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

Neurology

Preclinical

Phase 1

Phase 2

Phase 3

SOLIRIS for Guillain-Barre syndrome (GBS)

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). In February 2021, Alexion initiated a Phase 3 study of SOLIRIS in GBS in Japan and dosing is underway. 

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. The U.S. FDA granted priority review for ULTOMIRIS in children and adolescents with PNH and has set a Prescription Drug User Fee Act (PDUFA) target action date of June 7, 2021. 

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 global 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. 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. for the ULTOMIRIS SC formulation and device combination in PNH and aHUS in the third quarter of 2021, and in the EU in the first quarter of 2022. 

Hematology

Preclinical

Phase 1

Phase 2

Phase 3

ULTOMIRIS IV for generalized Myasthenia Gravis (gMG)

ULTOMIRIS is a long-acting C5 inhibitor. Enrollment is complete in the Phase 3 study of ULTOMIRIS in adults with gMG. Study results are expected in the second half of 2021.

Neurology

Preclinical

Phase 1

Phase 2

Phase 3

ULTOMIRIS IV for Neuromyelitis Optica Spectrum Disorder (NMOSD)

ULTOMIRIS is a long-acting C5 inhibitor. In March 2021, Alexion completed enrollment in the Phase 3 study of ULTOMIRIS in NMOSD. 

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. Phase 3 studies of ULTOMIRIS in adults and children with HSCT-TMA are underway. 

Nephrology

Preclinical

Phase 1

Phase 2

Phase 3

ULTOMIRIS IV for Amyotrophic Lateral Sclerosis (ALS)

ULTOMIRIS is a long-acting C5 inhibitor. In March 2021, Alexion completed enrollment in the Phase 3 study of ULTOMIRIS in ALS. Study results are expected in the first half of 2022. 

Neurology

Preclinical

Phase 1

Phase 2

Phase 3

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

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 second quarter of 2021.

Nephrology

Preclinical

Phase 1

Phase 2

Phase 3

ULTOMIRIS IV for Severe COVID-19

ULTOMIRIS is a long-acting C5 inhibitor. Further enrollment in a Phase 3 trial of ULTOMIRIS in adults hospitalized with severe COVID-19 requiring mechanical ventilation is paused, due to lack of efficacy, pending further analysis of the data. 

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 and dosing are complete in a Phase 3 study of ALXN1840 in Wilson disease. Study results are expected in the third quarter 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. 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. Two parallel Phase 3 studies - one in patients with Mayo stage IIIa disease and one in patients with Mayo stage IIIb disease - are underway. 

Cardiology

Preclinical

Phase 1

Phase 2

Phase 3

ALXN2060 (AG10) for transthyretin amyloidosis

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). Alexion is conducting a Phase 3 bridging study of ALXN2060 for patients with ATTR-CM in Japan.

Cardiology

Preclinical

Phase 1

Phase 2

Phase 3

ALXN2040 (Danicopan) for PNH with extravascular hemolysis (EVH)

ALXN2040 is an investigational, oral, factor D inhibitor. A Phase 3 study of ALXN2040 as an add-on therapy for PNH patients with EVH is underway. 

Hematology

Preclinical

Phase 1

Phase 2

Phase 3

ANDEXXA for Acute Intracranial Hemorrhage (ICH)

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. A supplemental Biologics License Application (sBLA) is under review by the U.S. FDA to enable the addition of edoxaban and enoxaparin to the U.S. label. In February 2021, Alexion filed for regulatory approval of ONDEXXYA in Japan. 

Hematology

Preclinical

Phase 1

Phase 2

Phase 3

ULTOMIRIS for Dermatomyositis (DM)

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.

Neurology

Preclinical

Phase 1

Phase 2

Phase 3

ALXN2040 - Geographic Atrophy (GA)

ALXN2040 is an investigational, oral, factor D inhibitor. In March 2021, Alexion submitted an Investigational New Drug (IND) application for ALXN2040 in GA and plans to initiate a Phase 2 study in the second half of 2021. 

Other

Preclinical

Phase 1

Phase 2

Phase 3

ALXN2050 PNH

ALXN2050 is an investigational, oral, factor D inhibitor. Alexion has re-initiated additional enrollment in the Phase 2 study of ALXN2050 monotherapy in PNH patients, following the receipt of Phase 1 data that support further dose escalation in the Phase 2 study.

Hematology

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 study of ALXN2050 in patients with various renal diseases in 2021, pending regulatory feedback.

Nephrology

Preclinical

Phase 1

Phase 2

Phase 3

ULTOMIRIS IV Renal Diseases

ULTOMIRIS is a long-acting C5 inhibitor. A proof-of-concept study of ULTOMIRIS in patients with IgA nephropathy and lupus nephritis is underway. 

Nephrology

Preclinical

Phase 1

Phase 2

Phase 3

ANDEXXA Urgent Surgery

ANDEXXA is currently being evaluated in a single-arm, open-label Phase 2 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 Phase 3 clinical trial to expand the label in this population.

Other

Preclinical

Phase 1

Phase 2

Phase 3

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 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 March 2021, Alexion initiated a new Phase 1 study of subcutaneous ALXN1830 in healthy volunteers. Following successful completion of this Phase 1 study, Alexion plans to initiate Phase 2 studies of subcutaneous ALXN1830 in gMG and WAIHA in 2021, pending regulatory feedback. 

Other

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. The Phase 1 healthy volunteer study of ALXN1720 has been paused for a second time due to COVID-19, but is expected to resume in the second quarter of 2021. 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.

Other

Preclinical

Phase 1

Phase 2

Phase 3

ALXN2075 (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. Data are expected in the second quarter of 2021. 

Other

Preclinical

Phase 1

Phase 2

Phase 3

ALXN1820

ALXN1820 is a bi-specific anti-properdin mini-body. A Phase 1 study of ALXN1820 is underway in healthy volunteers.

Other

Preclinical

Phase 1

Phase 2

Phase 3

ALXN1850 - Hypophosphatasia (HPP)

ALXN1850 is Alexion’s next generation asfotase alfa asset. Alexion plans to initiate a Phase 1 study of ALXN1850 in adults with HPP in the second quarter of 2021, pending regulatory feedback.

Metabolics

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

Last updated 05/03/21
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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