-- Separate analysis published in the Annals of Neurology assesses a potential new biomarker for NMOSD attack risk, severity and treatment effects --

Horizon Therapeutics plc (Nasdaq: HZNP) today announced the publication of a new analysis of the pivotal Phase 2/3 N-MOmentum trial for UPLIZNA (inebilizumab-cdon), assessing the potential for reduced risk of worsening disability in those living with NMOSD. These data are published in the May issue of Neurology Neuroimmunology & Neuroinflammation. UPLIZNA is the first and only FDA-approved anti-CD19 B-cell depleting humanized monoclonal antibody for the treatment of adult patients with anti-aquaporin-4 (AQP4) antibody positive NMOSD.

A separate analysis of the Phase 2/3 N-MOmentum trial published in the Annals of Neurology highlights a potential new biomarker of disease activity in NMOSD. Currently there is no blood test that can effectively monitor disease activity, severity of attacks or treatment impact for NMOSD patients. But research has indicated that serum glial fibrillary acidic protein (sGFAP) may be a relevant biomarker for the disease.

“Over the past few years, there has been remarkable progress in the scientific understanding and treatment of NMOSD that has improved patient outcomes,” said Quinn Dinh, M.D., vice president, medical affairs, Horizon. “These two publications assess the clinical impact of UPLIZNA on the challenging, progressive effects of NMOSD. Through continued research, we have an opportunity to further enhance our knowledge and ability to support the NMOSD community.”

About UPLIZNA

IMPORTANT SAFETY INFORMATION

UPLIZNA is contraindicated in patients with:

• A history of life-threatening infusion reaction to UPLIZNA
• Active hepatitis B infection
• Active or untreated latent tuberculosis

WARNINGS AND PRECAUTIONS

Infusion Reactions: UPLIZNA can cause infusion reactions, which can include headache, nausea, somnolence, dyspnea, fever, myalgia, rash, or other symptoms. Infusion reactions were most common with the first infusion but were also observed during subsequent infusions. Administer pre-medication with a corticosteroid, an antihistamine, and an anti-pyretic.

Infections: The most common infections reported by UPLIZNA-treated patients in the randomized and open-label periods included urinary tract infection (20%), nasopharyngitis (13%), upper respiratory tract infection (8%), and influenza (7%). Delay UPLIZNA administration in patients with an active infection until the infection is resolved.

Increased immunosuppressive effects are possible if combining UPLIZNA with another immunosuppressive therapy.

The risk of Hepatitis B Virus (HBV) reactivation has been observed with other B-cell-depleting antibodies. Perform HBV screening in all patients before initiation of treatment with UPLIZNA. Do not administer to patients with active hepatitis.

Although no confirmed cases of Progressive Multifocal Leukoencephalopathy (PML) were identified in UPLIZNA clinical trials, JC virus infection resulting in PML has been observed in patients treated with other B-cell-depleting antibodies and other therapies that affect immune competence. At the first sign or symptom suggestive of PML, withhold UPLIZNA and perform an appropriate diagnostic evaluation.

Patients should be evaluated for tuberculosis risk factors and tested for latent infection prior to initiating UPLIZNA.

Vaccination with live-attenuated or live vaccines is not recommended during treatment and after discontinuation, until B-cell repletion.

Reduction in Immunoglobulins: There may be a progressive and prolonged hypogammaglobulinemia or decline in the levels of total and individual immunoglobulins such as immunoglobulins G and M (IgG and IgM) with continued UPLIZNA treatment. Monitor the level of immunoglobulins at the beginning, during, and after discontinuation of treatment with UPLIZNA until B-cell repletion especially in patients with opportunistic or recurrent infections.

Fetal Risk: May cause fetal harm based on animal data. Advise females of reproductive potential of the potential risk to a fetus and to use an effective method of contraception during treatment and for 6 months after stopping UPLIZNA.

Adverse Reactions: The most common adverse reactions (at least 10% of patients treated with UPLIZNA and greater than placebo) were urinary tract infection and arthralgia.

For additional information on UPLIZNA, please see Prescribing Information at www.UPLIZNA.com.

About Neuromyelitis Optica Spectrum Disorder (NMOSD)

NMOSD is a rare, severe, autoimmune disease that attacks the optic nerve, spinal cord and brain stem, which leads to loss of vision and paralysis. In Japan, it has a low prevalence rate of 2 to 4 cases per 100,000 population.³ The disease is primarily associated with anti-aquaporin-4 (AQP4) antibodies, which are detected in approximately 73%-90% of patients with NMOSD.⁴

About Horizon

Horizon is focused on the discovery, development and commercialization of medicines that address critical needs for people impacted by rare, autoimmune and severe inflammatory diseases. Our pipeline is purposeful: 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, please visit www.horizontherapeutics.com and follow us on Twitter, LinkedIn, Instagram and Facebook.

Forward-Looking Statements

This press release contains forward-looking statements, including, but not limited to, statements related to the potential benefits of UPLIZNA and of sGFAP as a biomarker for NMOSD; 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 such forward-looking statements as a result of these risks and uncertainties, which include, without limitation, risks related to clinical trials, including the fact that prior results may not predict future clinical trial outcomes; regulatory obligations and oversight, including any changes in the legal and regulatory environment in which Horizon operates; Horizon’s ability to successfully commercialize UPLIZNA for NMOSD; and those risks detailed from time-to-time under the caption "Risk Factors" and elsewhere in Horizon’s filings and reports with the SEC. Horizon undertakes no duty or obligation to update any forward-looking statements contained in this press release as a result of new information.

References

1. Marignier R, et al. Disability Outcomes in the N-MOmentum Trial of Inebilizumab in Neuromyelitis Optica Spectrum Disorder. Neurol Neuroimmunol Neuroinflamm. 2021;8:e978. Available at: https://nn.neurology.org/content/8/3/e978.
2. Serum Glial Fibrillary Acidic Protein: A Neuromyelitis Optica Spectrum Disorder Biomarker. Ann Neurol. 2021;00:1-16. Available at: https://onlinelibrary.wiley.com/doi/full/10.1002/ana.26067.
3. Fujihara, Kazuo. 2017 Japanese guidelines for multiple sclerosis and neuromyelitis optica: Achievements and challenges. Clinical and Experimental Neuroimmunology. 2018;9. 10.1111/cen3.12450.
4. Chang VTW, Chang HM. Review: Recent advances in the understanding of the pathophysiology of neuromyelitis optica spectrum disorder. NAN. 2020;46:199-218.

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