A Pipeline Built With Purpose

Efficiently developing new compounds and identifying additional indications is how we deliver medicines that substantially improve the lives of people with marginalized, overlooked or forgotten diseases.

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

KRYSTEXXA® (pegloticase)

Combination with Immunomodulation in Uncontrolled Gout*

Phase 4

A recombinant uricase enzyme that breaks down urate.

MIRROR RCT: Phase 4 trial* evaluating KRYSTEXXA with methotrexate to increase the complete response rate in patients with uncontrolled gout.

UPLIZNA® (inebilizumab-cdon)

Myasthenia Gravis

Phase 3

IgG4-Related Disease

Phase 3

Kidney Transplant Desensitization

Phase 2

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

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

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

Kidney Transplant Desensitization: Phase 2 trial evaluating UPLIZNA, HZN-4920 or both, in highly-sensitized patients waiting for a kidney transplant.

HZN-825

Diffuse Cutaneous Systemic Sclerosis

Phase 2

Idiopathic Pulmonary Fibrosis

Phase 2

A molecule that blocks lysophosphatidic acid receptor 1 (LPAR1), preventing gene activation. We are initiating a Phase 2b pivotal trial in diffuse cutaneous systemic sclerosis and a Phase 2b pivotal trial in interstitial lung disease.

HZN-4920

Sjögren’s Syndrome

Phase 2

Rheumatoid Arthritis

Phase 2

Kidney Transplant Rejection

Phase 2

A fusion protein binding CD40L on T cells, blocking their interaction with CD40-expressing B cells. We are conducting Phase 2 clinical trials in Sjögren’s syndrome, rheumatoid arthritis and kidney transplant rejection.

HZN-7734

Systemic Lupus Erythematosus

Phase 2

COVID-19 Acute Lung Injury

Phase 1

A monoclonal antibody against ILT7 that depletes certain dendritic cells. We are conducting a Phase 1 clinical trial in COVID-19 acute lung injury and plan to start a Phase 2 clinical trial in systemic lupus erythematosus.

TEPEZZA® (teprotumumab-trbw)

Subcutaneous Administration

Phase 1

Diffuse Cutaneous Systemic Sclerosis

Phase 1

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

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

Diffuse Cutaneous Systemic Sclerosis: Phase 1 trial exploring TEPEZZA in diffuse cutaneous systemic sclerosis.

HZN-1116

Autoimmune Diseases

Phase 1

We plan to start a Phase 1 trial in autoimmune diseases.

HZN-003

Next-Gen Uncontrolled Gout

Pre-clinical

We are conducting preclinical research with this molecule.

HZN-007

Next-Gen Uncontrolled Gout

Pre-clinical

We are conducting preclinical research with this molecule.

HemoShear

Novel Gout Targets

Pre-clinical

Horizon entered into an exclusive collaboration to discover novel gout targets, by combining HemoShear’s REVEAL-Tx platform 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 urate.

PROTECT: Phase 4 trial evaluating KRYSTEXXA to improve management of uncontrolled gout in kidney transplant patients.

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.

Retreatment: Phase 4 trial evaluating KRYSTEXXA with methotrexate in patients who have previously failed KRYSTEXXA monotherapy.

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

Chronic TED: Phase 4 trial evaluating TEPEZZA in chronic TED to generate additional clinical data in this patient population.

Clinical Trials

Clinical trials are essential to the research and development process. People who participate in clinical trials play a vital role in helping physicians and investigators understand how an investigational medicine may safely and effectively treat a specific disease or condition.

For a comprehensive list of current clinical trials involving Horizon medicines, please visit: www.clinicaltrials.gov.

For more information about expanded access programs, read our Expanded Access Policy Statement.

This information was last updated on May 4, 2021.

C-HZN-00316-4