Reneo Pharmaceuticals(RPHM) - 2023 Q3 - Quarterly Report

Part I — Financial Information Item 1. Consolidated Financial Statements (Unaudited) The unaudited consolidated financial statements detail the company's financial position, operations, and cash flows Consolidated Balance Sheets Assets and Liabilities (in thousands) | Assets and Liabilities | Sep 30, 2023 | Dec 31, 2022 | | :--- | :--- | :--- | | Assets: | | | | Cash and cash equivalents | $11,737 | $19,927 | | Short-term investments | $113,877 | $81,246 | | Prepaid expenses and other current assets | $3,158 | $5,180 | | Total current assets | $128,772 | $106,353 | | Property and equipment, net | $529 | $453 | | Right-of-use assets | $1,006 | $1,292 | | Other non-current assets | $81 | $84 | | Total assets | $130,388 | $108,182 | | Liabilities: | | | | Accounts payable | $2,473 | $1,893 | | Accrued expenses | $10,765 | $4,827 | | Operating lease liabilities, current portion | $325 | $404 | | Total current liabilities | $13,563 | $7,124 | | Operating lease liabilities, less current portion | $812 | $1,059 | | Performance award | $1,069 | $29 | | Total liabilities | $15,444 | $8,212 | | Stockholders' equity: | | | | Common stock | $3 | $3 | | Additional paid-in capital | $305,479 | $236,693 | | Accumulated deficit | $(190,517) | $(136,683) | | Accumulated other comprehensive loss | $(21) | $(43) | | Total stockholders' equity | $114,944 | $99,970 | | Total liabilities and stockholders' equity | $130,388 | $108,182 | Consolidated Statements of Operations and Comprehensive Loss Operating Expenses and Loss (in thousands, except per share data) | Operating Expenses and Loss | Three Months Ended Sep 30, 2023 | Three Months Ended Sep 30, 2022 | Nine Months Ended Sep 30, 2023 | Nine Months Ended Sep 30, 2022 | | :--- | :--- | :--- | :--- | :--- | | Research and development | $13,622 | $9,938 | $39,009 | $27,348 | | General and administrative | $7,266 | $3,902 | $19,038 | $11,938 | | Total operating expenses | $20,888 | $13,840 | $58,047 | $39,286 | | Loss from operations | $(20,888) | $(13,840) | $(58,047) | $(39,286) | | Other income | $1,692 | $833 | $4,213 | $931 | | Net loss | $(19,196) | $(13,007) | $(53,834) | $(38,355) | | Unrealized gain (loss) on short-term investments | $10 | $(194) | $22 | $(60) | | Comprehensive loss | $(19,186) | $(13,201) | $(53,812) | $(38,415) | | Net loss per share, basic and diluted | $(0.57) | $(0.53) | $(1.81) | $(1.57) | | Weighted-average shares used in computing net loss per share, basic and diluted | 33,807,945 | 24,496,313 | 29,718,689 | 24,472,974 | Consolidated Statements of Changes in Stockholders' Equity Stockholders' Equity (in thousands, except share data) | Stockholders' Equity | Dec 31, 2022 | Mar 31, 2023 | Jun 30, 2023 | Sep 30, 2023 | | :--- | :--- | :--- | :--- | :--- | | Common Stock (Shares) | 24,699,553 | 25,107,430 | 33,800,788 | 33,872,166 | | Common Stock (Amount) | $3 | $3 | $3 | $3 | | Additional Paid-In Capital | $236,693 | $238,859 | $303,877 | $305,479 | | Accumulated Other Comprehensive Income (Loss) | $(43) | $12 | $(31) | $(21) | | Accumulated Deficit | $(136,683) | $(151,790) | $(171,321) | $(190,517) | | Total Stockholders' Equity | $99,970 | $87,084 | $132,528 | $114,944 | - Issuance of common stock in public offering, net of offering costs: $58,862 thousand (7,906,250 shares) in Q2 2023[17][48] - Issuance of common stock in private placement, net of offering costs: $4,667 thousand (625,000 shares) in Q2 2023[17][49] Consolidated Statements of Cash Flows Cash Flows (in thousands) | Cash Flows | Nine Months Ended Sep 30, 2023 | Nine Months Ended Sep 30, 2022 | | :--- | :--- | :--- | | Net cash used in operating activities | $(43,929) | $(31,749) | | Net cash used in investing activities | $(29,254) | $(30,925) | | Net cash provided by financing activities | $64,993 | $146 | | Net decrease in cash and cash equivalents | $(8,190) | $(62,528) | | Cash and cash equivalents, beginning of period | $19,927 | $124,660 | | Cash and cash equivalents, end of period | $11,737 | $62,132 | Notes to Consolidated Financial Statements (Unaudited) 1. Organization and Business The company is a clinical-stage pharmaceutical firm facing substantial doubt about its ability to continue as a going concern - Reneo Pharmaceuticals, Inc. is a clinical-stage pharmaceutical company focused on developing therapies for rare genetic mitochondrial diseases, with mavodelpar (REN001) as its lead product candidate[25] - The company incurred a net loss of $53.8 million for the nine months ended September 30, 2023, with an accumulated deficit of $190.5 million[26] - Management has concluded that substantial doubt exists about the company's ability to continue as a going concern for more than one year from the filing date[28] - Plans to alleviate going concern risk include raising additional capital through equity or debt financings and the ability to defer certain commercial development activities[28] 2. Summary of Significant Accounting Policies The financial statements are prepared under GAAP, consistent with prior annual reports, with recent accounting standard updates noted - The unaudited consolidated financial statements are prepared in accordance with GAAP and include accounts of the Company and its wholly-owned subsidiaries[30][32] - The Company adopted ASU 2016-13 (Financial Instruments - Credit Losses) as of January 1, 2023, with no material impact on its financial statements[34] - The Company is currently evaluating the impact of ASU 2022-03 (Fair Value Measurement of Equity Securities Subject to Contractual Sale Restrictions)[35] 3. Net Loss Per Share Basic loss per share is presented, while diluted loss per share excludes anti-dilutive common stock equivalents - Basic loss per share is calculated by dividing net loss by the weighted average number of common shares outstanding[37] - Diluted net loss per share excludes common stock equivalents (stock options and restricted stock units) because their inclusion would be anti-dilutive[37] Anti-Dilutive Securities | Anti-Dilutive Securities | As of September 30, 2023 | As of September 30, 2022 | | :--- | :--- | :--- | | Common stock options outstanding | 6,095,807 | 4,470,120 | | Unvested restricted stock units | 441,500 | 309,500 | | Total | 6,537,307 | 4,779,620 | 4. Fair Value Measurements Financial instruments are classified using a three-tier hierarchy, with a significant Level 3 liability for a performance award - The company categorizes its money market funds as Level 1 and commercial paper and U.S. treasury securities as Level 2[41] - The CEO's special performance award is a Level 3 liability, measured at fair value using a Monte Carlo simulation[42] Fair Value Measurements (in thousands) | Fair Value Measurements | Level 1 (Sep 30, 2023) | Level 2 (Sep 30, 2023) | Level 3 (Sep 30, 2023) | Total (Sep 30, 2023) | | :--- | :--- | :--- | :--- | :--- | | Assets: | | | | | | Money market investments | $4,946 | — | — | $4,946 | | U.S. treasury securities | — | $113,877 | — | $113,877 | | Total Assets | $4,946 | $113,877 | — | $118,823 | | Liabilities: | | | | | | Performance award | — | — | $1,069 | $1,069 | | Total Liabilities | — | — | $1,069 | $1,069 | | Fair Value Measurements | Level 1 (Dec 31, 2022) | Level 2 (Dec 31, 2022) | Level 3 (Dec 31, 2022) | Total (Dec 31, 2022) | | :--- | :--- | :--- | :--- | :--- | | Assets: | | | | | | Money market investments | $9,365 | — | — | $9,365 | | Commercial paper | — | $4,978 | — | $4,978 | | U.S. treasury securities | — | $76,253 | — | $76,253 | | Commercial paper | — | $4,993 | — | $4,993 | | Total Assets | $9,365 | $86,224 | — | $95,589 | | Liabilities: | | | | | | Performance award | — | — | $29 | $29 | | Total Liabilities | — | — | $29 | $29 | Performance Award (in thousands) | Performance Award | Amount | | :--- | :--- | | Balance as of January 1, 2023 | $29 | | Change in fair value | $1,040 | | Balance as of September 30, 2023 | $1,069 | 5. Marketable Debt Securities The company's available-for-sale debt securities, primarily U.S. treasuries, are carried at fair value with minor unrealized losses - Investments in debt securities are classified as current assets available-for-sale and are carried at fair value[45] Available-for-sale securities (in thousands) | Available-for-sale securities | Amortized Cost (Sep 30, 2023) | Gross Unrealized Gains (Sep 30, 2023) | Gross Unrealized Losses (Sep 30, 2023) | Fair Market Value (Sep 30, 2023) | | :--- | :--- | :--- | :--- | :--- | | U.S. treasury securities | $113,898 | $1 | $(22) | $113,877 | | Total | $113,898 | $1 | $(22) | $113,877 | | Available-for-sale securities | Amortized Cost (Dec 31, 2022) | Gross Unrealized Gains (Dec 31, 2022) | Gross Unrealized Losses (Dec 31, 2022) | Fair Market Value (Dec 31, 2022) | | :--- | :--- | :--- | :--- | :--- | | U.S. treasury securities | $76,297 | $2 | $(46) | $76,253 | | Commercial paper | $4,993 | — | — | $4,993 | | Total | $81,290 | $2 | $(46) | $81,246 | 6. Accrued Expenses Accrued expenses more than doubled, driven by increased clinical, regulatory, and compensation-related costs Accrued Expenses (in thousands) | Accrued Expenses | As of Sep 30, 2023 | As of Dec 31, 2022 | | :--- | :--- | :--- | | Accrued clinical and regulatory | $5,176 | $1,872 | | Accrued contract manufacturing cost | $1,005 | $1,583 | | Accrued compensation | $3,830 | $807 | | Accrued other | $754 | $565 | | Total accrued expenses | $10,765 | $4,827 | 7. Leases The company holds operating leases for office space in the U.S. and U.K. with a weighted-average remaining term of 3.4 years - The Company leases office space in Irvine, California (through November 30, 2026) and Sandwich, United Kingdom (through October 23, 2027)[47] Lease Information | Lease Information | As of Sep 30, 2023 | As of Sep 30, 2022 | | :--- | :--- | :--- | | Weighted incremental borrowing rate | 5% | 5% | | Weighted average remaining lease term (in years) | 3.4 | 3.8 | | Cash paid for amounts included in the measurement of lease liabilities (in thousands) | $430 | $397 | | Lease expense (in thousands) | $354 | $338 | Maturities of Lease Liabilities (in thousands) | Maturities of Lease Liabilities | As of Sep 30, 2023 | | :--- | :--- | | 2023 (remaining three months) | $94 | | 2024 | $381 | | 2025 | $381 | | 2026 | $343 | | 2027 | $34 | | Total lease payments | $1,233 | | Less: Imputed interest | $(96) | | Present value of lease liabilities | $1,137 | 8. Stockholders' Equity The company raised approximately $63.6 million in net proceeds through a public offering and a concurrent private placement in May 2023 - On May 8, 2023, the Company completed a public offering, selling 7,906,250 shares of common stock at $8.00 per share, generating approximately $58.9 million in net proceeds[48] - On May 9, 2023, the Company completed a concurrent private placement, selling 625,000 shares of common stock to Abingworth Bioventures 8 L.P. at $8.00 per share, generating approximately $4.7 million in net proceeds[49] 9. Stock-Based Compensation Stock-based compensation expense increased, driven by awards under the 2021 Equity Incentive Plan and a CEO performance award Shares Reserved for Future Issuance (as of Sep 30, 2023) | Shares Reserved for Future Issuance | Shares | | :--- | :--- | | Common stock options outstanding | 6,095,807 | | Unvested restricted stock units | 441,500 | | Available for future grants under the 2021 Equity Incentive Plan | 1,300,869 | | Available for future grants under the 2021 Employee Stock Purchase Plan | 546,341 | | Total shares of common stock reserved | 8,384,517 | Stock Option Activity (nine months ended Sep 30, 2023) | Stock Option Activity | Options Outstanding | Weighted Average Exercise Price | | :--- | :--- | :--- | | Outstanding at December 31, 2022 | 5,877,745 | $4.47 | | Granted | 548,915 | $6.88 | | Exercised | (149,190) | $2.15 | | Forfeited/Expired | (181,663) | $5.60 | | Outstanding at September 30, 2023 | 6,095,807 | $4.71 | | Vested at September 30, 2023 | 2,946,079 | $4.94 | | Exercisable at September 30, 2023 | 3,416,089 | $4.52 | - Unrecognized stock-based compensation expense at September 30, 2023, was $9.5 million, expected to be recognized over a weighted-average vesting term of 2.4 years[56] - The CEO's $7.5 million Performance Award resulted in $1.0 million in compensation expense for the nine months ended September 30, 2023, recognized in general and administrative expense[64][65] Stock-Based Compensation Expense (in thousands) | Stock-Based Compensation Expense | Three Months Ended Sep 30, 2023 | Three Months Ended Sep 30, 2022 | Nine Months Ended Sep 30, 2023 | Nine Months Ended Sep 30, 2022 | | :--- | :--- | :--- | :--- | :--- | | Research and development | $537 | $411 | $1,504 | $1,184 | | General and administrative | $892 | $579 | $2,289 | $1,919 | | Total | $1,429 | $990 | $3,793 | $3,103 | 10. License Agreement The company holds an exclusive worldwide license for mavodelpar from vTv Therapeutics, involving milestone and royalty payment obligations - In December 2017, the Company in-licensed mavodelpar (REN001) from vTv Therapeutics LLC, obtaining an exclusive, worldwide license for PPARδ agonists[25][69] - The license agreement included an initial upfront fee of $3.0 million and the issuance of 576,443 shares of common stock to vTv Therapeutics[69] - The Company is obligated to pay up to $64.5 million in development and regulatory milestones and up to $30.0 million in sales-based milestones, plus tiered royalty payments[70] 11. Subsequent Events Post-quarter end, the company repurchased shares from vTv Therapeutics and established a new $100.0 million ATM equity offering facility - On October 30, 2023, the Company repurchased 576,443 shares of its common stock from vTv Therapeutics for approximately $4.4 million and subsequently retired them[71] - On November 13, 2023, the Company entered into a new At-The-Market (ATM) equity offering sales agreement to sell up to $100.0 million in common stock[72] - Concurrently, the 2022 ATM Facility with Leerink, under which $1.2 million in shares had been sold, was terminated[72][73] Item 2. Management's Discussion and Analysis of Financial Condition and Results of Operations Management discusses financial results, highlighting increased net losses driven by higher R&D and G&A expenses, and addresses liquidity risks Overview - Reneo Pharmaceuticals is a clinical-stage company developing mavodelpar, a PPARδ agonist, for rare genetic mitochondrial diseases[77][80] - Mavodelpar has received orphan drug and Fast Track designations in the U.S. and Europe for various indications[82] - The pivotal Phase 2b STRIDE study for PMM completed enrollment in March 2023, with topline data expected in December 2023[84][87] Components of Our Results of Operations - Research and development expenses are expected to increase substantially due to advancing product candidates through clinical trials and regulatory processes[98] - General and administrative expenses are expected to increase to support corporate infrastructure growth and commercial development activities[102] Research and Development Expenses (in thousands) | Research and Development Expenses | Three Months Ended Sep 30, 2023 | Three Months Ended Sep 30, 2022 | Nine Months Ended Sep 30, 2023 | Nine Months Ended Sep 30, 2022 | | :--- | :--- | :--- | :--- | :--- | | Clinical and regulatory | $7,373 | $5,555 | $20,408 | $14,143 | | Contract manufacturing cost | $2,609 | $2,469 | $7,761 | $6,802 | | Nonclinical | $632 | $577 | $3,719 | $3,060 | | Medical affairs | $2,217 | $193 | $4,883 | $409 | | Research and development-other expense | $791 | $1,144 | $2,238 | $2,934 | | Total | $13,622 | $9,938 | $39,009 | $27,348 | Results of Operations Comparison of Three Months Ended September 30, 2023 and 2022 | (in thousands) | Sep 30, 2023 | Sep 30, 2022 | Change | | :--- | :--- | :--- | :--- | | Research and development | $13,622 | $9,938 | $3,684 | | General and administrative | $7,266 | $3,902 | $3,364 | | Total operating expenses | $20,888 | $13,840 | $7,048 | | Loss from operations | $(20,888) | $(13,840) | $(7,048) | | Other income | $1,692 | $833 | $859 | | Net loss | $(19,196) | $(13,007) | $(6,189) | - Research and development expenses increased by $3.7 million for the three months ended September 30, 2023, due to increased clinical development and medical affairs costs[105] - General and administrative expenses increased by $3.4 million for the three months ended September 30, 2023, due to commercial development and personnel costs[107] Comparison of Nine Months Ended September 30, 2023 and 2022 | (in thousands) | Sep 30, 2023 | Sep 30, 2022 | Change | | :--- | :--- | :--- | :--- | | Research and development | $39,009 | $27,348 | $11,661 | | General and administrative | $19,038 | $11,938 | $7,100 | | Total operating expenses | $58,047 | $39,286 | $18,761 | | Loss from operations | $(58,047) | $(39,286) | $(18,761) | | Other income | $4,213 | $931 | $3,282 | | Net loss | $(53,834) | $(38,355) | $(15,479) | - Research and development expenses increased by $11.7 million for the nine months ended September 30, 2023, driven by clinical development and medical affairs costs[110] - General and administrative expenses increased by $7.1 million for the nine months ended September 30, 2023, due to commercial development and personnel costs[111] - Other income increased for both periods due to higher interest rates and increased short-term investment balances[108][112] Liquidity and Capital Resources - As of September 30, 2023, the company had $125.6 million in cash, cash equivalents, and short-term investments[113] - Current funds are not sufficient for more than one year, raising substantial doubt about its ability to continue as a going concern[121][122] - Financing activities provided $65.0 million in cash for the nine months ended September 30, 2023, primarily from a public offering and private placement[128] Cash Flows (in thousands) | Cash Flows | Nine Months Ended Sep 30, 2023 | Nine Months Ended Sep 30, 2022 | | :--- | :--- | :--- | | Net cash used in operating activities | $(43,929) | $(31,749) | | Net cash used in investing activities | $(29,254) | $(30,925) | | Net cash provided by financing activities | $64,993 | $146 | | Net decrease in cash and cash equivalents | $(8,190) | $(62,528) | Critical Accounting Policies and Estimates - The company's critical accounting policies and estimates remain consistent with those in its 2022 Annual Report, with no material changes[134] Recent Accounting Pronouncements - Refer to Note 2 of the Notes to Consolidated Financial Statements for a description of recent accounting pronouncements[135] Item 3. Quantitative and Qualitative Disclosures about Market Risk This section is not required for smaller reporting companies, and thus no disclosures about market risk are provided - Quantitative and Qualitative Disclosures about Market Risk are not required for smaller reporting companies[136] Item 4. Controls and Procedures Management concluded that the company's disclosure controls and procedures were effective as of September 30, 2023 Evaluation of Disclosure Controls and Procedures - Management concluded that disclosure controls and procedures were effective as of September 30, 2023[137] Changes in Internal Control over Financial Reporting - There were no material changes in internal control over financial reporting during the quarter ended September 30, 2023[138] Part II — Other Information Item 1. Legal Proceedings The company is not currently involved in any material legal proceedings - The Company is not currently a party to any material legal proceedings[140] Item 1A. Risk Factors The company faces numerous risks including ongoing losses, going concern doubt, and dependence on its sole product candidate, mavodelpar Risks Related to Our Business and Industry - The company has incurred significant net losses since inception and anticipates continued losses, raising substantial doubt about its ability to continue as a going concern[150][151][153] - Substantial additional capital is required to develop and commercialize mavodelpar; failure to secure financing may force delays[154][157][163] - The company is entirely dependent on the success of mavodelpar, its only product candidate[164][166][167] - Clinical trials may fail to adequately demonstrate mavodelpar's safety and efficacy, potentially preventing or delaying regulatory approval[168][171][172] - The regulatory approval process is lengthy, expensive, and uncertain, and denial or delay would adversely impact revenue generation[188][190][193] - The COVID-19 pandemic has adversely affected and could continue to affect operations and clinical trials[195][197][198] - Mavodelpar may not gain market acceptance among physicians, patients, and healthcare payors, even if approved[222][225] - The company faces significant competition from other biotechnology and pharmaceutical companies with greater resources[248][250][253] Risks Related to Our Reliance on Third Parties - The company's business is dependent on the vTv License Agreement; its termination would result in the loss of significant rights[305][307] - Reliance on third-party Contract Research Organizations (CROs) for clinical trials poses risks if they fail to meet contractual duties[308][309][310] - Complete reliance on third parties for manufacturing carries risks of failure to obtain regulatory approval for facilities or insufficient quantities[313][314][315][316] Risks Related to Our Intellectual Property - Commercial success depends on obtaining and maintaining sufficient intellectual property protection for mavodelpar[319][320][321][322] - Patent applications may not result in issued patents, and existing patents may be challenged, invalidated, or rendered unenforceable[324][325][326] - Patent terms may be inadequate to protect product candidates for a sufficient time, leading to earlier competition from generic products[331][333][335] - Protecting intellectual property rights globally is challenging due to differing laws and enforcement[363][364][365][366] - Third-party claims alleging intellectual property infringement could prevent or delay drug development and incur substantial damages[396][397][399][401][410][412] - Inability to protect the confidentiality of trade secrets would harm the business and competitive position[425][427][428] Risks Related to Ownership of Our Common Stock - The trading price of the common stock is likely to be highly volatile, and investors could lose all or part of their investment[439][441] - Principal stockholders and management own a significant percentage of the stock, enabling them to exert substantial control[444] - Future sales and issuances of common stock could result in additional dilution for existing stockholders and cause the stock price to fall[445][459][460] - As an emerging growth company, reduced reporting requirements may make the common stock less attractive to investors[446][449] General Risk Factors - The company is subject to U.S. and foreign export/import controls, sanctions, and anti-corruption laws[470][471] - Business disruptions from natural disasters, epidemics, or man-made events could seriously harm future revenues[472][473] - Compromised information technology systems due to cyberattacks could lead to regulatory actions, litigation, and reputational harm[474][476][477][478][481][482][486] Item 2. Unregistered Sales of Equity Securities and Use of Proceeds The company repurchased shares from vTv Therapeutics and details the use of proceeds from its 2021 IPO Unregistered Sales of Equity Securities - There were no unregistered sales of equity securities during the period[491] Issuer Purchases of Equity Securities - On October 30, 2023, the Company repurchased 576,443 shares of its common stock from vTv Therapeutics for approximately $4.4 million[492] Use of Proceeds - The company received net proceeds of $84.6 million from its April 2021 IPO[493] - As of September 30, 2023, approximately $23.8 million of the IPO net proceeds have been used[494] - Remaining IPO proceeds are allocated to fund continued research and development of mavodelpar and for general purposes[495] Item 3. Defaults Upon Senior Securities The company reported no defaults upon senior securities during the period - There were no defaults upon senior securities[496] Item 4. Mine Safety Disclosures The company reported no mine safety disclosures - There were no mine safety disclosures[497] Item 5. Other Information The company established a new $100.0 million At-The-Market equity offering facility and terminated a prior agreement Entry into New Sales Agreement - On November 13, 2023, the Company entered into a new At-The-Market (ATM) equity offering sales agreement, allowing it to sell up to $100.0 million in common stock[498] Termination of Prior Sales Agreement - On November 13, 2023, the prior 2022 ATM Facility, under which $1.2 million in shares had been sold, was terminated[500] Item 6. Exhibits This section lists all exhibits filed as part of the Quarterly Report on Form 10-Q - The exhibit index includes corporate documents, legal opinions, sales agreements, and Sarbanes-Oxley Act certifications[502] Signatures The report is duly signed by the principal executive and financial officers on November 13, 2023 - The report is signed by Gregory J. Flesher, President and Chief Executive Officer, and Jennifer P. Lam, Senior Vice President, Finance and Administration[506]