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Annexon Biosciences' ANX005 Shows Promise in Guillain-Barré Syndrome Treatment

• Annexon Biosciences completed a pivotal Phase 3 trial for ANX005, an immunotherapy targeting C1q, showing statistically significant results on key endpoints for Guillain-Barré Syndrome (GBS). • ANX005 has received FDA fast track and orphan drug designations, with plans to file a biologics license application in the first half of 2025, potentially becoming the first FDA-approved treatment for GBS. • Unlike current treatments like IVIG, ANX005 is administered via a quick IV infusion, rapidly blocking C1q to halt the disease process, offering a potentially more effective approach. • Annexon is also exploring ANX005's potential in other complement-mediated diseases like geographic atrophy, Huntington’s disease, ALS, and lupus nephritis, highlighting its broad applicability.

Los Angeles Dodgers' first baseman Freddie Freeman's son's battle with Guillain-Barré syndrome (GBS) has brought increased attention to this rare neurological disorder, as Annexon Biosciences advances its lead candidate, ANX005, as a potential treatment.
GBS is a rare autoimmune condition where the immune system attacks the peripheral nervous system, leading to rapid onset of weakness and potential paralysis. According to the CDC, GBS impacts 3,000-6,000 people in the U.S. each year. Current treatments, like off-label intravenous immunoglobulin therapy (IVIG), have limited effectiveness, especially in severe cases.

ANX005: A Novel Approach

Annexon Biosciences has successfully completed a pivotal Phase 3 trial for ANX005, an immunotherapy designed to block C1q, a key molecule in the classical complement pathway associated with GBS. This approach differs from other complement programs by targeting upstream complement activity, localizing and activating complement or inflammation right on disease tissue.
According to Douglas Love, president and CEO of Annexon Biosciences, ANX005 is designed to block C1q immediately upon administration, quickly shutting down the disease process and allowing patients to move from a state of decline. A June Phase 3 readout showed statistically significant results on key endpoints, including the GBS disability scale.

Clinical and Commercial Implications

Unlike IVIG, which is administered over five days, ANX005 is given via a quick IV infusion. Annexon plans to file its biologics license application for ANX005 in the first half of 2025. If approved, ANX005 would be the first FDA-approved treatment for GBS.
Love noted the high compliance rate in treating GBS (90%) and the concentration of cases in a few large states and hospitals, allowing for an efficient commercial footprint.

Beyond GBS

ANX005 isn't the only product in Annexon’s pipeline targeting C1q. Other programs are in geographic atrophy and complement-mediated autoimmune diseases, as well as programs in Huntington’s disease, ALS and lupus nephritis.
Love stated that by targeting C1q, Annexon can pursue a wide range of indications not typically seen with other complement programs.
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Reference News

[1]
The Dodgers' World Series run brought attention to a rare disease. Annexon is stepping up ...
pharmavoice.com · Nov 19, 2024

Freddie Freeman's son's illness with Guillain-Barré syndrome raised awareness of the rare neurological disorder. Annexon...

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