Regulus Provides Update on 'Road to the Clinic' Strategy and Reports First Quarter 2013 Financial Results and Recent Highlights

  Regulus Provides Update on 'Road to the Clinic' Strategy and Reports First
             Quarter 2013 Financial Results and Recent Highlights

- Nominated RG-101 in HCV as First microRNA Candidate for Clinical
Development-

- Maintained Strong Financial Position with Over $90 million in Cash-

PR Newswire

LA JOLLA, Calif., May 14, 2013

LA JOLLA, Calif., May 14, 2013 /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 microRNA therapeutic pipeline under the company's 'Road to the
Clinic' strategy and reported financial results for the quarter ended March
31, 2013.

Regulus Continues to Execute on 'Road to the Clinic' Strategy:
Nominated RG-101 as First microRNA Candidate for Clinical Development

  oRegulus announced today that it has nominated its first microRNA candidate
    for clinical development, RG-101, a GalNAc-conjugated microRNA antagonist
    or anti-miR, which targets microRNA-122 (miR-122) for the treatment of
    patients with chronic hepatitis C virus (HCV) infection. Regulus is
    performing additional pre-clinical studies and finalizing development
    plans for RG-101 in HCV and expects to submit an application with
    regulatory authorities in early 2014.
  oRegulus plans to develop RG-101 independently of its strategic alliance
    with GlaxoSmithKline (GSK). The broad strategic alliance between Regulus
    and GSK remains intact and GSK retains its interest in the miR-122 program
    in HCV, as miR-122 will remain a Collaboration Target under the
    alliance. As such, the companies are in the process of amending the
    Product Development and Commercialization Agreement to clarify that RG-101
    is fully owned by Regulus.

"The nomination of RG-101 as our first microRNA candidate for clinical
development marks a tremendous milestone for Regulus and represents
significant achievement of our initial goals under our 'Road to the Clinic'
strategy," said Kleanthis G. Xanthopoulos, Ph.D., President and CEO of
Regulus. "We continue to focus our efforts on preparing for a successful
transition to a clinical-stage company and remain on track to nominate our
second microRNA candidate for clinical development by the end of the year.
Our recent progress is encouraging and we look forward to realizing the
potential that our microRNA technology may have in transforming the field of
drug discovery."

Neil W. Gibson, Ph.D., Chief Scientific Officer, added, "We believe that
RG-101 is a novel, pan-genotypic agent that may play an important role in
future HCV therapy. We are very encouraged by the pre-clinical data seen to
date, which includes activity against some of the known mutations that lead to
resistance to the current HCV therapies and a favorable potency and PK profile
which supports a once-a-month dosing paradigm. We believe that RG-101 may be
useful in difficult-to-treat HCV patients in combination with existing and
emerging therapies and we look forward to exploring its clinical utility."

Garry E. Menzel, Ph.D., Chief Operating Officer and Executive Vice President
of Finance, said, "As we prepare for clinical activity with RG-101, Regulus
continues to maintain a strong financial position with over $90 million in
cash at quarter-end. We remain on track to achieve our stated goals of
finishing 2013 with at least $60 million in cash, while maintaining a burn
rate of approximately $30 million to $35 million."

Recent Highlight

  oSelected New Opportunity in Oncology. Regulus also announced today that
    it has selected microRNA-221 (miR-221) as an attractive target for
    potential clinical evaluation in patients with hepatocellular carcinoma
    (HCC). Regulus is currently conducting target validation activities and
    expects to report further progress on this opportunity in the second half
    of 2013.

First Quarter 2013 Financial Results & Highlights

Regulus reported a net loss of $7.2 million for the quarter ended March 31,
2013, compared to a net loss of $2.2 million for the quarter ended March 31,
2012. The quarter ended March 31, 2013, included non-cash charges of $1.8
million from the change in value of the amended and restated convertible
promissory note originally issued to GlaxoSmithKline in 2010, which was
attributable to increases in value of the underlying common stock of the
Company since December 31, 2012. Basic and diluted net loss per share was
$0.20 for the quarter ended March 31, 2013, compared to net loss per share of
$13.06 for the quarter ended March 31, 2012. The comparison of net loss per
share for the periods presented is impacted by the initial public offering and
concurrent common stock issuances in October 2012.

Regulus recognized revenue of $3.2 million for the quarter ended March 31,
2013, compared to $3.3 million for the quarter ended March 31, 2012. 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 $6.9 million for the quarter ended
March 31, 2013, compared to $4.6 million for the quarter ended March 31,
2012. The increase is attributable to an expansion of our research and
development team, expertise and capabilities, in addition to increasing
pre-clinical study activities in the first quarter of 2013, compared to the
same period in 2012.

General and administrative expenses were $1.9 million for the quarter ended
March 31, 2013, compared to $0.9 million for the quarter ended March 31,
2012. The increase is attributable to costs associated with increases related
to additional personnel required to support the growth of the business, in
addition to an increase in overall operating costs associated with being an
SEC registrant in the first quarter of 2013, compared to the same period in
2012.

As of March 31, 2013, Regulus had $90.7 million in cash, cash equivalents and
short-term investments, debt with a principal balance of $5.4 million and
approximately 36 million shares of common stock outstanding. The Company
expects its current cash position to be sufficient to fund operations into
2016 and to finish 2013 with at least $60.0 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 for an update on the 'Road to the Clinic' Strategy and to discuss
its first quarter 2013 financial results and recent highlights. A live webcast
of the call will be available online at www.regulusrx.com. A replay will also
be available approximately one hour after completion of the call. To access
the replay, dial (855) 859-2056 (domestic) or (404) 537-3406 (international),
passcode 59485223. The webcast and replay will be archived on the company's
website following the call. 

About the 'Road to the Clinic' Strategy in 2013

Launched in February 2013, the 'Road to the Clinic' Strategy outlines certain
corporate goals that seek to advance our microRNA therapeutic pipeline toward
the clinic this year. Specifically, Regulus set the goal of nominating two
microRNA candidates for clinical development in 2013. We announced the first
candidate today as RG-101 for the treatment of HCV. The company also expects
to file its first applications with regulatory authorities in 2014, with the
first being for RG-101 in early 2014. In order to achieve these stated goals
and advance our programs toward clinical development, Regulus expects to
maintain its strong financial position and end 2013 with at least $60 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 leveraging a mature therapeutic platform based on technology that
has been developed over 20 years. Regulus works with a broad network of
academic collaborators and leverages the oligonucleotide drug discovery and
development expertise of its founding companies, Alnylam Pharmaceuticals and
Isis Pharmaceuticals. Regulus has nominated RG-101 for the treatment of HCV as
a candidate for clinical development and is advancing other microRNA
therapeutics toward clinical development in several areas, including oncology,
fibrosis and metabolic diseases. Regulus has formed strategic alliances with
AstraZeneca, GlaxoSmithKline and Sanofi and a research collaboration with
Biogen Idec.

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, the projected sufficiency of Regulus'
capital position for future periods, the expected ability of Regulus to
undertake certain activities and accomplish certain goals, 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'
SEC filings. 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,
                                                       2013          2012
                                                       (Unaudited)
Revenues:
Revenues under strategic alliances and collaborations  $ 3,238       $ 3,344
Operating expenses:
Research and development                               6,883         4,603
General and administrative                             1,905         921
Total operating expenses                               8,788         5,524
Loss from operations                                   (5,550)       (2,180)
Other income (expense), net                            (1,689)       (66)
Loss before income taxes                               (7,239)       (2,246)
Income tax (benefit) expense                             (10)          1
Net loss                                               $ (7,229)     $ (2,247)
Basic and diluted net loss per share                   $ (0.20)      $ (13.06)
Shares used to compute basic and diluted net loss per    35,872,606    171,998
share





Regulus Therapeutics Inc.
Condensed Balance Sheets
(In thousands)
                                                   March 31,  December 31,

                                                   2013       2012
                                                   (Unaudited)
Assets
Cash, cash equivalents and short-term investments  $  90,715  $   98,100
Other current assets                               1,054      829
Noncurrent assets                                  5,258      4,589
 Total assets                                   $  97,027  $   103,518
Liabilities and stockholders' equity
Current liabilities                                $  3,492   $   2,317
Current portion of deferred revenue                9,201      10,451
Convertible notes payable, at fair value           11,895     10,134
Other long-term liabilities                        804        767
Deferred revenue, less current portion             15,768     17,756
Stockholders' equity                               55,867     62,093
 Total liabilities and stockholders' equity     $  97,027  $   103,518



SOURCE Regulus Therapeutics Inc.

Website: http://www.regulusrx.com
Contact: Amy Conrad, Director, Investor Relations and Corporate
Communications, Regulus Therapeutics Inc., 858-202-6300,
aconrad@regulusrx.com; or Media, David Schull, Russo Partners LLC,
212-845-4271, david.schull@russopartnersllc.com
 
Press spacebar to pause and continue. Press esc to stop.