BRISBANE, Calif. - Annexon, Inc. (NASDAQ: ANNX), a clinical-stage biopharmaceutical company, announced positive results from its Phase 3 trial of ANX005 in patients with Guillain-Barré syndrome (GBS), a rare neurological disorder. The trial met its primary endpoint, showing a significant improvement in the GBS disability scale at week 8 with a 2.4-fold increase versus placebo.
The study, which enrolled 241 subjects in Bangladesh and the Philippines, tested two dosage levels of ANX005, 30 mg/kg and 75 mg/kg. The 30 mg/kg dose demonstrated rapid and complete suppression of complement activity, leading to early and sustained improvements in muscle strength, nerve damage, and reduced need for artificial ventilation.
While the 75 mg/kg dose showed multiple benefits, it did not achieve statistical significance on the primary endpoint. Still, the 30 mg/kg dose was associated with a 31-day reduction in median time to walk independently and 28 fewer days on artificial ventilation through week 26 compared to placebo.
ANX005 also achieved a reduction in serum levels of neurofilament light chain, a biomarker of nerve damage. The safety profile of ANX005 was generally well-tolerated, with the majority of adverse events being mild to moderate.
GBS affects over 22,000 people annually in the U.S. and Europe and currently has no FDA-approved treatments. The economic impact on the U.S. healthcare system is significant, with costs running into billions annually.
The company plans to present detailed Phase 3 data at the Peripheral Nerve Society Annual Meeting on June 25, 2024, and expects to submit a Biologics License Application (BLA) in the first half of 2025. Real-world evidence comparability data is also anticipated in the first half of 2025.
ANX005 has received Fast Track and Orphan Drug Designations from the FDA and Orphan Drug Designation from the European Medicines Agency. This announcement is based on a press release statement from Annexon.
In other recent news, Annexon Biosciences has been the subject of analyst attention, with Wells Fargo adjusting its outlook on the company's shares. The firm lowered its stock price target from $12.00 to $10.00, while maintaining an Overweight rating on the stock. The adjustment follows an update from Annexon's management that the last patient visit for a 24-week endpoint trial has occurred, with a database lock expected soon.
Wells Fargo anticipates that the trial data will be available in late May to early June, coinciding with a presentation at the upcoming Peripheral Nerve Society meeting. The analyst suggests a probability of success for the trial greater than 50% and believes that even if the trial does not meet its primary endpoints, Annexon's ANX005 treatment might still be viable for approval.
The firm also previously reiterated its Overweight rating on Annexon shares, expressing confidence in the company's ANX005 program for the treatment of Guillain-Barre Syndrome. These recent developments underscore the significance of the forthcoming trial data and its potential impact on Annexon's future in the market.
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