Regulus Reports Third Quarter 2016 Financial Results Conference Call Today at 5:00 p.m.

Regulus Reports Third Quarter 2016 Financial Results

Conference Call Today at 5:00 p.m. ET

LA JOLLA, Calif., November 1, 2016 Regulus Therapeutics Inc. (Nasdaq: RGLS), a biopharmaceutical company leading the discovery and

development of innovative medicines targeting microRNAs, today reported financial results for the three and nine months ended September 30, 2016 and provided a summary of corporate highlights.

Our focus in the third quarter included the advancement of the RG-012 development program and addressing the clinical hold for RG-101 while expanding

the pre-clinical pipeline, said Paul Grint, M.D., President and Chief Executive Officer of Regulus. We continue to be excited with the progress of our research programs, and look forward to sharing an update at our first R&D day on

Revenue was $0.2 million and $1.2 million for the three and nine months ended September 30, 2016, respectively, compared to $1.9 million and $9.9 million for the same periods in 2015. Revenue for the three and nine months ended September 30,

2016 and 2015 consisted of amortization of up-front payments from Regulus strategic alliances and collaborations. Revenue for the three and nine months ended September 30, 2015 included $0.9 million and $4.1 million, respectively, for research

services under Regulus strategic alliances and collaborations. Preclinical milestones earned under Regulus strategic alliances and collaborations were $0.3 million and $3.2 million for the three and nine months ended September 30,

Research and Development (R&D) Expenses: R&D expenses were $14.6 million and $49.3 million for the three

and nine months ended September 30, 2016, respectively, compared to $11.0 million and $43.6 million for the same periods in 2015. The increases in R&D expenses were primarily driven by the advancement of our clinical programs and increased

investment in our preclinical pipeline.

General and Administrative (G&A) Expenses: G&A expenses were $4.8 million and $13.6

million for the three and nine months ended September 30, 2016, respectively, compared to $4.2 million and $13.7 million for the same periods in 2015.

Net Loss: Net loss was $19.5 million, or $0.37 per share, and $61.8 million, or $1.17 per share, for the three and nine months ended September 30,

2016, respectively, compared to a net loss of $13.0 million, or $0.25 per share, and $48.5 million, or $0.95 per share, for the same periods in 2015.

Cash Position: Cash, cash equivalents, and short-term investments were $91.7 million at September 30, 2016, compared with $108.0 million at June

30, 2016 and $115.3 million at December 31, 2015.

Conference Call Details

Regulus will host a conference

call and webcast at 5:00 p.m. Eastern Time today to discuss third quarter financial results and provide a general business update. 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 99499252. To access the replay of the call, dial (855) 859-2056 (domestic) or (404) 537-3406 (international), passcode 99499252. The webcast and telephone

replay will be archived on the company s website following the call.

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 leadership in the microRNA field. 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 entered Phase I/IIa

clinical development through its strategic alliance with AstraZeneca. Regulus is also advancing several programs toward clinical development in renal, hepatic and central nervous systems diseases. 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. 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 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), 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

and biomarkers 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.

Investor Relations Contact:

Regulus Therapeutics Inc.

Selected Financial Information

Condensed Statement of Operations

(In thousands, except share and per share data)

Regulus Therapeutics Inc.

Condensed Balance Sheets