The primary endpoint of the Phase 3 study is GBS Disability Score at Week 8, and secondary endpoints include safety, duration of ventilation support, duration of ICU stays, muscle strength, mortality, and patient global impression of change scores. The primary endpoint will utilize a proportional odds methodology to assess the proportion of patients who shift to better outcomes on the GBS Disability Score with ANX005 treatment compared to placebo at week 8.
More information about the study is available at ClinicalTrials.gov (NCT Number: NCT04701164).
The company expects to report data from this pivotal study in the first half of 2024.
About Guillain-Barré Syndrome
GBS is a serious and urgent autoimmune condition of the nervous system that can lead to sudden paralysis and even death in otherwise healthy individuals. GBS is caused by antibodies generated in response to a seemingly routine illness, such as flu or diarrhea, that cross-react with components of a patient’s own peripheral nerves. After the infection resolves, the antibodies quietly continue to build until they trigger the immune protein C1q to unleash a powerful inflammatory response that damages peripheral nerves and nerve roots coming from the spinal cord. Within days of feeling normal, patients are sent to the emergency room with weakness or paralysis and, in 1 in 4 cases, need ventilator support with IVIg treatment because of severe muscle weakness that impacts their ability to breathe. GBS impacts approximately 12,000 people annually in the U.S. and EU, and there are currently no approved therapies for GBS in the U.S. The development of targeted treatments for GBS is crucial to improve outcomes and quality of life for those affected by this debilitating condition.
About ANX005
Annexon’s lead investigational therapy, ANX005, is a first-of-its kind selective, targeted and rapid-acting agent designed to reduce inflammation and nerve damage by fully stopping C1q activity in the peripheral and central nervous systems. In GBS, ANX005 seeks out C1q and selectively blocks it from binding to its target on nerves in the arms and legs, while allowing other complement system pathways and the rest of the immune system to function normally. ANX005 is administered intravenously and has been observed to act almost immediately – with the aim in GBS to rapidly stop the autoimmune damage of nerve cells, allowing the patient to regain their muscle strength more quickly with greater ability to return to pre-illness activities.
ANX005 is being evaluated in clinical trials for the treatment of GBS, Huntington’s disease and ALS. It has received both fast track and orphan drug designations from the FDA as well as orphan drug designation by the EMA for the treatment of GBS.
About Annexon
Annexon Biosciences (Nasdaq: ANNX) is a clinical-stage biopharmaceutical company utilizing a distinctive scientific approach to stop the initiator of classical complement-mediated inflammation, C1q, before it starts. As the only company solely focused on shutting down C1q, Annexon is developing a purposeful pipeline of investigational medicines designed to provide meaningful benefits across multiple diseases of the body, brain and eye. With proof-of concept data in both Guillain-Barré syndrome and geographic atrophy, Annexon is rigorously advancing mid-to late-stage clinical trials to bring first-of-their kind therapies to millions of people living with devastating inflammatory-related diseases. 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,” “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: ability of ANX005 to stop C1q activity; the timing of completion of Phase 3 trial of ANX005 in patients with GBS; the potential therapeutic benefit of ANX005 or any other product candidates on GBS or geographic atrophy; potential benefit of ANX005, if approved, compared to existing therapies; market size; plans to
