La compañía biotecnológica Horizon Therapeutics plc ocupa el primer lugar en cuanto a integridad, accesibilidad y transparencia en el sector, según las asociaciones de pacientes

 

  Horizon Therapeutics plc ocupa el primer lugar en cuanto al rol central que ocupan los pacientes en su trabajo, su integridad, accesibilidad y transparencia, según los resultados de la encuesta anual de PatientView. Esta edición incluye a más de 2200 grupos de pacientes que evaluaron a más de 40 compañías biotecnológicas y farmacéuticas. Asimismo, Horizon ocupa el segundo lugar en cuanto a la reputación corporativa general entre estas mismas asociaciones. 

«Todo lo que hacemos en Horizon está motivado por facilitar el día a día de las personas que viven con enfermedades complejas y por los grupos de pacientes que les atienden», afirma Matt Flesch, vicepresidente de comunicaciones de productos y defensa de los pacientes de Horizon. «Tenemos en cuenta las aportaciones de las organizaciones de pacientes, desde el diseño de los ensayos clínicos hasta las iniciativas para la concienciación de estas enfermedades, y trabajamos para apoyar los programas que tienen el mayor impacto para ellos. En definitiva, tratamos de mejorar continuamente nuestro enfoque orientado a las necesidades de las comunidades de pacientes, y por eso los resultados positivos del informe de PatientView significan mucho para todos nosotros en Horizon».

 

En la encuesta de PatientView de 2022, 247 grupos de pacientes de todo el mundo afirmaron estar familiarizados con Horizon, un aumento de 67 grupos con respecto al 2021. Además, 130 grupos dijeron que habían trabajado con la compañía, un incremento de 43 grupos con respecto al 2021. Esta encuesta incluye 13 indicadores sobre reputación corporativa y, en cada uno de ellos, Horizon se situó entre los primeros puestos de las compañías farmacéuticas y biotecnológicas analizadas a nivel mundial.

 

Compromiso de Horizon con los pacientes y las asociaciones

 

• Organización de la primera cumbre de líderes emergentes en enfermedades autoinmunes raras, en la que participaron 26 representantes de pacientes en el ámbito de las enfermedades raras y autoinmunes para establecer sinergias y compartir aprendizajes entre las distintas organizaciones de pacientes.
• Primera exposición móvil sobre la enfermedad ocular tiroidea (TED, por sus siglas en inglés) en Atlanta (Georgia), con el objetivo de proporcionar recursos e información sobre esta patología a los asistentes, como parte del esfuerzo continuo de Horizon para favorecer la concienciación de este tipo de enfermedades minoritarias. 
• Encuentro de pacientes, profesionales sanitarios y representantes de las asociaciones para analizar los desafíos actuales a los que se enfrenta la comunidad con neuromielitis óptica (TENMO) y para intercambiar ideas y oportunidades para apoyar a estos pacientes.

• En Europa, organización de una exposición fotográfica para concienciar sobre el TENMO en el 17º Congreso mundial sobre controversias en neurología (CONy, por sus siglas en inglés): estos pacientes también asistieron al acto de apertura para hablar con los participantes del congreso sobre su experiencia con el TENMO.
• Apoyo a iniciativas de concienciación en Brasil sobre la importancia de la atención multidisciplinar para superar la carga social y económica de las enfermedades autoinmunes raras como los talleres artísticos «Fazendo Arte com Gustavo Rosa» para pacientes con TENMO y el cortometraje «Atrás dos Meus Olhos», que cuenta la historia de un paciente con TED.
• Único patrocinador nacional en la presentación del evento insignia de la Fundación contra la Artritis (Walk to Cure Arthritis), fomentando el voluntariado entre sus empleados y participando en más de 70 eventos.
• Avances en la campaña global #RAREis de Horizon, entre los que se incluyen:

• Programa internacional de subvenciones: Horizon ha otorgado 30 subvenciones de más de 4500 euros para apoyar a la comunidad de pacientes con enfermedades raras, proporcionando recursos a estas asociaciones para avanzar, educar y atender las necesidades de este colectivo.
• Fondo de becas: En asociación con la fundación EveryLife para enfermedades raras otorga una beca educativa única de más de 4500 euros a adultos que viven en los Estados Unidos con enfermedades raras. Hasta la fecha se han concedido 177 becas.
• Fondo de adopción: En asociación con Gift of Adoption, proporcionó ayuda a 54 niños que viven con enfermedades raras para ser adoptados.

 

New Analysis of UPLIZNA® (inebilizumab) Phase 3 Trial Data Demonstrates Importance of Reducing Plasmablasts to Help Prevent Neuromyelitis Optica Spectrum Disorder (NMOSD) Attacks

 

 

-- Analysis aims to clarify the relationship between peripheral B-cell subsets in the blood, AQP4-IgG levels and NMOSD attacks --

 

 Horizon Therapeutics plc (Nasdaq: HZNP) today announced new data from two analyses of the UPLIZNA Phase 3 pivotal trial being presented at the 38^th Congress of the European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS 2022), 26-28 October in Amsterdam. UPLIZNA is the first and only targeted B-cell depleting monotherapy approved by the European Commission and the U.S. Food and Drug Administration for the treatment of NMOSD in adults who are anti-aquaporin-4 immunoglobulin G seropositive (AQP4-IgG+). 

Plasmablasts and plasma cells are responsible for the secretion of pathogenic AQP4 antibodies, leading to NMOSD.¹ These data demonstrate how the mechanism of action of UPLIZNA effectively and distinctly addressed the underlying causes of the disease. ^2,3 UPLIZNA effectively depleted CD19+ B cells, including plasmablasts and plasma cells, which is believed to have a profound effect in controlling attacks in patients living with NMOSD.^2,3

Broad reduction of B cells, specifically plasmablasts, that correlate with NMOSD attacks
Plasmablasts and plasma cells (CD19+ expressing B cells) are considered a key driver of NMOSD attacks.¹ Until recently there has been limited knowledge about the association between attacks and b-cell subsets. This analysis from the N-MOmentum trial (NCT02200770) aimed to further clarify the relationship between peripheral B-cell subsets in the blood, AQP4-IgG levels and NMOSD attacks. To do so, absolute counts of CD20+ B cells and CD27+ memory B cells in the peripheral blood, plasma cell gene expression and AQP4-IgG titers were assessed.⁴

Increases in plasma cells were seen in over half (57%, 12/21) of placebo participants at time of attack relative to baseline compared to 20% (4/20) and 16% (3/19) for total CD20+ B cells and CD27+ memory B cells respectively.⁴ Increases in plasma cells were also observed at the preceding visit relative to baseline  (p < 0.01).⁴ No significant increases in any B cell subsets at time of attack were observed in participants treated with UPLIZNA relative to the preceding visit.⁴

Unexpectedly, in the placebo group, significant increases in AQP4+ titer were observed at time of attack relative to baseline (p = 0.02) but not in those treated with UPLIZNA (p=0.76); however, changes in
AQP4-IgG titer from baseline to attack were not significantly different between treatment groups (p = 0.15).⁴  Moreover, 85% of placebo participants had AQP4 titer increases and/or increased plasma cells, representing a potential attack signal.⁴

UPLIZNA significantly decreased AQP4+ titer relative to placebo.⁴ At the end of the randomised control period, 37% (59/159) of participants treated with UPLIZNA had a ≥2-fold decrease in AQP4-IgG titers from baseline compared to 18% (9/50) of those treated with placebo (p=0.014).⁴ For participants with high AQP4-IgG titers (>1:20,480), 51% (18/35) of participants treated with UPLIZNA had a ≥2-fold decrease in AQP4-IgG titers from baseline compared to 8% (1/12) of placebo participants.⁴

“Plasmablasts and plasma cells are thought to play a central role in the underlying physiological process associated with NMOSD, however the relationship between peripheral B-cell subsets in the blood, AQP4-IgG levels and NMOSD has not been widely studied,” said Professor Friedemann Paul, study author and Group Leader of the Clinical Neuroimmunology Department of the NeuroCure Clinical Research Centre at the Charité, Berlin. “These data aim to clarify this relationship and suggest that the critical mechanism of action of UPLIZNA effectively and distinctly treats NMOSD in patients who are AQP4-IgG+. Further studies may help elucidate the therapeutic impact associated with targeting CD19, particularly on plasmablasts and the associated reduction in AQP4-IgG, aimed at reducing attacks.”

Previous studies have shown that UPLIZNA targets an extended range of CD19 B cells, including plasmablasts and plasma cells, and reduces levels of plasmablasts as well as memory B cells.

“NMOSD is a devastating rare disease where just one attack can leave patients with severe, irreversible consequences like loss of sight or paralysis. This analysis offers a clearer picture of UPLIZNA’s differentiated mechanism contributing to improved clinical outcomes,” said Karl Boegl, M.D., executive director, EMEA, regional medical lead, Horizon Therapeutics. “This is important as it helps healthcare professionals make more accurate treatment decisions for their NMOSD patients.”

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, brain and brain stem.^5,6 Approximately 80 percent of all patients with NMOSD test positive for anti-AQP4 antibodies.⁷ AQP4-IgG binds primarily to astrocytes in the central nervous system and triggers an escalating immune response that results in lesion formation and astrocyte death.⁸

 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 CD19.⁹ 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.^8,9,10 Loss of vision, paralysis, loss of sensation, bladder and bowel dysfunction, nerve pain and respiratory failure can all be manifestations of the disease.¹⁰ Each NMOSD attack can lead to further cumulative damage and disability.^12,13 NMOSD occurs more commonly in women and may be more common in individuals of African and Asian descent.^14,15

New Analysis Finds UPLIZNA® (inebilizumab) Effective Among European Populations with Neuromyelitis Optica Spectrum Disorder (NMOSD)

 Horizon Therapeutics plc (Nasdaq: HZNP) today announced new findings from a post hoc analysis of the N-MOmentum Phase 3 pivotal trial of UPLIZNA supporting the medicine’s efficacy in Europeans living with NMOSD. These data are being presented during the 8 th Congress of the European Academy of Neurology (EAN), June 25-28 in Vienna. UPLIZNA received marketing authorization from the European Commission (EC) on April 25, 2022 and is the first and only targeted CD19+ B-celldepleting monotherapy proven to reduce attacks in adult patients with NMOSD who are anti-aquaporin-4 immunoglobulin G seropositive (AQP4- IgG+). 

This post hoc analysis compared attack rates, disability-related outcomes and safety among 50 trial participants from the European Union (EU) (including participants from Bulgaria, Czech Republic, Estonia, Germany, Hungary and Poland) versus 163 non-EU participants. “People living with NMOSD in Europe need novel treatment options that have been shown to reduce attacks that can cause irreversible and debilitating damage, such as vision loss and paralysis,” said Friedemann Paul, M.D., study author and head, Clinical Neuroimmunology Research Group, NeuroCure Clinical Research Center, and head, Clinical and Experimental Neuroimmunology, Experimental and Clinical Research Center, Charité–Universitätsmedizin Berlin and Max Delbrueck Center for Molecular Medine, Berlin, Germany. “With UPLIZNA, physicians have a treatment option that can be given twice a year after initial dosing to help prevent NMOSD attacks by specifically targeting CD19 B-cells, which play a central role in the pathogenesis of the disease.” 

Key analysis findings:

- Participants in the EU who were treated with UPLIZNA experienced fewer attacks (12.5%) compared to those treated with placebo (30%), sharing similar results with non-EU participants receiving UPLIZNA (10.7%) or placebo (45.2%). 

-No significant differences in Expanded Disability Status Scale (EDSS) worsening were found between participants in the EU (15%) versus non-EU participants (14.9%). 

-Fewer NMOSD-related hospitalisations were reported among those receiving UPLIZNA compared to those treated with placebo (mean, EU: 1.0 vs 2.0; non-EU: 1.0 vs 1.33). 

 “The UPLIZNA pivotal trial is the largest in NMOSD and clearly demonstrates the merits of targeting CD19 B-cells, including plasmablasts and plasma cells, to provide broad, deep and durable B-cell depletion,” said Karl Boegl, M.D. Ph.D., executive director, EMEA regional medical affairs lead, Horizon. “We believe these data provide treating physicians with greater certainty that a targeted monotherapy like UPLIZNA can be a valuable option for the treatment of NMOSD patients in Europe.”