Aerovate Therapeutics(AVTE) - 2023 Q1 - Quarterly Report

PART I. FINANCIAL INFORMATION Item 1. Financial Statements (Unaudited) The company's unaudited condensed consolidated financial statements detail its financial position, performance, and cash flows Condensed Consolidated Balance Sheets Total assets and stockholders' equity decreased while total liabilities increased from December 2022 to March 2023 Condensed Consolidated Balance Sheets (in thousands) | Assets/Liabilities & Equity | March 31, 2023 | December 31, 2022 | | :-------------------------- | :------------- | :------------------ | | Assets: | | | | Cash and cash equivalents | $26,571 | $22,397 | | Short-term investments | $92,358 | $106,823 | | Total current assets | $120,569 | $131,496 | | Total assets | $124,393 | $135,301 | | Liabilities: | | | | Accounts payable | $4,726 | $2,575 | | Accrued & other current liabilities | $5,538 | $4,822 | | Total current liabilities | $10,637 | $7,782 | | Total liabilities | $11,298 | $8,558 | | Stockholders' Equity: | | | | Total stockholders' equity | $113,095 | $126,743 | | Total liabilities and stockholders' equity | $124,393 | $135,301 | Condensed Consolidated Statements of Operations and Comprehensive Loss The company's net loss widened in Q1 2023 due to higher R&D expenses, partially offset by increased interest income Condensed Consolidated Statements of Operations and Comprehensive Loss (in thousands) | Operating Expenses/Income | Three Months Ended March 31, 2023 | Three Months Ended March 31, 2022 | | :------------------------ | :-------------------------------- | :-------------------------------- | | Research and development | $13,488 | $7,255 | | General and administrative | $4,151 | $3,764 | | Total operating expenses | $17,639 | $11,019 | | Loss from operations | $(17,639) | $(11,019) | | Interest income | $1,120 | $109 | | Net loss | $(16,520) | $(10,910) | | Comprehensive loss | $(16,255) | $(11,495) | | Net loss per share, basic and diluted | $(0.67) | $(0.45) | - Research and development expenses increased by $6,233k (85.9%) year-over-year[26][103] - Interest income increased by $1,011k (927.5%) year-over-year[26][103] Condensed Consolidated Statements of Stockholders' Equity Total stockholders' equity decreased to $113.10 million, primarily driven by the net loss incurred during the period Condensed Consolidated Statements of Stockholders' Equity (in thousands) | Component | Balance at Dec 31, 2022 | Unrealized gain on investments | Stock based compensation | Net loss | Balance at Mar 31, 2023 | | :---------------------------------------- | :---------------------- | :----------------------------- | :----------------------- | :------- | :---------------------- | | Common Stock Amount | $2 | $— | $— | $— | $2 | | Additional Paid-In Capital | $215,110 | $— | $2,384 | $— | $217,717 | | Accumulated Other Comprehensive Gain/(Loss) | $(466) | $265 | $— | $— | $(201) | | Accumulated Deficit | $(87,903) | $— | $— | $(16,520) | $(104,423) | | Total Stockholders' Equity | $126,743 | $265 | $2,384 | $(16,520) | $113,095 | Condensed Consolidated Statements of Cash Flows Cash and cash equivalents increased by $4.17 million, a significant improvement from the prior year's decrease Condensed Consolidated Statements of Cash Flows (in thousands) | Cash Flow Activity | Three Months Ended March 31, 2023 | Three Months Ended March 31, 2022 | | :----------------- | :-------------------------------- | :-------------------------------- | | Net cash used in operating activities | $(11,424) | $(5,661) | | Net cash provided by (used in) investing activities | $15,375 | $(20,399) | | Net cash provided by financing activities | $223 | $— | | Net increase (decrease) in cash and cash equivalents | $4,174 | $(26,060) | | Cash and cash equivalents at the beginning of the year | $22,397 | $54,197 | | Cash and cash equivalents at the end of the period | $26,571 | $28,137 | - Net cash used in operating activities increased by $5,763k (101.8%) year-over-year[34][124] - Net cash provided by investing activities shifted from a use of $20,399k in Q1 2022 to a provision of $15,375k in Q1 2023, primarily due to higher maturities of short-term investments[34][121][124] Notes to Unaudited Condensed Consolidated Financial Statements These notes provide detailed disclosures on accounting policies, fair value, balance sheet items, and equity transactions (1) Organization and Nature of Operations Aerovate Therapeutics is a clinical-stage biopharmaceutical company focused on developing AV-101 for pulmonary arterial hypertension - Clinical-stage biopharmaceutical company focused on developing AV-101, a dry powder inhaled formulation of imatinib, for the treatment of pulmonary arterial hypertension (PAH), and initiated a global Phase 2b/Phase 3 trial in December 2021[37] - Completed its initial public offering (IPO) on July 2, 2021, generating approximately $126.9 million in net proceeds[38] - Entered into an ATM Equity OfferingSM Sales Agreement on April 5, 2023, to sell up to $75.0 million of common stock; no shares were sold as of May 15, 2023[39] - As of March 31, 2023, the company had cash and short-term investments of $118.9 million, sufficient to fund operations into the second half of 2025[40][41] (2) Basis of Presentation and Significant Accounting Policies The financial statements are prepared under U.S. GAAP and reflect a reverse stock split completed in June 2021 - Unaudited condensed consolidated financial statements are prepared in conformity with U.S. GAAP for interim financial information[44] - Effected a 1-for-3.1060103 reverse stock split on June 22, 2021, retrospectively adjusting all common stock and related share data[46] - Key accounting estimates include accruals for research and development expenses, stock-based compensation, and fair value of investments[47] - Elected to "opt out" of the extended transition period for complying with new or revised financial accounting standards as an emerging growth company[129] (3) Fair Value of Financial Instruments Financial assets are measured at fair value, with unrealized losses attributed to market fluctuations rather than credit risk Fair Value of Financial Assets (in thousands) - March 31, 2023 | Assets | Fair Value | Level 1 | Level 2 | Level 3 | | :------------------------- | :--------- | :------ | :------ | :------ | | Money market funds | $26,049 | $26,049 | $— | $— | | Commercial paper | $53,686 | $— | $53,686 | $— | | U.S. Treasury bills | $12,468 | $12,468 | $— | $— | | Agency bonds | $26,204 | $— | $26,204 | $— | | Total fair value of assets | $118,407 | $38,517 | $79,890 | $— | Fair Value of Financial Assets (in thousands) - December 31, 2022 | Assets | Fair Value | Level 1 | Level 2 | Level 3 | | :------------------------- | :--------- | :------ | :------ | :------ | | Money market funds | $18,436 | $18,436 | $— | $— | | Commercial paper | $55,577 | $— | $55,577 | $— | | U.S. Treasury bills | $26,841 | $26,841 | $— | $— | | Agency bonds | $24,405 | $— | $24,405 | $— | | Total fair value of assets | $125,259 | $45,277 | $79,982 | $— | - Unrealized losses on available-for-sale securities were caused by fluctuations in market value and interest rates, not credit risk, and no impairment losses were recognized[60][61] (4) Balance Sheet Components This section details the composition of prepaid expenses, other current assets, and accrued current liabilities Prepaid Expenses and Other Current Assets (in thousands) | Component | March 31, 2023 | December 31, 2022 | | :------------------------------------ | :------------- | :---------------- | | Prepaid expenses | $1,114 | $1,503 | | Other current assets | $266 | $295 | | Prepaid research and development | $260 | $478 | | Total prepaid expenses and other current assets | $1,640 | $2,276 | Accrued and Other Current Liabilities (in thousands) | Component | March 31, 2023 | December 31, 2022 | | :------------------------------------ | :------------- | :---------------- | | Accrued research and development | $4,267 | $2,751 | | Accrued payroll and other employee benefits | $1,019 | $1,691 | | Other | $252 | $380 | | Total accrued and other current liabilities | $5,538 | $4,822 | (5) Commitments and Contingencies The company has lease agreements for office spaces with total future minimum lease payments of $1.034 million - Entered into a lease agreement for office space in Waltham, Massachusetts in August 2021, with an initial base rent of approximately $18,000 per month[66] - Entered into a lease agreement for office space in Foster City, California in April 2022, with a base rent of $22,600 per month[67] Future Minimum Annual Lease Payments (in thousands) as of March 31, 2023 | Year | Total Lease Minimum Payments | | :--- | :--------------------------- | | 2023 | $284 | | 2024 | $508 | | 2025 | $242 | | Total | $1,034 | (6) Stockholders' Equity The company has 150 million authorized common shares, with 24.8 million outstanding and 6.5 million reserved for future issuance - Authorized 150,000,000 shares of common stock and 10,000,000 shares of undesignated preferred stock[71] - 24,816,940 shares of common stock were issued and outstanding as of March 31, 2023[23] Shares of Common Stock Reserved for Future Issuance (as of March 31, 2023) | Category | Shares | | :----------------------------------------- | :------- | | Common stock options granted and outstanding | 5,107,865 | | Shares reserved for issuance under the 2021 Plan | 933,657 | | Reserved for vesting of outstanding restricted stock units | 31,881 | | Reserved for future ESPP issuances | 463,360 | | Total | 6,536,763 | (7) Stock-Based Compensation Total stock-based compensation expense increased to $2.4 million in Q1 2023, with $35.2 million in unrecognized expenses remaining - As of March 31, 2023, there was $34.6 million of total unrecognized stock-based compensation expense related to unvested stock options, expected to be recognized over approximately 2.8 years[78] - As of March 31, 2023, the company had unrecognized stock-based compensation expense of $0.6 million related to its unvested restricted stock units, expected to be recognized over 3.0 years[83] Stock-Based Compensation Expense by Operating Expense (in thousands) | Category | Three Months Ended March 31, 2023 | Three Months Ended March 31, 2022 | | :----------------------------- | :-------------------------------- | :-------------------------------- | | Research and development | $1,299 | $768 | | General and administrative | $1,085 | $256 | | Total | $2,384 | $1,024 | Stock-Based Compensation Expense by Award Type (in thousands) | Award Type | Three Months Ended March 31, 2023 | Three Months Ended March 31, 2022 | | :----------------------------- | :-------------------------------- | :-------------------------------- | | Stock options | $2,289 | $1,024 | | Restricted stock awards and units | $48 | $— | | Employee stock purchase plan awards | $47 | $— | | Total | $2,384 | $1,024 | Item 2. Management's Discussion and Analysis of Financial Condition and Results of Operations Management discusses the company's financial condition, clinical-stage focus, and operational results for Q1 2023 Overview Aerovate Therapeutics is a clinical-stage biopharmaceutical company developing AV-101 for Pulmonary Arterial Hypertension - Clinical-stage biopharmaceutical company focused on developing AV-101, a dry powder inhaled formulation of imatinib, for the treatment of pulmonary arterial hypertension (PAH)[89] - Currently enrolling patients in a global Phase 2b/Phase 3 trial of AV-101 in adults with PAH[89] - Has no products approved for sale and has incurred significant operating losses since inception[89] COVID-19 Pandemic Impact The COVID-19 pandemic continues to pose uncertainties and potential adverse impacts on clinical development timelines - Continues to closely monitor the impact of the COVID-19 pandemic on its business, operations, and clinical development timelines[90] - Experienced delays in activating new trial sites and patient enrollment due to staffing shortages and interruptions[176] - Patient populations targeted by AV-101 (PAH patients) may be particularly susceptible to COVID-19, potentially making enrollment more difficult[177] Components of Results of Operations The company generates no revenue and expects substantial R&D and G&A expenses to continue for the foreseeable future - Currently has no products approved for sale and has not generated any revenue to date[92][94] - Research and development expenses are primarily related to the development of AV-101 and are expected to increase substantially[95][97] - General and administrative expenses are anticipated to increase due to public company compliance and pre-commercial activities[100] - Interest income is earned on cash, cash equivalents, and short-term investments[101] Results of Operations (Comparison of Q1 2023 and Q1 2022) The company's net loss increased to $16.5 million in Q1 2023, driven by a significant rise in R&D expenses Results of Operations (in thousands) | Operating Expenses/Income | 2023 | 2022 | Change | | :------------------------ | :------- | :------- | :------- | | Research and development | $13,488 | $7,255 | $6,233 | | General and administrative | $4,151 | $3,764 | $387 | | Total operating expenses | $17,639 | $11,019 | $6,620 | | Loss from operations | $(17,639) | $(11,019) | $(6,620) | | Interest income | $1,120 | $109 | $1,011 | | Other expense | $(1) | $— | $(1) | | Total other income | $1,119 | $109 | $1,010 | | Net loss | $(16,520) | $(10,910) | $(5,610) | - Research and development expenses increased by $6.2 million, primarily due to a $3.8 million increase in clinical costs and a $3.3 million increase in headcount-related costs[104] - Interest income increased by $1.0 million due to interest earned on cash and short-term investments[106] Liquidity and Capital Resources The company had $118.9 million in cash and investments, projected to fund operations into the second half of 2025 - As of March 31, 2023, the company had cash and short-term investments of $118.9 million[107] - Existing cash and investments are expected to fund planned operations into the second half of 2025[109] - Future capital requirements are substantial and depend on the costs of clinical trials, regulatory approvals, and commercialization of AV-101[110][112] - Entered into an ATM Equity OfferingSM Sales Agreement on April 5, 2023, to sell up to $75.0 million of common stock, with no shares sold as of May 15, 2023[108] Cash Flows (Comparison of Q1 2023 and Q1 2022) Net cash used in operations increased, while investing activities provided cash due to maturities of short-term investments Summary of Net Cash Flow Activity (in thousands) | Cash Flow Activity | Three Months Ended March 31, 2023 | Three Months Ended March 31, 2022 | | :------------------------------------ | :-------------------------------- | :-------------------------------- | | Net cash used in operating activities | $(11,424) | $(5,661) | | Net cash provided by (used in) investing activities | $15,375 | $(20,399) | | Net cash provided by financing activities | $223 | $— | | Net increase (decrease) in cash and cash equivalents | $4,174 | $(26,060) | - Net cash used in operating activities increased to $11.4 million in Q1 2023, primarily due to the net loss incurred[119] - Net cash provided by investing activities was $15.4 million in Q1 2023, driven by maturities of short-term investments, a reversal from $20.4 million used in Q1 2022[121] Critical Accounting Estimates Financial statements rely on key estimates for R&D expenses and stock-based compensation, with no significant policy changes - Preparation of financial statements requires management to make estimates, particularly for research and development expenses and stock-based compensation[128][47] - No significant changes in critical accounting policies and estimates occurred during the three months ended March 31, 2023[127] - As an emerging growth company, the company elected to "opt out" of the extended transition period for complying with new accounting standards[129] Item 3. Quantitative and Qualitative Disclosures About Market Risk The company is exposed to market risks from interest rates, foreign currency exchange, and inflation - Exposed to interest rate fluctuation risk on cash and investments; no material effect on Q1 2023 results[131] - Exposed to foreign currency fluctuation risk due to contracts with international vendors; no material effect on Q1 2023 results[132][135] - Inflation affects labor and R&D costs; no material effect on Q1 2023 results, but potential adverse impact in the near future[136] Item 4. Controls and Procedures Management concluded that disclosure controls and procedures were effective as of March 31, 2023 - Management concluded that disclosure controls and procedures were effective at a reasonable assurance level as of March 31, 2023[137] - No material changes in internal control over financial reporting occurred during the fiscal quarter ended March 31, 2023[138] PART II. OTHER INFORMATION Item 1. Legal Proceedings The company was not party to any material legal proceedings as of March 31, 2023 - As of March 31, 2023, the company was not party to any legal proceedings that it would expect to have a material adverse impact on its financial position[139] Item 1A. Risk Factors The company faces numerous material risks related to its operations, finances, competition, and regulatory environment Risks Related to Our Limited Operating History, Financial Position, and Capital Requirements The company has a limited operating history, significant losses, and will require substantial additional capital - The company is a clinical-stage biopharmaceutical company with a limited operating history and no products approved for commercial sale[144][152] - Incurred significant operating losses since inception, with an accumulated deficit of $104.4 million as of March 31, 2023[146] - Will require substantial additional capital to finance operations, which may not be available on acceptable terms[155][160] Risks Related to the Development of AV-101 The company's business is entirely dependent on the successful development and approval of its sole product candidate, AV-101 - The business is entirely dependent on the successful development, regulatory approval, and commercialization of AV-101, its only product candidate[166][167] - Experienced and may continue to experience difficulties with site activation and patient enrollment in clinical trials[185][186] - Clinical development is a lengthy and expensive process with an uncertain outcome; AV-101 may cause undesirable side effects[190][202][206] - Intends to use the 505(b)(2) regulatory pathway for AV-101, but the FDA may determine it does not meet requirements[212][213] Risks Related to Commercialization The company faces significant competition, market acceptance hurdles, and reimbursement uncertainties for AV-101 - Faces significant competition for AV-101, if approved, from established companies with greater financial and marketing resources[214][218] - Commercial success depends on market acceptance, coverage, and adequate reimbursement from third-party payors, which are uncertain[226][231][234] - Currently has no sales organization and must build or partner for marketing, sales, and distribution capabilities[237][238] - Faces an inherent risk of product liability from clinical testing and potential commercialization of AV-101[239][241] Risks Related to Our Reliance on Third Parties The company is highly dependent on third-party suppliers and contract research organizations for its operations - Highly dependent on qualified third parties to supply all components of AV-101, some of which are sole-source suppliers[243][244] - Relies entirely on third parties (medical institutions, CROs) to conduct all clinical trials, making the company vulnerable to their performance[251][252] - May seek to establish collaborations for development and commercialization, but faces significant competition and uncertainty in negotiating favorable terms[255][260] Risks Related to Our Intellectual Property The company's success relies on securing and enforcing intellectual property rights, which is uncertain and costly - Success depends on securing, enforcing, and defending intellectual property rights for AV-101; no assurance that patent applications will result in issued patents[262][263][268] - Intellectual property litigation is expensive, time-consuming, and could divert resources, potentially leading to loss of exclusivity[275][276] - May not identify relevant third-party patents, leading to infringement claims and potential commercialization prohibitions[290][291] - Relies on trade secrets and confidentiality agreements to protect unpatented know-how, but cannot guarantee protection[305][308] Risks Related to Government Regulation The company faces significant regulatory risks, including potential denial of approval and extensive ongoing requirements - May be unable to obtain regulatory approval for AV-101 from the FDA or comparable foreign regulatory authorities[313][314][316] - AV-101 is a drug-device combination product, which may result in additional regulatory risks and increased complexity[318] - Even if approved, AV-101 will be subject to extensive ongoing regulatory requirements for manufacturing, labeling, and promotion[322][323][329] - Healthcare legislative reforms, such as the Inflation Reduction Act of 2022, could adversely affect business, pricing, and reimbursement[351][352][356][357] - Has received orphan drug designation for AV-101, but this does not guarantee faster development or approval[334][339] Risks Related to Employee Matters and Managing Growth The company faces challenges in managing growth, attracting and retaining personnel, and mitigating operational risks - Will need to increase the size of its organization and may experience difficulties in managing growth and integrating additional employees[366][368][369] - Insurance policies may be inadequate, potentially exposing the company to unrecoverable risks from significant claims[370] - Relies on information technology systems and faces risks from cyberattacks, which could lead to data breaches and significant financial exposure[371][372] Risks Related to Ownership of Our Common Stock Ownership of common stock is subject to risks from tax law limitations, anti-takeover provisions, and designated legal forums - Ability to utilize net operating loss carryforwards (NOLs) may be limited by an "ownership change" under Section 382 of the Internal Revenue Code[377] - Comprehensive tax reform legislation could adversely affect the business and financial condition[378][381] - Anti-takeover provisions in charter documents and under Delaware law could make an acquisition more difficult[382][383] - Amended bylaws designate certain courts as the sole and exclusive forum for specific stockholder actions, potentially limiting favorable judicial forums[387] General Risk Factors The company is susceptible to risks from global economic conditions, financial industry instability, and data privacy compliance - Unfavorable global economic or political conditions, including rising inflation and interest rates, could adversely affect business and financial condition[389][390][400] - Adverse developments affecting the financial services industry, such as bank failures, could impair access to funding[392][394][395] - Employees and independent contractors may engage in misconduct or other improper activities, including noncompliance with regulatory standards[401][402] - Failures to comply with U.S. and foreign privacy and data protection laws could result in significant liability and reputational damage[404][405][406][410][414] - As an "emerging growth company," the company avails itself of reduced disclosure requirements, which could make its common stock less attractive[418][419][425] Item 2. Unregistered Sales of Equity Securities and Use of Proceeds The company completed its IPO in July 2021, raising $126.9 million, with no material change in the planned use of proceeds - Completed its IPO on July 2, 2021, issuing 9,984,463 shares of common stock at $14.00 per share[439] - Aggregate net proceeds from the IPO were approximately $126.9 million[440] - No material change in the planned use of IPO proceeds from that described in the final Prospectus[440] Item 3. Defaults Upon Senior Securities The company reported no defaults upon senior securities during the period - No defaults upon senior securities[440] Item 4. Mine Safety Disclosures This item is not applicable to the company - Mine Safety Disclosures are not applicable to the company[440] Item 5. Other Information No other information was reported under this item - No other information to report[440] Item 6. Exhibits This section lists the exhibits filed with the Form 10-Q, including organizational documents and officer certifications - Key exhibits include the Certificate of Incorporation, Bylaws, ATM Equity OfferingSM Sale Agreement, and certifications from executive officers[442]