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

Hematology

Preclinical

Phase 1

Phase 2

Phase 3

Neurology

Preclinical

Phase 1

Phase 2

Phase 3

Nephrology

Preclinical

Phase 1

Phase 2

Phase 3

Bone Metabolism

Preclinical

Phase 1

Phase 2

Phase 3

ALXN1840 for Wilson Disease

ALXN1840 (bis-choline tetrathiomolybdate) is an investigational, oral, targeted de-coppering therapy. 

Metabolics

Preclinical

Phase 1

Phase 2

Phase 3

CAEL-101 for AL Amyloidosis

CAEL-101 is an investigational first-in-class amyloid fibril targeted therapy.

Cardiology

Preclinical

Phase 1

Phase 2

Phase 3

Acoramidis (ALXN2060) for Transthyretin Amyloid Cardiomyopathy (ATTR-CM)

Acoramidis (ALXN2060) is an investigational, oral, small molecule. Alexion holds an exclusive license to develop and commercialize acoramidis in Japan.

Cardiology

Preclinical

Phase 1

Phase 2

Phase 3

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

Danicopan (ALXN2040) is an investigational, oral, factor D inhibitor.

Hematology

Preclinical

Phase 1

Phase 2

Phase 3

ULTOMIRIS for Dermatomyositis (DM)

ULTOMIRIS is a long-acting C5 inhibitor.

Neurology

Preclinical

Phase 1

Phase 2

Phase 3

Danicopan (ALXN2040) for Geographic Atrophy (GA)

Danicopan (ALXN2040) is an investigational, oral, factor D inhibitor.

Ophthalmology

Preclinical

Phase 1

Phase 2

Phase 3

Vemircopan (ALXN2050) for PNH

Vemircopan (ALXN2050) is an investigational, oral, factor D inhibitor.

Hematology

Preclinical

Phase 1

Phase 2

Phase 3

Vemircopan (ALXN2050) for gMG

Vemircopan (ALXN2050) is an investigational, oral, factor D inhibitor.

Neurology

Preclinical

Phase 1

Phase 2

Phase 3

Vemircopan (ALXN2050) for Lupus Nephritis (LN) or Immunoglobulin A Nephropathy (IgAN)

Vemircopan (ALXN2050) is an investigational, oral, factor D inhibitor.

Nephrology

Preclinical

Phase 1

Phase 2

Phase 3

ULTOMIRIS IV for Renal Diseases

ULTOMIRIS is a long-acting C5 inhibitor.

Nephrology

Preclinical

Phase 1

Phase 2

Phase 3

Gefurulimab (ALXN1720) for gMG

Gefurulimab (ALXN1720) is an investigational, anti-C5 albumin-binding humanized bispecific VHH antibody optimized for sub-cutaneous delivery. 

Neurology

Preclinical

Phase 1

Phase 2

Phase 3

ALXN1820

ALXN1820 is an investigational, anti-properdin humanized bispecific VHH antibody.

Hematology

Preclinical

Phase 1

Phase 2

Phase 3

ALXN1850 for Hypophosphatasia (HPP)

ALXN1850 is an investigational, next-generation asfotase alfa asset.

Bone Metabolism

Preclinical

Phase 1

Phase 2

Phase 3

NI006 for ATTR-CM

Alexion has entered into an exclusive global collaboration and license agreement with Neurimmune AG for NI006, an investigational human monoclonal antibody. 

Cardiology

Preclinical

Phase 1

Phase 2

Phase 3

ALXN1910

ALXN1910 is an investigational, next-generation, tissue-nonspecific alkaline phosphatase (TNSALP) enzyme replacement therapy (ERT).

Bone Metabolism

Preclinical

Phase 1

Phase 2

Phase 3

ALXN2030

ALXN2030 is an siRNA targeting the C3 complement protein.

Nephrology

Preclinical

Phase 1

Phase 2

Phase 3

ALXN2080

ALXN2080 is an investigational, oral, factor D inhibitor.

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.

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.

Other

Preclinical

Phase 1

Phase 2

Phase 3

Last updated 11/10/22
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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