LA JOLLA, Calif., May 8, 2014 /PRNewswire/ -- Regulus Therapeutics
Inc. (NASDAQ:RGLS), a biopharmaceutical company leading the
discovery and development of innovative medicines targeting
microRNAs, today reported financial results and highlights for the
quarter ended March 31, 2014 and
provided a summary of recent corporate highlights.
"In the first quarter of 2014, we continued to demonstrate our
leadership in the microRNA therapeutics field by advancing our
therapeutics pipeline with the initiation of the RG-101 clinical
trial, bolstering our intellectual property portfolio, and
strengthening our existing partnerships with the renewal of our
alliance with Sanofi," said Kleanthis G.
Xanthopoulos, Ph.D., President and CEO of Regulus. "Over the
next few quarters, we aim to build on this momentum and execute on
our 'Clinical Map Initiative' strategy. We have a unique
opportunity to demonstrate our first human proof of concept results
with a microRNA therapeutic, which if favorable, may set the stage
for success in future clinical trials across the pipeline. To that
end, we aim to nominate a third candidate for clinical development
by the end of the year, potentially giving us three clinical-stage
programs by 2015."
Neil W. Gibson, Ph.D., Chief
Scientific Officer of Regulus, added, "To date in 2014, we've
demonstrated significant scientific progress - we dosed our first
human subject with RG-101, advanced our preclinical oncology
programs, and initiated IND-enabling activities for the RG-012
program. In addition, we continued to maximize our discovery
efforts across our technology platform by pursuing new microRNA
therapeutic targets and advancing the biomarker work in Regulus'
microMarkers™ division."
Recent Highlight
- Advanced Oncology Portfolio; Presented Positive Preclinical
Data on miR-21 and miR-221 Programs at AACR. At the
American Association for Cancer Research Annual Meeting, Regulus
presented three posters and its strategic alliance partner, Sanofi,
presented an oral presentation on the therapeutic potential of
targeting microRNA-21 ("miR-21") and microRNA-221 ("miR-221") for
the treatment of hepatocellular carcinoma ("HCC"), the most common
form of liver cancer. In addition, Regulus presented data
from biomarker discovery studies evaluating the pharmacodynamics
signature for assessing miR-221 inhibition and microRNA expression
in tumor tissues in mouse models of HCC. Regulus believes
these data provide further evidence that miR-21 and miR-221 are
validated targets for the treatment of HCC and is currently
optimizing anti-miRs in each program for potential clinical
development.
First Quarter 2014 Highlights
- Commenced Dosing Human Subjects in Phase I Clinical Study of
RG-101. Regulus commenced dosing human subjects in the
Phase I clinical study of RG-101, a GalNAc-conjugated anti-miR
targeting microRNA-122 ("miR-122") for the treatment of chronic
hepatitis C virus infection ("HCV"). The Phase I clinical
study is being conducted in the
Netherlands and has four parts (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. Regulus expects to demonstrate
human proof-of-concept results from Part IV of the Phase I study of
RG-101 by the end of 2014, which is a key corporate goal under the
'Clinical Map Initiative'.
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.
- Renewed Strategic Alliance with Sanofi. Regulus and
Sanofi are now focused on specific orphan disease and oncology
targets, miR-21 for fibrosis and HCC and miR-221/222 for HCC.
Regulus intends to advance RG-012 (targeting miR-21 for kidney
fibrosis/Alport syndrome), and its programs targeting miR-21 and
miR-221/222 for HCC to proof-of-concept. Sanofi has an
exclusive option, exercisable after proof-of-concept, to take over
further development and commercialization of each program.
Regulus is eligible to receive milestone payments of up to
$101.8 million for proof-of-concept
option exercises, $15.0 million for
clinical milestones and up to $300.0
million for regulatory and commercial milestones. In
addition, Regulus has an option to co-promote in the United States or receive royalty payments
in the mid 10% to 20% range on any products.
- Partnered with Alport Syndrome Foundation to Raise Awareness
of Alport Syndrome; Regulus' RG-012 Program Expected to Enter the
Clinic in 1H 2015. Regulus partnered with the Alport
Syndrome Foundation, a non-profit corporation, to raise awareness
of the orphan disease during National Kidney Month and the
inaugural Alport Syndrome Awareness Month. Alport syndrome, a
life-threatening genetic kidney disease with no approved therapy,
is a genetic condition that impacts the body's ability to create a
specific type of collagen highly expressed in the kidney and
essential to normal kidney structure. In the absence of this
type of collagen, the kidneys are unable to effectively filter
toxins and waste products, resulting in end-stage renal disease and
also hearing loss or effects on vision. Regulus has discovered that
miR-21 is highly overexpressed in mouse models of Alport syndrome.
Regulus is developing RG-012, an anti-miR targeting miR-21, which
has demonstrated potent inhibition of miR-21 in vitro and
in vivo, a decrease in the rate of progression of renal
fibrosis, an increase in the lifespan of mice by up to fifty
percent, and a favorable pharmacokinetic profile that supports the
potential for a once per week dosing regimen. In the third
quarter of 2014, Regulus expects to initiate a natural history of
disease study to gather greater information about the progression
of Alport syndrome and to inform future clinical development
plans. In addition to this study, Regulus expects to initiate
a Phase I clinical study of RG-012 in the first half of 2015, which
is a key corporate goal under the 'Clinical Map Initiative'.
- Pursued Several Additional microRNA Targets, Independently
and with Strategic Partners. Regulus continued to pursue
several undisclosed microRNA targets, namely for orphan disease and
oncology indications, to expand its therapeutics pipeline.
Regulus and AstraZeneca have focused their collaborative efforts on
microRNA-103/107 for the treatment of metabolic diseases and
microRNA-19 for oncology indications and AstraZeneca has
contractual rights to a third target under the terms of the
alliance agreement. Regulus expects to nominate a third
microRNA candidate for clinical development (either independently
or with a strategic partner) by the end of 2014, which is a key
corporate goal under the 'Clinical Map Initiative'.
- Established Regulus microMarkers™
R&D Division. Regulus continued its
microRNA biomarker efforts within its newly established R&D
division, which utilizes a clinically-validated, highly
reproducible, proprietary technology platform to identify microRNAs
as potential biomarkers for disease.
First Quarter 2014 Financial Results &
Highlights
Regulus reported a net loss of $12.7
million for the quarter ended March
31, 2014, compared to a net loss of $7.2 million for the quarter ended March 31, 2013. Basic and diluted net loss
per share was $0.30 for the quarter
ended March 31, 2014, compared to net
loss per share of $0.20 for the
quarter ended March 31, 2013.
Regulus recognized revenue of $1.6
million for the quarter ended March
31, 2014, compared to $3.2
million for the quarter ended March
31, 2013. Revenue during these periods consisted
primarily of amortization of up-front payments received from our
strategic alliances and collaborations, which is recognized over
the estimated period of performance.
Research and development expenses were $9.6 million for the quarter ended March 31,
2014, compared to $6.9 million for
quarter ended March 31, 2013. This increase was
primarily driven by the initiation of a Phase I clinical study for
RG-101 and the continued advancement of other preclinical
development programs.
General and administrative expenses were $2.7 million for the quarter ended March 31,
2014, compared to $1.9 million for
the quarter ended March 31, 2013. This increase was primarily
driven by an increase in salaries and related employee costs and
other operating costs.
Non-cash charges recorded from changes in the value of
convertible notes payable were $2.1
million for the quarter ended March
31, 2014, compared to $1.8
million for the quarter ended March
31, 2013.
In February 2014, we entered into
a Common Stock Purchase Agreement with Aventis Holdings, Inc., an
entity affiliated with Sanofi, in which we sold 1,303,780 shares of
our common stock in a private placement at a price per share of
$7.67 for an aggregate purchase price
of $10.0 million. As of
March 31, 2014, Regulus had
$114.6 million in cash, cash
equivalents and short-term investments, debt with a principal
balance of $5.4 million and
43,346,014 shares of common stock outstanding. Regulus
expects to maintain its strong financial position and finish 2014
with at least $75.0 million in cash,
cash equivalents and short-term investments.
Conference Call & Webcast Information
Regulus will host a conference call and webcast at 5:00 p.m. Eastern Daylight Time today to
discuss its first quarter 2014 financial results, recent company
highlights and its expectations for the remainder of 2014. 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 33745994. To access the telephone replay of the
call, dial (855) 859-2056 (domestic) or (404) 537-3406
(international), passcode 33745994. The webcast and telephone
replay will be archived on the company's website for ninety days
following the call.
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.
Regulus Therapeutics Inc.
|
Selected Financial
Information
|
Condensed Statement of
Operations
|
(In thousands, except share and per share
data)
|
|
|
|
|
|
|
|
|
Three months
ended
March
31,
|
|
|
2014
|
|
2013
|
|
|
|
|
|
(Unaudited)
|
Revenues:
|
|
|
|
|
|
|
Revenues under strategic alliances and collaborations
|
|
$
1,631
|
|
$
3,238
|
Operating
expenses:
|
|
|
|
|
Research and
development
|
|
9,604
|
|
6,883
|
General and
administrative
|
|
2,732
|
|
1,905
|
Total operating
expenses
|
|
12,336
|
|
8,788
|
Loss from
operations
|
|
(10,705)
|
|
(5,550)
|
Other income
(expense), net
|
|
(2,035)
|
|
(1,689)
|
Loss before income
taxes
|
|
(12,740)
|
|
(7,239)
|
Income tax (benefit)
expense
|
|
|
1
|
|
|
(10)
|
Net loss
|
|
$
|
(12,741)
|
|
$
|
(7,229)
|
Basic and diluted net
loss per share
|
|
$
|
(0.30)
|
|
$
|
(0.20)
|
Shares used to
compute basic and diluted net loss per share
|
|
|
42,690,200
|
|
|
35,872,606
|
Regulus Therapeutics Inc.
|
Condensed Balance
Sheets
|
(In
thousands)
|
|
|
|
March 31,
2014
|
|
December 31,
2013
|
|
|
(Unaudited)
|
Assets
|
|
|
|
|
Cash, cash
equivalents and short-term investments
|
|
$
|
114,597
|
|
$
|
114,005
|
Other current
assets
|
|
3,632
|
|
3,177
|
Non-current
assets
|
|
5,878
|
|
5,883
|
Total
assets
|
|
$
|
124,107
|
|
$
|
123,065
|
Liabilities and
stockholders' equity
|
|
|
|
|
Current
liabilities
|
|
$
|
6,829
|
|
$
|
5,482
|
Total deferred
revenue
|
|
10,227
|
|
11,388
|
Convertible notes
payable, at fair value
|
|
13,403
|
|
11,279
|
Other long-term
liabilities
|
|
1,361
|
|
1,459
|
Stockholders'
equity
|
|
92,287
|
|
93,457
|
Total
liabilities and stockholders' equity
|
|
$
|
124,107
|
|
$
|
123,065
|
SOURCE Regulus Therapeutics Inc.