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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 longer-acting anti-C5 antibody. 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.

Eculizumab for Neuromyelitis Optica Spectrum Disorder (NMOSD)  |  See Clinical Trials

Alexion has completed enrollment in the PREVENT (Prevention of Relapses and EValuation of Eculizumab in NMO Treatment) study to evaluate the safety and efficacy of eculizumab as a potential treatment for patients with neuromyelitis optica spectrum disorder (NMOSD). NMOSD is a life-threatening, ultra-rare autoimmune neurological disorder in which complement activation by antibodies against aquaporin-4 on astrocyte cell surfaces causes damage in the central nervous system, including the spinal cord and optic nerve.1-3 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 for Paroxysmal Nocturnal Hemoglobinuria (PNH)  |  See Clinical Trials

Alexion is evaluating ALXN1210, a highly innovative, longer-acting anti-C5 antibody, in two Phase 3 trials 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). One study compares ALXN1210 administered intravenously every eight weeks to Soliris in complement inhibitor treatment-naive patients with PNH; the second is a switch study of ALXN1210 administered intravenously every eight weeks compared to patients currently treated with Soliris. Alexion has completed enrollment in both studies.

ALXN1210 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.

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
 

References:

  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.