Financial Overview - The company has received $420.9 million from equity and convertible debt securities, $101.8 million from collaborations, and $19.8 million from a Term Loan as of December 31, 2022[29]. - As of December 31, 2022, the company had cash, cash equivalents, and short-term investments totaling $39.2 million[29]. - As of December 31, 2022, the company had approximately $39.2 million in cash, cash equivalents, and short-term investments, with $6.0 million in outstanding debt obligations[131]. - The company expects its research and development expenses to substantially increase as it advances product candidates through clinical trials[132]. - The company will need to raise additional capital to fund operations and service debt obligations, with existing resources projected to last into early Q1 2024[131]. - The company incurred net losses of $28.3 million and $27.8 million for the years ended December 31, 2022, and 2021, respectively, with an accumulated deficit of $483.2 million as of December 31, 2022[142]. - The company has not generated any revenue from product sales and does not anticipate doing so for the foreseeable future[146]. Clinical Development - The lead product candidate, RGLS8429, is in Phase 1 clinical development for the treatment of autosomal dominant polycystic kidney disease (ADPKD) and has received orphan drug designation from the FDA[23]. - The Phase 1 single-ascending dose study of RGLS8429 in healthy volunteers has been completed, showing it was well-tolerated with no serious adverse events reported[36]. - Enrollment is ongoing in the Phase 1b multiple-ascending dose study for RGLS8429 in adult patients with ADPKD, with top-line data expected in the second half of 2023[36]. - The company is conducting clinical trials in three phases to evaluate the safety and efficacy of its drug candidates, with Phase 1 focusing on safety and dosage, Phase 2 on efficacy and dosage tolerance, and Phase 3 on broader patient populations[79]. - The FDA may require post-marketing clinical trials (Phase 4) to further assess the safety and effectiveness of approved drugs[83]. - Adverse effects (AEs) from product candidates could delay or prevent regulatory approval, impacting market acceptance and increasing commercialization costs[162]. - Regulatory authorities may impose restrictions on product distribution or require additional clinical trials, which could lead to significant delays in obtaining marketing approval[164]. Intellectual Property - The company has a comprehensive intellectual property estate with approximately 145 U.S. and foreign patents and patent applications related to microRNA drug products, expected to expire between 2024 and 2042[46]. - The company holds approximately 145 patents and patent applications related to microRNA and microRNA drug products, along with numerous patents licensed from Alnylam and Ionis related to RNA technologies[51]. - The company may face challenges in negotiating licenses for intellectual property rights from academic institutions, potentially blocking program pursuits[172]. - Patent applications may not result in enforceable patents, affecting collaboration and commercialization[194]. - The company may need to obtain licenses from third parties, which could hinder product development if not secured[204]. - Litigation to protect patents may be expensive and time-consuming, with uncertain outcomes[205]. Regulatory Environment - The FDA regulates the drug development process, which requires substantial time and financial resources, and non-compliance can lead to severe sanctions[69]. - The company must submit preclinical test results and other data to the FDA as part of the IND process, which becomes effective 30 days after submission unless a clinical hold is imposed[71]. - The FDA has a 10-month review period for standard New Drug Applications (NDAs) and a 6-month period for priority NDAs, although these timelines may be extended if additional information is required[78]. - The company must submit annual progress reports and IND safety reports to the FDA for any serious adverse events during clinical trials[74]. - The company is subject to ongoing FDA regulation post-approval, including record-keeping and reporting of adverse experiences[91]. - The company must ensure compliance with FDA regulations regarding advertising and promotion, particularly concerning off-label uses of approved drugs[91]. - The company is subject to various regulations governing clinical trials and commercial sales in different jurisdictions, impacting product development timelines[117]. Market and Competition - The biotechnology industry is characterized by intense competition, with potential competitors having greater financial resources and expertise in research and development[63]. - The company faces significant competition for talent in the life sciences field, which may affect its ability to attract and retain employees[124]. - The biotechnology and pharmaceutical industries are highly competitive, with established companies having greater financial and technical resources, which could adversely affect the company's operating results[209]. - The company's ability to compete will depend on its experience in drug discovery and development, as well as the acceptance of its product candidates by the medical community[212]. - The commercial success of product candidates is contingent upon demonstrating clinical safety and efficacy compared to existing products[212]. Collaboration and Partnerships - The collaboration with Sanofi was terminated in January 2023, which included the only product candidate advanced into the clinic, lademirsen[33]. - The company relies on third-party collaborations for the development and commercialization of certain microRNA product candidates, which may limit control over development processes[178]. - Collaborations with third parties for marketing and sales are essential, especially in markets outside the United States, to mitigate resource limitations[215]. Compliance and Legal Risks - The company is subject to increased civil and criminal penalties under HIPAA and HITECH for violations related to the privacy and security of health information[102]. - The company may face significant civil and criminal penalties for violations of the U.S. Foreign Corrupt Practices Act, which prohibits bribery of foreign officials[97]. - The company must report annually to CMS information related to payments or transfers of value made to healthcare professionals under the Physician Payments Sunshine Act[104]. - The company could be adversely affected by challenges under federal and state healthcare laws, which may result in penalties and reputational harm[101]. - The company anticipates potential expansion into the EU and UK, which would subject it to GDPR and UK GDPR regulations[103]. - The company may face increased compliance costs and legal risks due to various state privacy laws, such as the California Consumer Privacy Act[103]. - The company is affected by ongoing healthcare reform initiatives that seek to reduce healthcare costs, which could impact future operations[107]. Manufacturing and Supply Chain - The company contracts with third parties for manufacturing and does not own or operate its own manufacturing facilities[61]. - The company relies on third-party manufacturers to produce clinical and commercial quantities of its products, which must comply with FDA's current good manufacturing practices (cGMP)[92]. - The company is dependent on third-party manufacturers for producing clinical supplies, and any disruptions in the supply chain could delay product development and commercialization[183]. - Limited sources of supply for drug substances may cause delays in developing and commercializing product candidates if disruptions occur[184]. - Manufacturing issues may increase product and regulatory approval costs or delay commercialization[188]. Financial Obligations and Future Outlook - The company is required to maintain a cash balance of no less than $5.0 million under the Loan Agreement and is in compliance with all covenants as of the filing date[136]. - The outstanding amounts under the Term Loan mature on May 1, 2024[137]. - The company may incur additional indebtedness in the future, which could contain provisions as restrictive as those in the current Loan Agreement[140]. - The transition from LIBOR to alternative reference rates like SOFR could result in higher interest rates on the outstanding principal amount under the Term Loan, affecting the company's financial position[138]. - The company anticipates significant expenses and increasing operating losses for the foreseeable future, with fluctuations in net losses expected from quarter to quarter[145].

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 ...

Regulus Therapeutics, Inc. (NASDAQ:RGLS) Q2 2022 Earnings Conference Call August 11, 2022 5:00 PM ET Company Participants Crispina Calsada - CFO Joseph Hagan - President & CEO Conference Call Participants Yi Chen - H.C. Wainwright & Co. Operator Good morning, and welcome to the Regulus Therapeutics Second Quarter Financial Results Conference Call. All participants will be in a listen-only mode. [Operator Instructions] After today's presentation, there will be an opportunity to ask questions. [Operator instr ...

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 JUNE 30, 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) (State or Other Jurisdiction of Incorporat ...

Regulus Therapeutics, Inc. (NASDAQ:RGLS) Q1 2022 Earnings Conference Call May 12, 2022 5:00 PM ET Company Participants Crispina Calsada - CFO Joseph Hagan - President, CEO & Director Conference Call Participants Yi Chen - H.C. Wainwright & Co. Operator Good day, and welcome to the Regulus Therapeutics Inc. First Quarter 2022 Conference Call. [Operator Instructions]. Please note, this event is being recorded. I would now like to turn the conference over to Cris Calsada, Chief Financial Officer. Please go ahe ...

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 (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 FOR THE QUARTERLY PERIOD ENDED MARCH 31, 2022 or ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 FOR THE TRANSITION ...

Regulus Therapeutics Inc. (NASDAQ:RGLS) Q4 2022 Earnings Conference Call March 10, 2022 5:00 PM ET Company Participants Cris Calsada - Chief Financial Officer Jay Hagan - President and Chief Executive Officer Denis Drygin - Chief Scientific Officer Conference Call Participants Brian Cheng - Cantor Fitzgerald Yi Chen - H.C. Wainwright Operator Ladies and gentlemen thank you for standing by and welcome to the Regulus Therapeutics Q4 2021 Conference Call. [Operator Instructions] I’d now like to turn the confer ...

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, 2021 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 2021 Earnings Conference Call November 10, 2021 5:00 PM ET Company Participants Cris Calsada - Chief Financial Officer Jay Hagan - President and Chief Executive Officer Denis Drygin - Chief Scientific Officer Conference Call Participants Brian Cheng - Cantor Fitzgerald Yi Chen - H.C. Wainwright Operator Good afternoon, ladies and gentlemen and welcome to the Regulus Therapeutics Inc. Quarter 3 2021 Conference Call. [Operator Instructions] As a reminder, this confer ...