Industry Council

Alexion

Alexion is a global biopharmaceutical company focused on serving patients and families affected by rare diseases through the discovery, development and commercialization of life-changing medicines. As the global leader in complement biology and inhibition for more than 20 years, Alexion has developed and commercializes two approved complement inhibitors to treat patients with paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS), as well as the first and only approved complement inhibitor to treat anti-acetylcholine receptor (AChR) antibody-positive generalized myasthenia gravis (gMG) and neuromyelitis optica spectrum disorder (NMOSD). Alexion also has two highly innovative enzyme replacement therapies for patients with life-threatening and ultra-rare metabolic disorders, hypophosphatasia (HPP) and lysosomal acid lipase deficiency (LAL-D). In addition, the company is developing several mid-to-late-stage therapies, including a copper-binding agent for Wilson disease, an anti-neonatal Fc receptor (FcRn) antibody for rare Immunoglobulin G (IgG)-mediated diseases and an oral Factor D inhibitor as well as several early-stage therapies, including one for light chain (AL) amyloidosis, a second anti-FcRn therapy, a second oral Factor D inhibitor and a third complement inhibitor. Alexion focuses its research efforts on novel molecules and targets in the complement cascade and its development efforts on the core therapeutic areas of hematology, nephrology, neurology, metabolic disorders and cardiology. Headquartered in Boston, Massachusetts, Alexion has offices around the globe and serves patients in more than 50 countries. Further information about Alexion can be found at: www.alexion.com.

About Genentech

  Mechanism of Action SA237 is a humanized anti-human IL-6R neutralizing monoclonal antibody that was designed by application of recycling antibody technology to the approved anti-IL6 receptor antibody, tocilizumab, which is currently marketed as a treatment for rheumatoid arthritis (RA), systemic juvenile idiopathic arthritis, polyarticular juvenile idiopathic arthritis and Castleman’s disease. The recycling antibody technology enabled SA237 to bind to IL-6 receptor multiple times and be slowly cleared from plasma, which is expected to contribute to improvement of patient’s convenience by once monthly dosing frequency. The longer plasma half-life of SA237 compared with tocilizumab was confirmed based on the result of non-clinical study and Phase 1 study in healthy volunteers.

Study Details This research is being conducted to evaluate the efficacy, safety, pharmacodynamic, pharmacokinetic and immunogenic profiles of a humanized anti-human IL-6R neutralizing monoclonal antibody (SA237) in patients with Neuromyelitis Optica (NMO) and Neuromyelitis Optica Spectrum Disorder (NMOSD). This research is being conducted in the US and Canada. Ninety (90) patients are going to participate in this research.

Eligible participants Inclusion Criteria: 1. NMO or NMOSD 2. Age 18 to 74 years, inclusive at the time of informed consent. Exclusion Criteria: 1. Pregnancy or lactation. 2. Evidence of other demyelinating disease or PML. 3. Known active infection (excluding fungal infections of nail beds or caries dentium) within 4 weeks prior to baseline. Contact information If you are interested in participating, please contact: Contact: Clinical trials information clinical-trials@chugai-pharm.co.jp