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 with NMOSD in the second half of 2020.

Neurology

Preclinical

Phase 1

Phase 2

Phase 3

SOLIRIS for Guillain-Barre syndrome (GBS)

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. 

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. 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.

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 HSCT-TMA 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. A phase 3 study of ULTOMIRIS in ALS is underway. 

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 first half of 2021, pending regulatory feedback. 

Nephrology

Preclinical

Phase 1

Phase 2

Phase 3

ULTOMIRIS IV for Severe COVID-19

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.  

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 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. 

Cardiology

Preclinical

Phase 1

Phase 2

Phase 3

AG10 for transthyretin amyloidosis

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. 

Cardiology

Preclinical

Phase 1

Phase 2

Phase 3

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

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.

Hematology

Preclinical

Phase 1

Phase 2

Phase 3

ANDEXXA for Acute Intracranial Hemorrhage (ICH)

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.

Hematology

Preclinical

Phase 1

Phase 2

Phase 3

ALXN2050 (ACH-5228) PNH

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

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 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. 

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 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.

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

ALXN2050 Renal Diseases

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.

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. 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.

Other

Preclinical

Phase 1

Phase 2

Phase 3

ANDEXXA Urgent Surgery

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.

Other

Preclinical

Phase 1

Phase 2

Phase 3

Cerdulatinib

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.

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 7/30/2020
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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