Horizon Therapeutics plc initiates Phase 3 clinical trial (OPTIC-J) in Japan evaluating teprotumumab for the treatment of active thyroid eye disease (TED)

— Teprotumumab is marketed under the brand name TEPEZZA® in the USA —

DUBLIN, February 23, 2022–(BUSINESS WIRE)–Horizon Therapeutics plc (Nasdaq: HZNP) today announced that the first patient has been enrolled in a Phase 3 clinical trial (OPTIC-J) in Japan evaluating teprotumumab for the treatment of TED active. PDD is a rare, serious, progressive, and potentially vision-threatening autoimmune disease that can cause exophthalmos (swollen eyes), diplopia (double vision), eye pain, redness, and swelling.1 Teprotumumab, marketed under the brand name TEPEZZA in the United States (US), was approved by the United States Food and Drug Administration (FDA) in January 2020 as the first and only drug for PDD, and has received Priority Review, Orphan Drug, Fast Track, and Breakthrough Therapy designations. Teprotumumab has not been approved for commercial use in Japan.

The OPTIC-J trial is a randomized, double-blind, placebo-controlled, parallel-group study that will evaluate the efficacy, tolerability, and safety of teprotumumab in the treatment of patients in Japan with moderate to severe active PDD . The trial methodology is based on the Phase 3 OPTIC trial conducted in the United States and Europe and will include approximately 50 adults who meet trial eligibility criteria at trial sites across Japan. . Patients will be randomized 1:1 to receive teprotumumab or placebo once every three weeks for a total of eight infusions (10 mg/kg for the first infusion and 20 mg/kg for the remaining seven infusions). This dosage and administration is based on approval in the United States and may not be the same if approved in Japan.

The primary efficacy endpoint is the proptosis response rate at week 24, measured by the percentage of participants with at least a 2 mm reduction in proptosis from baseline in the study eye, without deterioration of the other eye (increase ≥ 2 mm). The trial will also assess overall responder rate, percentage of patients with a clinical activity score (CAS) of 0 or 1 at week 24 in the study eye, change from baseline at week 24 in the measure of proptosis in the study eye, diplopia responder rate, and change from baseline to week 24 in the Graves’ Ophthalmopathy Quality of Life Questionnaire ( GO-QoL). Study participants who complete the treatment period and do not respond to proptosis at week 24 or who relapse during the 48-week follow-up may choose to enter an open-label extension period to receive eight additional infusions of teprotumumab.

“There is a significant medical need in Japan for a drug that can effectively treat proptosis and diplopia, which are two of the most debilitating symptoms of thyroid eye disease,” said Yuji Hiromatsu, MD, professor emeritus at Kurume Medical Center. and co-coordinator. trial investigator. “Steroids are commonly used in Japan to help relieve inflammation, but they can have serious side effects and have not been shown to reduce proptosis or improve diplopia. If left untreated , thyroid eye disease can lead to loss of vision, severe pain, and other symptoms that make it difficult to work, sleep, and spend time with family and friends.”

“Given the unmet need in Japan for a drug that treats thyroid eye disease at the source, as well as the robust clinical response with teprotumumab treatment that has been observed in previous clinical trials and experience post-marketing in the United States, we look forward to understanding how this therapy can help patients in Japan in particular,” said Elizabeth HZ Thompson, Ph.D., executive vice president, research and development, Horizon. We are committed to collaborating with Japanese researchers and regulators on this important initiative, with the ultimate goal of bringing a valuable new treatment option to patients.”

The trial was designed in consultation with Japan’s Pharmaceuticals and Medical Devices Agency (PMDA). OPTIC-J stands for Treatment of Graves’ Orbitopathy (Thyroid Eye Disease) to Reduce Proptosis with Teprotumumab Infusions in a Randomized, Placebo-Controlled Clinical Study – Japan. Further information about the trial, including eligibility criteria, is available on the Japanese Clinical Trials Registry website (Trial ID number jRCT2031210453).

About Thyroid Eye Disease (TED)

PDD is a rare, serious, progressive and potentially vision-threatening autoimmune disease.1 PDD often occurs in people with Graves’ disease, but it is a distinct disease caused by autoantibodies activating an IGF-1R-mediated signaling complex on cells in the retrospace -orbital.2.3 This leads to a cascade of negative effects, which can cause long-term irreversible damage, including blindness.4.5 PDD begins with an acute (active) phase where inflammatory signs and symptoms, such as eye pain, swelling, proptosis, and diplopia, progress over time.1.4 The disease then enters a chronic phase where the inflammation is no longer present or has markedly diminished, but significant signs and symptoms may remain.

About teprotumumab

Teprotumumab is a fully human monoclonal antibody (mAb) and targeted inhibitor of the insulin-like growth factor receptor (IGF-1R). Teprotumumab is a biologic drug that is given to patients once every three weeks for a total of eight infusions. It has received Priority Review, Orphan Drug, Fast Track, and Breakthrough Therapy designations from the United States FDA. In previous clinical trials, the most common adverse reactions (incidence ≥ 5% and greater than placebo) were muscle spasms, nausea, alopecia, diarrhea, fatigue, hyperglycaemia, impaired hearing, dysgeusia, headaches, dry skin and menstrual disorders.

About Horizon

Horizon is focused on the discovery, development and commercialization of drugs that address the critical needs of people affected by rare, autoimmune and serious inflammatory diseases. Our pipeline is useful: we apply scientific expertise and courage to bring clinically meaningful therapies to patients. We believe science and compassion must work together to transform lives. For more information on how we go to incredible lengths to impact lives, visit www.horizontherapeutics.com and follow us on TwitterLinkedIn, Instagram and Facebook.

Forward-looking statements

This press release contains forward-looking statements, including, but not limited to, statements regarding the potential benefits of TEPEZZA (teprotumumab); the expected scope, endpoints and timing of the OPTIC-J clinical trial and other statements that are not historical facts. These forward-looking statements are based on Horizon’s current expectations and inherently involve significant risks and uncertainties. Actual results and the timing of events could differ materially from those anticipated in these forward-looking statements due to such risks and uncertainties, which include, but are not limited to, risks related to clinical trials, including that past results may not predict future results. results of clinical trials; the impacts of the COVID-19 pandemic and measures taken to slow its spread, including potential delays in clinical trials; regulatory obligations and oversight, including any changes in the legal and regulatory environment in Japan and the risks detailed from time to time under “Risk Factors” and elsewhere in Horizon’s filings and reports with the SEC. Horizon assumes no obligation or obligation to update any forward-looking statements contained in this press release as a result of new information.

The references

  1. Barrio-Barrio J, et al. Graves’ ophthalmopathy: classification, evaluation and management VISA versus EUGOGO. Journal of Ophthalmopathy. 2015;2015:249125.

  2. Weightman DR, et al. Autoantibodies against IGF-1 binding sites in thyroid-associated ophthalmopathy. Autoimmunity. 1993;16(4):251–257.

  3. Pritchard J, et al. Immunoglobulin activation of T-cell chemotactic expression in fibroblasts from Graves’ disease patients is mediated through the insulin-like growth factor receptor 1 pathway. J Immunol. 2003;170:6348-6354.

  4. Bartalena L, Kahaly GJ, Baldeschi L, et al. European Group on Graves’ Orbitopathy (EUGOGO) 2021 Clinical Practice Guidelines for the Medical Management of Graves’ Orbitopathy [published online ahead of print]. Eur J Endocrinol. July 1, 2021: EJE-21-0479.R1. doi: 10.1530/EJE-21-0479.

  5. McKeag D, et al. Clinical features of dysthyroid optic neuropathy: a survey of the European Graves Orbitopathy Group (EUGOGO). Br J Ophthalmol. 2007;91:455-458.

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