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SOLIRIS, a first-in-class complement inhibitor, is approved for the treatment of adults with generalized myasthenia gravis (gMG). A Phase 3 study of SOLIRIS in children and adolescents who have gMG is underway.

SOLIRIS, a first-in-class complement inhibitor, is approved for the treatment of adults with anti-aquaporin-4 (AQP4) antibody-positive NMOSD. Alexion plans to initiate a Phase 2/3 study in children and adolescents with NMOSD in the second half of 2020.

SOLIRIS is a first-in-class complement inhibitor. In June 2020, Japan's Ministry of Health, Labour and Welfare granted SAKIGAKE designation for SOLIRIS in GBS. Alexion plans to initiate a Phase 3 study of SOLIRIS in GBS in Japan in 2021, pending regulatory feedback. 

ULTOMIRIS, a long-acting C5 inhibitor, is approved for the treatment of adults with PNH, a severe and 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). A Phase 3 study in children and adolescents who have PNH is underway.

ULTOMIRIS is a long-acting C5 inhibitor approved for the treatment of adults and pediatric patients one month of age and older with aHUS to inhibit complement-mediated thrombotic microangiopathy (TMA). Atypical HUS is a chronic, ultra-rare, and life-threatening disease. A Phase 3 study in children and adolescents who have aHUS is underway. 

ULTOMIRIS is a long-acting C5 inhibitor. In June 2020, Alexion announced that the Phase 3 study of weekly subcutaneous (SC) ULTOMIRIS demonstrated PK-based non-inferiority versus intravenous ULTOMIRIS. Pending collection of 12-month safety and drug-device combination data, Alexion plans to file for approval in the U.S. and EU for the ULTOMIRIS SC formulation and device combination in PNH and aHUS in the third quarter of 2021.

ULTOMIRIS is a long-acting C5 inhibitor. A Phase 3 study of ULTOMIRIS in adults who have gMG is underway.

ULTOMIRIS is a long-acting C5 inhibitor. A Phase 3 study of ULTOMIRIS in NMOSD is underway.

ULTOMIRIS is a long-acting C5 inhibitor. Alexion plans to initiate limited dose-ranging studies of ULTOMIRIS in HSCT-TMA in the second half of 2020, followed by Phase 3 trials in 2021, pending regulatory feedback.

ULTOMIRIS is a long-acting C5 inhibitor. A phase 3 study of ULTOMIRIS in ALS is underway. 

ULTOMIRIS is a long-acting C5 inhibitor. Alexion plans to initiate a Phase 3 study of ULTOMIRIS in CM-TMA in the first half of 2021, pending regulatory feedback. 

ULTOMIRIS is a long-acting C5 inhibitor. A Phase 3 randomized controlled trial of ULTOMIRIS in adults with COVID-19 who are hospitalized with severe pneumonia or acute respiratory distress syndrome is underway.  

ALXN1840 (bis-choline tetrathiomolybdate) is a novel oral copper-protein binding agent with a unique mechanism of action, under investigation for Wilson disease, a rare, chronic, genetic, and potentially life-threatening liver disorder of impaired copper transport. Enrollment is complete in a Phase 3 study of ALXN1840 in Wilson disease. Study results are expected in the first half of 2021. 

Alexion is collaborating with Caelum Biosciences to develop CAEL-101 for light chain (AL) amyloidosis, a rare systemic disorder that causes misfolded immunoglobulin light chain protein to build up in and around tissues, resulting in progressive and widespread organ damage. A pivotal Phase 2/3 program is underway to investigate CAEL-101 as an add-on to current standard-of-care therapy.Dosing is complete in the Phase 2 dose selection portion of the program; the Phase 3 portion of the program is planned to begin in the third quarter of 2020, pending dose selection. 

Alexion holds an exclusive license to develop and commercialize AG10 in Japan. Eidos is currently evaluating AG10 in a Phase 3 study in the U.S. and Europe for ATTR cardiomyopathy (ATTR-CM) and plans to begin a Phase 3 study in ATTR polyneuropathy (ATTR-PN) in the second half of 2020. Alexion plans to expand the AG10 program into Japan in 2020, pending regulatory feedback. 

ALXN2040 is an investigational, oral, factor D inhibitor. Alexion plans to initiate a Phase 3 study of ALXN2040 as an add-on therapy for PNH patients with extravascular hemolysis (EVH) by the end of 2020.

In July 2020, Alexion announced the completion of its acquisition of Portola. The acquisition added ANDEXXA to the company's commercial and development portfolios. ANDEXXA has conditional approval in the U.S. and EU (marketed as ONDEXXYA in the EU) for the reversal of anticoagulation in patients experiencing life-threatening or uncontrolled bleeding who are treated with rivaroxaban or apixaban. The Phase 4 ANNEXA-I study - designed to provide clinical data supporting full approval - is underway to assess ANDEXXA compared to usual standard of care in patients presenting with acute intracranial hemorrhage while taking an oral Factor Xa inhibitor.

ALXN2050 is an investigational, oral, factor D inhibitor. A Phase 2 study of ALXN 2050 monotherapy in PNH is underway.

ALXN1830 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. Due to COVID-19, Alexion discontinued the Phase 2 study of ALXN1830, administered intravenously, in warm autoimmune hemolytic anemia (WAIHA). A new Phase 2 study of subcutaneous ALXN1830 in WAIHA is planned to begin in 2021. 

ALXN1830 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. Due to COVID-19,Alexion paused the Phase 1 study of a subcutaneous formulation of ALXN1830 in healthy volunteers. This trial and the planned Phase 2 study of subcutaneous ALXN1830 in gMG are planned to begin in 2021.

ULTOMIRIS is a long-acting C5 inhibitor. Alexion plans to initiate a Phase 2 proof-of-concept trial of ULTOMIRIS in patients with various renal diseases in 2020.

ALXN 2050 is an investigational, oral, factor D inhibitor. Alexion plans to initiate a proof-of-concept trial of ALXN2050 in patients with various renal diseases in 2021.

ALXN1720 is a novel anti-C5 albumin-binding bi-specific mini-body optimized for sub-cutaneous delivery that binds and prevents activation of human C5. Seven of nine cohorts are complete in a Phase 1 healthy volunteer study of ALXN1720. Due to COVID-19, the study was temporarily paused but is planned to restart in the third quarter of 2020.

ANDEXXA is currently being evaluated in a single-arm, open-label study in patients taking apixaban, rivaroxaban, edoxaban, or enoxaparin who require urgent surgery. The results of this study will inform the design of a randomized controlled clinical trial to expand the label in this population.

Acquired as part of the Portola acquisition, cedulatinib is a dual spleen tyrosine kinase and janus kinase (SYK/JAK) inhibitor being evaluated in a Phase 1/2a study in patients with relapsed/refractory chronic lymphocytic leukemia or B-cell or T-cell non-Hodgkin lymphoma.