1. Targeting the Pathways of Human Disease
Kleanthis G. Xanthopoulos, Ph.D.
SD Venture Group Panel
May 22, 2013
NASDAQ: RGLS RegulusRx.com

2. Targeting the Pathways of Human Disease
Safe Harbor Statement
Statements contained in this presentation 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 in the future, the
expected ability of Regulus to undertake certain activities and accomplish certain goals, the projected timeline of
clinical development activities and regulatory approvals, 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’ programs are described in additional detail in Regulus’ SEC filings. All forward-looking
statements contained in this presentation 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.

3. Targeting the Pathways of Human Disease3
Experience with Introducing 2 Innovative
Companies to the Public Markets
• Innovative science in microRNAs
• IPO in October 2012 - raised $80.9M
• 1st San Diego life science IPO in 2012
• 2nd preclinical IPO in last 5 years
• 6 large strategic partners who are
also shareholders (no VCs)
• Innovative anti-viral company
• Discovered Best-in-Class HCV non-nuc
• IPO in 2004 - raised $49M
• Sold to Roche in 2011 for $230M
NASDAQ: RGLS
October 2012
NASDAQ: ANDS
March 2004
(acquired by Roche)

4. Targeting the Pathways of Human Disease
 The life science IPO windows over the last 15 years have each
offered different, unique characteristics:
 Duration of window
 Varying typical company profiles
 Different pricing terms
 Performance of deal
 Current IPO window has been “OPEN” for 45 months
 2009 – Present, 46 IPOs have priced, raising $6.0B
 Backlog is moderate (currently 13 IPOs)
 JOBS has allowed for confidential filings
4
Recent Life Sciences IPO Windows –
Broad Perspective
Source: Lazard ECM Analytics and FactSet as of 05/17/13.
Note: Excludes SPACs and transactions less than $10mm.

5. Targeting the Pathways of Human Disease5
Source: ECM Analytics and FactSet as of 05/17/13.
Note: Excludes SPACs and transactions less than $10mm.
Key IPO Stats Similar Across 3 Recent Windows

6. Targeting the Pathways of Human Disease
6
Pricing Conditions Remain Challenging in
Current “OPEN” Window
Source: ECM Analytics and FactSet as of 05/17/13.
Note: Excludes SPACs and transactions less than $10mm.
Median amount raised $58.0M/Median Pre-Money $126.4M

7. Targeting the Pathways of Human Disease7
Insider Participation Approx. 70% for
Current “OPEN” Window
In the Current “Window” Median % of
Proceeds from Insiders is 34.1%

8. Targeting the Pathways of Human Disease
 Current “open” window vs. prior two windows:
1. Key difference is significant insider participation
− Expected by public investors but also by Board and management teams
2. Later-stage companies (meaning Phase II or later)
− Approximately 90% in Phase II or later
− Regulus is the exception to the rule in this trend
3. Increase in filing/offer discount results in lower pre-money valuation
− Median discount 28.1%/pre-money value $126M
4. Deals remain club deals
− Type of investor buying life sciences IPOs is same across windows
− Increase in median number of institutional buyers:
 Median of 25 buyers in current window vs. 11 and 17 in prior two windows
8
Current Life Sciences IPO Window –
4 Key Takeaways
Source: Lazard Analytics, VentureXpert and FactSet pricing as of 05/17/13
Note: Excludes transactions less than $10mm

9. Targeting the Pathways of Human Disease
 IPO is the most difficult financing
 Get well prepared
 12 months of preparation to draft your message
 Board and Governance
 Visit the key investors (about three dozen) more than twice prior to
the IPO
 Choose bankers wisely!
− Don’t choose banks; choose individuals
− Bulge bracket not always needed; no one will remember who took you
public
 Have faith, prepare for a marathon!
9
Some Key Points to Consider

10. Targeting the Pathways of Human Disease10
First-in-Class
Medicines
Regulus Going Public – Case Study
 Challenging Environment:
 RGLS is the exception to the rule
− Pre-clinical vs. later stage
− Unique capital structure – formed by 2 public companies (ISIS/ALNY), no VC
funding; over 70% insider participation in IPO deal
− Only San Diego based life science companies to go public in 2012
 Raised $80.9M in IPO and Related Transactions
 Significant insider participation
 AstraZeneca, Biogen Idec, Sanofi, GSK and Isis Pharmaceuticals
 Chose investment banks that fit Regulus’ needs
 Lazard, Cowen, BMO, Needham and WedBush
 Pre-IPO preparation was critical
 Conduct multiple meetings with public investors prior to IPO

11. Targeting the Pathways of Human Disease11
Not Much Has Meaningfully Changed in last 15 Years
In the IPO markets,
the more things
change, the more they
stay the same…