[PART I. FINANCIAL INFORMATION](index=6&type=section&id=PART%20I.%20FINANCIAL%20INFORMATION) [Item 1. Financial Statements](index=6&type=section&id=Item%201.%20Financial%20Statements) Regulus Therapeutics reported a $9.6 million net loss for Q1 2025, with $65.4 million in cash and investments, raising going concern doubts despite a pending Novartis acquisition Condensed Balance Sheet Data (in thousands) | Account | March 31, 2025 (Unaudited, in thousands) | December 31, 2024 (in thousands) | | :--- | :--- | :--- | | **Assets** | | | | Cash and cash equivalents | $49,460 | $38,528 | | Short-term investments | $15,908 | $37,249 | | Total current assets | $71,694 | $82,345 | | Total assets | $74,826 | $84,181 | | **Liabilities & Equity** | | | | Total current liabilities | $4,397 | $7,499 | | Total liabilities | $5,798 | $7,773 | | Total stockholders' equity | $69,028 | $76,408 | Condensed Statement of Operations (in thousands, except per share data) | Metric | Three months ended March 31, 2025 (in thousands) | Three months ended March 31, 2024 (in thousands) | | :--- | :--- | :--- | | Research and development | $6,819 | $6,040 | | General and administrative | $3,719 | $2,786 | | Loss from operations | $(10,538) | $(8,826) | | Net loss | $(9,634) | $(8,469) | | Net loss per share, basic and diluted | $(0.15) | $(0.29) | Condensed Statement of Cash Flows (in thousands) | Activity | Three months ended March 31, 2025 (in thousands) | Three months ended March 31, 2024 (in thousands) | | :--- | :--- | :--- | | Net cash used in operating activities | $(10,613) | $(8,742) | | Net cash provided by (used in) investing activities | $21,500 | $(78,544) | | Net cash provided by financing activities | $45 | $93,105 | | Net increase in cash, cash equivalents and restricted cash | $10,932 | $5,819 | - The company's financial statements are prepared on a going concern basis, but **current liquidity, recurring losses, and negative cash flows raise substantial doubt** about its ability to continue operations for one year[27](index=27&type=chunk) - Regulus entered a merger agreement with Novartis AG on April 29, 2025, for a cash tender offer of **$7.00 per share plus a $7.00 CVR per share** upon milestone achievement[82](index=82&type=chunk)[83](index=83&type=chunk) [Item 2. Management's Discussion and Analysis of Financial Condition and Results of Operations](index=24&type=section&id=Item%202.%20Management%27s%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) Management discusses microRNA therapeutics, positive farabursen Phase 1b results, a **$9.6 million net loss** for Q1 2025, and going concern doubts without the Novartis acquisition - The company is a clinical-stage biopharmaceutical firm focused on developing microRNA-targeting drugs, with lead candidate **farabursen in Phase 1b clinical development** for ADPKD[98](index=98&type=chunk) - Positive topline results from the farabursen Phase 1b study's fourth cohort showed a **mean halting of height-adjusted total kidney volume (htTKV) growth** over four months[118](index=118&type=chunk) Comparison of Operating Expenses (in thousands) | Expense Category | Q1 2025 (in thousands) | Q1 2024 (in thousands) | Change ($ in thousands) | Change (%) | | :--- | :--- | :--- | :--- | :--- | | Research and development | $6,819 | $6,040 | $779 | 13% | | General and administrative | $3,719 | $2,786 | $933 | 33.5% | - Increased R&D expenses were primarily due to **ADPKD program clinical advancement costs** and higher non-cash stock-based compensation, which also drove the increase in G&A expenses[131](index=131&type=chunk)[132](index=132&type=chunk) - As of March 31, 2025, the company held approximately **$65.4 million in cash and investments**, sufficient to fund operations only through Q1 2026, raising substantial doubt about its going concern ability if the Novartis merger is not completed[135](index=135&type=chunk)[136](index=136&type=chunk) [Item 3. Quantitative and Qualitative Disclosures about Market Risk](index=31&type=section&id=Item%203.%20Quantitative%20and%20Qualitative%20Disclosures%20about%20Market%20Risk) Market risk primarily stems from interest rate fluctuations on short-term cash and investments, with a **10% interest rate change** not expected to materially affect asset fair value - Excess cash is invested in **money market funds and U.S. Treasury securities** to ensure liquidity and preserve principal[147](index=147&type=chunk) - Due to the short-term nature of investments, a hypothetical **10% change in interest rates** would not materially impact the portfolio's realized value[148](index=148&type=chunk) [Item 4. Controls and Procedures](index=31&type=section&id=Item%204.%20Controls%20and%20Procedures) Management concluded disclosure controls and procedures were effective as of March 31, 2025, with no material changes to internal control over financial reporting during the quarter - Disclosure controls and procedures, evaluated by the CEO and CFO, were concluded to be **effective at a reasonable assurance level** as of March 31, 2025[150](index=150&type=chunk) - No material changes to internal control over financial reporting were identified during the last fiscal quarter[151](index=151&type=chunk) [PART II. OTHER INFORMATION](index=31&type=section&id=PART%20II.%20OTHER%20INFORMATION) [Item 1. Legal Proceedings](index=31&type=section&id=Item%201.%20Legal%20Proceedings) The company is not currently a party to any material legal proceedings - As of the report date, Regulus Therapeutics is **not a party to any material legal proceedings**[152](index=152&type=chunk) [Item 1A. Risk Factors](index=32&type=section&id=Item%201A.%20Risk%20Factors) The company faces significant risks including the uncertainty of the Novartis acquisition, a history of losses, the unproven nature of microRNA therapeutics, potential clinical trial failures, reliance on third parties, and intellectual property and data security concerns - A primary risk is the potential failure to complete the pending Novartis acquisition, which could adversely affect the business and stock price, and may require a **$27.3 million termination fee**[154](index=154&type=chunk)[155](index=155&type=chunk)[156](index=156&type=chunk) - The company has a history of significant losses, with a **$569.2 million accumulated deficit** as of March 31, 2025, and current cash sufficient only through Q1 2026, raising substantial doubt about its going concern ability without the Novartis merger or additional capital[210](index=210&type=chunk)[217](index=217&type=chunk) - The company's novel microRNA technology drug discovery approach may not lead to marketable products, as **no microRNA-targeting therapeutic has yet received regulatory approval**[166](index=166&type=chunk) - Clinical trials for product candidates like farabursen may fail to demonstrate safety and efficacy, with no guarantee of successful final results or regulatory approval despite positive interim data[172](index=172&type=chunk)[188](index=188&type=chunk) - Reliance on third-party manufacturers with limited supply sources for clinical product candidates poses risks of **significant delays and increased costs** due to manufacturing or supply chain disruptions[232](index=232&type=chunk)[233](index=233&type=chunk) - The company faces risks of data security breaches and cyberattacks, with failure to protect sensitive data potentially leading to **regulatory actions, litigation, and reputational harm**[289](index=289&type=chunk)[297](index=297&type=chunk) [Item 2. Unregistered Sales of Equity Securities and Use of Proceeds](index=66&type=section&id=Item%202.%20Unregistered%20Sales%20of%20Equity%20Securities%20and%20Use%20of%20Proceeds) No unregistered sales of equity securities occurred during the period - No unregistered sales of equity securities occurred[342](index=342&type=chunk) [Item 3. Defaults Upon Senior Securities](index=66&type=section&id=Item%203.%20Defaults%20Upon%20Senior%20Securities) No defaults upon senior securities occurred during the period - No defaults upon senior securities occurred[343](index=343&type=chunk) [Item 4. Mine Safety Disclosures](index=66&type=section&id=Item%204.%20Mine%20Safety%20Disclosures) This item is not applicable to the company - This item is not applicable to the company[346](index=346&type=chunk) [Item 5. Other Information](index=67&type=section&id=Item%205.%20Other%20Information) No other information is reported for the period - No other information is reported for the period[347](index=347&type=chunk) [Item 6. Exhibits](index=68&type=section&id=Item%206.%20Exhibits) This section lists the exhibits filed with the Form 10-Q, including the Merger Agreement with Novartis, charter documents, and officer certifications - Key exhibits filed include the **Merger Agreement with Novartis AG**, various certificates of incorporation, and officer certifications[348](index=348&type=chunk)[349](index=349&type=chunk)

Exhibit 99.1 Regulus Therapeutics Reports First Quarter 2025 Financial Results and Recent Updates Entered into an agreement to be acquired by Novartis for $7.00 per share in cash, with potential to receive an additional $7.00 per share in cash through a contingent value right ("CVR") upon the achievement of a regulatory milestone, for a total equity value of up to approximately $1.7 billion; Expected to be completed in the second half of 2025, subject to customary closing conditions Positive topline data fr ...

Entered into an agreement to be acquired by Novartis for $7.00 per share in cash, with potential to receive an additional $7.00 per share in cash through a contingent value right ("CVR") upon the achievement of a regulatory milestone, for a total equity value of up to approximately $1.7 billion; Expected to be completed in the second half of 2025, subject to customary closing conditionsPositive topline data from the completed fourth cohort of patients in the Phase 1b multiple-ascending dose (MAD) study of f ...

Group 1 - Monteverde & Associates PC has recovered millions for shareholders and is recognized as a Top 50 Firm in the 2024 ISS Securities Class Action Services Report [1] - The firm is investigating Regulus Therapeutics Inc. regarding its proposed merger with Novartis AG, which includes an initial payment of $7.00 per share and a non-tradeable CVR of $7.00 per share contingent on regulatory approval [1] Group 2 - The firm operates from the Empire State Building in New York City and has a successful track record in trial and appellate courts, including the U.S. Supreme Court [2] - Monteverde & Associates PC emphasizes that no company, director, or officer is above the law, and offers free information to shareholders concerned about their investments [3]

智通财经APP获悉，诺华制药(NVS.US)周三发布声明称，已达成协议将收购美国生物技术公司Regulus Therapeutics Inc.(RGLS.US)，这笔交易最高价值可达17亿美元。报告显示，这家瑞士制药巨头将通过一 家子公司，以每股7美元的价格预付总计8亿美元现金。 受此消息影响，Regulus Therapeutics股价在周三美股盘前飙升，截至发稿，上涨134%。同日，诺华股 票在瑞士市场表现平稳，不过在过去12个月里，其股价已累计上涨近6%。 此外，Regulus Therapeutics的股东还将获得一项或有价值权(CVR)——如果用于治疗常染色体显性多囊 肾病(ADPKD，这是导致肾衰竭最常见的遗传病因)的药物farabursen获得监管批准，他们还可额外获得9 亿美元。 目前，这笔收购交易已获得双方董事会批准，预计将于2025年下半年完成。 诺华首席财务官哈里·基尔希本周表示，公司正在积极寻求并购机会，希望借此在2025年后提升销售额; 同时他也指出，生物技术领域的企业估值正呈现下行趋势。 Regulus Therapeutics专注于研发靶向微小核糖核酸(microRNA)的疗法， ...

智通财经4月30日电，诺华4月30日宣布达成协议收购美国生物技术公司Regulus Therapeutics，交易包括 8亿美元预付款，达到未来监管里程碑后可能额外支付至多9亿美元。该交易预计将于今年下半年完成。 Regulus致力于开发microRNA疗法，专注于治疗常染色体显性多囊肾病（ADPKD）。 诺华将斥资至多17亿美元收购生物技术公司Regulus Therapeutics ...

Core Insights - Regulus Therapeutics Inc. (RGLS) has reached a significant support level, indicating a potential investment opportunity from a technical perspective [1] - The recent breakout of RGLS's 50-day simple moving average above its 200-day moving average signifies a "golden cross," which is a bullish indicator [1] - RGLS has experienced a substantial price increase of 97.7% over the last four weeks, suggesting strong upward momentum [3] Technical Analysis - A golden cross event consists of three stages: the stock price bottoms out, the shorter moving average crosses above the longer moving average, and the stock maintains upward momentum [2] - The occurrence of a golden cross is contrasted with a death cross, which indicates potential bearish price movement [2] Earnings Expectations - RGLS's earnings expectations have shown positive changes, with two upward revisions and no downward revisions in the last 60 days, enhancing the bullish outlook [3] - The Zacks Consensus Estimate for RGLS has also moved upward, further supporting the positive sentiment around the stock [3] Investment Consideration - Given the technical indicators and positive earnings estimate movements, RGLS is recommended for investors to consider adding to their watchlist [5]

Core Insights - Regulus Therapeutics announced positive topline results from the fourth cohort of its Phase 1b MAD study of farabursen for treating Autosomal Dominant Polycystic Kidney Disease (ADPKD) [2][4] - The study demonstrated a significant halting of kidney volume growth in patients receiving a 300 mg fixed dose of farabursen over four months [1][4] - The company is on track to initiate a Phase 3 pivotal trial in Q3 2025, with a focus on a 12-month endpoint for kidney volume change [2][6] Study Details - The Phase 1b MAD study was a double-blind, placebo-controlled trial assessing the safety, tolerability, pharmacokinetics, and pharmacodynamics of farabursen in adult ADPKD patients [3] - The study included three weight-based dose levels and one fixed dose level, with primary endpoints focused on urinary PC1 and PC2 changes, and an exploratory examination of htTKV growth rate [3][4] - In the fourth cohort, 26 subjects received 300 mg of farabursen bi-weekly for three months, showing consistent mechanistic responses and halting htTKV growth [4][5] Efficacy and Safety - Patients treated with farabursen showed a mean htTKV growth rate of 0.05%, compared to 2.58% in placebo subjects, indicating a significant reduction in kidney volume growth [5] - Urinary levels of PC1 and PC2 increased significantly, correlating with the observed effects on kidney volume [5] - Farabursen demonstrated a favorable safety and tolerability profile, consistent with earlier cohorts, with no serious adverse events reported [5][9] Future Plans - The company has confirmed alignment with the FDA regarding the Phase 3 trial design, which includes a 12-month htTKV endpoint for Accelerated Approval and a 24-month eGFR endpoint for Full Approval [6][9] - Regulus aims to advance farabursen as a potential treatment option for ADPKD, addressing a significant unmet medical need [6][10] Background on ADPKD - ADPKD is a common genetic disorder characterized by the development of multiple cysts in the kidneys, leading to end-stage renal disease in approximately 50% of patients by age 60 [8] - The disease affects around 160,000 individuals in the U.S., with a global prevalence estimated between 4 to 7 million [8]

Core Viewpoint - Regulus (RGLS) has been upgraded to a Zacks Rank 2 (Buy), indicating a positive outlook on its earnings estimates, which is a significant factor influencing stock prices [1][3]. Earnings Estimates and Stock Price Impact - The Zacks rating system is based on changes in earnings estimates, which are strongly correlated with near-term stock price movements [4][6]. - Institutional investors often rely on earnings estimates to determine the fair value of stocks, leading to buying or selling actions that affect stock prices [4]. Regulus Earnings Outlook - For the fiscal year ending December 2025, Regulus is expected to report earnings of -$1.38 per share, reflecting a 68.3% decrease from the previous year [8]. - Despite the negative earnings outlook, analysts have raised their estimates for Regulus, with a 0.2% increase in the Zacks Consensus Estimate over the past three months [8]. Zacks Rank System - The Zacks Rank system classifies stocks into five groups based on earnings estimates, with a strong historical performance, particularly for Zacks Rank 1 stocks, which have averaged a +25% annual return since 1988 [7]. - Regulus's upgrade to Zacks Rank 2 places it in the top 20% of Zacks-covered stocks, suggesting potential for market-beating returns in the near term [10].

Financial Overview - The company has received a total of $530.1 million from equity and convertible debt securities, $101.8 million from collaborations, and $19.8 million from a Term Loan as of December 31, 2024[30]. - As of December 31, 2024, the company had cash, cash equivalents, and short-term investments totaling $75.8 million[30]. - The net loss for the year ended December 31, 2024, was $46.4 million, compared to a net loss of $30.0 million for the year ended December 31, 2023, with an accumulated deficit of $559.6 million[192]. - The company has incurred losses in each year since its inception in September 2007, and it does not anticipate generating revenues from product sales for the foreseeable future[191][196]. - The company may need to raise additional capital to fund operations, and if unable to do so, it may have to significantly delay or discontinue development of future product candidates[186][190]. - The company relies primarily on equity and/or debt financings for funding, which may create challenges if there is volatility in the equity markets[188]. Product Development - The product candidate farabursen is in Phase 1b clinical development for the treatment of autosomal dominant polycystic kidney disease (ADPKD) and has shown improvements in kidney function in preclinical models[31]. - In September 2023, positive top-line data was announced from the first cohort of farabursen-treated ADPKD patients, with no safety concerns reported[36]. - In March 2024, positive topline results were reported from the second cohort, showing greater biological activity at a 2 mg/kg dose compared to lower doses[37]. - The third cohort results in June 2024 indicated consistent increases in urinary PC1 and PC2 levels at the 3 mg/kg dose[39]. - An End of Phase 1 meeting with the FDA in December 2024 supported the potential for an accelerated approval pathway based on a single pivotal Phase 3 study of farabursen[41]. - The company announced the discontinuation of RGLS4326 development in October 2021, shifting focus to farabursen targeting miR-17[151]. - The FDA granted Orphan Drug Designation to farabursen for the treatment of ADPKD in June 2022, with potential for additional designations for other candidates[172]. - The company may seek accelerated approval for farabursen based on a single pivotal Phase 3 study, but there is no assurance that the FDA will agree to this pathway[165][167]. Regulatory Environment - The FDA review process for NDAs typically takes 10 months for standard applications and 6 months for priority applications, but may be extended[90]. - The FDA may require post-marketing clinical trials to further assess drug safety and effectiveness after initial approval[95]. - Orphan drug designation can provide exclusivity for 7 years if the product receives the first FDA approval for a rare disease[97]. - The FDA has expedited development pathways such as Fast Track and Breakthrough Therapy designations to accelerate the approval process for serious conditions[98]. - The Fast Track program expedites the development of drugs for serious conditions, allowing for rolling review of NDA sections as they are completed[99]. - Breakthrough Therapy designation provides intensive guidance on drug development, starting as early as Phase 1, for drugs showing substantial improvement over existing therapies[100]. - Accelerated Approval allows drugs to be approved based on surrogate endpoints, with post-marketing studies required to confirm clinical benefits[101]. - Priority Review is granted to products that offer significant improvements in treatment where no satisfactory alternatives exist, facilitating faster evaluation by the FDA[102]. Competition and Market Landscape - The biotechnology and pharmaceutical industries are characterized by intense competition, with the company facing potential competition from larger, better-funded pharmaceutical companies[74]. - Otsuka Pharmaceutical Co., Ltd. received FDA approval in 2018 for Jynarque® to slow kidney function decline in adults at risk of rapidly-progressing ADPKD[75]. - Vertex completed IND-enabling studies of VX-407 in 2024 for a specific PKD1 mutation and is recruiting for a Phase 1 clinical trial[75]. - PYC Therapeutics announced regulatory clearance to initiate a Phase 1 study of PYC-003 in February 2025 targeting the PKD1 gene[75]. - Competitors have greater financial resources and expertise in R&D, which may lead to resource concentration through collaborations and M&A[76]. - The commercial potential may be reduced if competitors develop safer, more effective, or less expensive products[77]. Intellectual Property - The company has a portfolio of approximately 145 U.S. and foreign patents and patent applications related to microRNA drug products, with expected patent protection expiration between 2025 and 2045[53]. - The company has exclusive rights to approximately 150 patents and patent applications related to microRNA and microRNA drug products, with key chemical modifications expected to expire in 2027 and 2029[58]. - Intellectual property protection is critical, as failure to secure patents or protect trade secrets may hinder competitive positioning in the market[216]. - The lifespan of patents is limited, and once expired, the company may face competition from generic medications, affecting revenue potential[220]. - The company may encounter significant challenges in protecting intellectual property rights in foreign markets, which could adversely affect business operations[222]. Clinical Trials and Risks - The company faces significant risks if preclinical and clinical studies of product candidates do not yield successful results[146]. - The marketability of product candidates may suffer if adequate coverage and reimbursement are not provided by government and third-party payors[127]. - The company is subject to various regulations governing clinical trials and commercial sales in different jurisdictions[128]. - The company experienced delays in clinical trials, including a voluntary pause in the Phase 1 MAD trial for RGLS4326 due to unexpected toxicity observations in a mouse study[151]. - Clinical trial delays could shorten exclusive commercialization rights and allow competitors to enter the market earlier, impacting revenue generation[155]. - Adverse effects observed in clinical trials could lead to interruptions, delays, or denials of regulatory approval, significantly affecting commercialization efforts[156][157]. - The company faces risks related to the interpretation of clinical data, which may differ from regulatory authorities, impacting the approval and commercialization of product candidates[163]. - The outcome of clinical trials is uncertain, and interim results may not predict final outcomes, which could materially affect business prospects[159][162]. Operational Challenges - Manufacturing is contracted out to third parties, and the company does not own or operate manufacturing facilities[71]. - The company depends on third-party manufacturers for producing preclinical and clinical product candidates, which introduces risks related to supply chain disruptions and regulatory compliance[204]. - Limited sources of supply for drug substances may cause delays in developing and commercializing product candidates if disruptions occur[207]. - Manufacturing issues could lead to increased costs and delays in regulatory approvals, impacting the commercialization timeline[210]. - The company relies on CROs and clinical trial sites for conducting clinical trials, and any unsatisfactory performance could harm business prospects[212]. Employee and Organizational Structure - As of December 31, 2024, the company had 34 full-time employees, with 25 engaged in research and development[134]. - The company focuses on microRNA technology for drug development, which is a novel approach with no prior regulatory approvals for therapeutics targeting microRNAs[142]. - The success of the company's business depends on the ability to identify, develop, and commercialize microRNA therapeutics[145]. - The company relies on third-party collaborations for the development and commercialization of certain microRNA product candidates, which may limit control over development processes[199]. - There is a risk that third-party collaborators may not meet deadlines or perform satisfactorily, potentially delaying product development and revenue generation[201].