ANX-005

Single, Well-Tolerated Infusion of ANX005 Significantly Accelerated Recovery of GBS Patients vs. Placebo in Pivotal Phase 3 Trial; Potential to be First Targeted Therapy for GBS; Topline Real-World Evidence (RWE) Comparability Data Now Expected by Year-End 2024 Dosing Initiated in ARCHER II Registrational Trial for ANX007; Only Program to Demonstrate Significant Vision Protection and Structural Protection in Regions of the Eye Important for Vision Acuity in GA; Phase 3 Data Expected Second Half 2026 First-in-Kind Oral C1s Inhibitor ANX1502 Successfully Completed Bridging Study to Twice-Daily Tablet; Proof-of-Concept Data in Autoimmune Disease Expected Q4 2024 Robust Balance Sheet with Cash, Cash Equivalents, and Short-term Investments of Approximately $368.7 Million as of June 30, 2024, and Anticipated Runway into Second Half 2026 BRISBANE, Calif., Aug. 12, 2024 (GLOBE NEWSWIRE) -- Annexon, Inc. (Nasdaq: ANNX), a biopharmaceutical company advancing a late-stage clinical platform of novel therapies for people living with devastating classical complement-mediated neuroinflammatory diseases of the body, brain, and eye, today highlighted portfolio progress and reported second quarter 2024 financial results. “Annexon is at the cross section where decades of pioneering research and development are translating into clinical wins for scores of patients. This was exemplified in the first half of 2024 by the robust functional and biomarker data from two of our flagship programs: Guillain-Barré syndrome (GBS) and geographic atrophy (GA). These data further highlight the powerful disease modifying potential of our platform approach by blocking neuroinflammation where it starts in diverse acute and chronic conditions of high unmet need,” said Douglas Love, president and chief executive officer of Annexon. “In that regard, ANX005 helped GBS patients suffering from an acute neuromuscular emergency get better sooner and more completely in the first placebo-controlled pivotal study in 40 years. Moreover, ANX007 is the only program shown to help GA patients significantly preserve their vision while protecting associated retina structures critical for vision. We’re also pleased to report that the bridging study for our oral inhibitor ANX1502, the first clinical stage oral inhibitor of the classical pathway, has completed and the safety and pharmacokinetics profile confirmed the findings from the healthy volunteer trial.” Mr. Love continued, “With these data, we’re actively working to build on our strong momentum across our flagship programs with several key catalysts over the next 24-months that have the potential to drive significant benefit for millions of patients and our shareholders. Importantly, we now expect earlier topline data by year-end 2024 for our real-world evidence comparability study for ANX005 in GBS to support our BLA submission targeted for the first half of 2025. Additionally, our ~630 patient potentially best-in-disease global Phase 3 GA program has been initiated, and we anticipate pivotal data in the second half of 2026. Lastly, we remain on pace for proof-of-concept data for ANX1502 in Cold Agglutinin Disease (CAD) in the latter part of the year, which has the potential to enable advancement in an array of autoimmune indications. With a strong cash position, we are more excited than ever and remain sharply focused on delivering multiple first-in-kind targeted therapies to improve the lives of millions of patients living with devastating neuroinflammatory diseases.” Recent Clinical Program Updates Flagship Programs ANX005 in Guillain-Barré syndrome (GBS): First-in-kind monoclonal antibody designed to block C1q and the entire classical complement pathway in both the body and the brain. ANX005 demonstrated a highly clinically relevant and statistically significant effect on multiple measures of the primary endpoint GBS-DS; positive Phase 3 topline results and additional analyses reported at the June 2024 Peripheral Nerve Society (PNS) Annual MeetingIncreased likelihood of being in a better state of health seen by week 1, and observed at all subsequent time points through 26-weeksEarly, robust and durable treatment effects expedited recovery and led to patients walking and off ventilation approximately one month earlierSingle infusion of ANX005 was generally well-tolerated with a profile similar to placebo and adverse events balanced across groupsPatients with baseline characteristics similar to those of patients in North America and Europe had greater responses to ANX005 over placeboInitiated a real-world evidence (RWE) comparability study with global experts in GBS using the International Guillain-Barré Syndrome Outcomes Study (IGOS), a global, prospective, observational, multicenter cohort study that has enrolled 2,000 patients who were followed for one to three years ANX007 in Geographic Atrophy (GA): First-in-kind, non-pegylated antigen-binding fragment (Fab) designed to block C1q and activation of the classical complement cascade locally in the eye with an intravitreal formulation. Patient dosing initiated in the global registrational Phase 3 ARCHER II trial, a well-powered, sham-controlled study with a robust safety database expected to enroll approximately 630 patientsRegulatory alignment with the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) on key study elements of Phase 3 program, including first use of best corrected visual acuity (BCVA) protection against ≥15-letter loss as the primary outcome measure in GANew Phase 2 ARCHER data on the protection of vision and vision-related retinal structure presented at the 2024 American Society of Retina Specialists (ASRS) Annual Scientific meetingANX007 is the only investigational medicine for GA to date to show significant vision protection in both standard and low light conditions, and protection of photoreceptors in the central fovea, the region of the retina needed for important activities such as reading, driving and recognizing facesTreatments for GA have primarily focused on addressing lesion growth by measuring the protection of supportive retinal pigment epithelium (RPE) and have not translated to protection of clinically meaningful vision for patients ANX1502 for Autoimmune Conditions: First-in-kind oral small molecule inhibitor of the classical complement pathway designed to target chronic autoimmune diseases. Completed bridging study in healthy volunteers from a liquid suspension formulation to a twice daily tablet with safety and pharmacokinetic profile similar or better than previous studiesOngoing proof-of-concept (POC) trial in CAD will characterize pharmacodynamics on complement and hemolysis measures, and is designed to enable advancement in multiple antibody-mediated autoimmune indications, many with clinical validation and treatments approved for weekly or every other week infusionsANX1502 has the potential to offer the advantages of selective upstream classical complement inhibition with the convenience and dosing flexibility of oral administration Key Anticipated Milestones for Flagship Programs ANX005 in GBS: Initial topline data from RWE comparability protocol with IGOS now expected by year-end 2024 to support a planned BLA submission in the first half of 2025ANX007 in GA: Phase 3 ARCHER II trial topline data expected in the second half of 2026. Plans for an injection-controlled study, ARROW, to assess the prevention of ≥15-letter loss of BCVA, are also ongoing ANX1502 in CAD: POC trial evaluating the pharmacodynamics and efficacy of an oral tablet formulation in CAD anticipated to provide initial data in the fourth quarter of 2024 Second Quarter 2024 Financial Results Cash and operating runway: Cash, cash equivalents and short-term investments were $368.7 million as of June 30, 2024. Annexon continues to expect its cash, cash equivalents and short-term investments as of June 30, 2024, to be sufficient to fund the company’s planned operating expenses into the second half of 2026Research and development (R&D) expenses: R&D expenses were $25.0 million for the quarter ended June 30, 2024, reflecting the advancement of the Company’s priority programs, including GBS, GA and ANX1502, compared to $30.3 million for the quarter ended June 30, 2023General and administrative (G&A) expenses: G&A expenses were $8.6 million for the quarter ended June 30, 2024, compared to $7.4 million for the quarter ended June 30, 2023Net loss: Net loss was $29.6 million or $0.23 per share for the quarter ended June 30, 2024, compared to $35.2 million or $0.47 per share for the quarter ended June 30, 2023 About Annexon Annexon Biosciences (Nasdaq: ANNX) is a biopharmaceutical company advancing a late-stage clinical platform of novel therapies for people living with devastating classical complement-mediated neuroinflammatory diseases of the body, brain, and eye. Annexon’s novel scientific approach targets upstream C1q to block the classical complement inflammatory cascade before it starts, and its therapeutic candidates are designed to provide meaningful benefits across multiple autoimmune, neurodegenerative and ophthalmic diseases. With proof-of concept data in Guillain-Barré syndrome, Huntington’s disease and geographic atrophy, Annexon is rigorously advancing its mid-to late-stage clinical trials to bring new potential treatments to patients as quickly as possible. To learn more visit annexonbio.com. Forward Looking Statements This press release contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. In some cases, you can identify forward-looking statements by terminology such as “aim,” “anticipate,” “assume,” “believe,” “contemplate,” “continue,” “could,” “design,” “due,” “estimate,” “expect,” “goal,” “intend,” “may,” “objective,” “plan,” “positioned,” “potential,” “predict,” “seek,” “should,” “target,” “will,” “would” and other similar expressions that are predictions of or indicate future events and future trends, or the negative of these terms or other comparable terminology. All statements other than statements of historical facts contained in this press release are forward-looking statements. These forward-looking statements include, but are not limited to, statements about: the timing of completion of Phase 3 trial of ANX005 in patients with GBS; the potential therapeutic benefit of ANX005, if approved, compared to existing therapies; anticipated timing of the completion of a RWE comparability study and BLA submission for ANX005; the potential therapeutic benefit of ANX007; timing of the ARCHER II trial and initiation of ARROW trial; ANX007’s distinct potential neuroprotective mechanism of action and potential to provide protection from vision loss; timing of proof-of-concept data for ANX1502; continued development of ANX007 and ANX1502; anticipated cash runway into the second half of 2026; the potential benefits from treatment with anti-C1q therapy; and continuing advancement of the company’s portfolio. Forward-looking statements are not guarantees of future performance and are subject to risks and uncertainties that could cause actual results and events to differ materially from those anticipated, including, but not limited to, risks and uncertainties related to: the ongoing off-treatment follow-up portion of the ARCHER trial and final results from the ARCHER trial; the company’s history of net operating losses; the company’s ability to obtain necessary capital to fund its clinical programs; the early stages of clinical development of the company’s product candidates; the effects of public health crises on the company’s clinical programs and business operations; the company’s ability to obtain regulatory approval of and successfully commercialize its product candidates; any undesirable side effects or other properties of the company’s product candidates; the company’s reliance on third-party suppliers and manufacturers; the outcomes of any future collaboration agreements; and the company’s ability to adequately maintain intellectual property rights for its product candidates. These and other risks are described in greater detail under the section titled “Risk Factors” contained in the company’s Annual Report on Form 10-K and Quarterly Reports on Form 10-Q and the company’s other filings with the SEC. Any forward-looking statements that the company makes in this press release are made pursuant to the Private Securities Litigation Reform Act of 1995, as amended, and speak only as of the date of this press release. Except as required by law, the company undertakes no obligation to publicly update any forward-looking statements, whether as a result of new information, future events or otherwise. Investor Contact: Joyce AllaireLifeSci Advisorsjallaire@lifesciadvisors.com Media Contact: Sheryl SeapyReal Chemistry949-903-4750sseapy@realchemistry.com ANNEXON, INC.Condensed Consolidated Statements of Operations(in thousands, except share and per share amounts) Three Months EndedJune 30, Six Months EndedJune 30, 2024 2023 2024 2023 Operating expenses: Research and development (1) $25,026 $30,251 $45,989 $62,596 General and administrative (1) 8,554 7,440 16,163 16,337 Total operating expenses 33,580 37,691 62,152 78,933 Loss from operations (33,580) (37,691) (62,152) (78,933)Interest and other income, net 3,970 2,503 7,366 5,069 Net loss $(29,610) $(35,188) $(54,786) $(73,864)Net loss per share, basic and diluted $(0.23) $(0.47) $(0.43) $(0.99)Weighted-average shares used in computing net loss per share, basic and diluted 130,132,960 75,230,003 126,403,081 74,546,995 (1) Includes the following stock-based compensation expense: Research and development $2,311 $2,307 $4,593 $4,558 General and administrative $2,631 $2,353 $5,009 $4,709 ANNEXON, INC.Condensed Consolidated Balance Sheets(in thousands) June 30, December 31, 2024 2023 (unaudited) Assets Current assets: Cash and cash equivalents $157,304 $225,110 Short-term investments 211,395 34,606 Prepaid expenses and other current assets 5,270 4,144 Total current assets 373,969 263,860 Restricted cash 1,032 1,032 Property and equipment, net 13,702 14,773 Operating lease right-of-use assets 17,382 18,009 Total assets $406,085 $297,674 Liabilities and Stockholders' Equity Current liabilities: Accounts payable $4,126 $5,487 Accrued liabilities 10,657 10,235 Operating lease liabilities, current 2,345 2,165 Other current liabilities 20 41 Total current liabilities 17,148 17,928 Operating lease liabilities, non-current 27,858 29,190 Total liabilities 45,006 47,118 Stockholders’ equity: Common stock 106 78 Additional paid-in capital 988,347 823,029 Accumulated other comprehensive loss (89) (52)Accumulated deficit (627,285) (572,499)Total stockholders' equity 361,079 250,556 Total liabilities and stockholders’ equity $406,085 $297,674

BILLERICA, Mass.--( BUSINESS WIRE )-- Quanterix Corporation (NASDAQ: QTRX), a company fueling scientific discovery through ultra-sensitive biomarker detection, today announced financial results for the second quarter ended June 30, 2024. “ Our research business once again delivered strong performance in the second quarter, growing double digits,” said Masoud Toloue, Chief Executive Officer of Quanterix. “ Demand for Simoa sensitivity continues, as highlighted by a 35% increase in Accelerator service revenue compared to the corresponding prior year period, as customers are able to access our technology without impacting their capital expenditure budgets. For the balance of 2024, we expect to continue to execute against our strategy: delivering new menu for our research customers, innovating toward a new platform, and building the infrastructure for blood-based Alzheimer’s Diagnostics.” Second Quarter Financial Highlights Revenue of $34.4 million, an increase of 10.8% compared to $31.0 million in the corresponding prior year period. GAAP gross margin of 58.3%, as compared to 61.7% in the prior year period. Non-GAAP gross margin of 52.3% as compared to 56.4% in the corresponding prior year period. Net loss of $9.5 million, an increase of $3.4 million compared to the corresponding prior year period. Net cash usage in the quarter was $5.1 million, and the Company ended the period with $299.5 million of cash, cash equivalents, marketable securities, and restricted cash. Operational and Business Highlights The Company presented data at the Alzheimer’s Association International Conference (AAIC), demonstrating successful validation of a multi-marker approach for Alzheimer’s disease detection. The findings showed a multi-marker test with an algorithm improved diagnostic certainty compared to a stand-alone p-Tau 217 test, reducing the test’s intermediate zone three-fold from 31.2% to 10.5%, while maintaining overall accuracy above 90%. The Company announced four new partnerships to provide access to Alzheimer’s disease diagnostic testing. These partners consist of three large domestic hospital systems, Mt. Sinai, Banner Health, and UCSF, and a leading laboratory in China, KingMed Diagnostics. Utilizing data produced using the Company’s Accelerator lab services, the biotechnology company Annexon Biosciences reported that they demonstrated positive topline results for their pivotal phase 3 trial in Guillain-Barre Syndrome. As part of the trial, Annexon reported that they demonstrated their therapy ANX005 reduced levels of neurofilament light (Nf-L) between weeks two and four of therapy. 2024 Full Year Business Outlook The Company has revised its 2024 revenue guidance range to $134 million to $138 million, compared to $139 million to $144 million previously due to what remains a constrained capital funding environment. This revenue range excludes revenue from Lucent Diagnostics testing, which we expect to be immaterial for 2024. The Company continues to expect GAAP gross margin percentage to be in the range of 57%-61%, and non-GAAP gross margin percentage in the range of 51%-55%. Finally, the Company anticipates 2024 cash usage (change in cash, cash equivalents, marketable securities, and restricted cash) to be at the higher end of its prior cash usage range of $25 million to $30 million. For additional information on the non-GAAP financial measures included in this press release, please see “Use of Non-GAAP Financial Measures” and “ Reconciliation of GAAP to Non-GAAP Financial Measures” below. Conference Call In conjunction with this announcement, the Company will host a conference call on August 8, 2024, at 4:30 p.m. E.T. Click here to register for the conference call and obtain your dial-in number and passcode. Interested investors can also listen to the live webcast from the Event Details page in the Investors section of the Quanterix at https://ir.quanterix.com . An archived webcast replay will be available on the Company’s website for one year. Financial Highlights Quanterix Corporation Consolidated Statements of Operations (Unaudited, amounts in thousands except per share data) Three Months Ended June 30, 2024 2023 Revenues: Product revenue $19,887 $19,692 Service and other revenue 13,511 10,552 Collaboration and license revenue 729 629 Grant revenue 254 156 Total revenues 34,381 31,029 Costs of goods sold and services: Cost of product revenue 8,851 7,236 Cost of service and other revenue 5,472 4,655 Total costs of goods sold and services 14,323 11,891 Gross profit 20,058 19,138 Operating expenses: Research and development 8,104 5,946 Selling, general and administrative 24,135 21,591 Other lease costs 927 1,162 Total operating expenses 33,166 28,699 Loss from operations (13,108) (9,561) Interest income 3,681 3,886 Other income (expense), net (9) (154) Loss before income taxes (9,436) (5,829) Income tax expense (37) (235) Net loss $(9,473) $(6,064) Net loss per common share, basic and diluted $(0.25) $(0.16) Weighted-average common shares outstanding, basic and diluted 38,338 37,494 Quanterix Corporation Consolidated Balance Sheets (Unaudited, amounts in thousands) June 30, 2024 December 31, 2023 ASSETS Current assets: Cash and cash equivalents $47,002 $174,422 Marketable securities 249,853 146,902 Accounts receivable, net of allowance for expected credit losses 31,784 25,414 Inventory 28,363 22,365 Prepaid expenses and other current assets 8,724 9,291 Total current assets 365,726 378,394 Restricted cash 2,607 2,604 Property and equipment, net 18,205 17,926 Intangible assets, net 4,981 6,034 Operating lease right-of-use assets 17,399 18,251 Other non-current assets 2,370 1,802 Total assets $411,288 $425,011 LIABILITIES AND STOCKHOLDERS' EQUITY Current liabilities: Accounts payable $7,273 $5,048 Accrued compensation and benefits 9,044 13,659 Accrued expenses and other current liabilities 6,577 6,041 Deferred revenue 10,121 9,468 Operating lease liabilities 4,524 4,241 Total current liabilities 37,539 38,457 Deferred revenue, net of current portion 928 1,227 Operating lease liabilities, net of current portion 35,052 37,223 Other non-current liabilities 1,017 1,177 Total liabilities 74,536 78,084 Total stockholders’ equity 336,752 346,927 Total liabilities and stockholders’ equity $411,288 $425,011 Quanterix Corporation Consolidated Statements of Cash Flows (Unaudited, amounts in thousands) Six Months Ended June 30, 2024 2023 Cash flows from operating activities: Net loss $(19,545) $(12,167) Adjustments to reconcile net loss to net cash used in operating activities: Depreciation and amortization expense 3,124 2,845 Credit losses on accounts receivable 676 324 Accretion of marketable securities (3,619) — Operating lease right-of-use asset amortization 840 1,002 Stock-based compensation expense 10,493 8,095 Other operating activity (13) 548 Changes in assets and liabilities: Accounts receivable (7,242) (5,750) Inventory (6,011) (1,181) Prepaid expenses and other current assets 597 (527) Other non-current assets (596) (965) Accounts payable 2,054 (631) Accrued compensation and benefits, accrued expenses, and other current liabilities (4,390) (1,326) Deferred revenue 354 1,666 Operating lease liabilities (1,876) (730) Other non-current liabilities 39 (72) Net cash used in operating activities (25,115) (8,869) Cash flows from investing activities: Purchases of marketable securities (189,344) — Proceeds from maturities of marketable securities 89,229 — Purchases of property and equipment (2,105) (784) Net cash used in investing activities (102,220) (784) Cash flows from financing activities: Proceeds from common stock issued under stock plans 2,421 777 Payments for employee taxes withheld on stock-based compensation awards (2,150) (87) Net cash provided by financing activities 271 690 Net decrease in cash, cash equivalents, and restricted cash (127,064) (8,963) Effect of exchange rate changes on cash, cash equivalents, and restricted cash (353) (163) Cash, cash equivalents, and restricted cash at beginning of period 177,026 341,337 Cash, cash equivalents, and restricted cash at end of period $49,609 $332,211 Use of Non-GAAP Financial Measures To supplement our financial statements presented on a U.S. GAAP basis, we present non-GAAP gross profit, non-GAAP gross margin, non-GAAP total operating expenses, and non-GAAP loss from operations. These non-GAAP measures are calculated by including shipping and handling costs for product sales within cost of product revenue instead of within selling, general, and administrative expenses. We use these non-GAAP measures to evaluate our operating performance in a manner that allows for meaningful period-to-period comparison and analysis of trends in our business and our competitors. We believe that presentation of these non-GAAP measures provides useful information to investors in assessing our operating performance within our industry and to allow comparability to the presentation of other companies in our industry where shipping and handling costs are included in cost of goods sold for products. The non-GAAP financial information presented here should be considered in conjunction with, and not as a substitute for, the financial information presented in accordance with U.S. GAAP. Set forth below is a reconciliation of non-GAAP gross profit, non-GAAP gross margin, non-GAAP total operating expenses, and non-GAAP loss from operations to their most directly comparable GAAP financial measures. Reconciliation of GAAP to Non-GAAP Financial Measures Quanterix Corporation Reconciliation of GAAP Financial Measures to Non-GAAP Financial Measures (Unaudited, amounts in thousands except percentages) Three Months Ended June 30, Six Months Ended June 30, 2024 2023 2024 2023 GAAP gross profit $20,058 $19,138 $39,684 $36,064 Shipping and handling costs (2,075) (1,623) (4,217) (3,451) Non-GAAP gross profit $17,983 $17,515 $35,467 $32,613 GAAP revenue $34,381 $31,029 $66,447 $59,485 GAAP gross margin (gross profit as % of revenue) 58.3% 61.7% 59.7% 60.6% Non-GAAP gross margin (non-GAAP gross profit as % of revenue) 52.3% 56.4% 53.4% 54.8% GAAP total operating expenses $33,166 $28,699 $66,758 $55,045 Shipping and handling costs (2,075) (1,623) (4,217) (3,451) Non-GAAP total operating expenses $31,091 $27,076 $62,541 $51,594 GAAP loss from operations $(13,108) $(9,561) $(27,074) $(18,981) Non-GAAP loss from operations $(13,108) $(9,561) $(27,074) $(18,981) About Quanterix From discovery to diagnostics, Quanterix’s ultra-sensitive biomarker detection is driving breakthroughs only made possible through its unparalleled sensitivity and flexibility. The Company’s Simoa technology has delivered the gold standard for earlier biomarker detection in blood, serum or plasma, with the ability to quantify proteins that are far lower than the Level of Quantification of conventional analog methods. Its industry-leading precision instruments, digital immunoassay technology and CLIA-certified Accelerator laboratory have supported research that advances disease understanding and management in neurology, oncology, immunology, cardiology and infectious disease. Quanterix has been a trusted partner of the scientific community for nearly two decades, powering research published in more than 2,900 peer-reviewed journals. Find additional information about the Billerica, Massachusetts-based company at https://www.quanterix.com or follow us on Twitter and LinkedIn . Forward-Looking Statements Quanterix’s current financial results, as discussed in this press release, are preliminary and unaudited, and subject to adjustment. This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as "may," "will," "expect," "plan," "anticipate," "estimate," "intend" and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances) are intended to identify forward-looking statements. These forward-looking statements include, but are not limited to, statements about Quanterix’s financial performance, including statements under the header “2024 Full Year Business Outlook” set forth above, and are subject to a number of risks, uncertainties and assumptions. Forward-looking statements in this press release are based on Quanterix’s expectations and assumptions as of the date of this press release. Each of these forward-looking statements involves risks and uncertainties. Factors that may cause Quanterix’s actual results to differ from those expressed or implied in the forward-looking statements in this press release include, but are not limited to, those described in our periodic reports filed with the U.S. Securities and Exchange Commission, including the "Risk Factors" sections contained therein. Except as required by law, Quanterix assumes no obligation to update any forward-looking statements contained herein to reflect any change in expectations, even as new information becomes available.