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Exploring the Promising Research Pipeline of Alexion Therapeutics



ALXN1210 for aHUS

Alexion Pharmaceuticals (ALXN) is conducting two Phase 3 clinical studies in patients suffering from aHUS (atypical hemolytic uremic syndrome), which leads to life-threatening damage to the kidney, brain, heart, and other critical organs.

These Phase 3 studies are ongoing and are continuing to enroll. They are separately evaluating the safety and efficacy of ALXN1210 administered by intravenous infusion every eight weeks in adolescent, adult, and pediatric patients with aHUS who have never been treated with a complement inhibitor.

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ALXN1210 for subcutaneous delivery

Alexion plans to begin a Phase 3 study of a subcutaneous formulation of ALXN1210 in late 2018. This plan entails a once-per-week dosing to PNH (paroxysmal nocturnal hemoglobinuria) patients to support registration in PNH as well as aHUS.

Data from a previous Phase 1 study of healthy volunteers for evaluating ALXN1210 supports progressing the development of the subcutaneous formulation of the drug. Notably, the FDA granted orphan drug designation to ALXN1210 in a subcutaneous formulation for treating aHUS in October 2017.


Alexion Pharmaceuticals is evaluating Soliris, or eculizumab, as a treatment for patients with relapsing neuromyelitis optica spectrum disorder (or NMOSD).

Relapsing NMOSD is a severe and ultra-rare autoimmune disease of the central nervous system that mainly affects the optic nerves and spinal cord. The drug has received orphan drug designation from the FDA. Alexion has completed patient enrolment, and dosing is ongoing in a global clinical trial of Soliris for treating patients with relapsing NMOSD.


Alexion Pharmaceuticals’ ALXN6000 or Samalizumab is a first-in-class immunomodulatory humanized monoclonal antibody. The drug blocks CD200, which is a key immune checkpoint protein expressed in hematologic and solid malignancies. Currently, Alexion is pursuing out-licensing opportunities for ALXN6000.

In the next article, we’ll take a look at Alexion Pharmaceuticals’ financial performance.


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