Development Programs

Alexion’s development programs focus on four core therapeutic areas: hematology, nephrology, neurology, and metabolic disorders. We are leveraging our global leadership in complement biology to expand Soliris® into new indications within these areas, while driving continued innovation with ALXN1210, our investigational long-acting C5 inhibitor, WTX101 for Wilson disease and SYNT001 for rare IgG-mediated diseases. We are also seeking business development opportunities to strengthen our clinical-stage pipeline in the four core areas.

Learn more about our current development programs below.

Soliris® (eculizumab) for Neuromyelitis Optica Spectrum Disorder (NMOSD)  |  See Clinical Trials

Alexion is evaluating Soliris® (eculizumab) in neuromyelitis optica spectrum disorder (NMOSD), a rare devastating, complement-mediated disorder of the central nervous system (CNS). Alexion has completed the Phase 3 PREVENT (Prevention of Relapses and Evaluation of Eculizumab in NMO Treatment) study of Soliris® in patients with anti-aquaporin-4 (AQP4) auto antibody-positive NMOSD. Complement activation by anti-AQP4 auto-antibodies leads to destruction of vital cells in the CNS, causing demyelination and death of neurons, predominantly in the spinal cord and optic nerve. The disease leads to severe weakness, paralysis, respiratory failure, loss of bowel and bladder function, blindness and premature death. Most patients experience an unpredictable, relapsing course of disease where each individual attack adds to cumulative neurologic disability.4-8

ALXN1210 IV for Paroxysmal Nocturnal Hemoglobinuria (PNH)  |  See Clinical Trials

Alexion is evaluating ALXN1210, an investigational, long-acting C5 inhibitor, in patients with PNH, an ultra-rare blood disorder in which chronic, uncontrolled activation of complement, a component of the normal immune system, results in hemolysis (destruction of the patient's red blood cells). We have completed two Phase 3 studies in adults with PNH — one study in complement inhibitor treatment-naïve patients and one study in patients who were stable on Soliris®. A Phase 3 study in children and adolescents under the age of 18 who have PNH is currently underway.

ALXN1210 IV for Atypical Hemolytic Uremic Syndrome (aHUS)  |  See Clinical Trials

Alexion is evaluating ALXN1210 administered intravenously every eight weeks in a Phase 3 trial of complement inhibitor treatment-naive adolescent and adult patients with aHUS, a genetic, chronic, ultra-rare disease associated with vital organ failure and premature death. The company has also initiated a Phase 3 trial in pediatric patients with aHUS.

ALXN1210 Subcutaneous |  See Clinical Trials

Initial pharmacokinetic and tolerability data from a Phase I study in healthy volunteers support progressing the development of a subcutaneous formulation of ALXN1210. Based on discussions with regulators, Alexion plans to initiate a single, PK-based Phase 3 study of ALXN1210 delivered subcutaneously once per week to support registration in PNH and aHUS in late 2018. A Phase 1 study of ALXN1210 co-administered with Halozyme's ENHANZE® drug-delivery technology, PH20, is underway. Pending co-formulation data, this next-generation subcutaneous formulation will be called ALXN1810 and has the potential to further extend the dosing interval to once every two weeks or once per month.

WTX101 for Wilson Disease |  See Clinical Trials

Alexion is evaluating WTX101 (bis-choline tetrathiomolybdate) in a Phase 3 clinical trial for the treatment of Wilson disease, a rare genetic disorder with devastating hepatic and neurological consequences for patients. WTX101 is novel oral copper-protein binding agent with a unique mechanism of action and ability to access and bind to serum copper and promote its removal from the liver.

SYNT001 for Warm Autoimmune Hemolytic Anemia (WAIHA), Pemphigus Vulgaris (PV) or Pemphigus Foliaceus (PF)  |  See Clinical Trials

Alexion is evaluating SYNT001 in Phase 1b/2a studies in patients with warm autoimmune hemolytic anemia (WAIHA) and in patients with pemphigus vulgaris (PV) or pemphigus foliaceus (PF) and has demonstrated proof of mechanism showing rapid IgG reduction. SYNT001 is a humanized monoclonal antibody that inhibits the interaction of neonatal Fc receptor (FcRn) with Immunoglobulin G (IgG) and IgG immune complexes and has the potential to improve treatment in a number of rare IgG-mediated diseases.

Our Clinical Trials

Alexion is investigating new therapies that have the potential to transform patients' lives. Learn more about some of our key clinical studies.

Alexion Clinical Trials


  1. Takemoto SK, Zeevi A, Feng S, et al. National conference to assess antibody-mediated rejection in solid organ transplantation. Am J Transplant. 2004; 4(7):1033-41.
  2. Collins AB, Schneeberger EE, Pascual MA, et al. Complement activation in acute humoral renal allograft rejection: diagnostic significance of C4d deposits in peritubular capillaries. J Am Soc Nephrol. 1999;10(10):2208-14.
  3. Jarius S, Aboul-Enein, Waters P, et al. Antibody to AQP4 in the long term course of neuromyelitis optica. Brain. 2008;131:3072-80.
  4. Hinson SR, Romero MF, Popescu BFG, et al. Molecular outcomes of neuromyelitis optica (NMO)-IgG binding to aquaporin-4 in astrocytes. Proc Nat Acad Sci. 2012;109(4):1245-50.
  5. Hinson SR, Pittock SJ, Lucchinetti CF, et al. Pathogenic potential of IgG binding to water channel extracellular domain in neuromyelitis optica. Neurology. 2007;69:2221-31.
  6. Wingerchuk DM, Lennon VA, Lucchinetti CF, Pittock SJ, Weinshenker BG. The spectrum of neuromyelitis optica. Lancet Neuro. 2007;6(9):805-15.
  7. Wingerchuk DM. Diagnosis and treatment of neuromyelitis optica. Neurologis. 2007;13(1):2-11.
  8. Wingerchuk DM, Weinshenker BG. Neuromyelitis optica (Devics Syndrome). Chapter 26, Handbook of Clinical Neurology, Vol. 122 (3rd series) Multiple Sclerosis and Related Disorders, Elsevier, 2014.