LA JOLLA, Calif., Feb. 27, 2014 /PRNewswire/ -- Regulus
Therapeutics Inc. (NASDAQ:RGLS), a biopharmaceutical company
leading the discovery and development of innovative medicines
targeting microRNAs, today provided an update on its corporate
strategy and reported financial results for the fourth quarter and
year ended December 31, 2013,
including a summary of recent corporate highlights.
Corporate Strategy Update:
Achieved All Goals on 'Road to the Clinic' and Launches
'Clinical Map Initiative'
- Regulus announced today that it has achieved all goals under
its 'Road to the Clinic' Strategy:
- Regulus has nominated two microRNA candidates for clinical
development, RG-101, a GalNAc-conjugated anti-miR targeting
miR-122 for the treatment of chronic hepatitis C virus infection
("HCV"), and RG-012, an anti-miR targeting microRNA-21 ("miR-21")
for the treatment of Alport Syndrome, a life-threatening kidney
disease driven by genetic mutations with no approved
therapy;
- Regulus has received regulatory approval to initiate the Phase
I clinical study of RG-101; and
- Regulus exceeded its previously issued cash guidance, ending
2013 with $114.0 million in cash,
cash equivalents and short-term investments.
- Regulus also announced today its 'Clinical Map Initiative',
which outlines certain corporate goals to advance its microRNA
therapeutics pipeline over the next several years:
- 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.
"In 2013, we demonstrated significant progress toward our goal
of building a leading biopharmaceutical company dedicated to
discovering and developing a new and major class of medicines
targeting microRNAs. Today, we are pleased to have achieved
our goals on the 'Road to the Clinic' and transition into a
clinical-stage company with the launch of our 'Clinical Map
Initiative," said Kleanthis G.
Xanthopoulos, Ph.D., President and CEO of Regulus. "We
believe that 2014 will be another exciting year for Regulus.
We expect to commence dosing in our Phase I clinical trial of
RG-101, advance RG-012 toward clinical development, potentially
nominate a third candidate for clinical development and maintain a
strong financial position to support these goals.
Importantly, we look forward to our first human proof-of-concept
study with RG-101 in HCV patients. If positive, these results
will go a long way toward validating our technology platform and
approach to treating disease. Overall, we are pleased with
our achievements and we look forward to building a meaningful
clinical portfolio based on microRNA therapeutics."
Recent Pipeline Highlights
- Regulatory Approval Received for Phase I Clinical Study of
RG-101.
- In November 2013 at the American
Association for the Study of Liver Diseases (AASLD) meeting,
Regulus presented data evaluating RG-101 for in vitro and
in vivo potency, pharmacokinetic/pharmacodynamics,
toxicology and safety pharmacology and inhibition of HCV
replication. In addition, Regulus has also tested RG-101 for
efficacy in a human chimeric liver mouse model infected with HCV
genotypes 1a and 3a. In this model, up to a 2 log reduction
in HCV viral load titer was observed in both genotypes after a
single dose of RG-101. The duration of action observed for
RG-101 supports the potential for a once-a-month dosing
regimen. To date, RG-101 has demonstrated an excellent
preclinical safety profile and has been well tolerated.
- The Phase I clinical study of RG-101 will have 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, which is designed to
demonstrate human proof-of-concept. 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, which is being
conducted in the Netherlands, and
dosing is expected to commence in the near term.
- Strengthened Focus on Orphan Diseases and Oncology
Indications.
- Nominated RG-012 as Clinical Development Candidate for the
Treatment of Alport Syndrome. In November 2013 at the American Society of
Nephrology (ASN) Kidney Week meeting, Regulus presented data
demonstrating that RG-012 is a potent inhibitor of miR-21 in both
in vitro and in vivo preclinical models and that
subcutaneous administration of RG-012 has significantly decreased
the rate of renal fibrosis and increased the lifespan of the mice
up to 50%. RG-012 has been well tolerated to date with a
favorable pharmacokinetic profile that supports the potential for a
once/week dosing regimen. Regulus expects to initiate a
natural history of disease study in the third quarter of 2014 and
to initiate a Phase I clinical study of RG-012 for the treatment of
Alport Syndrome in the first half of 2015.
- Renewed Strategic Alliance with Sanofi; Received Additional
$10 Million Equity
Investment. Regulus renewed its strategic alliance with
Sanofi to discover, develop, and commercialize microRNA
therapeutics to focus on specific orphan disease and oncology
targets. Regulus will lead development of its RG-012 program
for the treatment of Alport Syndrome, its program targeting miR-21
in oncology and its program targeting microRNA-221/microRNA-222 for
the treatment of hepatocellular carcinoma. Regulus is responsible
for advancing the clinical candidates in these programs to
proof-of-concept. Sanofi will have the exclusive option,
exercisable after proof-of-concept, to take over further
development and commercialization of each microRNA therapeutic
program. If Sanofi chooses to exercise its option on any
program, Sanofi will reimburse Regulus for a significant portion of
its preclinical and clinical development costs and will pay Regulus
an option exercise fee. Regulus is eligible to receive development
and commercialization milestone payments and will have an option to
co-promote in the United States or
receive royalty payments in the mid 10% to 20% range on any of
these programs.
- Discovery Efforts Continued to Support Therapeutic
Pipeline. Regulus continued to pursue several additional
undisclosed microRNA targets, namely for orphan disease and
oncology indications, with the goal of potentially nominating a
third candidate for clinical development by the end of 2014.
- AstraZeneca Collaboration to Focus on miR-103/107, miR-19
and Replacement Target for miR-33 Program.
- Under its collaboration with AstraZeneca to discover and
develop microRNA therapeutics for cardiovascular diseases,
metabolic diseases and oncology, Regulus announced today that the
ongoing efforts on two of the collaboration programs are targeting
microRNA-103/107 for the treatment of metabolic diseases and
microRNA-19 for oncology indications, a target selected by
AstraZeneca in October 2013. Additionally, Regulus announced
today that the companies have agreed to terminate the program
targeting microRNA-33 ("miR-33") for the treatment of
atherosclerosis as a collaboration target. AstraZeneca has a
contractual right to substitute a new target for miR-33.
"Our scientific accomplishments in 2013 have advanced our
therapeutic pipeline in a meaningful way and have prepared us to
enter the clinic," said Neil W.
Gibson, Ph.D., Chief Scientific Officer of Regulus.
"Additionally, we are pleased to have strengthened our focus on
orphan diseases and oncology indications and we look forward to
expanding our clinical portfolio in the coming year."
Recent Corporate Highlights
- Expanded microRNA Biomarkers Platform and Established
Regulus microMarkers™ R&D
Division. The division utilizes a
clinically-validated, highly reproducible, proprietary technology
platform to identify microRNAs as potential biomarkers for
disease. In 2012, Regulus formed a research collaboration
with Biogen Idec focused on the discovery of microRNAs as
biomarkers for multiple sclerosis and has entered into an
arrangement with another leading, commercial-stage pharmaceutical
company to explore microRNAs as biomarkers for specific patient
populations.
- Attracted Key Talent. Regulus strengthened its
executive management team with the addition of David Szekeres as Chief Business Officer and
General Counsel. Additionally, Martin
Beaulieu, Ph.D. joined Regulus as head of the Regulus
microMarkers™ division.
Fourth Quarter and Year-End 2013 Financial Results
and 2014 Financial Outlook
Regulus completed 2013 with $114.0
million in cash, cash equivalents and short-term
investments, compared to $98.1
million as of December 31,
2012.
Regulus reported a net loss of $1.9
million and $18.7 million for
the quarter and year ended December 31,
2013, respectively, compared to $6.9
million and $17.4 million for
the same periods in 2012.
Basic and diluted net loss per share was $0.05 and $0.11 for
the quarter ended December 31, 2013,
respectively, compared to basic and diluted net loss per share of
$0.22 for the same period in
2012. Basic and diluted net loss per share was $0.49 for the year ended December 31, 2013, compared to basic and diluted
net loss per share of $2.12 for
2012. Comparability of net loss per share for the periods
presented is impacted by the initial public offering and concurrent
common stock issuances in October
2012 and the public offering of common stock in July
2013.
Regulus reported revenue of $5.5
million for the quarter ended December 31, 2013, compared to $3.2 million for the same period in 2012.
Revenue for the quarter ended December 31,
2013 included $4.5 million
from the Sanofi collaboration and license agreement and
$1.0 million from other strategic
alliances and collaborations, including approximately $0.3 million in research milestones. The
increase in revenues in the fourth quarter 2013 compared to the
same period in 2012 was primarily due to the incremental
recognition of $3.3 million from the
change in Regulus' estimated period of performance for the research
term within the Sanofi collaboration and license agreement, which
extended through the end of the option period in early 2014.
Revenues were $19.6 million for the
year ended December 31, 2013 compared
to $12.7 million in 2012.
Revenues for the year ended December 31,
2013 included approximately $15.4
million from the Sanofi collaboration and license agreement
and $4.2 million from other strategic
alliances and collaborations. Revenue during these periods
consisted primarily of amortization of upfront payments received
from Regulus' strategic alliances and collaborations, which is
recognized over the estimated period of
performance.
Research and development expenses were $8.2 million and $29.9
million for the quarter and year ended December 31, 2013, compared to $5.6 million and $20.3
million for the same periods in 2012. These increases
were substantially attributable to IND-enabling activities for
RG-101 and other programs in 2013. Regulus expects its
research and development expenses to continue to increase to the
extent it commences clinical studies and initiates additional
IND-enabling activities.
General and administrative expenses were $1.9 million and $7.4
million for the quarter and year ended December 31, 2013, compared to $1.9 million and $4.9
million for the same periods in 2012. The increase in
2013 was primarily attributable to operating costs associated with
the growth of the company and incremental costs associated with
being a public reporting company.
Net loss in the quarter ended December 31, 2013 included a non-cash gain of
$2.6 million from the change in value
of the amended and restated convertible promissory note originally
issued to GlaxoSmithKline plc in 2010, with such change in value
attributable to a decrease in value of Regulus common stock during
the quarter. Net loss in the quarter ended December 31, 2012 included a non-cash charge of
$2.6 million, with such change in
value attributable to an increase in value of common stock of the
Company during the quarter. For the years ended December 31, 2013 and 2012, total net non-cash
charges associated with the convertible promissory note were
$1.1 million and $4.7 million, respectively.
Regulus expects to finish 2014 with at least $75.0 million in cash, cash equivalents and
short-term investments, which includes the $10.0 million common stock purchase made by its
strategic alliance partner Sanofi in February 2014.
Conference Call & Webcast
Information
Regulus will host a conference call and webcast at
5:00 p.m. Eastern Standard Time today
to discuss its fourth quarter and year-end 2013 financial results,
recent company highlights and its expectations for 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 2177234. To access the telephone replay of the
call, dial (855) 859-2056 (domestic) or (404) 537-3406
(international), passcode 2177234. The webcast and telephone replay
will be archived on the company's website following the
call.
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 therapeutic 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. 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 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
December
31,
|
|
Year
ended
December
31,
|
|
|
2013
|
|
2012
|
|
2013
|
|
2012
|
|
|
|
Revenues:
|
|
|
|
|
|
|
|
|
|
|
|
|
Revenue under
strategic
alliances
|
|
$
|
5,454
|
|
$
|
3,238
|
|
$
|
19,569
|
|
$
|
12,700
|
Total
revenues
|
|
5,454
|
|
3,238
|
|
19,569
|
|
12,700
|
Operating
expenses:
|
|
|
|
|
|
|
|
|
Research and
development
|
|
8,232
|
|
5,607
|
|
29,942
|
|
20,342
|
General and
administrative
|
|
1,884
|
|
1,934
|
|
7,429
|
|
4,932
|
Total operating
expenses
|
|
10,116
|
|
7,541
|
|
37,371
|
|
25,274
|
Loss from
operations
|
|
(4,662)
|
|
(4,303)
|
|
(17,802)
|
|
(12,574)
|
Other income
(expense), net
|
|
2,716
|
|
(2,555)
|
|
(889)
|
|
(4,844)
|
Loss before income
taxes
|
|
(1,946)
|
|
(6,858)
|
|
(18,691)
|
|
(17,418)
|
Income tax (benefit)
expense
|
|
(19)
|
|
|
18
|
|
(23)
|
|
(10)
|
Net loss
|
|
$
|
(1,927)
|
|
$
|
(6,876)
|
|
$
|
(18,668)
|
|
$
|
(17,408)
|
Net loss per
share:
|
|
|
|
|
|
|
|
|
|
|
|
|
Basic
|
|
$
|
(0.05)
|
|
$
|
(0.22)
|
|
$
|
(0.49)
|
|
$
|
(2.12)
|
Diluted
|
|
$
|
(0.11)
|
|
$
|
(0.22)
|
|
$
|
(0.49)
|
|
$
|
(2.12)
|
Weighted average
shares used to compute net loss per share:
|
|
|
|
|
|
|
|
|
|
|
|
|
Basic
|
|
|
41,779,422
|
|
|
31,907,152
|
|
|
38,479,447
|
|
|
8,212,538
|
Diluted
|
|
|
43,191,081
|
|
|
31,907,152
|
|
|
38,479,447
|
|
|
8,212,538
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Regulus
Therapeutics Inc.
Condensed Balance
Sheets
(In
thousands)
|
|
|
December 31,
2013
|
|
December 31,
2012
|
Assets
|
|
|
|
|
Cash, cash
equivalents and short-term investments
|
|
$
|
114,005
|
|
$
|
98,100
|
Other current
assets
|
|
3,177
|
|
829
|
Non-current
assets
|
|
5,883
|
|
4,589
|
Total
assets
|
|
$
|
123,065
|
|
$
|
103,518
|
Liabilities and
stockholders' equity
|
|
|
|
|
Current
liabilities
|
|
$
|
5,482
|
|
$
|
2,317
|
Total deferred
revenue
|
|
11,388
|
|
28,207
|
Convertible notes
payable, at fair value
|
|
11,279
|
|
10,134
|
Other long-term
obligations liabilities
|
|
1,459
|
|
767
|
Stockholders'
equity
|
|
93,457
|
|
62,093
|
Total
liabilities and stockholders' equity
|
|
$
|
123,065
|
|
$
|
103,518
|
SOURCE Regulus Therapeutics Inc.