Our Pipeline

We are relentless in pursuing breakthroughs that boldly change the lives of the communities we serve.

Our pipeline is built on purpose, and our scientific courage drives us to tackle many marginalized, overlooked or forgotten diseases.

Rare Diseases

MEDICINE / CANDIDATE

PHASE

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

  • Trial in chronic TED patients

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) by year-end 2020. 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.

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TEPEZZA® (teprotumumab-trbw) Thyroid Eye Disease

  • OPTIC-X: Phase 3 extension trial

OPEN-LABEL EXTENSION

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.

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HZN-825 Diffuse Cutaneous Systemic Sclerosis

  • Phase 2b pivotal trial

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.

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HZN-825 Interstitial Lung Disease

  • Phase 2b pivotal trial in IPF

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.

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TEPEZZA® (teprotumumab-trbw) Diffuse Cutaneous Systemic Sclerosis

  • Exploratory trial

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 year-end 2020.

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TEPEZZA® (teprotumumab-trbw) Subcutaneous Administration

  • Pharmacokinetic trial

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.

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

Rheumatic Diseases

MEDICINE / CANDIDATE

PHASE

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KRYSTEXXA® (pegloticase) Immunomodulation

  • MIRROR open-label (complete)
  • MIRROR randomized controlled trial

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.

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KRYSTEXXA® (pegloticase) Nephrology

  • PROTECT open-label trial in kidney transplant patients with uncontrolled gout

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. More than 75 percent enrollment has been achieved and complete enrollment is expected by the end of 2020.

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

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KRYSTEXXA® (pegloticase) Shorter-Infusion Duration

  • Open-label trial

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.

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

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HZN-003 Next-Gen Uncontrolled Gout

  • Exploration of optimized uricase and optimized PEGylation for uncontrolled gout

PRE-CLINICAL

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

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HZN-007 Next-Gen Uncontrolled Gout

  • Exploration of optimized uricase and PASylation for 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.

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HemoShear Gout Discovery Collaboration

  • Exploration of novel approaches to treating gout

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™.

Recent Clinical Milestones

MEDICINE

STATUS

PROCYSBI® (cysteamine bitartrate)

  • New dosage form: Delayed-Release Oral Granules in Packets

TEPEZZA® (teprotumumab-trbw)



Clinical Trials

Clinical trials are an important part of 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 be able to 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.

 

This information was last updated on November 6, 2020.

C-HZN-00275