LA JOLLA, Calif., Feb. 18, 2015 /PRNewswire/ -- Regulus
Therapeutics Inc. (NASDAQ: RGLS), a biopharmaceutical company
leading the discovery and development of innovative medicines
targeting microRNAs, today reported financial results for the
fourth quarter and year ended December 31,
2014, including a summary of recent corporate
highlights.
![Regulus Therapeutics Inc. Logo Regulus Therapeutics Inc. Logo](http://photos.prnewswire.com/prnvar/20150130/172637LOGO)
"In 2014 and the start of 2015, Regulus has 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," said
Kleanthis G. Xanthopoulos, Ph.D.,
President and CEO of Regulus. "In 2014, we achieved key goals
under our 'Clinical Map Initiative' to advance our microRNA
therapeutics pipeline, with the most significant being the human
proof-of-concept results demonstrated with RG-101, our wholly-owned
GalNAc-conjugated anti-miR-122 for the treatment of HCV. We
are pleased with our continued execution and our 'Clinical Map
Initiative' goals for 2015 remain on track."
Paul Grint, M.D., Chief Medical
Officer of Regulus, added, "Our scientific accomplishments in 2014
positioned Regulus for an exciting year focused on the growth of
our clinical portfolio. Currently, we are preparing to
initiate Phase II studies for RG-101 in multiple countries and our
first clinical studies for RG-012, an anti-miR targeting
microRNA-21, to treat a devastating orphan disease with no approved
therapy called Alport syndrome. Throughout the year, we look
forward to reporting progress on these goals and on the overall
advancement of our portfolio."
Fourth Quarter 2014 and Recent Highlights
- Achieved Human Proof-of-Concept with RG-101 in HCV.
Treatment with a single subcutaneous dose of either 2 mg/kg or 4
mg/kg of RG-101, a GalNAc-conjugated anti-miR targeting
microRNA-122 ("miR-122"), 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. At day 29, mean viral load
reductions of 4.8 log10 and 4.1 log10 were
demonstrated in the 4 mg/kg and 2 mg/kg single dose cohorts,
respectively. At day 57, 15 out of 28 patients treated with
one single administration of either 2 mg/kg or 4 mg/kg of RG-101
had HCV RNA levels below the limit of quantification and 12 out of
these 15 treated patients had HCV RNA levels that cannot be
detected. To date, RG-101 has a favorable safety profile with no
serious adverse events or discontinuations reported in the treated
HCV patients. Under the 'Clinical Map Initiative', Regulus expects
to initiate Phase II studies investigating RG-101 in combination
with oral direct-acting antiviral agents and further as a single
agent (single or multiple doses of RG-101) in the second quarter of
2015.
- Enhanced Preclinical Profile of RG-012 with Data at Kidney
Week. RG-012 is a single-stranded, chemically
modified oligonucleotide that binds to and inhibits the function of
microRNA-21 ("miR-21") for the treatment of renal dysfunction in
Alport syndrome patients. Alport syndrome is a
life-threatening, genetic kidney disease driven by mutations in
specific collagen. By inhibiting miR-21, which is highly
overexpressed in animal models of Alport syndrome, RG-012 is
intended to act by reducing the severity of fibrosis, which then
may reduce the rate of decline of renal function in Alport syndrome
patients. At the American Society of Nephrology Kidney Week
2014 meeting, Regulus and its strategic alliance partner, Genzyme
(a Sanofi company) presented preclinical data demonstrating that
treatment with RG-012 was shown to protect kidney function better
than Sanofi's ACE inhibitor, ramipril, alone, and an additive
therapeutic effect was observed in combination with ramipril.
Under the 'Clinical Map Initiative', Regulus plans to enroll up to
120 Alport syndrome patients in a global natural history of disease
study called ATHENA, designed to characterize the natural decline
of renal function as measured by established renal markers in
Alport syndrome patients over time. In the first half of 2015,
Regulus plans to initiate a Phase I study to evaluate the safety
and tolerability of RG-012 in healthy volunteers and a Phase II
proof-of-concept study thereafter.
- Advanced Preclinical microRNA Portfolio, Independently and
with Strategic Partners. Regulus continued to pursue
several undisclosed microRNA targets, mainly for oncology and
orphan disease indications. In addition to its internal
research efforts, Regulus advanced several programs with its
strategic alliance partners, microRNA-103/107 for the treatment of
metabolic diseases and microRNA-19 for oncology indications with
AstraZeneca, microRNA-221 and miR-21 for hepatocellular carcinoma
and miR-21 for renal fibrosis (RG-012) with Sanofi.
Under the 'Clinical Map Initiative', Regulus expects
to nominate at least one additional microRNA candidate for clinical
development in 2015, either independently or with a partner.
- Earned Milestone Payment from Biogen Idec for
Biomarkers. Under its research collaboration with Biogen
Idec to identify microRNAs as biomarkers for multiple sclerosis
("MS"), Regulus' microMarkers(SM) division used its robust
technology platform to successfully meet quality specifications for
extracting and reproducibly profiling microRNAs from an initial set
of whole blood samples of a recent Biogen Idec MS therapy
trial. Under the 'Clinical Map Initiative', Regulus
microMarkers(SM) aims to (i) identify a microRNA biomarker
signature with the potential to differentiate relapse remitting MS
patients from healthy volunteers and to compare with treatment
response profiles with Biogen Idec; (ii) profile serum samples from
healthy volunteers and HCV patients in the completed study of
RG-101 to identify potential microRNA signatures; and (iii) profile
urine and blood samples from Alport syndrome patients in ATHENA to
potentially identify a clinically useful microRNA signature.
"Our scientific research in 2014 and the recent period have
provided key data to support the growth of our clinical pipeline,
our early discovery efforts and our biomarkers work," said
Neil W. Gibson, Ph.D., Chief
Scientific Officer of Regulus. "We continue to pursue
opportunities where we can apply our oligonucleotide drug discovery
and development expertise to validated microRNA targets.
Under our 'Clinical Map Initiative' goals for 2015, we aim to
nominate one additional microRNA candidate for clinical development
and expand our biomarkers work to support our clinical pipeline and
our collaborators' programs."
Fourth Quarter and Year-End 2014 Financial Results
Regulus reported a net loss of $22.2
million and $56.7 million for
the quarter and year ended December 31,
2014, respectively, compared to a net loss of $1.9 million and $18.7
million for the same periods in 2013. Basic and
diluted net loss per share was $0.47
for the quarter ended December 31,
2014, respectively, compared to basic and diluted net loss
per share of $0.05 and $0.11 for the same period in 2013. Basic and
diluted net loss per share was $1.29
for the year ended December 31, 2014,
compared to $0.49 for the same period
in 2013.
Regulus recognized revenue of $4.2
million and $7.7 million for
the quarter and year ended December 31,
2014, respectively, compared to $5.5
million and $19.6 million for
the same periods in 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 $10.5 million and $41.0
million for the quarter and year ended December 31, 2014, respectively, compared to
$8.2 million and $29.9 million for the same periods in 2013.
This increase was primarily driven by Phase I clinical study costs
for RG-101, an increase in IND-enabling costs for RG-012 and the
continued advancement of other preclinical development
programs.
General and administrative expenses were $3.3 million and $11.5
million for the quarter and year ended December 31, 2014, respectively, compared to
$1.9 million and $7.4 million for the same periods in 2013. This
increase was primarily driven by an increase in salaries and
related employee costs and other operating expenses associated with
general business activities.
Net loss for the quarter and year ended December 31, 2014 included a non-cash charge of
$12.7 million and $12.1 million, respectively, from the change in
value of our convertible note payable. Net loss for the quarter and
year ended December 31, 2013 included
a non-cash gain of $2.6 million and a
non-cash charge of $1.1 million,
respectively. Changes in value were primarily driven by changes in
our stock price during the respective periods.
As of December 31, 2014, Regulus
had $159.7 million in cash, cash
equivalents and short-term investments, debt with a principal
balance of $5.4 million and
48,944,530 shares of common stock outstanding.
2015 Financial Guidance
Regulus expects to end 2015 with greater than $100.0 million in cash, cash equivalents and
short-term investments.
"Regulus continues to maintain a solid balance sheet, with
$159.7 million in cash at the end of
2014," said David Szekeres, Chief
Business Officer and General Counsel of Regulus. "We believe
this balance sheet and our disciplined financial strategy will
allow us to execute our 'Clinical Map Initiative' goals and provide
us with sufficient runway to grow our pipeline."
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 2014 financial results, recent
company highlights and its expectations for 2015. 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 87438466.
To access the telephone replay of the call, dial (855) 859-2056
(domestic) or (404) 537-3406 (international), passcode 87438466.
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 has
leveraged its oligonucleotide drug discovery and development
expertise to develop a well-balanced microRNA therapeutics pipeline
complemented by a maturing microMarkers(SM) 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. 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 Idec 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 financial estimates
(including Regulus' projected cash at the end of 2015), 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 or the Regulus microMarkers(SM) division 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,
|
|
|
2014
|
|
2013
|
|
2014
|
|
2013
|
|
|
(Unaudited)
|
Revenues:
|
|
|
|
|
|
|
|
|
|
|
|
|
Revenue under
strategic
alliances
|
|
$
|
4,219
|
|
$
|
5,454
|
|
$
|
7,669
|
|
$
|
19,569
|
Total
revenues
|
|
4,219
|
|
5,454
|
|
7,669
|
|
19,569
|
Operating
expenses:
|
|
|
|
|
|
|
|
|
Research and
development
|
|
10,474
|
|
8,232
|
|
41,046
|
|
29,942
|
General and
administrative
|
|
3,278
|
|
1,884
|
|
11,533
|
|
7,429
|
Total operating
expenses
|
|
13,752
|
|
10,116
|
|
52,579
|
|
37,371
|
Loss from
operations
|
|
(9,533)
|
|
(4,662)
|
|
(44,910)
|
|
(17,802)
|
Other (expense)
income, net
|
|
(12,635)
|
|
2,716
|
|
(11,769)
|
|
(889)
|
Loss before income
taxes
|
|
(22,168)
|
|
(1,946)
|
|
(56,679)
|
|
(18,691)
|
Income tax (benefit)
expense
|
|
|
—
|
|
|
(19)
|
|
1
|
|
(23)
|
Net loss
|
|
$
|
(22,168)
|
|
$
|
(1,927)
|
|
$
|
(56,680)
|
|
$
|
(18,668)
|
Net loss per
share:
|
|
|
|
|
|
|
|
|
|
|
|
|
Basic
|
|
$
|
(0.47)
|
|
$
|
(0.05)
|
|
$
|
(1.29)
|
|
$
|
(0.49)
|
Diluted
|
|
$
|
(0.47)
|
|
$
|
(0.11)
|
|
$
|
(1.29)
|
|
$
|
(0.49)
|
Weighted average
shares used to compute net loss per share:
|
|
|
|
|
|
|
|
|
|
|
|
|
Basic
|
|
|
46,863,382
|
|
|
41,779,422
|
|
|
44,090,165
|
|
|
38,479,447
|
Diluted
|
|
|
46,863,382
|
|
|
43,191,081
|
|
|
44,090,165
|
|
|
38,479,447
|
Regulus
Therapeutics Inc.
Condensed Balance
Sheets
(In
thousands)
|
|
|
December 31,
2014
|
|
December 31,
2013
|
|
|
(Unaudited)
|
Assets
|
|
|
|
|
Cash, cash
equivalents and short-term investments
|
|
$
|
159,743
|
|
$
|
114,005
|
Other current
assets
|
|
5,208
|
|
3,177
|
Non-current
assets
|
|
6,529
|
|
5,883
|
Total assets
|
|
$
|
171,480
|
|
$
|
123,065
|
Liabilities and
stockholders' equity
|
|
|
|
|
Current
liabilities
|
|
$
|
8,698
|
|
$
|
5,482
|
Total deferred
revenue
|
|
6,349
|
|
11,388
|
Convertible notes
payable, at fair value
|
|
23,397
|
|
11,279
|
Other long-term
liabilities
|
|
1,022
|
|
1,459
|
Stockholders'
equity
|
|
132,014
|
|
93,457
|
Total liabilities and stockholders' equity
|
|
$
|
171,480
|
|
$
|
123,065
|
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SOURCE Regulus Therapeutics Inc.