Horizon Pharma plc to Present Results from its Phase 3 Teprotumumab Trial (OPTIC) at the 2019 American Association of Clinical Endocrinologists (AACE) Scientific and Clinical Congress

DUBLIN--(BUSINESS WIRE)--Horizon Pharma plc (Nasdaq: HZNP) today announced that it will present additional results from its Phase 3 confirmatory clinical trial (OPTIC) evaluating teprotumumab for the treatment of active thyroid eye disease (TED) at the 2019 American Association of Clinical Endocrinologists (AACE) 28^th Annual Scientific & Clinical Congress. The late-breaking oral presentation will be presented on Friday, April 26, 2019.

Positive primary endpoint data from the clinical trial, titled OPTIC (Treatment of Graves’ Orbitopathy (Thyroid Eye Disease) to Reduce Proptosis with Teprotumumab Infusions in a Randomized, Placebo-Controlled, Clinical Study), was announced on February 28, 2019. Detailed new data will be presented at the meeting.

Presentation Details:

• Title: Teprotumumab treatment effect on proptosis in patients with active thyroid eye disease: Results from a Phase 3, randomized, double-masked, placebo-controlled, parallel-group, multicenter study
• Abstract: Late breaking general session
• Speaker: Raymond Douglas, M.D., OPTIC co-principal investigator and director of the orbital and thyroid eye disease program, Cedars-Sinai Medical Center
• Date: Friday, April 26, 2019
• Time: 11 a.m. – 12 p.m. PT

About Teprotumumab

Teprotumumab is a fully human monoclonal antibody (mAb) and a targeted inhibitor of the insulin-like growth factor 1 receptor (IGF-1R). Teprotumumab is an investigational medicine and its safety and efficacy have not been established. The Phase 3 OPTIC confirmatory clinical study was conducted at leading centers in the U.S., Germany and Italy, with co-principal investigators Raymond Douglas, M.D., Ph.D., Cedars-Sinai Medical Center; and George Kahaly, M.D., Johannes Gutenberg University Medical Center. In addition to the Phase 3 OPTIC trial, Horizon is conducting the OPTIC‐X extension trial to gather further insight into the long-term efficacy and safety of teprotumumab. The robust clinical development program for teprotumumab in the treatment of TED includes positive Phase 2 results published in The New England Journal of Medicine.

About Thyroid Eye Disease

TED is a serious, progressive, and debilitating autoimmune disease with a limited window of active disease during which it may respond to medical intervention.^1,2 While TED often occurs in people living with hyperthyroidism or Graves’ disease, it is a distinct disease that is caused by autoantibodies activating an IGF-1R-mediated signaling complex on cells within the orbit.^3,4 This leads to a cascade of negative effects, which cause long-term, irreversible damage. Active TED lasts for up to three years and is characterized by inflammation and tissue expansion behind the eye.^5,1 As TED progresses, it causes serious damage – including proptosis (eye bulging), strabismus (misalignment of the eyes), and diplopia (double vision) – and in some cases can lead to blindness.^2,6 Currently, patients must suffer through active TED until the disease becomes inactive – often left with permanent and sight-impairing consequences – before they can have complex and costly surgical procedures that may never fully restore vision or appearance.^5,1,7 People living with TED often experience long-term functional, psychological and economic burdens, including inability to work and perform activities of daily living.^7,8

About Horizon Pharma plc

Horizon Pharma plc is focused on researching, developing and commercializing innovative medicines that address unmet treatment needs for rare and rheumatic diseases. By fostering a growing pipeline of medicines in development and exploring all potential uses for currently marketed medicines, we strive to make a powerful difference for patients, their caregivers and physicians. For us, it’s personal: by living up to our own potential, we are helping others live up to theirs. For more information, please visit www.horizonpharma.com, follow us @HZNPplc on Twitter, like us on Facebook or explore career opportunities on LinkedIn.

References

1. Graves’ Ophthalmopathy: VISA versus EUGOGO Classification, Assessment, and Management. Journal of Ophthalmology. 2015. https://www.hindawi.com/journals/joph/2015/249125/cta/. Accessed Feb 22, 2019.
2. The 2016 European Thyroid Association/European Group on Graves' Orbitopathy Guidelines for the Management of Graves' Orbitopathy. European Thyroid Journal. 2 March 2016. https://www.ncbi.nlm.nih.gov/pubmed/27099835. Accessed Feb 22, 2019.
3. Graves' Ophthalmopathy. The New England Journal of Medicine. 25 February 2010. https://www.nejm.org/doi/full/10.1056/NEJMra0905750. Accessed Feb 22, 2019.
4. Igs from patients with Graves' disease induce the expression of T cell chemoattractants in their fibroblasts. The Journal of Immunology. 15 January 2002. https://www.ncbi.nlm.nih.gov/pubmed/11777993. Accessed Feb 22, 2019.
5. Update on thyroid eye disease and management. Clinical Ophthalmology. 19 October 2009. https://www.ncbi.nlm.nih.gov/pmc/articles/PMC2770865/. Accessed Feb 22, 2019.
6. Clinical features of dysthyroid optic neuropathy: a European Group on Graves' Orbitopathy (EUGOGO) survey. British Journal of Ophthalmology. 11 October 2006. https://www.ncbi.nlm.nih.gov/pubmed/17035276. Accessed Feb 22, 2019.
7. Quality of Life and Occupational Disability in Endocrine Orbitopathy. DA International. 24 April 2009. https://www.ncbi.nlm.nih.gov/pmc/articles/PMC2689575/. Accessed Feb 22, 2019.
8. Public health relevance of Graves' orbitopathy. The Journal of Clinical Endocrinology & Metabolism. 26 November 2012. https://www.ncbi.nlm.nih.gov/pubmed/23185037. Accessed Feb 22, 2019.