LA JOLLA, Calif., May 7, 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 first quarter
ended March 31, 2015, including a
summary of recent corporate highlights.
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"The highlights during the first quarter and recent period of
2015 demonstrate our ability to continually execute on our
'Clinical Map Initiative' goals and increase our confidence in our
approach to treating disease with microRNA therapeutics," said
Kleanthis G. Xanthopoulos, Ph.D.,
President and CEO of Regulus. "Most notably, we reported impressive
results with RG-101, our wholly-owned anti-miR-122 for the
treatment of HCV, advanced our clinical portfolio with
AstraZeneca's selection of RG-125, a novel insulin sensitizer, and
pursued new microRNA targets to maintain our scientific leadership.
All told, these accomplishments have positioned us for an exciting
year that will showcase the breadth of our opportunity with
microRNAs."
Paul Grint, M.D., Chief Medical
Officer of Regulus, added, "Our recent progress has positioned us
for an exciting period focused on the growth of our clinical
portfolio and we have several key catalysts fast-approaching.
Specifically, we are preparing to initiate Phase II studies for
RG-101 and our first clinical study for RG-012, an anti-miR-21 to
treat Alport syndrome, and we intend to advance additional programs
into clinical development."
First Quarter 2015 and Recent Highlights
- Strengthened Profile of RG-101 with Data at The
International Liver Congress™ (ILC 2015). Regulus presented
results highlighting the favorable preclinical and clinical profile
of RG-101 at ILC 2015 in Vienna,
Austria in multiple posters and an oral late-breaking
session. Extended follow-up results evaluating a single
subcutaneous administration of either 2 mg/kg or 4 mg/kg of RG-101
as monotherapy in HCV patients with varied genotypes, liver
fibrosis status and treatment history showed that 10/22 patients
had HCV RNA levels below the limit of quantification ("BLOQ") at 12
weeks and 70 percent of those patients remained BLOQ at 20 weeks
(7/10). Previously, Regulus reported results from the completed
clinical study evaluating a single administration of either 2 mg/kg
or 4 mg/kg of RG-101 in the same patient population over 8 weeks
(57 days). These results demonstrated that at day 57, 15 out of 28
patients had HCV RNA levels BLOQ and RG-101 was well tolerated 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, with interim data expected by the end of
2015.
- Advanced Clinical Portfolio with Selection of
RG-125(AZD4076) by AstraZeneca; Earned $2.5M Milestone. Regulus achieved a key
'Clinical Map Initiative' goal for 2015 with the selection of its
third candidate for clinical development, RG-125 (AZD4076), a
GalNAc-conjugated anti-miR targeting microRNA-103/107
("miR-103/107") for the treatment of Non Alcoholic Steatohepatitis
("NASH") in patients with type 2 diabetes/pre-diabetes. Regulus and
AstraZeneca aim to present key preclinical data on RG-125(AZD4076)
at a scientific meeting later this year and expect to initiate a
Phase I study of RG-125(AZD4076) in humans by the end of 2015.
- Achieved Orphan Drug Status for RG-012 in Europe. The European Commission granted
orphan medicinal product designation for RG-012, an anti-miR
targeting microRNA-21 ("miR-21") for the treatment of Alport
syndrome, a life-threatening genetic kidney disease with no
approved therapy. The European designation adds to the orphan drug
status received for RG-012 in the United
States in 2014. 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, which was initiated
in September 2014 and is designed to
characterize the natural decline of renal function as measured by
established renal markers in Alport syndrome patients over time. In
the near term, 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, miR-103/107 for the treatment of metabolic diseases
(RG-125/AZD4076) 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.
Neil W. Gibson, Ph.D., Chief
Scientific Officer of Regulus, added, "Our recent scientific
progress highlights the productivity of Regulus' technology
platform and the applicability of microRNA therapeutics to
potentially treat a wide range of diseases. We believe that new
approaches like microRNA may provide innovative treatment options
in areas of unmet medical need and we look forward to advancing our
research."
First Quarter 2015 Financial Results &
Highlights
Regulus reported a net loss of $14.5
million for the quarter ended March
31, 2015, compared to a net loss of $12.7 million for the quarter ended March 31, 2014. Basic and diluted net loss
per share was $0.29 for the quarter
ended March 31, 2015, compared to net
loss per share of $0.30 for the
quarter ended March 31, 2014.
Regulus recognized revenue of $4.2
million for the quarter ended March
31, 2015, compared to $1.6
million for the quarter ended March
31, 2014. Revenue for the quarter ended March 31, 2015 included a $2.5 million pre-clinical milestone upon
AstraZeneca's selection of RG-125(AZD4076) as a clinical
candidate. Other revenue during these periods consisted primarily
of amortization of up-front payments received, which is recognized
over the estimated period of performance, and payments for other
research services under our strategic alliances and
collaborations.
Research and development expenses were $13.4 million for the quarter ended
March 31, 2015, compared to $9.6
million for quarter ended March 31, 2014. This
increase was primarily driven by Phase I clinical study costs for
RG-101, IND-enabling costs for RG-125(AZD4076) and an increase in
personnel and research costs to support the growth of the
pipeline.
General and administrative expenses were $3.6 million for the quarter ended March 31,
2015, compared to $2.7 million for
the quarter ended March 31, 2014. This increase was primarily
driven by an increase in salaries and related employee costs,
including stock-based compensation.
Net loss for the quarter ended March 31,
2015 included a non-cash charge of $1.8 million from the change in value of our
convertible note payable, compared to a non-cash charge of
$2.1 million for the quarter ended
March 31, 2014. In January 2015, the principal balance of the
convertible note payable of $5.4
million was converted into 1,356,738 shares of common stock
at a conversion price of $4.00 per
share.
As of March 31, 2015, Regulus had
$148.8 million in cash, cash
equivalents and short-term investments and 50,762,489 shares of
common stock outstanding.
2015 Financial Guidance
Regulus' cash guidance remains unchanged and the company expects
to end 2015 with greater than $100.0
million in cash, cash equivalents and short-term
investments.
"Regulus continues to maintain a solid financial balance sheet,
ending the first quarter 2015 with approximately $148 million in cash, which includes milestone
payments received from our strategic alliance partners and
collaborators," said David Szekeres,
Chief Business Officer and General Counsel of Regulus. "We
believe that these relationships will provide opportunities to
recognize additional revenues as we develop our microRNA
therapeutics through clinical studies and eventually toward the
market."
Conference Call & Webcast Information
Regulus will host a conference call and webcast at 5:00 p.m. Eastern Standard Time today to discuss
its first quarter 2015 financial results, recent company highlights
and its expectations for the remainder of the year. 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 40363416.
To access the telephone replay of the call, dial (855) 859-2056
(domestic) or (404) 537-3406 (international), passcode 40363416.
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 microMarkersSM 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. 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 been selected for clinical
development. 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 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-101, RG-012 and
RG-125), 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
microMarkersSM 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
March
31,
|
|
|
2015
|
|
2014
|
|
|
(Unaudited)
|
Revenues:
|
|
|
|
|
|
|
Revenues under strategic alliances and collaborations
|
|
$
4,200
|
|
$
1,631
|
Operating
expenses:
|
|
|
|
|
Research and
development
|
|
13,427
|
|
9,604
|
General and
administrative
|
|
3,644
|
|
2,732
|
Total operating
expenses
|
|
17,071
|
|
12,336
|
Loss from
operations
|
|
(12,871)
|
|
(10,705)
|
Other expense,
net
|
|
(1,620)
|
|
(2,305)
|
Loss before income
taxes
|
|
(14,491)
|
|
(12,740)
|
Income tax (benefit)
expense
|
|
|
(4)
|
|
|
1
|
Net loss
|
|
$
|
(14,487)
|
|
$
|
(12,741)
|
Basic and diluted net
loss per share
|
|
$
|
(0.29)
|
|
$
|
(0.30)
|
Shares used to
compute basic and diluted net loss per share
|
|
|
50,071,165
|
|
|
42,690,200
|
Regulus
Therapeutics Inc.
|
Condensed Balance
Sheets
|
(In
thousands)
|
|
|
|
March 31,
2015
|
|
December 31,
2014
|
|
|
(Unaudited)
|
|
|
|
Assets
|
|
|
|
|
Cash, cash
equivalents and short-term investments
|
|
$
|
148,791
|
|
$
|
159,743
|
Other current
assets
|
|
9,726
|
|
5,208
|
Non-current
assets
|
|
6,331
|
|
6,529
|
Total
assets
|
|
$
|
164,848
|
|
$
|
171,480
|
Liabilities and
stockholders' equity
|
|
|
|
|
Current
liabilities
|
|
$
|
9,044
|
|
$
|
8,698
|
Total deferred
revenue
|
|
8,076
|
|
6,349
|
Convertible notes
payable, at fair value
|
|
—
|
|
23,397
|
Other long-term
liabilities
|
|
903
|
|
1,022
|
Stockholders'
equity
|
|
146,825
|
|
132,014
|
Total
liabilities and stockholders' equity
|
|
$
|
164,848
|
|
$
|
171,480
|
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SOURCE Regulus Therapeutics Inc.