PIPELINE

Advancing Our Pipeline Every Day

We’re building on our fundamental understanding of complement biology and advancing on our core therapeutic areas of hematology, nephrology, neurology, metabolics, and cardiology. We continue to evolve into new areas where there is great unmet need and opportunity to help people living with rare diseases and their families fully live their best lives.

FILTER

Therapeutic Area

Development Phases

Therapy Therapeutic Area Preclinical Phase 1 Phase 2 Phase 3

SOLIRIS® (eculizumab) for generalized Myasthenia Gravis (gMG) in children and adolescents

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.

Neurology

Preclinical

Phase 1

Phase 2

Phase 3

SOLIRIS for Neuromyelitis Optica Spectrum Disorder (NMOSD) in children and adolescents

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.

Neurology

Preclinical

Phase 1

Phase 2

Phase 3

ULTOMIRIS® (ravulizumab-cwvz) IV for Paroxysmal Nocturnal Hemoglobinuria (PNH) in children and adolescents

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.

Hematology

Preclinical

Phase 1

Phase 2

Phase 3

ULTOMIRIS IV for atypical hemolytic uremic syndrome (aHUS) in children and adolescents

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. 

Nephrology

Preclinical

Phase 1

Phase 2

Phase 3

ULTOMIRIS Subcutaneous QW

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.

Hematology

Preclinical

Phase 1

Phase 2

Phase 3

ULTOMIRIS IV for generalized Myasthenia Gravis (gMG)

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

Neurology

Preclinical

Phase 1

Phase 2

Phase 3

ULTOMIRIS IV for Neuromyelitis Optica Spectrum Disorder (NMOSD)

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

Neurology

Preclinical

Phase 1

Phase 2

Phase 3

ULTOMIRIS IV for Hematopoietic Stem Cell Transplant-Associated Thrombotic Microangiopathy (HSCT‑TMA)

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.

Nephrology

Preclinical

Phase 1

Phase 2

Phase 3

ULTOMIRIS IV for Amyotrophic Lateral Sclerosis (ALS)

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

Neurology

Preclinical

Phase 1

Phase 2

Phase 3

ULTOMIRIS IV for Complement Mediated Thrombotic Microangiopathy (CM-TMA)

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. 

Nephrology

Preclinical

Phase 1

Phase 2

Phase 3

ULTOMIRIS IV for Severe COVID-19

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.

Other

Preclinical

Phase 1

Phase 2

Phase 3

ALXN1840 for Wilson Disease

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. 

Metabolics

Preclinical

Phase 1

Phase 2

Phase 3

CAEL-101 for AL Amyloidosis

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. 

Cardiology

Preclinical

Phase 1

Phase 2

Phase 3

AG10 for transthyretin amyloidosis

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.

Cardiology

Preclinical

Phase 1

Phase 2

Phase 3

ALXN2040 (Danicopan/ACH-4471) for PNH with extravascular hemolysis (EVH)

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.

Hematology

Preclinical

Phase 1

Phase 2

Phase 3

Danicopan (ACH-4471) for C3 Glomerulopathy

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.

Nephrology

Preclinical

Phase 1

Phase 2

Phase 3

ALXN2050 (ACH-5228) PNH

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

Hematology

Preclinical

Phase 1

Phase 2

Phase 3

ALXN1830 for Warm Autoimmune Hemolytic Anemia (WAIHA)

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.

Hematology

Preclinical

Phase 1

Phase 2

Phase 3

ALXN1830 for generalized Myasthenia Gravis (gMG)

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.

Neurology

Preclinical

Phase 1

Phase 2

Phase 3

ULTOMIRIS IV Renal Diseases

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.

Nephrology

Preclinical

Phase 1

Phase 2

Phase 3

ALXN1720

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. 

Other

Preclinical

Phase 1

Phase 2

Phase 3

Zealand Pharma A/S

Alexion is collaborating with Zealand Pharma A/S to discover and develop novel peptide therapies for up to four targets in the complement pathway. Peptides offer a number of advantages, including being highly selective and potent, allowing low dosage volumes for ease of administration, and having the potential to treat a broad range of complement-mediated diseases. 

Other

Preclinical

Phase 1

Phase 2

Phase 3

Dicerna - GalXCTM

Alexion is collaborating with Dicerna to jointly discover and develop subcutaneously delivered GalXCTM RNA interface (RNAi) candidates, currently in pre-clinical development, for the treatment of complement-mediated diseases. In December 2019, Alexion exercised its options for exclusive rights to two additional targets, expanding the collaboration to now encompass four targets within the complement pathway. 

Other

Preclinical

Phase 1

Phase 2

Phase 3

CP010

Alexion is collaborating with Complement Pharma to co-develop CP010, a pre-clinical C6 complement inhibitor that has the potential to treat multiple neurological disorders.

Other

Preclinical

Phase 1

Phase 2

Phase 3

 Immune Pharma - anti-eotaxin-1 antibody

In November 2019, Alexion acquired an anti-eotaxin-1 antibody from Immune Pharma for potential development in inflammatory diseases.

Other

Preclinical

Phase 1

Phase 2

Phase 3

Last updated 5/6/2020
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MEDICINES

Roberta, diagnosed with gMG at 16 years old
This experience has taught me to look at the small things that count. Now, I feel that I have something to look forward to and I am who I want to be.”
ROBERTA LIVING WITH gMG