Annexon Highlights Leadership in Advancing Clinical Research and Education for Guillain-Barré Syndrome (GBS) at American Academy of Neurology (AAN) 2025 Annual Meeting

Rhea-AI Summary

Annexon (ANNX) is showcasing its leadership in Guillain-Barré Syndrome (GBS) research at the American Academy of Neurology Annual Meeting 2025. The company will present Phase 3 trial data for ANX005, their potential first targeted therapy for GBS, during an oral plenary session on April 8.

Key highlights include an educational symposium on GBS care and the classical complement pathway, featuring expert presenters from leading medical institutions. Additionally, Annexon is launching 'Move GBS Forward,' a disease education campaign at Booth #2133 to promote awareness of GBS's impact and encourage prompt diagnosis.

GBS affects approximately 150,000 people worldwide annually and currently has no FDA-approved therapies. ANX005, a first-in-kind monoclonal antibody, is designed to block C1q with a single infusion to stop neuroinflammation and nerve damage during acute GBS phases.

Positive

• Phase 3 trial data shows rapid and durable benefits across clinical measures
• ANX005 could become the first FDA-approved targeted therapy for GBS
• Company advancing first-in-kind monoclonal antibody treatment

Negative

• No current FDA approval for ANX005
• GBS market to 150,000 patients worldwide annually

Insights

Biotechnology Analyst

Annexon's announcement represents a significant milestone in their clinical development program with positive Phase 3 data for ANX005 in Guillain-Barré Syndrome (GBS). This achievement is particularly noteworthy as it marks the first placebo-controlled Phase 3 trial in GBS showing "rapid and durable benefit across clinical measures and time points."

The strategic importance cannot be overstated—ANX005 is positioned as the first potential targeted therapy for GBS, a condition affecting approximately 150,000 people worldwide annually with no FDA-approved treatments. This creates a clear path to address an unmet medical need in a market with competition.

ANX005's mechanism of action—blocking C1q with a single infusion—offers a differentiated approach to halting neuroinflammation in acute GBS. The selection for an oral plenary presentation at AAN signals the scientific community's recognition of this data's importance.

For a company with a market cap of approximately $203 million, positive late-stage data for their lead asset represents a crucial value inflection point. With GBS categorized as a neuromuscular emergency often requiring intensive care, a therapy demonstrating improved recovery could provide significant clinical and economic value, potentially strengthening Annexon's commercial position if approved.

Neurologist

The Phase 3 results for ANX005 in Guillain-Barré Syndrome represent a potential paradigm shift in GBS management. Currently, treatment is to supportive care, plasmapheresis, or intravenous immunoglobulin—all non-specific approaches with variable efficacy.

ANX005's targeted mechanism inhibiting C1q addresses the root cause of neuroinflammation in GBS. The classical complement pathway plays a central role in nerve damage during the acute phase, making it a logical therapeutic target. The reported "rapid and durable benefit across clinical measures" suggests ANX005 may accelerate recovery and potentially reduce long-term disability.

The single-infusion administration offers practical advantages in acute care settings, potentially simplifying treatment protocols. GBS progression can be rapid and devastating—patients often deteriorate from normal functioning to requiring mechanical ventilation within days.

Annexon's disease education campaign "Move GBS Forward" appropriately highlights both physical and mental impacts of GBS, addressing an often-overlooked aspect of the condition. The inclusion of prominent neurologists from major academic centers in their educational symposium reinforces the clinical significance of their approach and helps establish Annexon as a leader in advancing GBS care beyond current standards.

Oral Plenary Presentation on Tuesday, April 8 Features Phase 3 Data for ANX005, the First Potential Targeted Therapy for GBS, Showing Rapid and Durable Benefit Across Clinical Measures and Time Points

Educational Symposium Focuses on Advancing GBS Care and Role of Classical Complement Pathway

New Disease Education Campaign “Move GBS Forward” Draws Attention to Life-altering Physical and Mental Impact of GBS

BRISBANE, Calif., April 03, 2025 (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 highlights the company’s leadership in advancing clinical research and education for GBS at the AAN Annual Meeting taking place April 5–9, 2025, in San Diego, California.

Highlighted activities at the conference include:

• An oral plenary session, part of the “Clinical Trials Plenary” session, for the first placebo-controlled Phase 3 trial in GBS, “A Phase 3 Study to Evaluate Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of ANX005 in Patients with Guillain-Barré Syndrome (GBS)”.
  • Presenter: Jeff Allen, MD, Professor of Neurology at the University of Minnesota
  • Program Number: PL5.006
  • Date/Time: Tuesday, April 8, 10:30-10:45 a.m. Pacific Time

• An educational symposium, “Advancing GBS Care: Latest Insights into the role of classical complement pathway in GBS,” where experts will discuss the risks associated with GBS, current state of patient care and need for new targeted treatments.
  • Presenters: Jeff Allen, MD, Professor of Neurology at the University of Minnesota; Avni Kapadia, MD, Assistant Professor of Neurology ​at Baylor College of Medicine​; and Nick Silvestri, MD, FAAN​, Professor of Neurology, Associate Dean​ at University of Buffalo
  • Date/Time: Tuesday, April 8, 6:00-7:00 p.m. Pacific Time
    
    
• At AAN (Booth #2133), Annexon is launching a new disease education campaign for healthcare professionals called “Move GBS Forward,” which is designed to advance awareness and understanding of the sudden and long-term impact of GBS so as to encourage prompt diagnosis and care.
  

GBS is a neuromuscular emergency that affects at least 150,000 people worldwide each year, with no FDA-approved therapies. This rare autoimmune disease is characterized by rapidly progressing and severe weakness that can lead to complete paralysis, often requiring intensive care and mechanical ventilation.

ANX005 is a first-in-kind monoclonal antibody designed to block C1q, the initiating molecule of the classical complement cascade, with a single infusion to halt ongoing neuroinflammation and nerve damage in the acute phase of GBS to improve and expedite overall recovery.

About Annexon

Annexon Biosciences (Nasdaq: ANNX) is developing therapeutics that stop classical complement-driven neurodegeneration as first-in-kind treatments for millions of people living with serious neuroinflammatory diseases of the body, brain and eye. Our novel scientific approach focuses on C1q, the initiating molecule of classical complement’s potent inflammatory pathway that when misdirected can lead to tissue damage and loss. By targeting C1q, our immunotherapies are designed to stop this neuroinflammatory cascade in disease before it starts. Our pipeline spans three diverse therapeutic areas – autoimmune, neurodegenerative and ophthalmic diseases – and includes targeted investigational drug candidates designed to address the unmet needs of over 8 million people worldwide. Annexon’s mission is to deliver game-changing therapies to patients so that they can live their best lives. 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 ability of ANX005 to block C1q activity within a single infusion; the potential therapeutic benefit of ANX005; 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 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 Allaire
LifeSci Advisors
jallaire@lifesciadvisors.com

Media Contact:

Sheryl Seapy
Real Chemistry
949-903-4750
sseapy@realchemistry.com

FAQ

What are the key findings from Annexon's Phase 3 trial for ANX005 in GBS treatment?

The Phase 3 trial data for ANX005 will be presented on April 8, 2025, at the AAN Annual Meeting, showing rapid and durable benefit across clinical measures and time points.

How many people does Guillain-Barré Syndrome (GBS) affect globally each year?

GBS affects at least 150,000 people worldwide annually, with no current FDA-approved therapies.

What is ANX005 and how does it work in treating GBS?

ANX005 is a first-in-kind monoclonal antibody designed to block C1q with a single infusion, aiming to halt neuroinflammation and nerve damage during acute GBS phases.

When and where is Annexon presenting their GBS research at AAN 2025?

Annexon is presenting at the AAN Annual Meeting in San Diego from April 5-9, 2025, with their key presentation scheduled for April 8 at 10:30 AM PT.