Research and Development
Developing transformative therapies begins with our personal investment in the lives of people our medicines help wherever they are in their journey, from diagnosis through ongoing care. At Horizon, we aim to make a powerful difference for our patients, their caregivers and physicians through research of breakthrough medicines as well as exploration of new applications for existing medicines. Our long-term strategy focuses on building a pipeline of clinically meaningful development-stage medicines in order to bring new options forward for people living with rare and rheumatic diseases.
- OPTIC confirmatory Phase 3 trial
- OPTIC-X Phase 3 extension trial
HZN-001 (teprotumumab) is a fully human monoclonal antibody, insulin-like growth factor-1 receptor (IGF-1R) inhibitor being studied in a confirmatory Phase 3 clinical trial for the treatment of active thyroid eye disease (TED). Teprotumumab has received Breakthrough Therapy, Orphan Drug and Fast Track designations from the U.S. Food and Drug Administration.
In patients with active TED, IGF-1R is overexpressed on orbital fibroblasts, resulting in local inflammation and orbital tissue expansion, which can lead to proptosis, or bulging of the eye.
Teprotumumab is an investigational medicine and its safety and efficacy have not been established.
- Label expansion: birth to 2 months
A supplemental New Drug Application (sNDA) has been submitted to the U.S. Food and Drug Administration (FDA) to expand the approved indication for RAVICTI (glycerol phenylbutyrate) Oral Liquid to include infants younger than two months of age living with urea cycle disorders (UCDs). The sNDA submission is based off an open-label clinical study evaluating the safety, efficacy and pharmacokinetics of RAVICTI in pediatric subjects under two years of age with UCDs. The FDA is expected to complete their review during the second half of 2018.
(Submitted Feb. 2018 / U.S.)
RAVICTI is not approved for use in patients younger than two months of age by any Regulatory authority.
- Optimized uricase and optimized PEGylation for uncontrolled gout
HZN-003 is a genetically engineered uricase derivative, next generation gout biologic with optimized uricase and optimized PEGylation technology.
- Optimized uricase and PASylation for uncontrolled gout
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.
- MIRROR trial: immunomodulation (Phase 4)
- TRIPLE trial: tolerization and immunomodulation (IIT)
- RECIPE trial: immunomodulation (IIT)
- Methotrexate to Increase Response Rates in Patients with Uncontrolled GOut Receiving KRYSTEXXA (MIRROR): Horizon Pharma-sponsored multicenter, efficacy and safety study for methotrexate co-administered with KRYSTEXXA to evaluate the impact of methotrexate weekly for one month prior to dosing with KRYSTEXXA and then throughout the 24 weeks of treatment with KRYSTEXXA.
- REduCing Immunogenicity to PegloticasE (RECIPE): investigator-initiated double-blind, placebo controlled trial to evaluate if a 12-week course of immunomodulating therapy with daily doses of mycophenolate mofetil (MMF) can safely and significantly attenuate immunogenicity.
- Tolerization Reduces Intolerance to Pegloticase and Prolongs the Urate Lowering Effect (TRIPLE): investigator-initiated exploratory, open-label adaptive trial design with multiple patient cohorts, including a cohort to evaluate the impact of adding daily doses of the immunomodulator azathioprine for a two-week run-in period, followed by continued daily azathioprine with KRYSTEXXA.
These are ongoing investigational trials and have not yet been approved by any Regulatory authority.
- RIFLE trial: lupus
- A Crossover Study to Compare RAYOS to IR Prednisone to Improve Fatigue and Morning Symptoms for SLE (RIFLE): investigator-initiated randomized, double-blind, active comparator-controlled, crossover study to assess the capacity of RAYOS compared to immediate-release prednisone to improve fatigue and control morning symptoms in those living with systemic lupus erythematosus.
For more information on RAYOS, please see RAYOSRx.com.
This is an ongoing investigational trial and has not yet been approved by any Regulatory authority.
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.