A Pipeline Built With Purpose

Identifying and efficiently developing new medicines and indications is how we deliver breakthrough medicines that substantially improve the lives of people living with marginalized, overlooked or forgotten diseases.

Medicine / Candidate
  • Pre-clinical
  • Phase 1
  • Phase 2
  • Phase 3
  • Phase 3b/4

Rare Diseases

TEPEZZA® (teprotumumab-trbw)
Chronic (Inactive) Thyroid Eye Disease

Phase 3b/4

Horizon expects to initiate a randomized, placebo-controlled trial of TEPEZZA in patients with chronic Thyroid Eye Disease (TED) (previously referred to as inactive TED) in the second quarter of 2021. In chronic TED, the disease is no longer progressive; however, significant disease manifestations such as proptosis (eye bulging) and diplopia (double vision) remain.

For more information about Thyroid Eye Disease, please visit our disease information page. For more information on TEPEZZA, go to TEPEZZA.com.

TEPEZZA® (teprotumumab-trbw)
Thyroid Eye Disease
OPTIC-X: Open-Label Extension

Phase 3

Treatment of Graves' Orbitopathy to Reduce Proptosis With Teprotumumab Infusions in an Open-Label Clinical Extension Trial (OPTIC-X): An open-label extension trial in which patients who participated in the OPTIC Phase 3 clinical trial may receive eight infusions of TEPEZZA. The trial is designed to better understand whether certain patients may benefit from retreatment or longer treatment (more than six months) with teprotumumab. Patients who have longer disease duration (as they received placebo in OPTIC) are also eligible to receive a course of teprotumumab. The primary endpoint is proptosis responder rate (the percentage of participants with ≥2 mm reduction in proptosis in the study eye) without deterioration (≥2 mm increase) of proptosis in the fellow eye. Topline results were announced in July 2020.

For more information about Thyroid Eye Disease, please visit our disease information page. For more information on TEPEZZA, go to TEPEZZA.com.

HZN-825
Diffuse Cutaneous Systemic Sclerosis

Phase 2

HZN-825 is an LPAR1 antagonist in development for the treatment of diffuse cutaneous systemic sclerosis (dcSSc), a rare, chronic autoimmune disease. Diffuse cutaneous systemic sclerosis is a subtype of systemic sclerosis in which excess collagen production causes skin thickening and hardening, or fibrosis, over large areas of the body; usually the fingers, hands, arms, forearms, anterior trunk, legs and face. Compared with other forms of the disease, people with dcSSc are often at a higher risk of internal organ damage, including interstitial lung disease (ILD), gastrointestinal tract and kidney complications. Horizon expects to begin a Phase 2b pivotal trial in the first half of 2021.

HZN-825
Interstitial Lung Disease

Phase 2

As part of our strategy to further explore the potential fibrosis-mediating benefits of LPAR1 antagonism, Horizon is planning a clinical development program for HZN-825 in interstitial lung diseases (ILD), starting with idiopathic pulmonary fibrosis (IPF), which is a rare progressive lung disease with a median survival of less than five years. Horizon anticipates initiating a Phase 2b pivotal trial in the IPF indication in mid-2021.

TEPEZZA® (teprotumumab-trbw)
Diffuse Cutaneous Systemic Sclerosis

Phase 1

An exploratory trial in diffuse cutaneous systemic sclerosis (dcSSc), a rare, chronic autoimmune disease. Diffuse cutaneous systemic sclerosis is a subtype of systemic sclerosis in which excess collagen production causes skin thickening and hardening, or fibrosis, over large areas of the body; usually the fingers, hands, arms, forearms, anterior trunk, legs and face. Compared with other forms of the disease, people with dcSSc are often at a higher risk of internal organ damage, including interstitial lung disease (ILD), gastrointestinal tract and kidney complications. Available literature suggests that the mechanism of action of teprotumumab, which is to block the IGF-1 receptor, could have an impact on fibrotic processes, such as those that are relevant to dcSSc. Horizon is planning to initiate an exploratory trial in dcSSc by mid-2021.

TEPEZZA® (teprotumumab-trbw)
Subcutaneous Administration

Phase 1

A pharmacokinetic trial to explore subcutaneous dosing of TEPEZZA, which is currently administered by infusion. The objective of the trial is to inform the potential for additional administration options for TEPEZZA, which could provide greater flexibility for patients and physicians. This program includes evaluating Halozyme’s ENHANZE drug delivery technology for a subcutaneous formulation of TEPEZZA, with initial early clinical work expected to begin in 2021.

For more information about Thyroid Eye Disease, please visit our disease information page. For more information on TEPEZZA, go to TEPEZZA.com.

Rheumatic Diseases

KRYSTEXXA® (pegloticase)
Immunomodulation

Phase 3b/4

Methotrexate to Increase Response Rates in Patients with Uncontrolled Gout Receiving KRYSTEXXA (MIRROR Open Label [OL]): A small open-label trial for methotrexate co-administered with KRYSTEXXA to evaluate the impact of methotrexate weekly starting one month prior to dosing with KRYSTEXXA and then through Month 6 of treatment with KRYSTEXXA.

Methotrexate to Increase Response Rates in Patients with Uncontrolled Gout Receiving KRYSTEXXA (MIRROR Randomized Controlled Trial [RCT]): A randomized, double-blind, placebo-controlled efficacy and safety trial for methotrexate co-administered with KRYSTEXXA to evaluate the impact of methotrexate weekly starting one month prior to dosing with KRYSTEXXA and then through Month 6 of treatment with KRYSTEXXA. The trial has completed enrollment.

For more information about gout, please visit our disease information page. For more information on KRYSTEXXA, go to KRYSTEXXA.com.

KRYSTEXXA® (pegloticase)
Nephrology

Phase 3b/4

Prospective Study of Pegloticase in Transplant Patients (PROTECT): A multicenter, open-label trial which will evaluate the efficacy and safety of KRYSTEXXA among adults with uncontrolled gout who have received a kidney transplant. The trial has completed enrollment.

For more information about gout, please visit our disease information page. For more information on KRYSTEXXA, go to KRYSTEXXA.com.

KRYSTEXXA® (pegloticase)
Shorter-Infusion Duration

Phase 3b/4

Infusion Duration Study to Assess Tolerability of Pegloticase Administered With a Shorter Infusion Duration in Subjects With Uncontrolled Gout Receiving Methotrexate (AGILE): An open-label clinical trial evaluating the impact of administering KRYSTEXXA over a significantly shorter infusion duration. Currently, KRYSTEXXA is infused over a two-hour or longer timeframe. A shorter infusion duration administration could meaningfully impact the experience for patients, physicians and sites of care. Enrollment in this trial is ongoing.

For more information about gout, please visit our disease information page. For more information on KRYSTEXXA, go to KRYSTEXXA.com.

KRYSTEXXA® (pegloticase)
Monthly Dosing

Phase 3b/4

An open-label trial evaluating a monthly dosing regimen of KRYSTEXXA with methotrexate to treat people with uncontrolled gout. The current dosing schedule for KRYSTEXXA is every other week. The goal of the trial is to explore whether a monthly dosing regimen can provide similar outcomes as the current dosing schedule. The trial is expected to initiate in the first half of 2021.

For more information about gout, please visit our disease information page. For more information on KRYSTEXXA, go to KRYSTEXXA.com.

KRYSTEXXA® (pegloticase)
Retreatment

Phase 3b/4

An open-label trial evaluating KRYSTEXXA with methotrexate in patients who have previously failed KRYSTEXXA. The goal of the trial is to evaluate whether patients can benefit from KRYSTEXXA with methotrexate after developing an immune response to KRYSTEXXA when taken alone. Patients who have previously failed KRYSTEXXA have limited options available to address their uncontrolled gout. The trial is expected to initiate in the first half of 2021.

For more information about gout, please visit our disease information page. For more information on KRYSTEXXA, go to KRYSTEXXA.com.

HZN-003
Next-Gen Uncontrolled Gout

Pre-clinical

HZN-003 is a genetically engineered uricase derivative, next generation gout biologic with optimized uricase and optimized PEGylation technology.

HZN-007
Next-Gen Uncontrolled Gout

Pre-clinical

Collaboration agreement with XL-protein GmbH to identify clinical-stage product candidates that could be used to construct a next-generation gout biologic. If the collaboration agreement identifies clinical stage candidates, Horizon will have the right to license the candidates.

Novel Gout Targets

Pre-clinical

Collaboration with HemoShear Therapeutics, LLC, to explore novel approaches to identify and validate biological targets to treat gout using HemoShear’s proprietary disease modeling platform, REVEAL-Tx.


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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 March 15, 2021.

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