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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. A Phase 2/3 study of SOLIRIS in children and adolescents with NMOSD is underway.

SOLIRIS is a first-in-class complement inhibitor. SOLIRIS in GBS has been granted SAKIGAKE designation by Japan's Ministry of Health, Labour and Welfare (MHLW). Alexion plans to initiate a Phase 3 study of SOLIRIS in GBS in Japan in the first half of 2021.

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 global Phase 3 study in children and adolescents who have aHUS is underway. 

ULTOMIRIS is a long-acting C5 inhibitor. 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. In November 2020, Alexion completed enrollment in the Phase 3 study of ULTOMIRIS in adults with gMG. Study results are expected in the second half of 2021.

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

ULTOMIRIS is a long-acting C5 inhibitor. Dosing in underway in Phase 3 studies of ULTOMIRIS in adults and children with HSCT-TMA.

ULTOMIRIS is a long-acting C5 inhibitor. As completion of full enrollment nears, screening of new patients has closed for the Phase 3 study of ULTOMIRIS in ALS.

ULTOMIRIS is a long-acting C5 inhibitor. In January 2021, Alexion submitted an Investigational New Drug (IND) application for ULTOMIRIS in CM-TMA and plans to initiate a Phase 3 study in the first half of 2021.

ULTOMIRIS is a long-acting C5 inhibitor. In January 2021, Alexion paused further enrollment in a Phase 3 trial of ULTOMIRIS in adults hospitalized with severe COVID-19 requiring mechanical ventilation, due to lack of efficacy, pending further analysis of the data. This decision was made based on the recommendation of an independent data monitoring committee, following their review of data from a pre-specified interim analysis.

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. Alexion and Caelum Biosciences are conducting the Cardiac Amyloid Reaching for Extended Survival (CARES) Phase 3 clinical program to evaluate CAEL-101, a first-in-class amyloid fibril targeted therapy, in combination with standard-of-care therapy in AL amyloidosis. Dosing is underway in two parallel Phase 3 studies - one in patients with Mayo stage IIIa disease and one in patients with Mayo stage IIIb disease.

Alexion holds an exclusive license to develop and commercialize AG10 in Japan. Eidos is currently evaluating AG10 in two Phase 3 studies in the U.S. and Europe - one for ATTR cardiomyopathy (ATTR-CM) and one for ATTR polyneuropathy (ATTR-PN). In October 2020, Alexion initiated a Phase 3 bridging study of ALXN2060 for patients with ATTR-CM in Japan and dosing is underway.

ALXN2040 is an investigational, oral, factor D inhibitor. In December 2020, Alexion initiated a Phase 3 study of ALXN2040 as an add-on therapy for PNH patients with EVH and dosing is underway. 

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. In December 2020, Alexion submitted a supplemental Biologics License Application (sBLA) to the U.S. FDA to enable the addition of edoxaban and enoxaparin to the U.S. label. Alexion plans to file for regulatory approval of ANDEXXA in Japan in the first quarter of 2021. 

ULTOMIRIS is a long-acting C5 inhibitor. Alexion plans to initiate a Phase 2/3 study of ULTOMIRIS in DM in the second half of 2021, pending regulatory feedback.

ALXN 2040 is an investigational, oral, factor D inhibitor. Alexion plans to initiate a Phase 2 study of ALXN2040 in GA in the second half of 2021.

ALXN2050 is an investigational, oral, factor D inhibitor. Alexion has paused further enrollment in the Phase 2 study of ALXN2050 monotherapy in PNH patients, pending the receipt of further Phase 1 data (expected in the second quarter of 2021) that will allow for dose escalation in the Phase 2 study.

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

ULTOMIRIS is a long-acting C5 inhibitor. In November 2020, Alexion initiated a proof-of-concept study of ULTOMIRIS in patients with IgA nephropathy and lupus nephritis.

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) and the Phase 1 study of a subcutaneous formulation of ALXN1830 in healthy volunteers. In January 2021, a Clinical Trial Application (CTA) for a new Phase 1 study of subcutaneous ALXN1830 was approved in New Zealand; study initiation is planned for the first quarter of 2021. Following successful completion of the Phase 1 study, Alexion plans to initiate Phase 2 studies of subcutaneous ALXN1830 in gMG and WAIHA in 2021, pending regulatory feedback. 

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. The Phase 1 healthy volunteer study of ALXN1720 has been paused for a second time due to COVID-19. Additional cohorts have been added to the study to explore higher doses and enable the initiation of a Phase 3 study in gMG, pending successful completion of the Phase 1 study as has been agreed with the FDA. Data from the Phase 1 study are expected in the second half of 2021. Alexion also plans to initiate a study of ALXN1720 in DM.

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. Data are expected in the first half of 2021. 

ALXN1820 is a bi-specific anti-properdin mini-body. In January 2021, Alexion initiated a Phase 1 study of ALXN1820 in healthy volunteers.

ALXN1850 is Alexion’s next generation asfotase alfa asset. In November 2020, Alexion submitted an IND application to the FDA for ALXN1850. Initiation of a Phase 1 study in adults with HPP is planned for the second quarter of 2021.