Financial Overview - The company has received $530.1 million from equity and convertible debt securities, $101.8 million from collaborations, and $19.8 million from a Term Loan as of March 31, 2024[86]. - As of March 31, 2024, the company had cash, cash equivalents, and investments totaling $107.7 million[86]. - Cash, cash equivalents, and investments totaled $107.7 million as of March 31, 2024, following a private placement financing that raised approximately $93.9 million in net proceeds[109]. - Net cash used in operating activities was $8.7 million for the three months ended March 31, 2024, an increase from $8.1 million in the same period of 2023[115]. - Net cash used in investing activities was $78.5 million for the three months ended March 31, 2024, compared to net cash provided of $15.0 million in the same period of 2023[116]. - Net cash provided by financing activities was $93.1 million for the three months ended March 31, 2024, compared to net cash used of $0.8 million in the same period of 2023[117]. - The outstanding principal amount of the Term Loan was $0.6 million as of March 31, 2024, with a floating interest rate[121]. - The company anticipates needing to raise additional capital to fund future operations, which may include equity or debt financings[112]. Research and Development - The company has incurred approximately $436.8 million in research and development expenses since inception through March 31, 2024[98]. - Research and development expenses increased to $6.0 million for the three months ended March 31, 2024, up 23% from $4.9 million in the same period of 2023[104]. - The company is focused on developing therapeutics targeting microRNAs, which may provide a new class of drugs for complex diseases[85]. Clinical Development - RGLS8429, targeting autosomal dominant polycystic kidney disease (ADPKD), is in Phase 1b clinical development, with positive top-line data showing 36%-41% increases in mean PC1 levels at Day 85 and Day 86[90]. - The second cohort of the Phase 1b study showed greater biological activity of RGLS8429 at 2 mg/kg, with encouraging imaging-based biomarker results indicating potential impact on kidney volume[91]. - The ongoing Phase 1b study is evaluating RGLS8429 at 3 mg/kg in cohort 3 and a 300 mg fixed dose in cohort 4, with an increased sample size to up to 30 patients in cohort 4[92]. - The FDA granted orphan drug designation to RGLS8429 for the treatment of ADPKD in June 2022[81]. - The company aims to explore an accelerated approval pathway for RGLS8429 based on a single pivotal Phase 2 study, confirmed in a Type D meeting with the FDA in December 2023[94]. - RGLS8429 was well-tolerated in clinical trials, with no serious adverse events reported[89]. Expenses - General and administrative expenses rose to $2.8 million for the three months ended March 31, 2024, compared to $2.4 million in the same period of 2023, primarily due to non-cash stock-based compensation[107]. - Net interest and other income was $0.4 million for the three months ended March 31, 2024, compared to $0.2 million for the same period in 2023[108].

Exhibit 99.1 Regulus Therapeutics Reports First Quarter 2024 Financial Results and Recent Updates Initiated the fourth cohort of patients in the Phase 1b multiple-ascending dose (MAD) clinical trial of RGLS8429 for the treatment of autosomal dominant polycystic kidney disease (ADPKD) Topline data from the third cohort of patients in the Phase 1b MAD study of RGLS8429 expected in mid-2024 Ended first quarter 2024 with cash, cash equivalents, and investments of $107.7 million; Cash runway into H1 2026 SAN DIE ...

Table of Contents UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, D.C. 20549 FORM 10-K (Mark One) ☒ ANNUAL REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 FOR THE FISCAL YEAR ENDED DECEMBER 31, 2023 or ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 FOR THE TRANSITION PERIOD FROM TO Commission file number: 001-35670 Regulus Therapeutics Inc. (Exact name of registrant as specified in its charter) Delaware 26-4738379 (State ...

Exhibit 99.1 Regulus Therapeutics Reports Fourth Quarter and Year-End 2023 Financial Results and Recent Updates Positive topline data from the second cohort of patients in Phase 1b Multiple-Ascending Dose (MAD) Clinical Trial of RGLS8429 for the Treatment of Autosomal Dominant Polycystic Kidney Disease (ADPKD) Oversubscribed $100 million private placement; expected to extend cash runway into H1 2026 Completed enrollment of the third cohort of patients in the MAD study; Data anticipated mid-2024 Completed Ty ...

UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, D.C. 20549 FORM10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 FOR THE QUARTERLY PERIOD ENDED SEPTEMBER 30, 2023 or TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 FOR THE TRANSITION PERIOD FROM TO Commission file number: 001-35670 Regulus Therapeutics Inc. (Exact name of registrant as specified in its charter) (State or Other Jurisdiction of Incorpo ...

FORM10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 FOR THE QUARTERLY PERIOD ENDED JUNE 30, 2023 UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, D.C. 20549 or TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 FOR THE TRANSITION PERIOD FROM TO Commission file number: 001-35670 Regulus Therapeutics Inc. (Exact name of registrant as specified in its charter) (State or Other Jurisdiction of Incorporatio ...

UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, D.C. 20549 FORM10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 FOR THE QUARTERLY PERIOD ENDED MARCH 31, 2023 FOR THE TRANSITION PERIOD FROM TO Commission file number: 001-35670 Regulus Therapeutics Inc. (Exact name of registrant as specified in its charter) (State or Other Jurisdiction of Incorporation or Organization) 4224 Campus Point Court, Suite 210 92121 San Diego CA (Address of Princi ...

Table of Contents UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, D.C. 20549 FORM 10-K (Mark One) ☒ ANNUAL REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 FOR THE FISCAL YEAR ENDED DECEMBER 31, 2022 or ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 FOR THE TRANSITION PERIOD FROM TO Commission file number: 001-35670 Regulus Therapeutics Inc. (Exact name of registrant as specified in its charter) Delaware 26-4738379 (State ...

Regulus Therapeutics Inc. (NASDAQ:RGLS) Q3 2022 Earnings Conference Call November 10, 2022 5:00 PM ET Company Participants Crispina Calsada - CFO Jay Hagan - President, CEO & Director Denis Drygin - Chief Scientific Officer Conference Call Participants Whitney Ijem - Canaccord Genuity Yanan Zhu - Wells Fargo Securities Operator Hello, and welcome to the Regulus Therapeutics Inc. Q3 2022 Earnings Conference Call. [Operator Instructions]. I would now like to turn the conference over to Chief Financial Officer ...

DECIII is | --- | --- | --- | --- | |--------------------------------------|-------|-------|-------| | | | | | | Corporate Presentation. August 2022 | | | | | | | | | Safe Harbor Statement 2 • 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 the Company's RGLS8429 program, including the potential outcome of clinical develop ...