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 ...

PART I. FINANCIAL INFORMATION [Financial Statements](index=5&type=section&id=Item%201.%20Financial%20Statements) The unaudited condensed financial statements for the nine months ended September 30, 2021, reveal a net loss of $20.7 million, an increase from the prior year, and raise substantial doubt about the company's ability to continue as a going concern - The company has substantial doubt about its ability to continue as a going concern, as its cash and cash equivalents may not be sufficient to fund operations for one year following the issuance of these financial statements and intends to seek additional capital to continue operations[27](index=27&type=chunk)[28](index=28&type=chunk) [Condensed Balance Sheets](index=5&type=section&id=Condensed%20Balance%20Sheets) As of September 30, 2021, the company's cash and cash equivalents increased to $35.8 million, with total assets reaching $43.0 million and total liabilities $14.0 million Condensed Balance Sheet Data (in thousands) | | September 30, 2021 (Unaudited) | December 31, 2020 | | :--- | :--- | :--- | | **Assets** | | | | Cash and cash equivalents | $35,848 | $31,087 | | Total current assets | $39,986 | $36,730 | | Total assets | $43,042 | $37,604 | | **Liabilities and Stockholders' Equity** | | | | Total current liabilities | $11,460 | $11,578 | | Total liabilities | $14,030 | $11,578 | | Total stockholders' equity | $29,012 | $26,026 | | Total liabilities and stockholders' equity | $43,042 | $37,604 | [Condensed Statements of Operations and Comprehensive Loss](index=7&type=section&id=Condensed%20Statements%20of%20Operations%20and%20Comprehensive%20Loss) For the nine months ended September 30, 2021, the company reported zero revenue and an increased net loss of $20.7 million due to higher operating expenses Condensed Statements of Operations (in thousands, except per share data) | | Nine months ended Sep 30, 2021 | Nine months ended Sep 30, 2020 | | :--- | :--- | :--- | | Revenue under collaborations | $0 | $5,006 | | Research and development | $13,385 | $11,396 | | General and administrative | $7,471 | $6,736 | | Total operating expenses | $20,856 | $18,132 | | Loss from operations | ($20,856) | ($13,126) | | Net loss | ($20,675) | ($14,408) | | Net loss per share, basic and diluted | ($0.26) | ($0.47) | [Condensed Statements of Changes in Stockholders' Equity (Deficit)](index=8&type=section&id=Condensed%20Statements%20of%20Changes%20in%20Stockholders%27%20Equity%20%28Deficit%29) Stockholders' equity increased to $29.0 million by September 30, 2021, primarily driven by capital raised through the At-The-Market offering - Issuance of common stock through the At-The-Market (ATM) offering was a primary driver of changes in equity, contributing **$5.7 million** in the first quarter and **$14.8 million** in the second quarter of 2021[18](index=18&type=chunk) [Condensed Statements of Cash Flows](index=10&type=section&id=Condensed%20Statements%20of%20Cash%20Flows) Net cash used in operating activities was $16.3 million, offset by $21.3 million provided by financing activities, resulting in a $4.8 million net increase in cash Summary of Cash Flows (in thousands) | | Nine months ended Sep 30, 2021 | Nine months ended Sep 30, 2020 | | :--- | :--- | :--- | | Net cash used in operating activities | ($16,297) | ($15,725) | | Net cash used in investing activities | ($210) | ($11) | | Net cash provided by (used in) financing activities | $21,330 | ($542) | | Net increase (decrease) in cash | $4,823 | ($16,278) | | Cash and cash equivalents at end of period | $35,910 | $17,843 | - Financing activities in the first nine months of 2021 provided **$21.3 million** in cash, primarily from the issuance of common stock, a significant reversal from the **$0.5 million** cash used in the same period of 2020[23](index=23&type=chunk) [Notes to Condensed Financial Statements](index=11&type=section&id=Notes%20to%20Condensed%20Financial%20Statements) Key notes include the 'going concern' assumption, details on the $4.7 million Term Loan, forgiveness of the $0.7 million PPP loan, and the Sanofi collaboration - The company's financial statements are prepared on a 'going concern' basis, but management has identified substantial doubt about its ability to continue as a going concern due to insufficient cash to fund operations for the next year[27](index=27&type=chunk) - As of September 30, 2021, **$4.7 million** was outstanding under the Term Loan with Oxford Finance, with principal payments scheduled to begin on January 1, 2022[58](index=58&type=chunk)[60](index=60&type=chunk) - The company received full forgiveness of its approximately **$0.7 million** Paycheck Protection Program (PPP) loan in the second quarter of 2021, which was recorded as a gain in other income[64](index=64&type=chunk) - In August 2020, an amendment to the Sanofi agreement resulted in recognizing **$5.0 million** in revenue in Q3 2020, and as of September 30, 2021, an additional **$25.0 million** in potential development milestone payments from Sanofi is fully constrained and not recognized as revenue[87](index=87&type=chunk)[88](index=88&type=chunk) [Management's Discussion and Analysis of Financial Condition and Results of Operations](index=23&type=section&id=Item%202.%20Management%27s%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) Management discusses the company's clinical-stage focus on microRNAs, the advancement of RGLS8429, and critical liquidity challenges including zero collaboration revenue and increased R&D expenses [Overview](index=24&type=section&id=OVERVIEW) Regulus, a clinical-stage biopharmaceutical company, is prioritizing RGLS8429 for ADPKD, with an IND submission planned for Q2 2022, while Sanofi develops RG-012 - The company's lead product candidate is RGLS8429, an anti-miR targeting miR-17 for the treatment of ADPKD, following the discontinuation of the first-generation compound, RGLS4326[102](index=102&type=chunk)[111](index=111&type=chunk) - Regulus plans to have a pre-IND meeting with the FDA for RGLS8429 in late 2021 and submit an IND to initiate a Phase 1 clinical study in the second quarter of 2022[111](index=111&type=chunk) - The RG-012 program for Alport syndrome was transitioned to Sanofi, which is now responsible for all development costs and is currently enrolling patients in a Phase 2 trial[102](index=102&type=chunk)[110](index=110&type=chunk) [Results of Operations](index=27&type=section&id=RESULTS%20OF%20OPERATIONS) Collaboration revenue was zero for the nine months ended September 30, 2021, while R&D expenses increased by 17% and G&A expenses rose by 12% Comparison of Results of Operations (in thousands) | | Nine months ended Sep 30, 2021 | Nine months ended Sep 30, 2020 | | :--- | :--- | :--- | | Revenue under collaborations | $0 | $5,006 | | Research and development expenses | $13,385 | $11,396 | | General and administrative expenses | $7,471 | $6,736 | - Revenue was **zero** for the first nine months of 2021, compared to **$5.0 million** in the same period of 2020, which was recognized upon completion of material transfers to Sanofi[125](index=125&type=chunk) - R&D expenses increased by **17%** for the nine-month period, primarily driven by a **28%** increase in third-party and outsourced expenses related to drug manufacturing for the RG8429 product candidate[127](index=127&type=chunk) - Net interest and other income for the nine months ended September 30, 2021 included a **$0.7 million** gain on the forgiveness of the company's PPP loan[129](index=129&type=chunk) [Liquidity and Capital Resources](index=28&type=section&id=LIQUIDITY%20AND%20CAPITAL%20RESOURCES) As of September 30, 2021, the company had $35.8 million in cash, but faces substantial doubt about its going concern ability due to historical losses and significant future capital needs - The company had cash and cash equivalents of **$35.8 million** as of September 30, 2021[130](index=130&type=chunk) - The financial statements have been prepared on a 'going concern' basis, but there is substantial doubt about the company's ability to continue as such due to its financial condition[131](index=131&type=chunk)[132](index=132&type=chunk) Summary of Cash Flows (in thousands) | | Nine months ended Sep 30, 2021 | Nine months ended Sep 30, 2020 | | :--- | :--- | :--- | | Net cash used in operating activities | ($16,297) | ($15,725) | | Net cash provided by (used in) financing activities | $21,330 | ($542) | [Quantitative and Qualitative Disclosures about Market Risk](index=30&type=section&id=Item%203.%20Quantitative%20and%20Qualitative%20Disclosures%20about%20Market%20Risk) The company's primary market risk is interest rate exposure from its $4.7 million floating-rate Term Loan, with potential impacts from LIBOR phase-out - The company has interest rate exposure from its outstanding Term Loan, which has a floating rate based on LIBOR, and the planned phase-out of LIBOR may require renegotiation and could result in higher interest rates[142](index=142&type=chunk) - Due to the short-term nature of cash equivalents, management believes a **10%** change in interest rates would not have a material effect on their fair value or on interest expense[141](index=141&type=chunk)[143](index=143&type=chunk) [Controls and Procedures](index=30&type=section&id=Item%204.%20Controls%20and%20Procedures) Management concluded that disclosure controls and procedures were effective as of September 30, 2021, with no material changes to internal control over financial reporting - The principal executive officer and principal financial officer concluded that the company's disclosure controls and procedures were effective as of September 30, 2021[145](index=145&type=chunk) - No material changes to the company's internal control over financial reporting were identified during the most recent fiscal quarter[146](index=146&type=chunk) PART II. OTHER INFORMATION [Legal Proceedings](index=31&type=section&id=Item%201.%20Legal%20Proceedings) The company is not currently a party to any legal proceedings that would materially affect its financial position or operations - The company is not currently a party to any material legal proceedings[147](index=147&type=chunk) [Risk Factors](index=31&type=section&id=Item%201A.%20Risk%20Factors) The company faces substantial risks including novel drug discovery challenges, clinical trial failures, regulatory uncertainty, financial instability, reliance on third parties, intellectual property issues, and stock volatility - The company's approach to drug discovery using microRNA technology is novel and may never lead to marketable products[149](index=149&type=chunk) - There is substantial doubt about the company's ability to continue as a going concern, and it will need to raise additional capital to fund operations[179](index=179&type=chunk) - The company depends on collaborations, such as with Sanofi, for the development and commercialization of certain product candidates, and the termination or failure of these collaborations could materially harm the business[200](index=200&type=chunk) - The company may be unable to comply with Nasdaq's continued listing requirements, including the minimum **$1.00** bid price, which could result in delisting[291](index=291&type=chunk)[292](index=292&type=chunk) [Risks Related to Discovery and Development](index=31&type=section&id=RISKS%20RELATED%20TO%20THE%20DISCOVERY%20AND%20DEVELOPMENT%20OF%20PRODUCT%20CANDIDATES) Risks include the potential failure of novel microRNA drug discovery, clinical trial failures, unpredictable regulatory approval, and challenges in securing intellectual property - The company's drug discovery approach is novel and may never lead to marketable products, and its focus solely on microRNA technology increases this risk[149](index=149&type=chunk)[150](index=150&type=chunk) - Clinical trials may fail to demonstrate safety and efficacy, and early positive results are not predictive of final outcomes, as exemplified by the discontinuation of RGLS4326 after discussions with the FDA and data from its Phase 1b trial[155](index=155&type=chunk)[158](index=158&type=chunk) - Obtaining regulatory approval is uncertain and unpredictable, and even if granted, the FDA may impose significant restrictions, post-marketing studies, or withdraw the product if new problems arise[166](index=166&type=chunk)[167](index=167&type=chunk) [Risks Related to Financial Condition and Capital Needs](index=36&type=section&id=RISKS%20RELATED%20TO%20OUR%20FINANCIAL%20CONDITION%20AND%20NEED%20FOR%20ADDITIONAL%20CAPITAL) The company faces significant financial risks including a history of losses, substantial doubt about its going concern ability, and the need for additional capital to fund operations and service debt - The company will need to raise additional capital to continue as a going concern, as current cash is insufficient to fund operations and service debt obligations[179](index=179&type=chunk) - The company has incurred significant losses since inception, with a net loss of **$20.7 million** for the first nine months of 2021 and an accumulated deficit of **$447.7 million**[191](index=191&type=chunk)[192](index=192&type=chunk) - The company has never generated revenue from product sales and its ability to achieve profitability is uncertain and dependent on successful drug development and commercialization[197](index=197&type=chunk) [Risks Related to Reliance on Third Parties](index=39&type=section&id=RISKS%20RELATED%20TO%20OUR%20RELIANCE%20ON%20THIRD%20PARTIES) The company's business heavily relies on third-party collaborators like Sanofi, limited manufacturers, and CROs, posing risks of development delays and commercialization failures - The company depends on collaborations with partners like Sanofi for financial and scientific resources, and the failure or termination of these collaborations could prevent the commercialization of product candidates[200](index=200&type=chunk) - The company relies on a limited number of suppliers for raw materials and drug substance manufacturing, and any disruption in this supply chain could cause significant delays in development and commercialization[209](index=209&type=chunk) - The company relies on third-party CROs to conduct clinical trials and is responsible for ensuring their compliance with GCPs, and poor performance by CROs could lead to unreliable data, trial delays, and failure to obtain regulatory approval[214](index=214&type=chunk)[215](index=215&type=chunk) [Risks Related to Intellectual Property](index=43&type=section&id=RISKS%20RELATED%20TO%20OUR%20INTELLECTUAL%20PROPERTY) Success depends on protecting intellectual property, but patent applications may be challenged, and the company faces infringement claims and risks from license agreement non-compliance - The company's ability to compete effectively depends on protecting its intellectual property through patents and trade secrets, but its patent applications may fail to issue or be invalidated if challenged[218](index=218&type=chunk) - The company faces the risk of third-party intellectual property infringement claims, which could block product commercialization and result in substantial litigation expenses and damages[225](index=225&type=chunk)[227](index=227&type=chunk) - The company relies on license agreements with its founding companies, Alnylam and Ionis, and other third parties, and failure to comply with these agreements could lead to the loss of important intellectual property rights[228](index=228&type=chunk) [Risks Related to Commercialization](index=45&type=section&id=RISKS%20RELATED%20TO%20COMMERCIALIZATION%20OF%20PRODUCT%20CANDIDATES) Commercial success is uncertain due to reliance on partners, intense industry competition, challenges in market acceptance, and the need to establish sales and marketing capabilities - Commercial success for partnered programs, like the miR-21 program with Sanofi, depends heavily on the partner's efforts, over which Regulus has little control[234](index=234&type=chunk)[235](index=235&type=chunk) - The company faces significant competition from major pharmaceutical and biotechnology companies with greater financial and technical resources[236](index=236&type=chunk) - Market acceptance of any approved products will depend on factors like clinical safety, convenience, pricing, and the ability to secure adequate reimbursement from government and private payors[240](index=240&type=chunk)[248](index=248&type=chunk) [Risks Related to Business Operations and Industry](index=48&type=section&id=RISKS%20RELATED%20TO%20OUR%20BUSINESS%20OPERATIONS%20AND%20INDUSTRY) Operational risks include dependence on key personnel, compliance with complex healthcare and data privacy laws, cybersecurity threats, and potential disruptions from catastrophic events like pandemics - The company is highly dependent on its principal executive team and faces intense competition for qualified scientific and technical personnel[252](index=252&type=chunk) - The business is subject to numerous federal and state healthcare fraud and abuse laws, as well as data privacy regulations like GDPR and CCPA, with violations carrying significant penalties[258](index=258&type=chunk)[259](index=259&type=chunk) - The COVID-19 pandemic poses risks of disruption to clinical trials, manufacturing supply chains, and access to capital, with the ultimate impact remaining uncertain[285](index=285&type=chunk)[288](index=288&type=chunk) - The company is exposed to significant cybersecurity risks, including ransomware and supply chain attacks, which could interrupt operations, compromise sensitive data, and result in financial loss and reputational harm[271](index=271&type=chunk)[272](index=272&type=chunk) [Risks Related to Common Stock](index=55&type=section&id=RISKS%20RELATED%20TO%20OUR%20COMMON%20STOCK) Risks related to common stock include high price volatility, potential Nasdaq delisting due to non-compliance, future equity dilution, and limitations on net operating loss carryforwards - The market price of the company's common stock has been and is expected to continue to be highly volatile[290](index=290&type=chunk) - The company may be unable to comply with Nasdaq's continued listing requirements, including the **$1.00** minimum bid price, and faces the risk of delisting, having received a notice of non-compliance from Nasdaq on August 9, 2021[291](index=291&type=chunk)[292](index=292&type=chunk) - Future sales of equity securities to raise needed capital will result in substantial dilution to existing stockholders[298](index=298&type=chunk) - The company's ability to use its significant net operating loss (NOL) carryforwards of over **$300 million** may be limited due to past and future 'ownership changes' under Section 382 of the tax code[302](index=302&type=chunk)[303](index=303&type=chunk) [Unregistered Sales of Equity Securities and Use of Proceeds](index=59&type=section&id=Item%202.%20Unregistered%20Sales%20of%20Equity%20Securities%20and%20Use%20of%20Proceeds) The company reported no unregistered sales of equity securities during the period - None[307](index=307&type=chunk) [Defaults Upon Senior Securities](index=60&type=section&id=Item%203.%20Defaults%20Upon%20Senior%20Securities) The company reported no defaults upon senior securities during the period - None[308](index=308&type=chunk) [Mine Safety Disclosures](index=60&type=section&id=Item%204.%20Mine%20Safety%20Disclosures) This item is not applicable to the company - Not applicable[309](index=309&type=chunk) [Other Information](index=60&type=section&id=Item%205.%20Other%20Information) The company reported no other information required to be disclosed - None[310](index=310&type=chunk) [Exhibits](index=61&type=section&id=Item%206.%20Exhibits) This section lists exhibits filed with the Form 10-Q, including corporate governance documents, securities forms, and CEO/CFO certifications - The exhibits include various corporate governance documents, forms of securities, and required certifications by the CEO and CFO[312](index=312&type=chunk)[313](index=313&type=chunk)