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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 Refractory Generalized Myasthenia Gravis (gMG)  |  See Clinical Trials

Alexion has submitted marketing applications in the U.S., EU, and Japan for Soliris for the treatment of patients with refractory gMG who are anti-acetylcholine receptor (AChR) antibody-positive. These patients represent an ultra-rare segment of patients with MG—a debilitating, complement-mediated neuromuscular disease—who continue to suffer from severe disease symptoms and complications despite available treatment options. Patients with refractory gMG may have difficulty walking, talking, swallowing and breathing normally. Exacerbations and crises of their disease may require hospitalization and intensive care, and may be life-threatening.1-10

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

Alexion is enrolling patients 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.11-13 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.14-18

Eculizumab for Antibody-Mediated Rejection (AMR)  |  See Clinical Trials

Alexion is investigating eculizumab in patients receiving kidney transplants who are at elevated risk of acute antibody-mediated rejection (AMR), a severe and potentially life-threatening condition that can lead to severe kidney allograft damage, resulting in rapid loss of function and possible loss of the transplanted kidney.19 Research suggests that uncontrolled complement activation, triggered by the binding of donor-specific antibodies (DSAs) to target proteins in the donor kidney, may be the primary cause of acute AMR in kidney transplant recipients.19,20 Currently, there are no approved therapies for the prevention or treatment of AMR.

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.

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 also plans to initiate a Phase 3 trial in pediatric patients with aHUS.

ALXN1210 Subcutaneous  |  See Clinical Trials

Alexion is in discussions with regulators to progress the development of a subcutaneous formulation of ALXN1210, based on initial pharmacokinetic and tolerability data from a Phase I study in healthy volunteers.

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. Carr A, Cardwell C, McCarron P, et al. A systemic review of population based epidemiological studies in Myasthenia Gravis. BMC Neurology. 2010, 10:46.
  2. Silvestri N, Wolfe G. Treatment-refractory myathenia gravis. J. Clin Neuromuscul Dis. 2014;15(4):167-178.
  3. Regulation (EU) No 536/2014 of the European Parliament and of the Council of 16 April 2014 on clinical trials on medicinal products for human use, and repealing Directive 2001/20/EC. http://eur-lex.europa.eu/legal-content/EN/TXT/PDF/?uri=CELEX:32000R0141&qid=1421232987002&from=EN.
  4. Huda R, Tüzün E, Christadoss P. Targeting complement system to treat myasthenia gravis Rev. Neurosci. 2014; 25(4): 575–583.
  5. Howard JF, Barohn RJ, Cutter GR, et al. A randomized, double-blind, placebo-controlled phase II study of eculizumab in patients with refractory generalized myasthenia gravis. Muscle Nerve. 2013;48(1):76-84.
  6. Meriggioli MN, Sanders DB. Autoimmune myasthenia gravis: emerging clinical and biological heterogeneity. Lancet Neurol. 2009-8(5): 475-490.
  7. Howard J. Targeting the Complement System in Refractory Myasthenia Gravis. Supplement to Neurology Reviews. February 2016.
  8. National Institute of Neurological Disorders and Stroke. Myasthenia Gravis Fact Sheet. Last modified May 10, 2016. http://www.ninds.nih.gov/disorders/myasthenia_gravis/detail_myasthenia_gravis.htm. Accessed May 31, 2016.4
  9. Sathasivam S. Diagnosis and management of myasthenia gravis. Progress in Neurology and Psychiatry. January/February 2014.
  10. Howard JF. Myasthenia gravis: A manual for the healthcare provider. Myasthenia Gravis Foundation of America, Inc. 2008.
  11. 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.
  12. 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.
  13. Jarius S, Aboul-Enein, Waters P, et al. Antibody to AQP4 in the long term course of neuromyelitis optica. Brain. 2008;131:3072-80.
  14. 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.
  15. 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.
  16. Wingerchuk DM, Lennon VA, Lucchinetti CF, Pittock SJ, Weinshenker BG. The spectrum of neuromyelitis optica. Lancet Neuro. 2007;6(9):805-15.
  17. Wingerchuk DM. Diagnosis and treatment of neuromyelitis optica. Neurologis. 2007;13(1):2-11.
  18. 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.
  19. Tuzun E, Kurtuncu M, Turkoglu R, et al. Enhanced complement consumption in neuromyelitis optica and Behcet’s disease patients. J Neuroimmunol 2011;233(1-2):211-5.
  20. Kuroda H, Fujihara K, Takano R, et al. Increase of complement fragment C5a in cerebrospinal fluid during exacerbation of neuromyelitis optica. J Neuroimmunol 2013;254(1-2):178-82.