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) Thyroid Eye Disease

  • OPTIC-X trial: 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): A 48-week, open-label clinical extension trial in which patients who participated in the OPTIC Phase 3 clinical trial may receive up to eight additional 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. 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.

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

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TEPEZZA™ (teprotumumab-trbw) Diffuse Cutaneous Scleroderma

  • Exploratory trial

PHASE 1

An exploratory trial in diffuse cutaneous scleroderma, a rare fibrotic disease with no treatment options, evaluating additional indications for TEPEZZA. Diffuse cutaneous scleroderma is a subtype of scleroderma in which excess collagen production causes skin thickening and hardening, or fibrosis, over large areas of the body, usually the fingers, hands, arms, anterior trunk, legs and face. There can be significant associated organ damage, including to the gastrointestinal tract, kidneys, lungs and heart. 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 diffuse cutaneous scleroderma. Horizon expects to conduct an exploratory trial beginning in the first half of 2020 to evaluate objective biomarker and clinical endpoints to inform potential subsequent larger and longer duration clinical trials.

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 for one month prior to dosing with KRYSTEXXA and then throughout 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 for one month prior to dosing with KRYSTEXXA and then throughout Month 6 of treatment with KRYSTEXXA. The MIRROR RCT follows the MIRROR open-label evaluation that was initiated in fall 2018 and is now fully enrolled.

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

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KRYSTEXXA® (pegloticase) Nephrology

  • PROTECT 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.

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

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KRYSTEXXA® (pegloticase) Shorter-Infusion Duration

  • Open-label trial

PHASE 3b / 4

Evaluating the impact of administering KRYSTEXXA over a significantly shorter infusion duration. The initial proof-of-concept work will begin mid-2020. Currently, KRYSTEXXA is infused over a two-hour or longer timeframe.  A shorter infusion duration could meaningfully improve the experience and convenience for patients, physicians and sites of care.

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

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HZN-003 Next-Gen Uncontrolled Gout

  • 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

  • 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.

Recent Clinical Milestones

MEDICINE

STATUS

PROCYSBI® (cysteamine bitartrate)

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

TEPEZZA™ (teprotumumab-trbw)

Discovery Collaborations

MEDICINE / CANDIDATE

PHASE

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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™. The platform combines physiological and computational models of disease to identify novel treatment approaches. This collaboration may lead to potential clinical targets or new studies of which Horizon will have the rights to commercialize any successful candidates.



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 March 17, 2020.

C-HZN-00203