1. BRISBANE, Calif., Jan. 20, 2022 (GLOBE NEWSWIRE) -- Annexon, Inc. ("Annexon") (NASDAQ:ANNX), a clinical-stage biopharmaceutical company developing a new class of complement medicines for patients with classical complement-mediated autoimmune, neurodegenerative, and ophthalmic disorders, today announced the appointment of biotech executive Bettina M. Cockroft, M.D., senior vice president and chief medical officer of Sangamo Therapeutics, Inc., to the company's board of directors.

    "We are very pleased to welcome Bettina to the Annexon board of directors, as she brings extensive experience leading clinical organizations with a focus on neurodegenerative and movement disorders," said Douglas Love, Esq., president and chief executive officer…

    BRISBANE, Calif., Jan. 20, 2022 (GLOBE NEWSWIRE) -- Annexon, Inc. ("Annexon") (NASDAQ:ANNX), a clinical-stage biopharmaceutical company developing a new class of complement medicines for patients with classical complement-mediated autoimmune, neurodegenerative, and ophthalmic disorders, today announced the appointment of biotech executive Bettina M. Cockroft, M.D., senior vice president and chief medical officer of Sangamo Therapeutics, Inc., to the company's board of directors.

    "We are very pleased to welcome Bettina to the Annexon board of directors, as she brings extensive experience leading clinical organizations with a focus on neurodegenerative and movement disorders," said Douglas Love, Esq., president and chief executive officer of Annexon. "This is an exciting period for Annexon, and Bettina's expertise will be invaluable as we work toward advancing our novel complement-focused therapies for patients in need."

    "Annexon has built an impressive pipeline of therapies for patients with classical compliment-mediated disorders in the body, brain, and eye," said Dr. Cockroft. "The data generated across the pipeline to-date, including in two very challenging indications like Guillain–Barré Syndrome (GBS) and Huntington's Disease (HD), is promising, and with multiple data readouts anticipated over the next two years, I am excited to partner with the board and senior management team to bring forward important therapies that address complement-mediated diseases."

    Dr. Cockroft joins the Annexon board of directors with nearly 20 years of experience in senior management roles in the biopharmaceutical industry and has worked across multiple therapeutic areas and led programs in several countries. Dr. Cockroft has served as senior vice president and chief medical officer of Sangamo since September 2019 and oversees all clinical development activities and operations. Prior to Sangamo, Dr. Cockroft served on the senior leadership team at Cytokinetics, Inc., where she was responsible for clinical development of fast skeletal muscle troponin activators in diseases such as amyotrophic lateral sclerosis and spinal muscular atrophy. Before that, Dr. Cockroft served as chief medical officer of Auris Medical AG, where she led and grew the clinical development team responsible for two Phase 3 programs. Dr. Cockroft also held roles of increasing responsibility at Merck Serono S.A., Novartis Consumer Health and Menarini Ricerche earlier in her career. Dr. Cockroft earned her MBA at MIT Sloan School of Management and her M.D. from the University of Genova.

    About Annexon

    Annexon (NASDAQ:ANNX) is a clinical-stage biopharmaceutical company developing a new class of complement medicines for patients with classical complement-mediated autoimmune, neurodegenerative, and ophthalmic disorders of the body, brain, and eye. The company's pipeline is based on its platform technology addressing a broad spectrum of well-researched classical complement-mediated autoimmune and neurodegenerative diseases triggered by aberrant activation of C1q, the initiating molecule of the classical complement pathway. Annexon is advancing a portfolio of innovative product candidates designed to block the activity of C1q and the entire classical complement pathway: ANX005 (intravenous administration), ANX007 (intravitreal administration), and ANX009 (subcutaneous administration). Annexon is deploying a disciplined, biomarker-driven strategy designed to improve the probability of technical success of its portfolio. For more information, visit www.annexonbio.com.

    Contacts:

    Investors:

    Chelcie Lister

    THRUST Strategic Communications

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    Sheryl Seapy

    Real Chemistry

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  2. ANX005 Has Been Generally Well-Tolerated and Has Shown Full Target Engagement of C1q in the CSF

    Improvements in Clinical Outcome Measures Observed in Greater than 50% of All Evaluable Patients and 75% of Evaluable Patients with Excess Baseline Complement Activity

    NfL Levels Remained Consistent and Were Comparable to Published Natural History Data for HD Patients

    Phase 2 Trial Ongoing with Full Data Expected in the Second Quarter of 2022

    Company to Host Conference Call Today at 4:30 p.m. ET

    BRISBANE, Calif., Jan. 04, 2022 (GLOBE NEWSWIRE) -- Annexon, Inc. (NASDAQ:ANNX), a clinical-stage biopharmaceutical company developing medicines that stop destructive immune activity in complement-mediated autoimmune, neurodegenerative and ophthalmic…

    ANX005 Has Been Generally Well-Tolerated and Has Shown Full Target Engagement of C1q in the CSF

    Improvements in Clinical Outcome Measures Observed in Greater than 50% of All Evaluable Patients and 75% of Evaluable Patients with Excess Baseline Complement Activity

    NfL Levels Remained Consistent and Were Comparable to Published Natural History Data for HD Patients

    Phase 2 Trial Ongoing with Full Data Expected in the Second Quarter of 2022

    Company to Host Conference Call Today at 4:30 p.m. ET

    BRISBANE, Calif., Jan. 04, 2022 (GLOBE NEWSWIRE) -- Annexon, Inc. (NASDAQ:ANNX), a clinical-stage biopharmaceutical company developing medicines that stop destructive immune activity in complement-mediated autoimmune, neurodegenerative and ophthalmic disorders, today announced interim data from its ongoing, open-label Phase 2 clinical trial of ANX005 in patients with Huntington's disease (HD) who completed the 24-week treatment period. Annexon is developing ANX005, its lead monoclonal antibody candidate, for the treatment of a range of complement-mediated disorders, including HD.

    HD is a fatal, progressive movement disorder involving the activation of the classical complement pathway. C1q, the initiator of the classical pathway, is recognized as a major driver of a destructive immune response that leads to synapse loss and neurodegeneration. ANX005 is designed to disrupt the disease course, stopping the start of damaging complement activation by blocking C1q and the entire classical complement pathway.

    Interim data from the ongoing Phase 2 trial show that treatment with ANX005 has been generally well-tolerated, with full target engagement of C1q in both serum and cerebrospinal fluid (CSF) observed in evaluable patients through the dosing period. Evaluable patients maintained clinical function, as measured by changes in mean Composite Unified Huntington's Disease Rating Scale (cUHDRS), relative to baseline after six months of treatment, and improvement in cUHDRS was observed in more than half of all evaluable patients and in 75% of evaluable patients who showed excess complement activity at baseline. NfL levels observed after six months of treatment remained generally consistent and were comparable to NfL levels described in published natural history data for HD patients. Overall, these interim findings appear to build on the scientific hypothesis of Annexon scientific founder, the late Ben Barres, who believed that blocking C1q protects synaptic loss and can lead to rapid functional impact on clinical outcomes in neurodegenerative diseases.

    "People with HD face a devastating condition, with no cure or approved disease-modifying treatments available," said Edward Wild, FRCP, Ph.D., consultant neurologist, NHNN Queen Square and associate director, UCL Huntington's Disease Centre. "I believe the interim data from this open-label trial of ANX005 are encouraging, showing complete CSF target engagement and that ANX005 has been generally well-tolerated, with no concern regarding the NfL levels seen in this early readout. The apparent stabilization of cUHDRS observed relative to normal disease progression, together with the potential improvement seen in patients with elevated baseline C4a, supports the hypothesis that protecting synapses via C1q inhibition could produce meaningful functional benefit in HD, and justifies the continued development of ANX005 for this indication."

    ANX005 Interim Safety and Target Engagement Data

    ANX005 has been generally well-tolerated in the study (n=28). As of the data cutoff date of October 17, 2021, the most common adverse events (AEs) reported were first dose-associated infusion-related reactions, including transient skin rash, consistent with the experience observed in the company's Phase 1b trial of ANX005 in patients with Guillain-Barré Syndrome (GBS). In the HD trial, five patients discontinued ANX005 treatment, three of whom discontinued due to a drug-related AE. Two patients experienced a drug-related serious adverse event, including one event of systemic lupus erythematosus (mucocutaneous), whose symptoms resolved post-study drug discontinuation, and one event of idiopathic pneumonitis, which stabilized post-study drug discontinuation. Of note, no cases of serious infection were identified, and no deaths were reported.

    Interim data show that treatment with ANX005 has led to full target engagement of C1q in both serum and CSF through the dosing period in patients who were evaluable as of the cutoff date of October 17, 2021 (n=13).

    ANX005 Interim Efficacy and Biomarker Data

    Patients evaluable as of the cutoff date of December 14, 2021 (n=23) experienced improvements in clinical measures, assessed by cUHDRS, a clinical rating scale with four domains measuring the progression of HD consisting of motor, cognitive and functional capacity. Overall, patients maintained clinical function relative to baseline in cUHDRS after six months of treatment. Published natural history data show that HD patients typically experienced a decline of approximately 1 point over one year1, or 0.5 points over six months. Additionally, as of the cutoff date, 56% of patients showed improvement from baseline in cUHDRS and several subdomains of cUHDRS over six months of treatment. Moreover, in a sub-analysis of patients assessed according to baseline complement activity (C4a), 75% of patients with excess baseline complement activity demonstrated a statistical improvement in cUHDRS over six months of treatment versus 36% with low baseline complement activity, consistent with the scientific hypothesis of rapid response to anti-C1q therapy via enhanced synapse function. Elevated C4a is an objective measurement of excess complement activity in CSF that has been found to correlate with disease progression and multiple clinical endpoints in HD.2 These findings suggest that patients with excess complement activity may be more likely to respond to anti-C1q therapy in future clinical trials and may inform future trial designs.

    NfL, a protein component of the neuronal cytoskeleton, has been shown to increase in the CSF with disease severity in HD patients. Interim data assessing NfL from patients evaluable as of the cutoff date of October 17, 2021 (n=16) who completed 24 weeks of treatment showed that mean plasma and CSF NfL levels remained generally consistent and were comparable to NfL levels described in published natural history data for HD patients.3 Published data suggest that in slowly progressive neurodegenerative diseases like HD, synapse loss is associated with progressive functional decline, preceding the loss of neurons4 and NfL changes. As such, changes in NfL may require treatment durations longer than six months and Annexon will continue to assess NfL levels in patients over the three-month follow-up period.

            

    "We are quite encouraged by the interim data generated with ANX005 in HD. We are particularly excited to see a heightened clinical response in patients with excess baseline complement activity, suggesting that the classical complement pathway plays a key role in the neurodegenerative disease process and that ANX005 has the potential to provide meaningful benefit to HD patients," remarked Douglas Love, Esq., president and chief executive officer of Annexon. "These early data in HD patients, coupled with prior proof-of-concept data in GBS, provide a growing body of evidence for the potential role of anti-C1q in treating complement mediated neurodegenerative and autoimmune diseases, and we look forward to continuing to assess the full potential of our approach in several ongoing trials in diseases of high unmet need."

    The Phase 2 trial remains ongoing, and Annexon anticipates reporting full data from all patients enrolled, including data from the three-month follow-up period, in the second quarter of 2022. Pending results from the full dataset, Annexon plans to evaluate the opportunity for a Phase 3 trial of ANX005 in HD patients.

    Conference Call Information

    Annexon management will host a conference call today at 4:30 p.m. ET. To participate in the conference call, please dial (833) 649-1187 (domestic) or (281) 206-0036 (international) and refer to conference ID 6297344. The webcast and accompanying slides can be accessed under the ‘Events & Presentations' section on the Investors page at www.annexonbio.com. A replay of the webcast will be archived on the Annexon website for 30 days.

    About the Phase 2 Trial (ANX005-HD-01)

    The Phase 2 multi-center, open-label trial is evaluating ANX005 administered intravenously for a 24-week (six-month) dosing period in patients with, or at risk for, early manifest HD. The study enrolled a total of 28 patients in May of 2021, and 23 patients completed the 24-week treatment period. The interim data reported today include safety data for all 28 patients enrolled, efficacy data as measured by UHDRS in all 23 evaluable patients, pharmacokinetics (PK) and pharmacodynamics (PD) data for the first 13 patients, and NfL for the first 16 patients who completed the 24-week treatment period based on cutoff dates required to obtain data for this interim analysis.

    About Annexon

    Annexon (NASDAQ:ANNX) is a clinical-stage biopharmaceutical company developing a new class of complement medicines for patients with classical complement-mediated autoimmune, neurodegenerative, and ophthalmic disorders of the body, brain and eye. The company's pipeline is based on its platform technology addressing a broad spectrum of well-researched classical complement-mediated autoimmune and neurodegenerative diseases triggered by aberrant activation of C1q, the initiating molecule of the classical complement pathway. Annexon is advancing a portfolio of innovative product candidates designed to block the activity of C1q and the entire classical complement pathway: ANX005 (intravenous administration), ANX007 (intravitreal administration), and ANX009 (subcutaneous administration). Annexon is deploying a disciplined, biomarker-driven strategy designed to improve the probability of technical success of its portfolio. For more information, visit www.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," "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, statements about: initial findings and observations related to the interim data from the company's ongoing, open-label Phase 2 clinical trial of ANX005 in patients with HD; the potential benefits from treatment with anti-C1q therapy; timing of full data from the Phase 2 trial of ANX005 in HD patients; plans to evaluate the opportunity for a Phase 3 trial of ANX005 in HD patients; and continuing advancement of the company's innovative 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 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 COVID-19 or other 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:

    Chelcie Lister

    THRUST Strategic Communications

    Media Contact:

    Sheryl Seapy

    Real Chemistry

    949-903-4750

    1 Schobel 2017

    2 Suri, et al., HSG conference 2021

    3 Tabrizi 2019 and Rodrigues 2020

    4 Delva, et al., Neurology, 2021; Albin, et al., Ann Neurol, 1991; Gomez-Tortosa, Ann Neurol, 1991



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  3. BRISBANE, Calif., Jan. 04, 2022 (GLOBE NEWSWIRE) -- Annexon, Inc. (NASDAQ:ANNX), a clinical-stage biopharmaceutical company developing a new class of complement medicines for patients with classical complement-mediated autoimmune, neurodegenerative and ophthalmic disorders, today announced that Douglas Love, Esq., President & Chief Executive Officer, will present at the 40th Annual J.P. Morgan Healthcare Conference on Tuesday, January 11, 2022, at 3:45 p.m. ET.

    A live webcast of the event can be accessed under the ‘Events & Presentations' section on the Investors page at www.annexonbio.com. A replay of the webcast will be archived on the Annexon website for 30 days following the presentation.

    About Annexon

    Annexon (NASDAQ:ANNX) is a clinical-stage…

    BRISBANE, Calif., Jan. 04, 2022 (GLOBE NEWSWIRE) -- Annexon, Inc. (NASDAQ:ANNX), a clinical-stage biopharmaceutical company developing a new class of complement medicines for patients with classical complement-mediated autoimmune, neurodegenerative and ophthalmic disorders, today announced that Douglas Love, Esq., President & Chief Executive Officer, will present at the 40th Annual J.P. Morgan Healthcare Conference on Tuesday, January 11, 2022, at 3:45 p.m. ET.

    A live webcast of the event can be accessed under the ‘Events & Presentations' section on the Investors page at www.annexonbio.com. A replay of the webcast will be archived on the Annexon website for 30 days following the presentation.

    About Annexon

    Annexon (NASDAQ:ANNX) is a clinical-stage biopharmaceutical company developing a new class of complement medicines for patients with classical complement-mediated autoimmune, neurodegenerative, and ophthalmic disorders of the body, brain, and eye. The company's pipeline is based on its platform technology addressing a broad spectrum of well-researched classical complement-mediated autoimmune and neurodegenerative diseases triggered by aberrant activation of C1q, the initiating molecule of the classical complement pathway. Annexon is advancing a portfolio of innovative product candidates designed to block the activity of C1q and the entire classical complement pathway: ANX005 (intravenous administration), ANX007 (intravitreal administration), and ANX009 (subcutaneous administration). Annexon is deploying a disciplined, biomarker-driven strategy designed to improve the probability of technical success of its portfolio. For more information, visit www.annexonbio.com.

    Contact:

    Chelcie Lister

    THRUST Strategic Communications



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  4. BRISBANE, Calif., Dec. 13, 2021 (GLOBE NEWSWIRE) -- Annexon, Inc. (NASDAQ:ANNX), a clinical-stage biopharmaceutical company developing a new class of complement medicines for patients with classical complement-mediated autoimmune, neurodegenerative and ophthalmic disorders, announced safety and dose-response data from its Phase 1 clinical trial of ANX009, the company's subcutaneously administered product candidate that is designed to block the activity of C1q and the entire classical complement pathway. In addition, Annexon reported preclinical data supporting the role of the complement pathway in warm autoimmune hemolytic anemia (wAIHA). The data were presented during two poster sessions at the 63rd American Society of Hematology (ASH) Annual…

    BRISBANE, Calif., Dec. 13, 2021 (GLOBE NEWSWIRE) -- Annexon, Inc. (NASDAQ:ANNX), a clinical-stage biopharmaceutical company developing a new class of complement medicines for patients with classical complement-mediated autoimmune, neurodegenerative and ophthalmic disorders, announced safety and dose-response data from its Phase 1 clinical trial of ANX009, the company's subcutaneously administered product candidate that is designed to block the activity of C1q and the entire classical complement pathway. In addition, Annexon reported preclinical data supporting the role of the complement pathway in warm autoimmune hemolytic anemia (wAIHA). The data were presented during two poster sessions at the 63rd American Society of Hematology (ASH) Annual Meeting & Exposition.

    "We are pleased to present these data at ASH, which further support our approach of targeting C1q in order to fully block the downstream components of the complement pathway," said Sanjay Keswani, MBBS, FRCP, executive vice president and chief medical officer of Annexon. "ANX009 was shown to be well-tolerated with complete and sustained C1q inhibition, supporting its continued clinical advancement. In addition, as we continue to progress our clinical program with ANX005 for the treatment of wAIHA, these in vitro analyses provide important insights into the role that complement activation plays in wAIHA and the potential to enrich for patients most likely to respond to anti-C1q therapy in our clinical studies."

    Poster Title: Safety, Tolerability, and Clinical Pharmacology of ANX009, an Inhibitory Antibody Fab Fragment Against C1q, Administered Subcutaneously to Healthy Volunteers (3166)

    Data Summary: ANX009 is an antigen-binding fragment (Fab) that disrupts autoantibody complement activation through the inhibition of C1q. It is being developed as subcutaneously administered treatment for antibody-mediated autoimmune diseases of blood and vascular tissues. Data reported in the poster are from a single and multiple ascending dose Phase 1 trial of ANX009 in 48 healthy volunteers. Findings demonstrated that ANX009 was well-tolerated across all doses with no drug-related safety, dose-limiting toxicities, serious adverse events, or adverse events leading to discontinuations. Further, a dose-response was observed across dose cohorts with notable reductions in C1q in serum. Taken together, the findings support the clinical advancement of ANX009 in patients with complement-mediated autoimmune disorders.

    Poster Title: Evidence of Classical Complement Pathway Involvement in a Subset of Patients with Warm Autoimmune Hemolytic Anemia (2001)

    Data Summary: Autoimmune hemolytic anemia (AIHA) is a constellation of diseases caused by autoantibodies targeting red blood cells (RBCs) with or without complement activation, with the two main types being cold agglutinin disease (CAD) and wAIHA. To understand additional methods to suggest evidence of complement activation in AIHA patients, Annexon conducted a series of in vitro studies using patient blood samples. Findings from a modified in vitro complement deposition assay suggest that sera from CAD patients and a subset of wAIHA patients possess autoantibodies capable of triggering classical pathway C4 deposition on the surface of healthy human RBC. The company believes multiple factors may affect complement deposition in this assay and is assessing how these and other in vitro assay results may be translated to demonstrate classical complement pathway involvement in AIHA patients in vivo in an ongoing Phase 0 non-interventional trial. Annexon is also currently evaluating ANX005 in a Phase 2 clinical trial for the treatment of wAIHA patients with evidence of classical complement activity.

    About Annexon

    Annexon (NASDAQ:ANNX) is a clinical-stage biopharmaceutical company developing a new class of complement medicines for patients with classical complement-mediated autoimmune, neurodegenerative, and ophthalmic disorders of the body, brain, and eye. The company's pipeline is based on its platform technology addressing a broad spectrum of well-researched classical complement-mediated autoimmune and neurodegenerative diseases triggered by aberrant activation of C1q, the initiating molecule of the classical complement pathway. Annexon is advancing a portfolio of innovative product candidates designed to block the activity of C1q and the entire classical complement pathway: ANX005 (intravenous administration), ANX007 (intravitreal administration), and ANX009 (subcutaneous administration). Annexon is deploying a disciplined, biomarker-driven strategy designed to improve the probability of technical success of its portfolio. For more information, visit www.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," "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, statements about: continuing advancement of the company's innovative portfolio; timing of data from clinical trials; timing of completion of clinical studies and clinical development milestones; the company's ability to deliver on its objectives; the implementation of the company's business model and strategic plans for its business and product candidates, including potential treatment indications and additional indications that the company may pursue. 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 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 COVID-19 or other 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.

    Contact:

    Chelcie Lister

    THRUST Strategic Communications



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  5. SOUTH SAN FRANCISCO, Calif., Nov. 09, 2021 (GLOBE NEWSWIRE) -- Annexon, Inc. (NASDAQ:ANNX), a clinical-stage biopharmaceutical company developing a new class of complement medicines for patients with classical complement-mediated autoimmune, neurodegenerative and ophthalmic disorders, today announced recent highlights and reported third quarter 2021 financial results.

    "The third quarter marks another quarter of meaningful progress with our portfolio, including effectively advancing three clinical-stage product candidates in five clinical trials," said Douglas Love, Esq., president and chief executive officer of Annexon. "We're also pleased to have been granted Orphan Drug designation by the FDA for ANX005, underscoring the Agency's recognition…

    SOUTH SAN FRANCISCO, Calif., Nov. 09, 2021 (GLOBE NEWSWIRE) -- Annexon, Inc. (NASDAQ:ANNX), a clinical-stage biopharmaceutical company developing a new class of complement medicines for patients with classical complement-mediated autoimmune, neurodegenerative and ophthalmic disorders, today announced recent highlights and reported third quarter 2021 financial results.

    "The third quarter marks another quarter of meaningful progress with our portfolio, including effectively advancing three clinical-stage product candidates in five clinical trials," said Douglas Love, Esq., president and chief executive officer of Annexon. "We're also pleased to have been granted Orphan Drug designation by the FDA for ANX005, underscoring the Agency's recognition of ANX005's potential as a novel therapy for the treatment of Huntington's disease. Additionally, in collaboration with multiple academic and physician key opinion leaders, we conducted two C1q Series R&D Events that highlighted the applicability of our platform and differentiated scientific approach targeting the classical complement cascade in a range of autoimmune and neurodegenerative diseases. The combination of a rich set of milestones, a strong balance sheet, and a talented team, lays the foundation for a transformational year ahead. We look forward to sharing the continued progress across our portfolio, including initial data from our Huntington's disease clinical trial, as we work to bring treatments to patients suffering from serious complement-mediated diseases."

    Program Highlights

    • Second C1q Series R&D Event Highlights Neurodegeneration Franchise: Annexon hosted its second C1q Series event, which spotlighted the company's neurodegeneration franchise and approach to treating complement-mediated neurodegenerative diseases. During the event, Annexon's leadership team and participating key opinion leaders shared their perspectives on:
      • The role of C1q and the classical complement pathway in neurodegenerative diseases, including Huntington's disease (HD) and amyotrophic lateral sclerosis (ALS);
      • Research on neurofilament light chain (NfL) and natural history data showing the association of a rise in NfL with clinical progression in both HD and ALS; and
      • Internal data demonstrating the ability to reduce NfL with ANX005, both clinically in Guillain-Barré syndrome (GBS), and preclinically in HD and ALS.
    • Initial Data from Phase 2 trial of ANX005 in HD Expected in the Fourth Quarter of 2021: Annexon is studying its lead compound, ANX005, a clinical-stage investigational monoclonal antibody intended to treat patients with complement-mediated disorders, in a Phase 2 trial in patients with HD. The company anticipates initial data from the trial in the fourth quarter of 2021, which is expected to include data from a subset of 16 patients who have completed the six-month treatment period, including safety and tolerability, C1q target engagement in cerebral spinal fluid and serum, and impact on NfL levels. The full dataset from all patients who complete both the six-month on-treatment and three-month off-treatment periods are expected to be reported in the first half of 2022.
    • ANX005 Granted Orphan Drug Designation for the Treatment of HD: The U.S. Food and Drug Administration (FDA) has granted Orphan Drug designation for ANX005 for the treatment of patients with HD. Orphan Drug designation provides orphan status to drugs or biologics that are intended to treat rare diseases or disorders affecting fewer than 200,000 people in the United States, and certain incentives to the product sponsor, including tax credits for qualified clinical trials and fee waivers. The designation confers eligibility for seven years of market exclusivity following FDA product approval.

    Business Highlights

    • Leadership Team Strengthened by Appointment of Ted Yednock, Ph.D., as Chief Innovation Officer and Larry Mattheakis, Ph.D., as Chief Scientific Officer: In the third quarter, Annexon appointed Ted Yednock, Ph.D., as chief innovation officer (CIO), and Larry Mattheakis, Ph.D., as chief scientific officer (CSO). Dr. Ted Yednock served as Annexon's CSO since 2013. As CIO, he will serve as chairman of the Scientific Advisory Board and continue to enhance and integrate Annexon's key scientific findings and collaborations. Dr. Larry Mattheakis brings decades of drug discovery and development expertise to the CSO role, supporting the advancement of Annexon's current and future pipeline.

    Third Quarter 2021 Financial Results

    • Cash and Operating Runway: Cash and cash equivalents and short-term investments were $271.4 million as of September 30, 2021. Annexon expects that its current cash position is sufficient to fund its operating plans into 2024.
    • Research and development (R&D) expenses: R&D expenses were $27.6 million for the quarter ended September 30, 2021, compared to $11.8 million for the quarter ended September 30, 2020.
    • General and administrative (G&A) expenses: G&A expenses were $8.1 million for the quarter ended September 30, 2021, compared to $3.8 million for the quarter ended September 30, 2020.
    • Net loss: Net loss was $35.6 million or $0.93 per share for the quarter ended September 30, 2021, compared to $15.6 million or $0.77 per share for the quarter ended September 30, 2020.

    About Annexon

    Annexon (NASDAQ:ANNX) is a clinical-stage biopharmaceutical company developing a new class of complement medicines for patients with classical complement-mediated autoimmune, neurodegenerative, and ophthalmic disorders of the body, brain, and eye. The company's pipeline is based on its platform technology addressing a broad spectrum of well-researched classical complement-mediated autoimmune and neurodegenerative diseases triggered by aberrant activation of C1q, the initiating molecule of the classical complement pathway. Annexon is advancing a portfolio of innovative product candidates designed to block the activity of C1q and the entire classical complement pathway: ANX005 (intravenous administration), ANX007 (intravitreal administration), and ANX009 (subcutaneous administration). Annexon is deploying a disciplined, biomarker-driven strategy designed to improve the probability of technical success of its portfolio. For more information, visit www.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," "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, statements about: continuing advancement of the company's innovative portfolio; timing of data from clinical trials; timing of completion of clinical studies and clinical development milestones; the company's ability to deliver on its objectives and the potential for a transformational year ahead; the implementation of the company's business model and strategic plans for its business and product candidates, including potential treatment indications and additional indications that the company may pursue; cash position sufficient to fund its operating plans into 2024; the benefits of Orphan Drug designation; and leadership team service. 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 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 COVID-19 or other 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.

    Contact:

    Chelcie Lister

    THRUST Strategic Communications

    ANNEXON, INC.

    Condensed Consolidated Statements of Operations

    (in thousands, except share and per share amounts)

    (unaudited)

      Three Months Ended

    September 30,
      Nine Months Ended

    September 30,
     
      2021  2020  2021  2020 
    Operating expenses:                
    Research and development (1) $27,581  $11,775  $72,849  $31,279 
    General and administrative (1)  8,099   3,810   20,406   8,999 
    Total operating expenses  35,680   15,585   93,255   40,278 
    Loss from operations  (35,680)  (15,585)  (93,255)  (40,278)
    Other income (expense), net  82   (52)  303   64 
    Net loss before taxes  (35,598)  (15,637)  (92,952)  (40,214)
    Provision for income taxes     1      5 
    Net loss  (35,598)  (15,638)  (92,952)  (40,219)
    Accretion on redeemable convertible preferred stock     (145)     (705)
    Deemed dividend – beneficial conversion feature on redeemable

    convertible preferred stock
         (6,219)     (6,219)
    Net loss attributable to common stockholders $(35,598) $(22,002) $(92,952) $(47,143)
    Net loss per share attributable to common stockholders,

    basic and diluted
     $(0.93) $(0.77) $(2.43) $(4.79)
    Weighted-average shares used in computing net loss per

    share attributable to common stockholders, basic and diluted
      38,341,110   28,465,156   38,261,359   9,845,754 
                     
                     
    (1) Includes the following stock-based compensation expense:                
    Research and development $2,382  $624  $6,330  $1,284 
    General and administrative $2,046  $847  $5,577  $1,613 
                     

    ANNEXON, INC.

    Condensed Consolidated Balance Sheets

    (in thousands)

      September 30,

    2021
      December 31,

    2020
     
      (Unaudited)     
    Assets        
    Current assets:        
    Cash and cash equivalents $68,519  $268,565 
    Short-term investments  202,848   82,641 
    Prepaid expenses and other current assets  4,131   2,805 
    Total current assets  275,498   354,011 
    Restricted cash  1,166    
    Property and equipment, net  12,219   1,935 
    Operating lease right-of-use assets  20,680    
    Other assets  593    
    Total assets $310,156  $355,946 
    Liabilities and Stockholders' Equity        
    Current liabilities:        
    Accounts payable $4,797  $3,734 
    Accrued liabilities  7,059   6,497 
    Deferred rent, current     391 
    Operating lease liabilities, current  63    
    Total current liabilities  11,919   10,622 
    Deferred rent     1,046 
    Operating lease liabilities, non-current  33,760    
    Total liabilities  45,679   11,668 
    Commitments and contingencies        
    Stockholders' equity:        
    Common stock  38   38 
    Additional paid-in capital  523,458   510,309 
    Accumulated other comprehensive loss  (75)  (77)
    Accumulated deficit  (258,944)  (165,992)
    Total stockholders' equity  264,477   344,278 
    Total liabilities and stockholders' equity $310,156  $355,946 



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