of RG-101 in combination with simeprevir (OLYSIO™), an
approved direct acting antiviral; and (iv) a single-dose study in
which HCV patients received 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 direct acting antiviral, such as simeprevir
(OLYSIO™), may have on the pharmacokinetics of
RG-101.
'Clinical Map Initiative' Goals for RG-101
Regulus' 'Clinical Map Initiative' outlines certain corporate
goals to advance its microRNA therapeutics pipeline over the next
several years. Regulus plans to investigate RG-101 in
combination with oral agents to potentially shorten treatment
durations, optimize clinical outcomes and potentially improve
responses in certain underserved HCV patient populations, and
further as a single agent (multiple doses of RG-101 in specific
populations). In the near term, Regulus expects to initiate
the above described studies in the second quarter of 2015 and to
report interim data by the end of 2015.
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
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.
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
domain dominant leadership in the microRNA field. Under its
'Clinical Map Initiative', 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 been selected for clinical
development. 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 and a research collaboration with
Biogen focused on microRNA biomarkers. 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.
OLYSIO™ is a registered trademark of Janssen Therapeutics.
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SOURCE Regulus Therapeutics Inc.