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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 who have NMOSD in mid-2020.

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. Enrollment is complete in a single, PK-based Phase 3 study of ULTOMIRIS delivered subcutaneously once per week to support registration in PNH and aHUS. Data are expected in the first half of 2020.

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 the second half of 2020, followed by Phase 3 trials in 2021, pending regulatory feedback.

ULTOMIRIS is a long-acting C5 inhibitor. In March 2020, Alexion began dosing patients in a Phase 3 study of ULTOMIRIS in ALS.  

ULTOMIRIS is a long-acting C5 inhibitor. Alexion plans to initiate a Phase 3 study of ULTOMIRIS in CM-TMA in the second half of 2020, pending regulatory feedback. 

In April 2020, Alexion announced U.S. Food and Drug Administration (FDA) acceptance of an investigational new drug application for ULTOMIRIS in severe COVID-19 and initiated a Phase 3 randomized controlled trial in adults with COVID-19 who are hospitalized with severe pneumonia or acute respiratory distress syndrome.

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 will investigate CAEL-101 as an add-on to current standard-of-care therapy. In March 2020, the companies began dosing patients in the Phase 2 dose selection portion of the program; the Phase 3 portion of the program is planned to begin later in 2020, pending dose selection. 

Alexion holds an exclusive license to develop and commercialize AG10 in Japan. AG10 is a small molecule designed to treat the root cause of transthyretin amyloidosis (ATTR) – destabilized and misfolded transthyretin (TTR) protein – by binding and stabilizing TTR in the blood. Eidos is currently evaluating AG10 in a Phase 3 study in the U.S. and Europe for ATTR cardiomyopathy (ATTR-CM) – a progressive, fatal disease caused by the accumulation of misfolded TTR amyloid in the heart – and plans to begin a Phase 3 study in ATTR polyneuropathy (ATTR-PN) – a progressive, fatal disease caused by the accumulation of misfolded TTR amyloid in the peripheral nervous system - in 2020. Alexion plans to expand the AG10 program into Japan in 2020, pending regulatory feedback.

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

Danicopan (ACH-4471) is an investigational, oral, factor D inhibitor in Phase 2 development for C3 glomerulopathy (C3G). Interim data are expected in the second quarter of 2020.

ALXN2050 is an investigational, oral, factor D inhibitor in Phase 2 development for PNH.

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 has temporarily paused the Phase 2 study of ALXN1830.

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 has temporarily 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 anticipated 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.

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. Due to COVID-19, the Phase 1 study in healthy volunteers is temporarily paused.