Axsome Therapeutics(AXSM) - 2024 Q2 - Quarterly Report

CAUTIONARY NOTE REGARDING FORWARD-LOOKING STATEMENTS This section highlights that the report contains forward-looking statements regarding the company's future financial results, product development, regulatory approvals, commercialization efforts, capital resources, and market acceptance - Forward-looking statements cover expectations for expenses, clinical development, manufacturing, regulatory approval, commercialization, capital resources, capital expenditures, revenue projections, profitability, partnership agreements, product acquisitions, intellectual property, product acceptance, stock price volatility, key personnel, third-party manufacturers, and strategic implementation[8] - Actual results may differ materially due to factors discussed under 'Risk Factors' and 'Management's Discussion and Analysis of Financial Condition and Results of Operations'[8] - All forward-looking statements are qualified by cautionary statements and claim the protection of the safe harbor provisions of the Private Securities Litigation Reform Act of 1995[9] PART I — FINANCIAL INFORMATION ITEM 1. FINANCIAL STATEMENTS This section presents the unaudited interim consolidated financial statements, including balance sheets, statements of operations, statements of stockholders' equity, and statements of cash flows, along with detailed notes explaining the company's accounting policies, financial instrument valuations, debt, equity, and revenue recognition Consolidated Balance Sheets Presents the company's financial position, detailing assets, liabilities, and stockholders' equity at specific reporting dates | Metric (in thousands) | June 30, 2024 (Unaudited) | December 31, 2023 | | :-------------------- | :------------------------ | :------------------ | | Cash and cash equivalents | $315,657 | $386,193 | | Accounts receivables, net | $120,342 | $94,820 | | Total current assets | $463,027 | $504,263 | | Total assets | $548,226 | $588,236 | | Total current liabilities | $186,570 | $138,854 | | Total liabilities | $445,374 | $397,259 | | Total stockholders' equity | $102,852 | $190,977 | - Total assets decreased from $588.2 million at December 31, 2023, to $548.2 million at June 30, 2024, primarily due to a decrease in cash and cash equivalents[12] - Total liabilities increased from $397.3 million to $445.4 million, driven by increases in accounts payable and accrued expenses[12] Consolidated Statements of Operations Details the company's revenues, expenses, and net loss over specific reporting periods, reflecting operational performance | Metric (in thousands) | Three months ended June 30, 2024 | Three months ended June 30, 2023 | Six months ended June 30, 2024 | Six months ended June 30, 2023 | | :-------------------- | :------------------------------- | :------------------------------- | :----------------------------- | :----------------------------- | | Product sales, net | $86,520 | $46,017 | $160,616 | $74,586 | | Total revenues | $87,166 | $46,700 | $162,165 | $141,276 | | R&D expenses | $49,853 | $20,581 | $86,683 | $38,374 | | SG&A expenses | $103,554 | $78,935 | $202,524 | $153,126 | | Total operating expenses | $165,212 | $111,757 | $307,486 | $212,707 | | Net loss | $(79,345) | $(67,170) | $(147,702) | $(78,388) | | Net loss per share | $(1.67) | $(1.54) | $(3.11) | $(1.80) | - Total revenues for the three months ended June 30, 2024, increased by 86.6% to $87.2 million from $46.7 million in the prior year, driven by higher product sales[14] - Net loss for the six months ended June 30, 2024, significantly increased to $147.7 million from $78.4 million in the prior year, primarily due to higher R&D and SG&A expenses, and the absence of the one-time license revenue recognized in Q1 2023[14] Consolidated Statements of Stockholders' Equity Outlines changes in the company's equity accounts, including common stock, additional paid-in capital, and accumulated deficit | Metric (in thousands) | Balance at Dec 31, 2023 | Balance at June 30, 2024 | | :-------------------- | :---------------------- | :----------------------- | | Common stock | $5 | $5 | | Additional paid-in capital | $1,026,543 | $1,086,120 | | Accumulated deficit | $(835,571) | $(983,273) | | Total stockholders' equity | $190,977 | $102,852 | - Total stockholders' equity decreased from $191.0 million at December 31, 2023, to $102.9 million at June 30, 2024, mainly due to the accumulated deficit from net losses, partially offset by increases in additional paid-in capital from stock-based compensation and stock issuances[17] Consolidated Statements of Cash Flows Summarizes cash inflows and outflows from operating, investing, and financing activities over specific periods | Metric (in thousands) | Six months ended June 30, 2024 | Six months ended June 30, 2023 | | :-------------------------------- | :----------------------------- | :----------------------------- | | Net cash used in operating activities | $(83,577) | $(61,170) | | Net cash used in investing activities | $(150) | $(204) | | Net cash provided by financing activities | $13,191 | $297,645 | | Net (decrease) increase in cash | $(70,536) | $236,271 | | Cash at end of period | $315,657 | $437,113 | - Net cash used in operating activities increased to $83.6 million for the six months ended June 30, 2024, compared to $61.2 million in the prior year, primarily due to higher commercial and clinical activities and the absence of the 2023 upfront license payment[21] - Net cash provided by financing activities significantly decreased to $13.2 million in 2024 from $297.6 million in 2023, mainly due to the absence of large debt drawdowns and public offering proceeds seen in the prior year[21] Note 1. Nature of Business and Basis of Presentation Describes the company's biopharmaceutical focus, product portfolio, and the foundational principles underlying its financial statement preparation - Axsome Therapeutics, Inc. is a biopharmaceutical company focused on developing and delivering novel therapies for central nervous system (CNS) conditions with limited treatment options[23] - The company's portfolio includes two approved products, Auvelity® (AXS-05) and Sunosi®, and three unapproved product candidates: AXS-07, AXS-12, and AXS-14, all being developed for multiple indications[23] - The company acquired U.S. and ex-U.S. rights to Sunosi in 2022 and announced FDA approval of Auvelity in August 2022[24] - The company has incurred operating losses since inception, with an accumulated deficit of $983.3 million as of June 30, 2024, and expects to continue incurring losses[27] - Existing cash is believed to be sufficient for anticipated operating cash requirements for at least twelve months, with future funding potentially sourced from equity offerings, debt financings, or strategic alliances[28] Note 2. Summary of Significant Accounting Policies Details the key accounting principles and methods applied in preparing the financial statements, covering revenue, R&D, and stock-based compensation - Revenue is recognized when the customer obtains control of a promised good or service, reflecting the consideration expected in exchange[33] - Product sales are recorded net of reserves for variable consideration, including rebates, discounts, and returns, which are estimated based on anticipated performance and available information[39][40] - Cost of revenue includes direct costs, overhead, and product royalties, with specific royalty commitments to SK Biopharmaceuticals, Aerial Biopharma, and Antecip Bioventures (related party)[49][50] - Research and development costs are expensed as incurred, including employee-related expenses, contract services, and product license fees[72] - Stock-based compensation expense is recognized over the vesting period for stock options (Black-Scholes model) and restricted stock units (market closing price)[77][78] Note 3. Accounts receivable, net Provides a breakdown of trade receivables and reserves for variable consideration, highlighting major customer concentrations | Metric (in thousands) | June 30, 2024 | December 31, 2023 | | :-------------------- | :------------ | :---------------- | | Trade receivables | $133,531 | $107,320 | | Less: Reserves for variable consideration | $(13,189) | $(12,500) | | Accounts receivable, net | $120,342 | $94,820 | - Accounts receivable, net, increased to $120.3 million as of June 30, 2024, from $94.8 million at December 31, 2023[89] - Approximately 95% of total accounts receivable as of June 30, 2024, were from Cardinal Health, Inc., Cencora, Inc., and McKesson Corporation[89] Note 4. Inventory Details the composition of inventory, including raw materials, work in process, and finished goods, and their classification | Metric (in thousands) | June 30, 2024 | December 31, 2023 | | :-------------------- | :------------ | :---------------- | | Raw materials | $7,771 | $5,534 | | Work in process | $10,536 | $10,287 | | Finished goods | $8,270 | $9,643 | | Total inventory | $26,577 | $25,464 | - Total inventory increased to $26.6 million at June 30, 2024, from $25.5 million at December 31, 2023, with an increase in raw materials[90] - Non-current inventory, consisting of raw materials and work in progress, is anticipated to be consumed beyond the normal operating cycle[90] Note 5. Intangible Asset Presents the carrying amount, accumulated amortization, and estimated future amortization of the company's finite-lived intangible asset | Metric (in thousands) | December 31, 2023 | June 30, 2024 | | :-------------------- | :---------------- | :------------ | | Gross carrying amount | $63,800 | $63,800 | | Accumulated amortization | $10,514 | $13,693 | | Net carrying amount | $53,286 | $50,107 | | Remaining weighted-average useful life | 9-years | 8-years | - The net carrying amount of the finite-lived intangible asset decreased to $50.1 million at June 30, 2024, from $53.3 million at December 31, 2023, due to amortization[95] - Estimated future amortization expense for 2024 is $3.2 million, and $6.4 million annually from 2025 to 2028[96] Note 6. Fair Value of Financial Instruments Discusses the valuation methodologies and inputs used for financial instruments, particularly contingent consideration liabilities - Contingent consideration liabilities are measured at fair value using the probability weighted income approach, classified as Level 3 inputs due to significant unobservable inputs[60][64] | Metric (in thousands) | June 30, 2024 | December 31, 2023 | | :-------------------- | :------------ | :---------------- | | Contingent consideration | $76,660 | $79,707 | - The fair value of contingent consideration decreased to $76.7 million at June 30, 2024, from $79.7 million at December 31, 2023, reflecting adjustments to fair value and payments[98][100] | Unobservable Input | As of June 30, 2024 Weighted average (range, if applicable) | As of December 31, 2023 Weighted average (range, if applicable) | | :----------------- | :---------------------------------------------------------- | :---------------------------------------------------------- | | Discount rate | 15.9% | 13.2% | | Revenue discount rate | 16.1% - 19.1% | 16.4% - 19.4% | Note 7. Accrued Expenses and Other Current Liabilities Details the components of accrued expenses and other current liabilities, including R&D, compensation, and sales allowances | Metric (in thousands) | June 30, 2024 | December 31, 2023 | | :-------------------- | :------------ | :---------------- | | Accrued research and development | $13,565 | $6,503 | | Accrued compensation | $16,212 | $20,457 | | Accrued selling, general and administrative | $14,743 | $9,242 | | Accrued sales discounts, rebates and allowances | $63,530 | $46,713 | | Total | $116,771 | $90,501 | - Total accrued expenses and other current liabilities increased to $116.8 million at June 30, 2024, from $90.5 million at December 31, 2023, primarily due to increases in accrued R&D and sales discounts, rebates, and allowances[103] Note 8. Loan and Security Agreement Outlines the terms and amendments of the company's loan agreement, including principal amounts, interest rates, and covenants - The Fourth Amendment to the Loan Agreement (May 2023) increased the cash holding limit for Axsome Malta Ltd. outside the U.S. to $15.0 million for 45 days, then $10.0 million, with a waiver allowing up to $12.5 million until December 31, 2023[105] - The Third Amendment (January 2023) extended the maturity date to January 1, 2028 (potentially 2029), increased the aggregate principal amount to $350.0 million, revised term loan advance amounts, adjusted the interest rate, and increased the minimum cash requirement to $30.0 million[106] - As of June 30, 2024, the company had $180.0 million in outstanding debt, with scheduled principal payments entirely in 2028[116][121] | Metric (in thousands) | Three months ended June 30, 2024 | Three months ended June 30, 2023 | Six months ended June 30, 2024 | Six months ended June 30, 2023 | | :-------------------- | :------------------------------- | :------------------------------- | :----------------------------- | :----------------------------- | | Interest expense | $4,869 | $4,067 | $9,738 | $7,702 | | Amortization of final payment fee | $360 | $272 | $709 | $522 | | Amortization of debt discount | $281 | $637 | $551 | $971 | Note 9. Commitments and Contingencies (Leases) Details the company's lease obligations, including operating and finance leases, and associated expenses and future payments - The company entered a ten-year sublease for its principal executive offices in February 2023, with a determined lease term of five years and a discount rate of 12.0%[123] - A fleet lease program initiated in Q1 2024 is classified as a finance lease, with a right-of-use asset and liability of $4.2 million as of June 30, 2024, and a weighted average discount rate of 9.6%[124] | Lease Expense (in thousands) | Three months ended June 30, 2024 | Three months ended June 30, 2023 | Six months ended June 30, 2024 | Six months ended June 30, 2023 | | :--------------------------- | :------------------------------- | :------------------------------- | :----------------------------- | :----------------------------- | | Operating lease expense | $583 | $689 | $1,166 | $1,007 | | Finance lease expense (amortization) | $263 | — | $373 | — | | Finance lease expense (interest) | $86 | — | $121 | — | | Future Minimum Lease Payments (in thousands) | Operating lease | Finance lease | | :------------------------------------------- | :-------------- | :------------ | | 2024 | $668 | $783 | | 2025 | $1,976 | $1,496 | | 2026 | $2,521 | $1,407 | | 2027 | $2,521 | $952 | | 2028 | $3,204 | $280 | | Total lease payments | $10,890 | $4,918 | | Present value of lease liabilities | $8,248 | $4,222 | Note 10. Stockholders' Equity Provides details on common stock activity, stock option and RSU grants, and stock-based compensation expense - Under the March 2022 Sales Agreement, the company received $11.3 million in gross proceeds from selling 148,875 shares for the three months ended June 30, 2024, with net proceeds of $11.0 million[131] - In June 2023, a public offering of 3.0 million shares at $75.00 per share generated $211.3 million in net proceeds, with an additional $31.7 million from underwriters' option exercise in July 2023[134] - As of June 30, 2024, there were 1,183,992 shares available for future grant under the 2015 Omnibus Incentive Compensation Plan[136] | Stock Option Activity | Number of shares | Weighted average exercise price | | :-------------------- | :--------------- | :------------------------------ | | Outstanding at Dec 31, 2023 | 8,462,294 | $41.48 | | Granted | 1,047,180 | $83.23 | | Exercised | (193,125) | $32.59 | | Forfeited/Canceled | (377,542) | $59.88 | | Outstanding at June 30, 2024 | 8,938,807 | $45.79 | | Exercisable at June 30, 2024 | 5,066,333 | $32.08 | - Total unrecognized compensation cost for non-vested stock options was $177.5 million as of June 30, 2024, to be recognized over 2.7 years[140] | RSU Activity | Number of shares | Weighted average grant date fair value | | :-------------------- | :--------------- | :------------------------------------- | | Outstanding at Dec 31, 2023 | 804,150 | $41.36 | | Granted | 397,237 | $75.23 | | Vested | (226,323) | $37.11 | | Forfeited | (82,009) | $49.83 | | Outstanding at June 30, 2024 | 893,055 | $56.74 | - Total unrecognized compensation cost for unvested RSUs was $44.8 million as of June 30, 2024, to be recognized over 2.8 years[141] | Stock-based Compensation Expense (in thousands) | Three months ended June 30, 2024 | Three months ended June 30, 2023 | Six months ended June 30, 2024 | Six months ended June 30, 2023 | | :---------------------------------------------- | :------------------------------- | :------------------------------- | :----------------------------- | :----------------------------- | | Research and development | $5,072 | $3,238 | $9,640 | $5,652 | | Selling, general and administrative | $16,310 | $12,684 | $31,932 | $23,213 | | Total | $21,382 | $15,922 | $41,572 | $28,865 | Note 11. Warrants Details the number and weighted average exercise price of outstanding warrants, and their accounting classification | Warrant Activity | Warrants | Weighted average exercise price | | :-------------------- | :------- | :------------------------------ | | Outstanding at Dec 31, 2023 | 79,220 | $56.80 | | Outstanding at June 30, 2024 | 79,220 | $56.80 | - The company had 79,220 warrants outstanding at June 30, 2024, with a weighted average exercise price of $56.80, unchanged from December 31, 2023[147] - Warrants issued in 2023, 2022, and 2020 in connection with debt agreements were classified as a component of stockholders' equity, with their fair value reducing the carrying value of the debt[148][149] Note 12. Net Loss per Common Share Presents the calculation of basic and diluted net loss per common share, and the impact of anti-dilutive securities | Metric | Three months ended June 30, 2024 | Three months ended June 30, 2023 | Six months ended June 30, 2024 | Six months ended June 30, 2023 | | :-------------------- | :------------------------------- | :------------------------------- | :----------------------------- | :----------------------------- | | Net loss | $(79,345) | $(67,170) | $(147,702) | $(78,388) | | Weighted average common shares outstanding | 47,573,229 | 43,669,820 | 47,482,602 | 43,597,131 | | Net loss per common share | $(1.67) | $(1.54) | $(3.11) | $(1.80) | - Basic and diluted net loss per common share increased to $(1.67) for the three months ended June 30, 2024, from $(1.54) in the prior year, and to $(3.11) for the six months ended June 30, 2024, from $(1.80) in the prior year[151] - Potentially dilutive securities, including stock options, RSUs, warrants, and ESPP shares, were excluded from diluted EPS computation as they were anti-dilutive due to net losses[152] Note 13. Revenues Breaks down total revenues by product sales and license/royalty revenue, highlighting growth drivers and geographical concentration | Revenue Type (in thousands) | Three months ended June 30, 2024 | Three months ended June 30, 2023 | Six months ended June 30, 2024 | Six months ended June 30, 2023 | | :-------------------------- | :------------------------------- | :------------------------------- | :----------------------------- | :----------------------------- | | Product sales, net Auvelity | $65,025 | $27,643 | $118,420 | $43,393 | | Product sales, net Sunosi | $21,495 | $18,374 | $42,196 | $31,193 | | Total product sales, net | $86,520 | $46,017 | $160,616 | $74,586 | | Sunosi license revenue | — | — | — | $65,735 | | Sunosi royalty revenue | $646 | $683 | $1,549 | $955 | | Total revenues | $87,166 | $46,700 | $162,165 | $141,276 | - Total revenues for the three months ended June 30, 2024, increased by 86.6% to $87.2 million, primarily driven by a 135.2% increase in Auvelity net sales and a 17.0% increase in Sunosi net sales[155] - For the six months ended June 30, 2024, total revenues increased by 14.8% to $162.2 million, despite the absence of the $65.7 million Sunosi license revenue recognized in the prior year, due to strong product sales growth[155] - Product sales, net, in the United States accounted for $85.4 million and $158.7 million for the three and six months ended June 30, 2024, respectively[156] Note 14. License Agreements Details key license agreements, including terms for commercialization rights, upfront payments, milestones, and royalties - In February 2023, Axsome Malta entered an exclusive license agreement with Pharmanovia to commercialize Sunosi in Europe and certain Middle East/North Africa countries, receiving a non-refundable upfront payment of €62.0 million ($65.7 million)[157][158] - The company will receive a mid-twenties royalty percentage on Sunosi net sales in the Territory and is eligible for up to €94.5 million in sales-based milestone payments from Pharmanovia[158] - In January 2020, the company licensed data and intellectual property from Pfizer for reboxetine (AXS-12) and esreboxetine (AXS-14), with Pfizer eligible for up to $323 million in regulatory and sales milestones and tiered royalties[160][162] - The company has three exclusive license agreements with Antecip Bioventures II LLC (owned by the CEO), requiring a 3.0% royalty on net sales of Auvelity (AXS-05)[164] Note 15. Royalty Agreements Outlines the company's royalty obligations under various agreements for its commercial products - Under the Asset Purchase Agreement with Jazz, the company agreed to pay Jazz high single-digit royalties for current indications and mid-single-digit royalties for future indications on U.S. net sales of Sunosi[167] - The company assumed Jazz's commitments to SK Biopharmaceuticals and Aerial Biopharma, including single-digit tiered royalties on Sunosi sales and up to $165 million in revenue milestones and $1 million in development milestones[167] Note 16. Income Taxes Discusses the company's income tax position, including tax benefits, expenses, and the impact of valuation allowances | Metric (in thousands) | Three months ended June 30, 2024 | Three months ended June 30, 2023 | Six months ended June 30, 2024 | Six months ended June 30, 2023 | | :-------------------- | :------------------------------- | :------------------------------- | :----------------------------- | :----------------------------- | | Loss before income taxes | $(79,345) | $(67,787) | $(147,702) | $(76,425) | | Income tax benefit (expense) | — | $617 | — | $(1,963) | | Effective tax rate | —% | (0.9)% | —% | 2.6% | - No income tax expense was recorded for the three and six months ended June 30, 2024, due to projected losses and a full valuation allowance against deferred tax assets[170] - A tax benefit of $0.6 million was recorded for Q2 2023, and a tax expense of $2.0 million for H1 2023, related to income earned in Malta from the Pharmanovia License Agreement[170] Note 17. Related Party Transactions Details transactions with related parties, specifically license agreements and royalty payments to an entity owned by the CEO - The company has three exclusive license agreements with Antecip Bioventures II LLC, an entity owned by Dr. Herriot Tabuteau, the CEO and Chairman of the Board[173] - Royalty expense of $2.0 million and $3.6 million was recorded for the three and six months ended June 30, 2024, respectively, to Antecip, representing 3.0% of Auvelity net sales[172] ITEM 2. MANAGEMENT'S DISCUSSION AND ANALYSIS OF FINANCIAL CONDITION AND RESULTS OF OPERATIONS This section provides management's perspective on the company's financial condition, results of operations, and future outlook, including discussions of commercial products, development programs, recent developments, revenue, expenses, liquidity, and capital resources Overview Provides a high-level introduction to the company's commercial-stage biopharmaceutical focus and key product portfolio - Axsome Therapeutics is a commercial-stage biopharmaceutical company focused on CNS conditions, with approved products Auvelity® and Sunosi®, and pipeline candidates AXS-07, AXS-12, and AXS-14[176] - Auvelity was FDA-approved in August 2022 for major depressive disorder, and Sunosi was acquired in 2022 for excessive daytime sleepiness associated with narcolepsy or obstructive sleep apnea[176][177] Development Programs Details the status and intellectual property of the company's clinical development pipeline for various CNS conditions - AXS-05 (Auvelity formulation) is in development for Alzheimer's disease agitation (ADVANCE-1, ACCORD-1, ADVANCE-2, ACCORD-2 trials) and smoking cessation (Phase 2 completed)[179] - AXS-07 for acute migraine has completed two Phase 3 trials (MOMENTUM, INTERCEPT) and an open-label safety study (MOVEMENT); NDA resubmission is expected to be Class 2[180] - AXS-12 for narcolepsy (Orphan Drug Designation) completed Phase 2 (CONCERT) and Phase 3 (SYMPHONY) trials, with SYMPHONY achieving its primary endpoint[181] - AXS-14 for fibromyalgia has in-licensed positive Phase 2 and Phase 3 data from Pfizer[182] - Solriamfetol (Sunosi active ingredient) is in Phase 3 trials for MDD (PARADIGM), binge eating disorder (ENGAGE), ADHD (FOCUS), and shift work disorder (SUSTAIN)[183] - Intellectual property for AXS-05, AXS-07, AXS-12, AXS-14, and Sunosi extends to various years, with a settlement agreement permitting generic Sunosi sales from June 30, 2042[185] Year to Date and Recent Developments Highlights key clinical trial initiations and positive top-line data reported during the current year - In May 2024, the ACCORD-2 study (Phase 3, double-blind, placebo-controlled, randomized withdrawal trial) for AXS-05 in AD agitation was initiated[188] - In March 2024, the PARADIGM study (Phase 3, randomized, double-blind, placebo-controlled, multicenter trial) for solriamfetol in MDD was initiated[188] - In April 2024, the ENGAGE study (Phase 3, randomized, double-blind, placebo-controlled, multicenter trial) for solriamfetol in BED was initiated[188] - In August 2024, the SUSTAIN study (Phase 3, randomized, double-blind, placebo-controlled, multicenter trial) for solriamfetol in SWD was initiated[188] - In March 2024, positive top-line data from the Phase 3 SYMPHONY study of AXS-12 in narcolepsy were reported[189] Financial Overview Summarizes key financial performance metrics, including product sales, license revenue, and expected trends in R&D and SG&A expenses | Metric (in millions) | Three months ended June 30, 2024 | Three months ended June 30, 2023 | Six months ended June 30, 2024 | Six months ended June 30, 2023 | | :------------------- | :------------------------------- | :------------------------------- | :----------------------------- | :----------------------------- | | Net product sales | $86.5 | $46.0 | $160.6 | $74.6 | - Net product sales increased significantly, with $86.5 million for Q2 2024 (vs. $46.0 million for Q2 2023) and $160.6 million for H1 2024 (vs. $74.6 million for H1 2023)[190] - The company recognized $65.7 million in license revenue in Q1 2023 from the Pharmanovia License Agreement, with ongoing royalty revenue from Sunosi sales in ex-U.S. markets[191][192] - Research and development expenses are expected to stabilize after significant increases due to ongoing clinical trials and manufacturing costs[195] - Selling, general and administrative expenses are expected to increase slightly due to expanded marketing, promotional, and advertising costs for Auvelity, Sunosi, and anticipated product launches[197] Results of Operations Analyzes the company's revenues, expenses, and net loss for the reported periods, detailing the drivers of financial performance | Metric (in thousands) | Three months ended June 30, 2024 | Three months ended June 30, 2023 | Six months ended June 30, 2024 | Six months ended June 30, 2023 | | :-------------------- | :------------------------------- | :------------------------------- | :----------------------------- | :----------------------------- | | Product sales, net Auvelity | $65,025 | $27,643 | $118,420 | $43,393 | | Product sales, net Sunosi | $21,495 | $18,374 | $42,196 | $31,193 | | Total revenues | $87,166 | $46,700 | $162,165 | $141,276 | | Cost of revenue | $8,055 | $4,599 | $14,352 | $12,155 | | Research and development | $49,853 | $20,581 | $86,683 | $38,374 | | Selling, general and administrative | $103,554 | $78,935 | $202,524 | $153,126 | | Net loss | $(79,345) | $(67,170) | $(147,702) | $(78,388) | - Auvelity net sales increased by 135.2% to $65.0 million (Q2 2024) and 172.8% to $118.4 million (H1 2024) year-over-year, while Sunosi net sales increased by 17.0% to $21.5 million (Q2 2024) and 35.3% to $42.2 million (H1 2024)[201] - Research and development expenses increased by $29.3 million (Q2) and $48.3 million (H1) year-over-year, driven by Phase 3 trials for solriamfetol, AXS-05, AXS-12, and higher manufacturing costs for AXS-07 and AXS-14[207] - Selling, general and administrative expenses increased by $24.6 million (Q2) and $49.4 million (H1) year-over-year, mainly due to greater commercial activities for Auvelity and Sunosi and higher personnel costs[208] - Net loss increased to $79.3 million (Q2 2024) and $147.7 million (H1 2024) primarily due to increased R&D and SG&A spend, and the absence of the $65.7 million license revenue from Pharmanovia in H1 2023[213] Liquidity and Capital Resources Discusses the company's funding sources, cash position, debt agreements, and future capital requirements - The company's operations have been financed primarily through equity offerings, debt borrowings, and product sales since inception[214] - Under the March 2022 Sales Agreement, the company received $11.0 million in net proceeds from common stock sales for the three months ended June 30, 2024[219] - In January 2023, the Third Amendment to the Loan Agreement increased the term loan advance to $350.0 million, with $180 million outstanding and $170 million remaining as of June 30, 2024[220] - The company believes current cash is sufficient to fund anticipated operations into cash flow positivity, but acknowledges the costly and uncertain nature of product commercialization and clinical trials[222] | Cash Flow Activity (in thousands) | Six months ended June 30, 2024 | Six months ended June 30, 2023 | | :-------------------------------- | :----------------------------- | :----------------------------- | | Operating activities | $(83,577) | $(61,170) | | Investing activities | $(150) | $(204) | | Financing activities | $13,191 | $297,645 | | Net increase (decrease) in cash | $(70,536) | $236,271 | - Cash used in operating activities increased by $22.4 million year-over-year, impacted by the absence of the $65.7 million upfront payment from Pharmanovia in Q1 2023[224] - Cash provided by financing activities decreased significantly due to the absence of large debt drawdowns and public offering proceeds from the prior year[227] Contractual Obligations and Commitments Outlines the company's significant contractual obligations, including license agreements, debt, royalties, and lease commitments - The company has license agreements with Pfizer (for AXS-12 and AXS-14) and Antecip Bioventures (for Auvelity/AXS-05), involving upfront payments, potential milestones, and tiered royalties[231][232] - The Loan Agreement with Hercules Capital, Inc. has been amended multiple times, increasing the principal amount to $350.0 million and revising terms, with covenants limiting additional indebtedness, liens, and requiring minimum cash balances[234][235] - Royalty agreements include non-refundable, non-creditable payments to Jazz on U.S. net sales of Sunosi and assumed commitments to SK Biopharmaceuticals and Aerial Biopharma for tiered royalties and milestones[236][237] - A ten-year sublease for corporate offices at One World Trade Center commenced in April 2023, with a one-time termination option on its fifth anniversary[238] Employees and Human Capital Management Describes the company's workforce, human capital management strategies, and efforts to attract and retain talent - As of July 29, 2024, the company had 589 full-time employees, none represented by a collective bargaining agreement[239] - The company emphasizes competitive compensation, equity ownership, development programs, and a robust benefits package to attract and retain highly skilled employees[239] Off-Balance Sheet Arrangements Confirms the absence of off-balance sheet arrangements as defined by SEC regulations during the reported periods - The company did not have any off-balance sheet arrangements during the periods presented, as defined by SEC regulations[240] Recent Accounting Pronouncements Discusses the company's evaluation of recently issued accounting standards updates and their potential impact - The company is evaluating the effect of ASU 2023-07 (Segment reporting, effective for years beginning after December 15, 2023) and ASU 2023-09 (Improvements to income tax disclosures, effective for annual periods beginning after December 15, 2024) on its consolidated financial statements[86][87] ITEM 3. QUANTITATIVE AND QUALITATIVE DISCLOSURE ABOUT MARKET RISK This section discusses the company's exposure to market risks, primarily interest rate fluctuations and foreign currency exchange risk, and assesses their potential impact on financial condition and results of operations - The company is exposed to interest rate risk due to its cash holdings ($315.7 million as of June 30, 2024) and variable interest rate debt, but does not expect a material effect from a sudden 100 basis point interest rate increase[243] - Foreign currency exchange risk arises from transactions denominated in Euros, Swiss Francs, and British Pounds, particularly royalty revenues from Pharmanovia, but no material effects have been incurred to date[244] - Inflation has not had a material effect on the company's business, financial condition, or results of operations during the six months ended June 30, 2024[245] ITEM 4. CONTROLS AND PROCEDURES Management, with the participation of the principal executive and financial officers, evaluated the effectiveness of disclosure controls and procedures, concluding they were effective as of June 30, 2024. No material changes in internal control over financial reporting occurred during the quarter - Disclosure controls and procedures were evaluated and deemed effective as of June 30, 2024, ensuring timely and accurate reporting of required information[246] - No changes in internal control over financial reporting materially affected, or are reasonably likely to materially affect, the company's internal control over financial reporting during the quarter ended June 30, 2024[247] PART II — OTHER INFORMATION ITEM 1. LEGAL PROCEEDINGS This section details ongoing legal proceedings, including a securities class action, a shareholder derivative action, and patent infringement lawsuits related to Auvelity and Sunosi, highlighting the company's efforts to defend its intellectual property - A Securities Class Action (Gru v. Axsome Therapeutics, Inc., et al.) alleging false statements regarding CMC practices and AXS-07 NDA was dismissed in September 2023, but plaintiffs filed a second amended complaint in February 2024[249][250] - A Shareholder Derivative Action (Engel v. Herriot Tabuteau, et al.) with similar allegations is stayed pending the Securities Class Action[251] - The company commenced three patent infringement actions against Teva relating to Teva's ANDA for Auvelity, with actions currently pending[252] - Multiple patent infringement actions were commenced against Hikma and five other drug companies regarding their ANDAs for Sunosi; a settlement with Unichem Laboratories Ltd. permits generic solriamfetol sales from June 30, 2042[253] ITEM 1A. RISK FACTORS This section outlines significant risks that could materially affect the company's business, including financial condition, operational challenges, dependence on third parties, intellectual property issues, legal and compliance matters, and risks related to common stock ownership Risk Factors Summary Provides a high-level overview of the most critical risks facing the company, spanning financial, operational, and competitive challenges - Key risks include significant losses and potential inability to achieve profitability, need for additional funding, restrictions from debt covenants, limited commercialization history, dependence on product success, and challenges in demonstrating product safety and efficacy[255][256][257] - Other risks involve intense competition, difficulties in establishing marketing and sales capabilities, potential lack of market acceptance, reliance on third-party services and manufacturers, intellectual property protection challenges, and compliance with healthcare laws[258][259][260][261] RISKS RELATED TO OUR FINANCIAL CONDITION AND CAPITAL REQUIREMENTS Details risks associated with the company's financial health, including operating losses, funding needs, debt covenants, and macroeconomic factors - The company has incurred significant operating losses since inception ($983.3 million accumulated deficit as of June 30, 2024) and expects continued losses due to commercialization and development costs, requiring additional financing[262][263][267] - Operating activities may be restricted by covenants in the loan agreement with Hercules, including minimum cash requirements and limitations on indebtedness, asset sales, and dividends[272][274] - The company's limited operating history in commercializing products (Auvelity and Sunosi launched in 2022) makes it difficult to accurately evaluate future business prospects and financial fluctuations[275][276][277] - Geopolitical instability (e.g., Russia-Ukraine conflict, Israel-Hamas conflict) and U.S. policy changes (e.g., regarding China manufacturing) could disrupt global markets, supply chains, and access to capital, adversely affecting the business[279][280][281][282][283][284] - Climate change poses physical risks (extreme weather, supply chain disruption) and transitional risks (new regulations, increased costs) that could negatively impact business operations and financial results[285][286][287] RISKS RELATED TO OUR BUSINESS AND THE DEVELOPMENT OF OUR PRODUCT CANDIDATES Covers risks inherent in product development, clinical trials, regulatory approvals, and the commercialization of pipeline candidates - The company's business is entirely dependent on the successful commercialization of Auvelity and Sunosi and the development/commercialization of pipeline candidates, which is uncertain and costly[288][289] - Reliance on the 505(b)(2) pathway for some product candidates (AXS-05, AXS-07) carries risks, including potential FDA disagreement, need for additional trials, and patent infringement lawsuits that could delay approval[305][307][309] - Clinical trials are expensive, lengthy, and uncertain; failures, delays, or negative results at any stage could prevent or delay regulatory approval and commercialization[316][317][321] - Undesirable side effects or toxicity profiles of product candidates could delay or prevent regulatory approval, lead to restrictive labeling (e.g., black box warnings), or require abandonment of development[325][326][327] - Delays or difficulties in patient enrollment for clinical trials, especially for rare indications, could significantly increase development costs and timelines, or lead to abandonment of programs[331][332][334] - Development of combination therapies (AXS-05, AXS-07) is more complex, requiring demonstration of each component's contribution and overall safety/efficacy, potentially increasing trial complexity and subject numbers[335][336] - Changes in manufacturing or formulation during development could alter product performance, require additional testing or regulatory approval, and cause delays or increased costs[337] - Failure to obtain marketing approval in international jurisdictions, which have varying regulatory requirements and approval timelines, would prevent products from being marketed abroad[338] - Breakthrough Therapy and Fast Track designations do not guarantee faster approval or increased likelihood of marketing approval, and can be rescinded (e.g., AXS-12's Breakthrough Therapy designation)[339][342][343] - Regulatory approval is limited to specific indications; promoting off-label uses could lead to significant liability, government fines, and damage to reputation[344][345][348][349][350] - Ongoing regulatory obligations and review post-approval can result in significant expenses, labeling restrictions, market withdrawal, and penalties for non-compliance (e.g., cGMP, GCP)[352][353][354][355][356][357] - International operations expose the company to risks such as differing regulatory requirements, parallel importing, data privacy laws (GDPR), intellectual property enforcement challenges, economic instability, and foreign currency fluctuations[358][359][360] - Failure or delay in obtaining FDA approval for proposed product names could require adopting alternative names, leading to loss of trademark benefits and increased costs[360][361] RISKS RELATED TO THE COMMERCIALIZATION OF OUR PRODUCTS Addresses risks concerning market competition, product acceptance, sales capabilities, generic competition, and product liability - The company faces significant competition from major pharmaceutical and biotechnology companies, academic institutions, and other research organizations in the CNS disorder market[363][364][365] - Competitors may develop safer, more effective, or less expensive products, or obtain regulatory approval faster, impacting the company's market position and commercial opportunities[366] - Generic forms of active ingredients in the company's product candidates are available and could be used off-label, adversely affecting profitability[367] - Approval of generic or similar versions of the company's products, or insufficient exclusivity periods, could significantly reduce sales and profitability[370][371][372][374] - Orphan Drug Designation for AXS-12 (narcolepsy) does not guarantee exclusivity or protection from competition, and changes in orphan drug regulations could harm the business[375][376][377] - Inability to establish effective marketing, sales, and distribution capabilities, or to recruit and retain personnel, could limit revenue generation from commercial products[378][379][380] - Lack of broad market acceptance by physicians, patients, and third-party payors for approved products could limit revenues, requiring significant resources for education and potentially leading to price concessions[381][382][383][384][385] - Product liability claims, arising from clinical trials or commercial sales, could result in substantial liabilities, decreased demand, reputational harm, and significant financial and management resource diversion[386][387][389] - Sunosi, as a Schedule IV controlled substance, is subject to U.S. federal and state controlled substance laws and regulations, with non-compliance potentially leading to enforcement actions, penalties, and adverse effects on business[390][391][392][393] RISKS RELATED TO OUR DEPENDENCE ON THIRD PARTIES Highlights risks arising from reliance on third-party contractors for clinical trials, manufacturing, distribution, and strategic collaborations - The company relies heavily on third parties (CROs, medical institutions, clinical investigators) for preclinical studies and clinical trials, and their failure to perform or comply with regulations could delay product development and commercialization[395][396][397][400] - Dependence on a limited number of contract manufacturers for products poses risks of supply delays, inability to meet demand, and non-compliance with cGMP regulations, which could jeopardize regulatory approval and commercialization[402][403][404][406][407][408][409] - Reliance on third-party service providers for warehousing, distribution, government price reporting, and adverse event reporting means their failures could impair product delivery, lead to regulatory enforcement, or False Claims Act liability[410][411][412] - Collaboration arrangements (e.g., with Pharmanovia, Duke University) may not be successful, as collaborators might not commit sufficient resources, terminate agreements, or develop competing products, impacting development and commercialization[413][414][417][418] RISKS RELATED TO INTELLECTUAL PROPERTY Discusses challenges in protecting intellectual property, including patent enforcement, infringement claims, and reliance on licensed technologies - Protecting proprietary rights is difficult and costly; patents have limited lifespans, and market exclusivity is limited, potentially allowing generic competition upon expiry[420][421][422][424] - Patent reform legislation (e.g., Leahy-Smith America Invents Act) and post-grant proceedings can increase uncertainties and costs, potentially reducing the scope or enforceability of patent rights[425][426] - Non-compliance with procedural, documentary, and fee payment requirements of patent agencies can lead to abandonment or lapse of patent rights[428][429] - Lawsuits for infringing third-party intellectual property rights are costly and time-consuming, potentially leading to substantial damages, injunctions, or the need for expensive licenses[430][431][432][433] - Lawsuits to protect or enforce the company's patents are expensive, time-consuming, and may be unsuccessful, risking invalidation or narrow interpretation of patents[434][435][436] - Reliance on licensed intellectual property from third parties (Pfizer, Antecip) means termination or unavailability of licenses on reasonable terms could materially harm the business[437][438][439][440][441] - Claims of wrongful use or disclosure of trade secrets by employees or consultants, or inability to prevent disclosure of trade secrets, could lead to litigation, substantial costs, and loss of competitive advantage[443][444] - Protecting intellectual property rights globally is expensive and challenging, as foreign laws may offer less protection, potentially allowing competitors to use technologies in other jurisdictions[445][446] RISKS RELATED TO LEGAL AND COMPLIANCE MATTERS Outlines risks associated with healthcare laws, regulatory compliance, pricing controls, and potential employee misconduct - Failure to comply with federal, state, and foreign healthcare laws (e.g., Anti-Kickback Statute, False Claims Act, HIPAA, GDPR) could result in substantial penalties, fines, exclusion from healthcare programs, and adverse effects on business[447][448][449][450][452] - Inadequate coverage and payment rates from government or third-party payors, or their preference for less expensive therapies, could limit revenue and profitability prospects[453][454][456][457][458][460][461][462][463] - New legislation, regulatory proposals, and healthcare payor initiatives (e.g., ACA, Inflation Reduction Act of 2022) may increase compliance costs, restrict marketing, and adversely affect revenue and capital raising[464][465][466][467][468][469][470] - Strict price controls in international markets could adversely affect revenues and profitability, as pricing negotiations can be lengthy and reimbursement levels may be inadequate[471] - Employee misconduct, including noncompliance with regulatory standards or fraud, could lead to regulatory sanctions, reputational harm, significant fines, and diversion of management attention[472][473] - Use of hazardous materials in manufacturing requires compliance with environmental laws, with violations potentially leading to substantial fines, penalties, business interruptions, and cleanup liabilities[474][475][476] RISKS RELATED TO OUR BUSINESS OPERATIONS Covers operational risks such as organizational growth, M&A integration, key personnel retention, internal controls, and system failures - Significant organizational growth (589 full-time employees as of July 29, 2024) may strain personnel resources and infrastructure, requiring effective management, hiring, training, and improved controls[477][478][479][480] - Acquiring businesses or products, or forming strategic alliances, may not realize expected benefits due to integration difficulties, higher costs, or diversion of management attention[481] - Inability to attract and retain key personnel, especially senior management, could seriously harm business strategy due to intense competition and the specialized skills required[482] - Failure to maintain an effective system of internal controls over financial reporting could lead to inaccurate financial reports, loss of investor confidence, stock price decline, and regulatory sanctions[483][484][485][486][487] - Disclosure controls and procedu