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.
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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 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.
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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SOURCE Regulus Therapeutics Inc.