A Pipeline Built With Purpose

Leading with science and compassion, our team purposefully builds our pipeline to improve patients’ lives, relentlessly pushing science forward by creatively investigating pathways to reveal uncharted scientific discoveries and deliver transformational medicines.

Program
Potential Indication
  • Preclinical
  • Phase 1
  • Phase 2
  • Phase 3

UPLIZNA® (inebilizumab-cdon)

IgG4-Related Disease

Phase 3

Myasthenia Gravis

Phase 3

A humanized, affinity-optimized, afucosylated IgG1 kappa (IgG1κ) monoclonal antibody (mAb) that binds to the B cell-specific surface antigen CD19.

IgG4-Related Disease: Phase 3 trial evaluating UPLIZNA for the prevention of flare in patients with IgG4-related disease.

Myasthenia Gravis (MINT): Phase 3 trial evaluating UPLIZNA for improving outcomes in patients with myasthenia gravis.

Dazodalibep

Sjögren’s Syndrome

Phase 3

Focal Segmental Glomerulosclerosis

Phase 2

A fusion protein binding CD40L on T cells, blocking their interaction with CD40-expressing B cells. We are investigating dazodalibep in Sjögren's syndrome and focal segmental glomerulosclerosis. We also completed a Phase 2 randomized, placebo-controlled clinical trial that established proof of concept in rheumatoid arthritis in May 2022 and completed a Phase 2 clinical trial in kidney transplant rejection in March 2023.

Watch this video to learn about the mechanism of action for dazodalibep.

HZN-825

Diffuse Cutaneous Systemic Sclerosis

Phase 2

Idiopathic Pulmonary Fibrosis

Phase 2

A molecule that blocks lysophosphatidic acid receptor 1 (LPAR1). We are conducting a Phase 2b pivotal trial in diffuse cutaneous systemic sclerosis and a Phase 2 trial in idiopathic pulmonary fibrosis.

Learn more about the HZN-825 trial in diffuse cutaneous systemic sclerosis here.

Watch this video to learn about the mechanism of action for HZN-825.

Daxdilimab

Alopecia Areata

Phase 2

Dermatomyositis or Anti-synthetase Inflammatory Myositis

Phase 2

Discoid Lupus Erythematosus

Phase 2

Lupus Nephritis

Phase 2

Systemic Lupus Erythematosus

Phase 2

A fully human monoclonal antibody against ILT7 that depletes certain dendritic cells. We are investigating daxdilimab in both dermatomyositis and anti-synthetase inflammatory myositis as well as conducting Phase 2 clinical trials in alopecia areata, discoid lupus erythematosus, lupus nephritis and systemic lupus erythematosus. We recently announced negative topline results from the systemic lupus erythematosus trial and are evaluating the full data set to determine next steps.

Learn more about the daxdilimab trial in discoid lupus erythematosus here.

Learn more about the daxdilimab trial in dermatomyositis and anti-synthetase inflammatory myositis here.

Learn more about the daxdilimab trial in lupus nephritis here.

Watch this video to learn about the mechanism of action for daxdilimab.

TEPEZZA® (teprotumumab-trbw)

Active TED in Japan (OPTIC-J)

Phase 3

Chronic/Low Clinical Activity Score (CAS) TED in Japan

Phase 3

Subcutaneous Administration

Phase 1

A monoclonal antibody against insulin-like growth factor-1 receptor (IGF-1R).

Active TED in Japan (OPTIC-J): Phase 3 randomized, placebo-controlled trial in Japan to evaluate TEPEZZA in patients with moderate-to-severe active TED.

Chronic/Low Clinical Activity Score (CAS) TED in Japan: Phase 3 randomized, placebo-controlled trial in Japan to evaluate TEPEZZA in patients with chronic/low clinical activity score (CAS) TED.

Subcutaneous Administration: Phase 1 pharmacokinetic trial to explore subcutaneous administration of TEPEZZA.

HZN-1116

Autoimmune Diseases

Phase 1

A fully human monoclonal antibody that binds and neutralizes the function of the FLT3-ligand, thereby reducing both conventional and plasmacytoid dendritic cells. We are conducting a Phase 1 trial in autoimmune diseases.

Bempikibart (ADX-914)

Alopecia Areata

Phase 2

Atopic Dermatitis

Phase 2

Collaboration and option agreement with Q32 Bio to develop bempikibart (ADX-914), a fully human anti-IL-7Rα antibody that re-regulates adaptive immune function by blocking signaling mediated by both IL-7 and TSLP, two key immune pathways.

Alopecia Areata: Phase 2 trial being conducted by Q32 Bio to evaluate bempikibart (ADX-914) in alopecia areata.

Atopic Dermatitis: Phase 2 trial being conducted by Q32 Bio to evaluate bempikibart (ADX-914) in atopic dermatitis.

Horizon has an option to acquire bempikibart (ADX-914) from Q32 Bio on pre-negotiated terms through the completion of Phase 2 clinical trials.

HZN-457

Next-Gen Uncontrolled Gout

Phase 1

Exclusive collaboration to investigate a short interfering RNA (siRNA) therapeutic targeting xanthine dehydrogenase in the liver (GalNAc), to reduce serum uric acid and treat uncontrolled gout. We are conducting a Phase 1 clinical trial in gout.

Alpine 

Autoimmune Diseases

Pre-clinical

Exclusive collaboration to develop novel, multi-specific fusion protein based therapies for autoimmune and inflammatory diseases.

HemoShear

Novel Gout Targets

Pre-clinical

Exclusive collaboration to discover new therapeutics for novel gout targets by combining HemoShear’s discovery expertise with Horizon’s rheumatology development and commercialization expertise.


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Moving On-Market Medicines Forward

Horizon is exploring the potential of its on-market medicines to identify paths for new treatment methods and improve patient outcomes.

A recombinant uricase enzyme that breaks down uric acid.

AGILE: Phase 4 trial evaluating the impact of administering KRYSTEXXA over a shorter infusion duration in patients with uncontrolled gout who are receiving methotrexate.

FORWARD OL: Phase 4 trial evaluating monthly dosing of KRYSTEXXA with methotrexate in patients with uncontrolled gout.

A monoclonal antibody against insulin-like growth factor-1 receptor (IGF-1R).

Chronic Thyroid Eye Disease (TED): Phase 4 trial evaluating TEPEZZA in chronic/low clinical activity score (CAS) TED to generate additional clinical data in this patient population.

This information was last updated on September 21, 2023.

C-HZN-00316-20