LA JOLLA, Calif., June 25, 2014 /PRNewswire/ -- Regulus
Therapeutics Inc. (NASDAQ: RGLS), a biopharmaceutical company
leading the discovery and development of innovative medicines
targeting microRNAs, today announced that the U.S. Patent and
Trademark Office ("USPTO") has granted a patent in the
company's exclusively licensed 'Sarnow' patent estate, for claims
related to targeting microRNA-122 ("miR-122") for the treatment of
HCV in combination with other anti-viral therapies. The Sarnow
patent estate, owned by Stanford
University and exclusively licensed to Regulus, has produced
patents in Europe, Japan and the United
States covering the use of a broad class of anti-miR
inhibitors of miR-122 for the treatment of HCV as monotherapy or in
combination with other anti-viral therapies. Regulus' lead
asset, RG-101, a GalNAc-conjugated anti-miR that targets miR-122,
is being evaluated in a Phase I clinical study for the treatment of
HCV. Regulus expects to demonstrate human proof-of-concept results
from Part IV of the Phase I study by the end of 2014. If
favorable, these results may positively advance Regulus' microRNA
technology platform and provide clinical support for its unique
approach to treating disease.
"We are pleased with the newly granted miR-122 claims in the US,
which represent the fourth U.S. patent to grant within the Sarnow
patent estate," said David L.
Szekeres, Chief Business Officer and General Counsel of
Regulus. "In the United States and
abroad, we continue to strengthen Regulus' already comprehensive
patent estate relating to miR-122 compositions-of-matter and
methods of use, which we believe is an important component for the
commercialization of therapies targeting the host factor miR-122 to
treat HCV infection."
The recently granted claims add to Regulus' already robust
patent estate relating to miR-122-specific compositions-of-matter
and methods of use, which includes:
- The Sarnow patent estate claiming methods related to the
treatment of HCV infection using anti-miR-122 oligonucleotides,
either as a monotherapy or in combination with other antiviral
therapies (including US Patent No. 7,307,067, US Patent No.
7,838,504, US Patent No. 8,217,020, US Patent No. 8,759,312,
European Patent No. 1747023; Japan Patent No. 4,943,322; allowed
Australia Application No. 2005240118; also pending in Canada and Israel);
- Regulus-owned US Patent No. 7,683,036 claiming methods of
inhibiting miR-122 activity using fully modified anti-miR-122
oligonucleotides and US Patent No. 8,546,350 claiming methods of
inhibiting miR-122 activity using various types of modified
anti-miR-122 oligonucleotides;
- US Patent No. 7,232,806, Japan Patent No. 4,371,812, Australia
Patent No. 2002347035, and Israel Patent No. 161100, exclusively
licensed to Regulus for therapeutic uses, claiming broad
anti-miR-122 compositions-of-matter (also pending in Europe and Canada); and
- Regulus-owned European Patent No. 1931782, Australia Patent No.
2006284855, and Japan Patent No. 5523705 claiming uses of a broad
class of anti-miR-122 oligonucleotides to lower cholesterol levels
(also pending in US and Canada).
About Regulus' Intellectual Property Estate
Regulus believes that it has a leading intellectual property
position and substantial know-how relating to the development and
commercialization of microRNA therapeutics, composed of
approximately 200 patents and patent applications that the company
owns or has in-licensed from academic institutions and third
parties including its founding companies, Alnylam Pharmaceuticals,
Inc. and Isis Pharmaceuticals, Inc., related to microRNA and
microRNA drug products. Regulus also has access to
approximately 850 patents and patent applications exclusively
related to RNA technologies, including patents and patent
applications relating to chemical modification of oligonucleotides
that are useful for the development of microRNA therapeutics.
About microRNAs and RG-101 for the Treatment of HCV
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 one-third 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.
microRNA-122 ("miR-122") is the most abundant microRNA in
hepatocytes and is a critical host factor for survival and
replication of all know HCV genotypes, and anti-miRs targeting
miR-122 have been shown to block HCV infection. RG-101 is a
novel anti-miR-122 oligonucleotide therapeutic that is effectively
targeted to hepatocytes for the treatment of HCV through
conjugation to GalNAc, a carbohydrate-based chemistry approach for
asialoglycoprotein receptor-mediated delivery of oligonucleotides
to hepatocyte cells of the liver. Utilizing the GalNAc conjugate
chemistry has significantly improved the potency of the active
oligonucleotide of RG-101 by achieving targeted delivery of the
oligonucleotide to the infected hepatocytes. In preclinical
studies, Regulus has observed significant HCV viral load reduction
in a human chimeric liver mouse model infected with genotypes 1a
and 3a, a long duration of action for RG-101 which supports the
potential for a once-a-month dosing regimen, and a favorable
preclinical safety profile in which RG-101 has been well
tolerated.
Currently, RG-101 is being evaluated in a four-part Phase I
clinical study: (i) a single ascending-dose study in healthy
volunteer subjects; (ii) a multiple-ascending dose study in healthy
volunteer subjects; (iii) a single-dose drug-drug interaction study
of RG-101 in combination with an approved oral direct-acting
antiviral ("DAA") in healthy volunteer subjects; and (iv) a
single-dose study in HCV patients to assess the safety and viral
load reduction. The primary objective of the Phase I clinical
study of RG-101 is to evaluate safety and tolerability and the
secondary objectives are to evaluate pharmacokinetics, viral load
reduction and any impact an oral DAA may have on the
pharmacokinetics of RG-101. Up to approximately 100
healthy volunteer subjects and HCV patients are planned to be
enrolled in the Phase I study. By the end of 2014, Regulus
expects to demonstrate human proof-of-concept results from Part IV
of the Phase I study of RG-101, which is a key corporate goal under
the company's 'Clinical Map Initiative'.
About 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. Regulus expects to demonstrate human proof-of-concept
results in the Phase I clinical study of RG-101 for the treatment
of HCV by the end of 2014, initiate a Phase I clinical study of
RG-012 for the treatment of Alport syndrome in the first half of
2015, 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,
GlaxoSmithKline and Sanofi and a research collaboration with Biogen
Idec focused on microRNA biomarkers. In addition, the Company
has established Regulus microMarkers™, a research and development
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 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 financial estimates
(including Regulus' projected cash at the end of 2014), the
projected sufficiency of Regulus' capital position for future
periods, the expected ability of Regulus to undertake certain
activities and accomplish certain goals (including with respect to
development and other activities related to RG-012 and RG-101 and
with respect to the nomination of a third microRNA candidate for
clinical development), 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.
SOURCE Regulus Therapeutics Inc.