argenx Advances Clinical Development of Efgartigimod SC in Idiopathic Inflammatory Myopathies
Argenx announced the continuation of efgartigimod SC development in the Phase 2/3 ALKIVIA study for idiopathic inflammatory myopathies (IIM) following successful Phase 2 results. The study met its primary endpoint, showing statistically significant improvement in total improvement score (TIS) at Week 24 compared to placebo. The trial will continue enrolling patients across three myositis subtypes: immune-mediated necrotizing myopathy (IMNM), anti-synthetase syndrome (ASyS), and dermatomyositis (DM). The ALKIVIA study aims to enroll 240 patients total, with safety and tolerability profile consistent with previous clinical trials.
Argenx ha annunciato il proseguimento dello sviluppo di efgartigimod SC nello studio di Fase 2/3 ALKIVIA per le miopatie infiammatorie idiopatiche (IIM), a seguito di risultati positivi della Fase 2. Lo studio ha raggiunto il suo obiettivo principale, mostrando un miglioramento statisticamente significativo nel punteggio totale di miglioramento (TIS) alla settimana 24 rispetto al placebo. La sperimentazione continuerà ad arruolare pazienti attraverso tre sottotipi di miostite: miopatia necrotizzante immune-mediata (IMNM), sindrome anti-sintetasi (ASyS) e dermatomiosite (DM). Lo studio ALKIVIA mira ad arruolare un totale di 240 pazienti, con un profilo di sicurezza e tollerabilità coerente con studi clinici precedenti.
Argenx anunció la continuación del desarrollo de efgartigimod SC en el estudio de Fase 2/3 ALKIVIA para las miopatías inflamatorias idiopáticas (IIM) tras los exitosos resultados de la Fase 2. El estudio cumplió con su objetivo principal, mostrando una mejora estadísticamente significativa en la puntuación total de mejora (TIS) a la semana 24 en comparación con el placebo. El ensayo continuará reclutando pacientes en tres subtipos de miositis: miopatía necrotizante inmunomediada (IMNM), síndrome anti-sintetasa (ASyS) y dermatomiositis (DM). El estudio ALKIVIA tiene como objetivo reclutar un total de 240 pacientes, con un perfil de seguridad y tolerabilidad consistente con ensayos clínicos previos.
Argenx는 efgartigimod SC의 개발을 특发성염증근육병(IIM)에 대한 2/3 상 ALKIVIA 연구에서 계속한다고 발표했습니다. 이 연구는 2상 결과가 성공적이었고, 제 1차 목표를 달성하여 24주차에 위약 대비 총 개선 점수(TIS)에서 통계적으로 유의미한 개선을 보여주었습니다. 이 시험은 세 가지 미오시스 하위 유형인 면역 매개 괴사성 근염(IMNM), 항합성효소 증후군(ASyS), 피부근염(DM)에서 환자를 계속 모집할 예정입니다. ALKIVIA 연구는 총 240명의 환자를 모집할 계획이며, 이전의 임상 시험과 일관된 안전성 및 내약성 프로필을 가지고 있습니다.
Argenx a annoncé la poursuite du développement de efgartigimod SC dans l'étude de Phase 2/3 ALKIVIA pour les myopathies inflammatoires idiopathiques (IIM) suite aux réussites des résultats de Phase 2. L'étude a atteint son objectif principal, montrant une amélioration statistiquement significative du score d'amélioration total (TIS) à la semaine 24 par rapport au placebo. L'essai continuera de recruter des patients dans trois sous-types de myosite : myopathie nécrosante à médiation immunitaire (IMNM), syndrome anti-synthétase (ASyS) et dermatomyosite (DM). L'étude ALKIVIA vise à recruter un total de 240 patients, avec un profil de sécurité et de tolérabilité cohérent avec les essais cliniques précédents.
Argenx hat die Fortsetzung der Entwicklung von efgartigimod SC in der Phase 2/3-Studie ALKIVIA für idiopathische inflammatory Myopathien (IIM) nach erfolgreichen Ergebnissen der Phase 2 bekannt gegeben. Die Studie erreichte ihr primäres Ziel und zeigte eine statistisch signifikante Verbesserung des Gesamtverbesserungspunkts (TIS) in der Woche 24 im Vergleich zur Placebogruppe. Die Studie wird weiterhin Patienten aus drei Myositis-Subtypen rekrutieren: immunvermittelte nekrotisierende Myopathie (IMNM), Anti-Synthetase-Syndrom (ASyS) und Dermatomyositis (DM). Die ALKIVIA-Studie zielt darauf ab, insgesamt 240 Patienten zu rekrutieren, wobei das Sicherheits- und Verträglichkeitsprofil mit früheren klinischen Studien konsistent ist.
- Met primary endpoint with statistically significant treatment effect
- Showed improvement across all six core set measures
- Safety and tolerability profile consistent with previous trials
- Potential to be first targeted treatment for myositis
- None.
Insights
The Phase 2 ALKIVIA trial results mark a significant milestone for argenx's efgartigimod SC in myositis treatment. The study's success in meeting its primary endpoint with statistical significance across all three subtypes (IMNM, ASyS and DM) represents a major advancement in the $33.6B market cap company's pipeline.
The trial's positive outcome in the Total Improvement Score (TIS) at Week 24 is particularly noteworthy given the current treatment landscape for myositis. The seamless transition to Phase 3, maintaining enrollment across all subtypes, indicates strong confidence in the drug's potential. This development could position efgartigimod SC as the first targeted therapy for myositis, addressing an significant unmet medical need in a market currently dominated by steroids and broad immunosuppressants.
This clinical advancement significantly strengthens argenx's market position in the autoimmune disease space. The potential market opportunity is substantial, as myositis represents an underserved indication with treatment options. Success in this indication would expand efgartigimod's commercial potential beyond its existing approvals, potentially driving future revenue growth.
The positive Phase 2 data and continuation into Phase 3 reduces development risk and enhances the probability of eventual regulatory approval. With a planned total enrollment of 240 patients, the robust study design adds credibility to the development program and could support a strong commercial launch if approved.
- Phase 2 data establish proof-of-concept of efgartigimod SC in myositis
- Enrollment to continue in Phase 3 across all three subtypes (IMNM, ASyS, DM) under evaluation in ALKIVIA
- Potential for efgartigimod SC to be first targeted approach for myositis patients who have limited treatment options
November 20, 2024, 7:00 AM CET
Amsterdam, the Netherlands – argenx SE (Euronext & Nasdaq: ARGX), a global immunology company committed to improving the lives of people suffering from severe autoimmune diseases, today announced the decision to continue development of efgartigimod subcutaneous (SC) (efgartigimod alfa and hyaluronidase-qvfc) in the ongoing Phase 2/3 ALKIVIA study in adults with idiopathic inflammatory myopathies (IIM or myositis), following analysis of topline data from the Phase 2 portion of the study. ALKIVIA will continue to enroll patients across each of the three myositis subtypes in the study, including immune-mediated necrotizing myopathy (IMNM), anti-synthetase syndrome (ASyS), and dermatomyositis (DM).
“Efgartigimod SC continues to show its promise for patients suffering from chronic autoimmune diseases,” said Luc Truyen, M.D., Ph.D., Chief Medical Officer of argenx. “Idiopathic inflammatory myopathies are debilitating diseases that can cause muscle weakness, affect multiple organs, and have a severe impact on patients’ quality of life, including increased morbidity and early mortality. We are excited to continue the development of efgartigimod SC across all three subtypes, allowing us to explore the broad potential of this precision therapy for those whose needs remain unmet by current treatments like steroids, plasma-derived therapies, and broad immunosuppressants. We are grateful for the patients and investigators participating in the ALKIVIA study, and hope to bring efgartigimod to patients living with myositis as soon as possible.”
The decision to continue clinical development of efgartigimod SC in each of the three myositis subtypes is supported by the efficacy and safety results from the Phase 2 portion of the seamless Phase 2/3 ALKIVIA study. Overall, the study met its primary endpoint, demonstrating a statistically significant treatment effect in mean total improvement score (TIS) at Week 24, and showed improvement across all six core set measures of the TIS in favor of efgartigimod SC compared to placebo. The observed safety and tolerability profile was consistent to that demonstrated with other clinical trials.
ALKIVIA Study Design
The ALKIVIA study is a randomized, double-blind, placebo-controlled, multicenter, operationally seamless Phase 2/3 study of efgartigimod SC for the treatment of idiopathic inflammatory myopathies (IIM or myositis) across three subtypes, including immune-mediated necrotizing myopathy (IMNM), anti-synthetase syndrome (ASyS), and dermatomyositis (DM). The ALKIVIA study will enroll 240 patients in total and is being conducted in two phases, with an analysis of the Phase 2 portion of the clinical trial after the first 90 patients completed the study, followed by a Phase 3 portion if a signal is observed in the Phase 2 portion. The primary endpoint is the mean total improvement score (TIS) at the end of the treatment period (24 weeks in Phase 2 and 52 weeks in Phase 3) of all treated patients (IMNM, ASyS, DM) compared to placebo. Key secondary endpoints include response rates at the end of treatment, time to response, and duration of response in TIS, as well as change from baseline in individual TIS components. Other secondary endpoints include quality of life and other functional scores.
About Idiopathic Inflammatory Myopathies
Idiopathic inflammatory myopathies (myositis) are a rare group of autoimmune diseases that can be muscle specific or affect multiple organs including the skin, joints, lungs, gastrointestinal tract and heart. Myositis can be very severe and disabling and have a material impact on quality of life. Initially, myositis was classified as either DM or polymyositis, but as the underlying pathophysiology of myositis has become better understood, including through the identification of characteristic autoantibodies, new polymyositis subtypes have emerged. Two of these subtypes are IMNM and ASyS. Proximal muscle weakness is a unifying feature of each subtype. IMNM is characterized by skeletal muscle weakness due to muscle cell necrosis. ASyS is characterized by muscle inflammation, inflammatory arthritis, interstitial lung disease, thickening and cracking of the hands (“mechanic’s hands”) and Raynaud’s phenomenon. DM is characterized by muscle inflammation and degeneration and skin abnormalities, including heliotrope rash, Gottron’s papules, erythematous, calcinosis and edema.
About Efgartigimod SC
Efgartigimod SC (efgartigimod alfa and hyaluronidase-qvfc) is a human IgG1 antibody fragment designed to reduce pathogenic immunoglobulin G (IgG) antibodies by binding to the neonatal Fc receptor (FcRn) and blocking the IgG recycling process. Efgartigimod SC is the first-approved FcRn blocker globally and is marketed as VYVGART® Hytrulo in the United States and China for the treatment of generalized myasthenia gravis (gMG) and chronic inflammatory demyelinating polyneuropathy (CIDP), and as VYVGART SC or VYVDURA (Japan) for gMG in other regions globally. Efgartigimod SC is currently being evaluated in more than 15 severe autoimmune diseases where pathogenic IgGs are believed to be mediators of disease.
About argenx
argenx is a global immunology company committed to improving the lives of people suffering from severe autoimmune diseases. Partnering with leading academic researchers through its Immunology Innovation Program (IIP), argenx aims to translate immunology breakthroughs into a world-class portfolio of novel antibody-based medicines. argenx developed and is commercializing the first approved neonatal Fc receptor (FcRn) blocker, globally in the U.S., Japan, Israel, the EU, the UK, China and Canada. The Company is evaluating efgartigimod in multiple serious autoimmune diseases and advancing several earlier stage experimental medicines within its therapeutic franchises. For more information, visit www.argenx.com and follow us on LinkedIn, X/Twitter, Instagram, Facebook, and YouTube.
Media:
Ben Petok
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Investors:
Alexandra Roy (US)
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Forward Looking Statements
The contents of this announcement include statements that are, or may be deemed to be, “forward-looking statements.” These forward-looking statements can be identified by the use of forward-looking terminology, including the terms “aim,” “continue,” “hope,” “potential,” or “will,” and include statements argenx makes concerning its continued development of efgartigimod SC (efgartigimod alfa and hyaluronidase-qvfc) in the ongoing Phase 2/3 ALKIVIA study; its plan to continue enrollment of patients across all three myositis subtypes (IMNM, ASyS, DM) under evaluation in the ALKIVIA study; the potential of efgartigimod SC to be the first targeted treatment approach for myositis patients who have limited other treatment options and whose needs remain unmet by current treatments; its hope to bring efgartigimod to patients living with myositis as soon as possible; its plan for the study design of the ALKIVIA study; and its goal of translating immunology breakthroughs into a world-class portfolio of novel antibody-based medicines. By their nature, forward-looking statements involve risks and uncertainties and readers are cautioned that any such forward-looking statements are not guarantees of future performance. argenx’s actual results may differ materially from those predicted by the forward-looking statements as a result of various important factors, including the results of argenx's clinical trials; expectations regarding the inherent uncertainties associated with the development of novel drug therapies; preclinical and clinical trial and product development activities and regulatory approval requirements in products and product candidates; the acceptance of argenx's products and product candidates by patients as safe, effective and cost-effective; the impact of governmental laws and regulations on our business; disruptions caused on our reliance of third parties suppliers, service provides and manufacturing; inflation and deflation and the corresponding fluctuations in interest rates; and regional instability and conflicts. A further list and description of these risks, uncertainties and other risks can be found in argenx’s U.S. Securities and Exchange Commission (SEC) filings and reports, including in argenx’s most recent annual report on Form 20-F filed with the SEC as well as subsequent filings and reports filed by argenx with the SEC. Given these uncertainties, the reader is advised not to place any undue reliance on such forward-looking statements. These forward-looking statements speak only as of the date of publication of this document. argenx undertakes no obligation to publicly update or revise the information in this press release, including any forward-looking statements, except as may be required by law.
FAQ
What were the Phase 2 results of efgartigimod SC in the ALKIVIA trial (ARGX)?
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