C1q

Only Program that has Demonstrated Significant Vision Protection in Standard and Low Light Conditions and Significant Preservation of Photoreceptors in the Fovea Region Critical for Visual Acuity ARCHER II Phase 3 Pivotal Program in GA Underway with Data Expected Second Half 2026 BRISBANE, Calif., Sept. 09, 2024 (GLOBE NEWSWIRE) -- Annexon, Inc. (Nasdaq: ANNX), a biopharmaceutical company advancing novel complement therapies for neuroinflammatory diseases of the body, brain, and eye, today announced the Company will present analyses of ANX007 from the completed Phase 2 ARCHER trial in geographic atrophy (GA) at the Retina Society’s 57th annual scientific meeting being held September 11-15 in Lisbon, Portugal, and at the 24th annual Euretina Congress being held September 19–22 in Barcelona, Spain. ANX007 is a first-in-kind, non-pegylated antigen-binding fragment (Fab) designed to block C1q locally in the eye with an intravitreal formulation. “The randomized ARCHER data for ANX007 in GA demonstrated broad-based protection of vision in standard and low light conditions, along with significant protection of visual structures in key regions of the eye important to visual acuity. Based on these data, we have initiated a Phase 3 pivotal ARCHER II trial to further evaluate the potential of ANX007 to be the first treatment to protect vision in GA,” said Douglas Love, president and chief executive officer of Annexon. “We look forward to sharing these findings as part of podium presentations at the upcoming annual meetings for the Retina Society and Euretina Congress in Europe, and hosting a symposium with leading global experts to discuss the mechanism of action and significant opportunity for ANX007 to transform the GA treatment landscape.” Details of the presentations are as follows: Retina Society’s 57th Annual Scientific Meeting“Preservation of Photoreceptors and Prevention of Visual Acuity Loss by ANX007 in Geographic Atrophy (GA) in the Phase 2 ARCHER Trial” Session: Podium presentationPresenter: Dr. Glenn J. Jaffe, Duke Department of Ophthalmology, North Carolina, USDate/Time: Saturday, September 14, 2024, 10:28 am CESTLocation: Four Seasons Hotel Ritz, Lisbon, Portugal 24th Annual Euretina Congress“Preservation of Vision by ANX007 in Geographic Atrophy: Clinical Results from the Phase 2 ARCHER Trial” Session: Podium presentationPresenter: Dr. Usha Chakravarthy, Royal Victoria Hospital (The Belfast Trust) and Queens University of Belfast, Northern IrelandDate/Time: Thursday, September 19, 2024, 4:30 pm CESTLocation: CCIB Barcelona, Spain “Effect of ANX007 on Protecting Central Macular Ellipsoid Zone and RPE and Association with Visual Acuity in the Phase 2 ARCHER GA Study” Session: E-poster presentationPresenter: Dr. Paulo Eduardo Stanga, The Retina Clinic London and Institute of Ophthalmology, University College London, UK Annexon Sponsored Symposium“C1q Driven Neurodegeneration in GA and Relationship to Structure” Dr. Jeff Heier, Ophthalmic Consultants of Boston, US and an investigator in ARCHER “ANX007: Visual Acuity Protection and Safety in the Phase 2 ARCHER Trial” Dr. Usha Chakravarthy, Royal Victoria Hospital (The Belfast Trust) and Queens University of Belfast, Northern Ireland “Linking Structure to Function: Protection of Vision Associated Structures with ANX007” Dr. Paulo Eduardo Stanga, The Retina Clinic London and Institute of Ophthalmology, University College London, UK Annexon Sponsored Symposium: “Protection of Vision and Structure in GA” Presenters: “C1q Driven Neurodegeneration in GA and Relationship to Structure” Dr. Jeff Heier, Ophthalmic Consultants of Boston, US and an investigator in ARCHER “ANX007: Visual Acuity Protection and Safety in the Phase 2 ARCHER Trial” Dr. Usha Chakravarthy, Royal Victoria Hospital (The Belfast Trust) and Queens University of Belfast, Northern Ireland “Linking Function to Structure: Protection of Vision-Associated Structures with ANX007” Dr. Paulo Eduardo Stanga, The Retina Clinic London and Institute of Ophthalmology, University College London, UK Date/Time: Saturday, September 21, 2024, 1:00 pm CESTLocation: CCIB Barcelona, Spain About ANX007 and Phase 2 ARCHER TrialANX007 is an antigen-binding fragment (Fab) antibody designed as a first-in-kind therapeutic to selectively inhibit C1q, the initiating molecule of the classical complement pathway and a key driver of neurodegeneration. In GA, C1q binds to photoreceptor synapses early in the disease process, causing aberrant activation of the classical pathway with synapse loss, inflammation and neuronal damage that results in vision loss. Intravitreal administration of ANX007 fully stops C1q and classical pathway activation. In animal models, the murine analog of ANX007 protects against loss of photoreceptor synapses and cells to preserve function. ANX007 has been granted Fast Track designation from the Food and Drug Administration and is the first therapeutic candidate for the treatment of GA to receive Priority Medicine (PRIME) designation in the EU, which provides early and proactive support to developers of promising medicines that may offer a major therapeutic advantage over existing treatments or benefit to patients without treatment options. In the randomized, multi-center, double-masked, sham-controlled Phase 2 ARCHER clinical trial, ANX007 demonstrated consistent protection against vision loss across multiple measures in a broad population of patients with GA. ANX007 provided statistically significant, time and dose-dependent protection from vision loss as measured by ≥ 15 letter loss on reading an eye chart with best corrected visual acuity (BCVA≥15), the widely accepted and clinically-meaningful functional endpoint. Significant protection from vision loss was also shown in other prespecified measures of BCVA and visual function, including low luminance visual acuity (LLVA) and low luminance visual deficit (LLVD). ANX007’s treatment effect increased over the course of the on-treatment portion of the study, suggesting that ANX007 may provide a growing and durable treatment effect over time. While benefit gained against vision loss was maintained during the subsequent six-month off-treatment period, the rate of decline for BCVA ≥ 15-letter vision after treatment termination began to parallel that of sham, providing additional support for the observed on-treatment protection. ANX007 was also shown to protect key retinal structures important for vision, including significant protection of photoreceptors as measured by optical coherence tomography (OCT) and supported by slowing of loss of retinal pigment epithelial cells (RPE) in the fovea, as measured by fundus autofluoresence (FAF). ANX007 was generally well-tolerated through month 12, with no increase in choroidal neovascularization (CNV) rates between the treated and sham arms and no events of retinal vasculitis reported. About Geographic AtrophyGeographic atrophy (GA) is an advanced form of dry age-related macular degeneration (AMD), an eye disease that is the leading cause of blindness in the elderly. GA is a chronic progressive neurodegenerative disorder of the retina involving the loss of photoreceptor synapses and cells in the outer retina. GA affects an estimated one million people in the United States and eight million people globally, severely limiting their independence and causing frustration, anxiety and emotional hardship. Effective treatments that preserve vision are still needed, as no currently approved therapies have been shown in clinical trials to significantly prevent vision loss. About AnnexonAnnexon 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 their potential treatments to patients as quickly as possible. To learn more visit annexonbio.com. Forward Looking StatementsThis 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,” “suggest,” “target,” “on track,” “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, the ability of ANX007 to block upstream C1q, the clinical and regulatory status of ANX007; ANX007’s distinct potential neuroprotective mechanism of action and potential to provide protection from vision loss; the potential therapeutic benefit of ANX007; and Annexon’s ability to rigorously advance mid- to late-stage clinical trials and continue development 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 Advisors, LLCjallaire@lifesciadvisors.com Media Contact:Sheryl SeapyReal Chemistry949-903-4750sseapy@realchemistry.com

BRISBANE, Calif., Aug. 29, 2024 (GLOBE NEWSWIRE) -- Annexon, Inc. (Nasdaq: ANNX), a biopharmaceutical company advancing novel complement therapies for neuroinflammatory diseases of the body, brain and eye, today announced that Douglas Love, Esq., president & chief executive officer, will participate in fireside chats during the following September investor conferences: Wells Fargo Healthcare Conference on Wednesday, September 4, 2024, at 9:30 a.m. ET in Boston, MA; and,Cantor Global Healthcare Conference on Tuesday, September 17, 2024 at 3:05 p.m. ET in New York, NY. Live webcasts of the presentations can be accessed under the ‘Events & Presentations’ section on the Investors page at www.annexonbio.com. Replays of the webcasts will be archived on the Annexon website for 30 days following the presentations. 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 their potential treatments to patients as quickly as possible. To learn more visit annexonbio.com. Investor Contact: Joyce AllaireLifeSci Advisorsjallaire@lifesciadvisors.com Media Contact: Sheryl SeapyReal Chemistry949-903-4750sseapy@realchemistry.com

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