Advancing Our Pipeline Every Day

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 the first quarter of 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. In December 2019, Alexion initiated a Phase 3 study of ULTOMIRIS in NMOSD.

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

ULTOMIRIS is a long-acting C5 inhibitor. In December 2019, Alexion submitted an investigational new drug application (IND) for ULTOMIRIS in ALS to the U.S. Food and Drug Administration (FDA), and in January 2020, announced the planned initiation of a pivotal Phase 3 study in the first quarter of 2020.

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. 

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. Alexion is in the process of completing enrollment 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. The Phase 2 dose selection portion of the program will initiate in the first half of 2020, with the Phase 3 portion of the program 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 the first quarter of 2020. Alexion plans to expand the AG10 program into Japan in 2020, pending regulatory feedback.

Phase 3 development is initiating for danicopan (ACH-4471), an investigational, oral, factor D inhibitor, as an add-on therapy for PNH patients with extravascular hemolysis (EVH).

Danicopan (ACH-4471) is an investigational, oral, factor D inhibitor in Phase 2 development for C3 glomerulopathy (C3G).

ACH-5228 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. Alexion plans to re-initiate a Phase 2 study of ALXN1830, administered previously, in warm autoimmune hemolytic anemia (WAIHA) in early 2020. In December 2019, Alexion initiated a Phase 1 study of a subcutaneous formulation of ALXN1830 in healthy volunteers. Pending successful completion of this Phase 1 study, Alexion plans to initiate a Phase 2 study of subcutaneous ALXN1830 in gMG in the second half of 2020. 

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. Alexion plans to re-initiate a Phase 2 study of ALXN1830, administered previously, in warm autoimmune hemolytic anemia (WAIHA) in early 2020. In December 2019, Alexion initiated a Phase 1 study of a subcutaneous formulation of ALXN1830 in healthy volunteers. Pending successful completion of this Phase 1 study, Alexion plans to initiate a Phase 2 study of subcutaneous ALXN1830 in gMG in the second half of 2020. 

In January 2020, Alexion announced the completion of its acquisition of Achillion Pharmaceuticals, Inc. The acquisition adds two oral factor D inhibitors to treat rare diseases associated with the complement alternative pathway to Alexion's clinical-stage pipeline - danicopan (ACH-4471) and ACH-5228. Phase 3 development is being initiated for danicopan as an add-on therapy for PNH patients with extravascular hemolysis (EVH). Danicopan is also in Phase 2 development for C3G, and ACH-5228 is in Phase 2 development for PNH. 

ULTOMIRIS is a long-acting C5 inhibitor. Alexion plans to initiate an exploratory clinical study of ULTOMIRIS in PPMS.

Alexion plans to initiate a proof-of-concept trial of ALXN1810 (subcutaneous ALXN1210 co-administered with Halozyme’s ENHANZE^® drug-delivery technology, recombinant human hyaluronidase enzyme (rHuPH20)) in patients with various renal diseases in 2020.

A Phase 1 study of ALXN1720, a novel anti-C5 albumin-binding bi-specific mini-body that binds and prevents activation of human C5, is underway in healthy volunteers.