Neurology
The complex structures and mechanisms of the nervous system are vulnerable to the
inflammatory effects of terminal complement. Alexion's research and development
teams have designed a study to examine Soliris® (eculizumab)
as a treatment for patients with myasthenia gravis, a rare neurologic disorders.
Independent investigators are also examining Soliris as a treatment for patients
with a separate rare neurologic disorder, Multifocal Motor Neuropathy (MMN).
Myasthenia Gravis (MG)
MG is a rare autoimmune syndrome characterized by auto-antibodies that recognize
a specific target in the nerve-muscle junction causing tissue damage and interference
with signaling between nerve and muscle fibers.39 Patients with MG initially
experience weakness in their ocular, or eye muscles, and the disease typically progresses
to head, spinal, limb and respiratory muscles. Symptoms can include drooping eyelid,
blurred vision, slurred speech, difficulty chewing or swallowing, weakness in the
arms and legs and difficulty breathing.
A study, published in the Journal of Immunology in 2007, demonstrated the
pre-clinical effectiveness of anti-C5 therapy in blocking this process in an animal
model.40 Alexion is now screening patients for a Phase II clinical trial of Soliris to evaluate the potential
of complement inhibition for the treatment of patients with myasthenia gravis that
have few treatment options.
Multifocal Motor Neuropathy (MMN)
MMN is a rare autoimmune disorder in which auto-antibodies attack the nerve-muscle
junctions.41 Patients with MMN demonstrate a slow progressive asymmetrical
weakness of limbs without sensory loss.42 Antibodies and complement activation
products have been identified at the nerve-muscle junctions in diseases similar
to MMN.43
In data published in the January 2008 issue of the journal Brain, anti-C5
therapy was shown to be protective in animal models of MMN.44 In the
fourth quarter of 2008, an investigator-initiated clinical trial protocol was filed
with regulators in the United Kingdom to initiate studies of Soliris in patients
with MMN. Independent investigators have started to dose patients in this study.