-- Information to be featured in three oral presentations during the virtual conference --

Horizon Therapeutics plc (Nasdaq: HZNP) today announced the presentation of company information on Neuromyelitis Optica Spectrum Disorder (NMOSD) in three oral sessions during the 7th Congress of the European Academy of Neurology (EAN), June 19 -22, 2021. Presentations will discuss information from the open-label extension period (OLP) of the pivotal N-MOmentum trial for people living with NMOSD.

“NMOSD is a chronic disease that requires lifelong management,” said Quinn Dinh, M.D., vice president, international medical affairs and pipeline launch strategy. “The EAN Congress provides an important opportunity to engage with the international neurology community to advance the understanding and further increase awareness of this rare disease.”

Presentation Details:

• Title: Long term safety outcomes in neuromyelitis optica spectrum disorder: N-MOmentum trial
  • Session: OS3003, B. Cree
  • Date: June 21, 17:00-17:15 CEST, 11-11:15 a.m. EDT
• Title: Long term efficacy outcomes in neuromyelitis optica spectrum disorder: N-MOmentum trial
  • Session: OS3003, B. Cree
  • Date: June 21, 17:15-17:30 CEST, 11:15-11:30 a.m. EDT
• Title: Immunoglobulin kinetics and infection risk after long-term treatment for NMOSD
  • Session: OS3003, B. Greenberg
  • Date: June 21, 17:30-17:45 CEST, 11:30-11:45 a.m. EDT

About Neuromyelitis Optica Spectrum Disorder (NMOSD)

NMOSD is a unifying term for neuromyelitis optica (NMO) and related syndromes. NMOSD is a rare, severe, relapsing, neuroinflammatory autoimmune disease that attacks the optic nerve, spinal cord as well as the brain and brain stem.^1,2 Approximately 80 percent of all patients with NMOSD test positive for anti-AQP4 antibodies.³ AQP4-IgG bind primarily to astrocytes in the central nervous system and trigger an escalating immune response that results in lesion formation and astrocyte death.⁴

Anti-AQP4 autoantibodies are produced by plasmablasts and plasma cells. These B cell populations are central to NMOSD disease pathogenesis, and a large proportion of these cells express CD19. Depletion of these CD19+ B cells is thought to remove an important contributor to inflammation, lesion formation and astrocyte damage. Clinically, this damage presents as an NMOSD attack, which can involve the optic nerve, spinal cord and brain.^4,5 Loss of vision, paralysis, loss of sensation, bladder and bowel dysfunction, nerve pain and respiratory failure can all be manifestations of the disease.⁶ Each NMOSD attack can lead to further cumulative damage and disability.^7,8 NMOSD occurs more commonly in women and may be more common in individuals of African and Asian descent.^9,10

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.

References

1. Ajmera MR, Boscoe A, Mauskopf J, Candrilli SD, Levy M. Evaluation of comorbidities and health care resource use among patients with highly active neuromyelitis optica. J Neurol Sci. 2018; 384:96-103.
2. What is NMO? Guthyjacksonfoundation.org. www.guthyjacksonfoundation.org/neuromyelitis-optica-nmo/ Accessed April 15, 2021.
3. Layman’s Guide to NMO. Sumairafoundation.org. https://www.sumairafoundation.org/laymans-guide-to-nmo/ Accessed March 9, 2020.
4. Liu Y, et al. A tract-based diffusion study of cerebral white matter in neuromyelitis optica reveals widespread pathological alterations. Mult Scler. 2011;18(7);1013-1021.
5. Duan T, Smith AJ, Verkamn AS. Complement-independent bystander injury in AQP4-IgG seropositive neuromyelitis optica produced by antibody-dependent cellular cytotoxity. Acta Neuropathologica Comm. 2019;7(112).
6. Beekman J, et al. Neuromyelitis optica spectrum disorder: patient experience and quality of life. Neural Neuroimmunol Neuroinflamm. 2019;6(4):e580.
7. Kimbrough DJ, et al. Treatment of neuromyelitis optica: review and recommendations. Mult Scler Relat Disord. 2012;1(4):180-187.
8. Baranello RJ, Avasarala, JR. Neuromyelitis optica spectrum disorders with and without aquaporin 4 antibody: Characterization, differential diagnosis, and recent advances. J Neuro Ther. 2015;1(1):9-14.
9. Wingerchuk DM. Neuromyelitis optica: effect of gender. J Neurol Sci. 2009;286(1-2):18-23.
10. Flanagan EP, et al. Epidemiology of aquaporin-4 autoimmunity and neuromyelitis optica spectrum. Ann Neurol. 2016;79(5):775-783.

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