Findings from pivotal nipocalimab Phase 3 study in a broad antibody positive population of people living with generalized myasthenia gravis (gMG) published in The Lancet Neurology
Johnson & Johnson (NYSE: JNJ) announced the publication of Phase 3 study results for nipocalimab in The Lancet Neurology. The study focused on treating generalized myasthenia gravis (gMG) in antibody-positive adults. The trial met its primary endpoint, showing significant improvement in MG-ADL scores over 24 weeks.
Key findings include up to 75% reduction in median pre-dose total IgG from baseline, with sustained disease control over 24 weeks. The therapy demonstrated a tolerable safety profile, with discontinuation rates similar to placebo (5.1% vs 7.1%). Nipocalimab received FDA Priority Review and was recently granted Breakthrough Therapy Designation for Sjögren's disease treatment.
The company submitted Biologics License Application (BLA) to FDA and Marketing Authorisation Application (MAA) to EMA in August and September 2024, respectively, seeking approval for gMG treatment.
Johnson & Johnson (NYSE: JNJ) ha annunciato la pubblicazione dei risultati dello studio di Fase 3 per nipocalimab su The Lancet Neurology. Lo studio si è concentrato sul trattamento della miastenia gravis generalizzata (gMG) in adulti positivi agli anticorpi. La sperimentazione ha raggiunto il suo obiettivo primario, mostrando un significativo miglioramento nei punteggi MG-ADL dopo 24 settimane.
I risultati chiave includono una riduzione fino al 75% della media totale di IgG pre-dose rispetto al basale, con un controllo della malattia sostenuto per 24 settimane. La terapia ha dimostrato un profilo di sicurezza tollerabile, con tassi di interruzione simili a quelli del placebo (5,1% contro 7,1%). Nipocalimab ha ricevuto la Priorità di Revisione da parte della FDA ed è stato recentemente insignito della Designazione di Terapia Innovativa per il trattamento della malattia di Sjögren.
L'azienda ha presentato la Domanda di Licenza Biologica (BLA) alla FDA e la Domanda di Autorizzazione al Mercato (MAA) all'EMA ad agosto e settembre 2024, rispettivamente, per richiedere l'approvazione per il trattamento della gMG.
Johnson & Johnson (NYSE: JNJ) anunció la publicación de los resultados del estudio de Fase 3 para nipocalimab en The Lancet Neurology. El estudio se centró en el tratamiento de la miastenia gravis generalizada (gMG) en adultos positivos a anticuerpos. El ensayo alcanzó su objetivo principal, mostrando una mejora significativa en las puntuaciones de MG-ADL a lo largo de 24 semanas.
Los hallazgos clave incluyen una reducción de hasta el 75% en la media total de IgG pre-dosis desde el inicio, con un control sostenido de la enfermedad durante 24 semanas. La terapia demostró un perfil de seguridad tolerable, con tasas de interrupción similares al placebo (5,1% frente a 7,1%). Nipocalimab recibió una Revisión Prioritaria por parte de la FDA y fue recientemente otorgada la Designación de Terapia Innovadora para el tratamiento de la enfermedad de Sjögren.
La empresa presentó la Solicitud de Licencia Biológica (BLA) a la FDA y la Solicitud de Autorización de Comercialización (MAA) a la EMA en agosto y septiembre de 2024, respectivamente, buscando la aprobación para el tratamiento de gMG.
존슨 앤드 존슨 (NYSE: JNJ)은 The Lancet Neurology에 nipocalimab에 대한 3상 연구 결과를 발표했습니다. 이 연구는 항체 양성 성인의 일반화된 근무력증 (gMG) 치료에 중점을 두었습니다. 이 시험은 주요 목표를 달성했으며, 24주 동안 MG-ADL 점수가 유의미하게 개선되었습니다.
주요 결과에는 기준선에 비해 평균 총 IgG가 사전 투여 시점에서 최대 75% 감소하고, 24주 동안 질병 조절이 지속되었다는 내용이 포함되어 있습니다. 이 요법은 허용 가능한 안전성 프로필을 보여주었으며, 중단율은 위약과 유사했습니다 (5.1% 대 7.1%). Nipocalimab는 FDA의 우선 리뷰를 받았고 최근에는 쇼그렌 증후군 치료를 위한 혁신 치료 지정도 받았습니다.
회사는 FDA에 생물학적 라이선스 신청(BLA)을 제출하고, EMA에는 2024년 8월과 9월에 각각 상업화 허가 신청(MAA)을 제출하여 gMG 치료 승인을 요청했습니다.
Johnson & Johnson (NYSE: JNJ) a annoncé la publication des résultats de l'étude de Phase 3 pour nipocalimab dans The Lancet Neurology. L'étude s'est concentrée sur le traitement de la myasthénie grave généralisée (gMG) chez les adultes positifs pour les anticorps. L'essai a atteint son objectif principal, montrant une amélioration significative des scores MG-ADL sur 24 semaines.
Les résultats clés incluent une réduction allant jusqu'à 75 % de l'IgG total médian avant la dose par rapport à la ligne de base, avec un contrôle de la maladie maintenu pendant 24 semaines. La thérapie a démontré un profil de sécurité tolérable, avec des taux d'abandon similaires à ceux du placebo (5,1 % contre 7,1 %). Nipocalimab a reçu une Examen Prioritaire de la FDA et a récemment été attribué le statut de Thérapie Innovante pour le traitement de la maladie de Sjögren.
L'entreprise a soumis une Demande de Licence Biologique (BLA) à la FDA et une Demande d'Autorisation de Mise sur le Marché (MAA) à l'EMA en août et septembre 2024, respectivement, demandant l'approbation pour le traitement de la gMG.
Johnson & Johnson (NYSE: JNJ) gab die Veröffentlichung der Ergebnisse der Phase-3-Studie zu nipocalimab in The Lancet Neurology bekannt. Die Studie konzentrierte sich auf die Behandlung von generalisierter Myasthenia gravis (gMG) bei antikörperpositiven Erwachsenen. Die Studie erreichte ihren Primärapunkt und zeigte eine signifikante Verbesserung der MG-ADL-Werte über einen Zeitraum von 24 Wochen.
Wichtige Erkenntnisse umfassen eine Reduktion von bis zu 75 % des medianen Gesamt-IgG vor der Dosis im Vergleich zur Ausgangssituation, mit anhaltender Krankheitskontrolle über 24 Wochen. Die Therapie zeigte ein akzeptables Sicherheitsprofil, mit Abbruchraten, die vergleichbar mit denen des Placebos waren (5,1 % gegenüber 7,1 %). Nipocalimab erhielt von der FDA eine Prioritätsprüfung und wurde kürzlich mit der Bezeichnung „Durchbruchstherapie“ für die Behandlung von Sjögren-Syndrom ausgezeichnet.
Das Unternehmen reichte im August und September 2024 eine Biologics License Application (BLA) bei der FDA und eine Marketing Authorisation Application (MAA) bei der EMA ein, um die Genehmigung zur Behandlung von gMG zu beantragen.
- Phase 3 study met primary endpoint with statistically significant improvement
- Up to 75% reduction in median pre-dose total IgG from baseline
- FDA Priority Review status granted
- Low discontinuation rate (5.1%) comparable to placebo
- FDA Breakthrough Therapy Designation received for Sjögren's disease treatment
- None.
Insights
The publication of J&J's Phase 3 nipocalimab results in The Lancet Neurology represents a significant milestone in the treatment of generalized myasthenia gravis (gMG), a rare autoimmune disorder affecting approximately 700,000 people worldwide.
Breaking down the complex science into simpler terms: Think of gMG as your immune system mistakenly attacking the communication pathway between nerves and muscles. Nipocalimab works like a selective vacuum cleaner, removing harmful antibodies while preserving other important immune components. The study showed it can reduce these harmful antibodies by up to 75% while maintaining other protective antibodies intact.
From a commercial perspective, this development is particularly noteworthy for several reasons:
- The drug demonstrated efficacy across all antibody-positive patient types, potentially capturing a broader market segment than some existing treatments
- The FDA's Priority Review status could accelerate market entry, potentially giving J&J a competitive advantage
- The safety profile, with discontinuation rates similar to placebo (5.1% vs 7.1%), suggests potential for favorable insurance coverage and adoption
Looking at the broader implications for J&J's business strategy, nipocalimab's success extends beyond gMG. Its recent Breakthrough Therapy Designation for Sjögren's disease indicates potential for multiple revenue streams. This aligns with J&J's strategy to expand its presence in the lucrative rare disease market, estimated to reach $400 billion by 2030.
The clinical results are particularly compelling because they show sustained disease control over 24 weeks - a key differentiator in the market. For investors, this suggests potential for strong market positioning and sustained revenue growth in J&J's immunology portfolio.
The first FcRn blocker to demonstrate sustained disease control over 24 weeks in antibody positive adult patients: anti-AChR+, anti-MuSK+, anti-LRP4+
Nipocalimab demonstrated a sustained reduction in autoantibody levels, one of the underlying causes of gMG, by up to
The investigational therapy was recently granted
"Nipocalimab has been shown in multiple clinical studies to help reduce IgG, including autoantibodies, among this broad population of antibody positive adults with gMG. The positive results from the Vivacity-MG3 study further support the potential of nipocalimab to address the underlying cause of this debilitating autoantibody disease," said Carlo Antozzi, M.D., Neuroimmunology and Muscle Pathology Unit of the Neurological Institute Foundation C. Besta of
gMG is a chronic, life-long, rare, autoantibody-driven disease, for which there currently is no cure. gMG impacts an estimated 700,000 people worldwide.2,3 Nipocalimab, a fully human IgG1 antibody, is an immunoselective investigational therapy that has been shown in clinical trials to lower immunoglobulin G (IgG), including pathogenic IgG, one of the root causes of autoantibody diseases.1,4 Data from the Phase 3 study showed up to a
Nipocalimab plus standard of care (SOC) demonstrated a significantly greater reduction in MG-ADL response (≥2-point improvement from baseline) compared with placebo plus SOC (p=0.0213).1 For someone living with gMG, a 1- to 2-point change on MG-ADL may be the difference between normal eating and frequent choking on food, or shortness of breath at rest and requiring the assistance of a ventilator.5
"The Phase 3 Vivacity-MG3 data demonstrates our steadfast pursuit of researching and developing potential innovative and transformational approaches for autoantibody-driven diseases, such as gMG," said Sindhu Ramchandren, M.D., Executive Medical Director, Neuroscience, Johnson & Johnson Innovative Medicine. "We are delighted by the publication of this robust Phase 3 data in The Lancet Neurology as well as the Priority Review granted by the FDA. People living with gMG require additional effective immunoselective therapeutic options that can potentially preserve the ability to maintain a protective immune response even after reduction of IgG."
Johnson & Johnson submitted a Biologics License Application (BLA) to the
Editor's notes:
a. MG-ADL (Myasthenia Gravis – Activities of Daily Living) provides a rapid clinical assessment of the patient's recall of symptoms impacting activities of daily living, with a total score range of 0 to 24; a higher score indicates greater symptom severity.5 |
b. Dr. Carlo Antozzi has provided consulting, advisory and speaking services to Johnson & Johnson. He has not been paid for any media work. |
ABOUT GENERALIZED MYASTHENIA GRAVIS (gMG)
Myasthenia gravis (MG) is an autoantibody disease in which the immune system mistakenly makes antibodies (e.g., anti-acetylcholine receptor [AChR], anti-muscle-specific tyrosine kinase [MuSK] or anti-low density lipoprotein-related protein 4 [LRP4]), which target proteins at the neuromuscular junction and can block or disrupt normal signaling from nerves to muscles, thus impairing or preventing muscle contraction.2,9,10 The disease impacts an estimated 700,000 people worldwide.2 Approximately 10 to
Initial disease manifestations are usually ocular but in
ABOUT THE PHASE 3 VIVACITY-MG3 STUDY
The Phase 3 Vivacity-MG3 study (NCT04951622) was specifically designed to measure sustained efficacy and safety with consistent dosing in this unpredictable chronic condition where unmet need remains high. Antibody positive or negative adult gMG patients with insufficient response (MG-ADL ≥6) to ongoing SOC therapy were identified and 199 patients, 153 of whom were antibody positive, enrolled in the 24-week double-blind placebo-controlled trial.23,24 Randomization was 1:1, nipocalimab plus current SOC (30 mg/kg IV loading dose followed by 15 mg/kg every two weeks) or placebo plus current SOC.23 Baseline demographics were balanced across arms (77 nipocalimab, 76 placebo).23 The primary endpoint of the study was mean change in MG-ADLa score from baseline over Weeks 22, 23 and 24 in antibody positive patients. A key secondary endpoint included change in QMG score. Long-term safety and efficacy were further assessed in an ongoing open-label extension (OLE) phase.24
ABOUT NIPOCALIMAB
Nipocalimab is an investigational monoclonal antibody, designed to bind with high affinity to block FcRn and reduce levels of circulating immunoglobulin G (IgG) antibodies potentially without impact on other immune functions. This includes autoantibodies and alloantibodies that underlie multiple conditions across three key segments in the autoantibody space including Rare Autoantibody diseases, Maternal Fetal diseases mediated by maternal alloantibodies and Rheumatic diseases.25,26,27,28,29,30,31,32,33 Blockade of IgG binding to FcRn in the placenta is also believed to limit transplacental transfer of maternal alloantibodies to the fetus.34,35
The
U.S. FDA Fast Track designation in hemolytic disease of the fetus and newborn (HDFN) and warm autoimmune hemolytic anemia (wAIHA) in July 2019, gMG in December 2021 and fetal neonatal alloimmune thrombocytopenia (FNAIT) in March 2024U.S. FDA Orphan drug status for wAIHA in December 2019, HDFN in June 2020, gMG in February 2021, chronic inflammatory demyelinating polyneuropathy (CIDP) in October 2021 and FNAIT in December 2023U.S. FDA Breakthrough Therapy designation for HDFN in February 2024 and for Sjögren's disease in November 2024U.S. FDA granted Priority Review in gMG in Q4 2024- EU EMA Orphan medicinal product designation for HDFN in October 2019
ABOUT JOHNSON & JOHNSON
At Johnson & Johnson, we believe health is everything. Our strength in healthcare innovation empowers us to build a world where complex diseases are prevented, treated, and cured, where treatments are smarter and less invasive, and solutions are personal. Through our expertise in Innovative Medicine and MedTech, we are uniquely positioned to innovate across the full spectrum of healthcare solutions today to deliver the breakthroughs of tomorrow and profoundly impact health for humanity.
Learn more at https://www.jnj.com/ or at www.innovativemedicine.jnj.com
Follow us at @JNJInnovMed.
Janssen Research & Development, LLC and Janssen Biotech, Inc. are Johnson & Johnson companies.
CAUTIONS CONCERNING FORWARD-LOOKING STATEMENTS
This press release contains "forward-looking statements" as defined in the Private Securities Litigation Reform Act of 1995 regarding product development and the potential benefits and treatment impact of nipocalimab. The reader is cautioned not to rely on these forward-looking statements. These statements are based on current expectations of future events. If underlying assumptions prove inaccurate or known or unknown risks or uncertainties materialize, actual results could vary materially from the expectations and projections of Janssen Research & Development, LLC, Janssen Biotech, Inc. and/or Johnson & Johnson. Risks and uncertainties include, but are not limited to: challenges and uncertainties inherent in product research and development, including the uncertainty of clinical success and of obtaining regulatory approvals; uncertainty of commercial success; manufacturing difficulties and delays; competition, including technological advances, new products and patents attained by competitors; challenges to patents; product efficacy or safety concerns resulting in product recalls or regulatory action; changes in behavior and spending patterns of purchasers of health care products and services; changes to applicable laws and regulations, including global health care reforms; and trends toward health care cost containment. A further list and descriptions of these risks, uncertainties and other factors can be found in Johnson & Johnson's Annual Report on Form 10-K for the fiscal year ended December 31, 2023, including in the sections captioned "Cautionary Note Regarding Forward-Looking Statements" and "Item 1A. Risk Factors," and in Johnson & Johnson's subsequent Quarterly Reports on Form 10-Q and other filings with the Securities and Exchange Commission. Copies of these filings are available online at www.sec.gov, www.jnj.com or on request from Johnson & Johnson. None of Janssen Research & Development, LLC, Janssen Biotech, Inc. nor Johnson & Johnson undertakes to update any forward-looking statement as a result of new information or future events or developments.
1 Antozzi, C et al.,Efficacy and safety of nipocalimab in adults with generalised myasthenia gravis (Vivacity MG3): a randomised, double-blind, placebo-controlled phase 3 study. The Lancet Neurology. Feb 2025; 24: 105–16. https://www.thelancet.com/journals/laneur/article/PIIS1474-4422(24)00498-8/fulltext. |
2 Chen J, Tian D-C, Zhang C, et al. Incidence, mortality, and economic burden of myasthenia gravis in |
3 National Institute of Neurological Disorders and Stroke. Myasthenia Gravis. Available at: https://www.ninds.nih.gov/health-information/disorders/myasthenia-gravis. Last accessed: January 2025. |
4 Boes M, et al. Accelerated development of IgG autoantibodies and autoimmune disease in the absence of secreted IgM. Proc Natl Acad Sci U S A. 2000 Feb 1;97(3):1184-9. doi: 10.1073/pnas.97.3.1184. |
5 Wolfe GI Myasthenia gravis activities of daily living profile. Neurology. 1999;22;52(7):1487-9. doi: 10.1212/wnl.52.7.1487. |
6 Johnson & Johnson. Nipocalimab granted |
7 Johnson & Johnson EMEA. Johnson & Johnson seeks first EU approval of nipocalimab to treat a broad population of patients living with antibody-positive generalised myasthenia gravis. Available at: https://innovativemedicine.jnj.com/emea/newsroom/immunology/johnson-johnson-seeks-first-eu-approval-of-nipocalimab-to-treat-a-broad-population-of-patients-living-with-antibody-positive-generalised-myasthenia-gravis. Last accessed: January 2025. |
8 Johnson & Johnson. Nipocalimab is the first and only investigational therapy granted |
9 Bacci ED et al. Understanding side effects of therapy for myasthenia gravis and their impact on daily life. BMC Neurol. 2019;19(1):335. |
10 Wiendl, H., et al., Guideline for the management of myasthenic syndromes. Therapeutic advances in neurological disorders, 16, 17562864231213240. https://doi.org/10.1177/17562864231213240. Last accessed: January 2025 |
11 Evoli A, Batocchi AP, Bartoccioni E, Lino MM, Minisci C, Tonali P. Juvenile myasthenia gravis with prepubertal onset. Neuromuscul Disord. 1998 Dec;8(8):561-7. doi: 10.1016/s0960-8966(98)00077-7. |
12 Evoli A. Acquired myasthenia gravis in childhood. Curr Opin Neurol. 2010 Oct;23(5):536-40. doi: 10.1097/WCO.0b013e32833c32af. |
13 Finnis MF, Jayawant S. Juvenile myasthenia gravis: a paediatric perspective. Autoimmune Dis. 2011;2011:404101. doi: 10.4061/2011/404101. |
14 Haliloglu G, Anlar B, Aysun S, Topcu M, Topaloglu H, Turanli G, Yalnizoglu D. Gender prevalence in childhood multiple sclerosis and myasthenia gravis. J Child Neurol. 2002 May;17(5):390-2. doi: 10.1177/088307380201700516. |
15 Parr JR, Andrew MJ, Finnis M, Beeson D, Vincent A, Jayawant S. How common is childhood myasthenia? The |
16 Mansukhani SA, Bothun ED, Diehl NN, Mohney BG. Incidence and Ocular Features of Pediatric Myasthenias. Am J Ophthalmol. 2019 Apr;200:242-249. doi: 10.1016/j.ajo.2019.01.004. |
17 Bever, C.T., Jr, Aquino, A.V., Penn, A.S., Lovelace, R.E. and Rowland, L.P. (1983), Prognosis of ocular myasthenia. Ann Neurol., 14: 516-519. https://doi.org/10.1002/ana.410140504 |
18 Kupersmith MJ, Latkany R, Homel P. Development of generalized disease at 2 years in patients with ocular myasthenia gravis. Arch Neurol. 2003 Feb;60(2):243-8. doi: 10.1001/archneur.60.2.243. PMID: 12580710. |
19 Myasthenia gravis fact sheet. Retrieved April 2024 from https://www.ninds.nih.gov/sites/default/files/migrate-documents/myasthenia_gravis_e_march_2020_508c.pdf. |
20 Myasthenia Gravis: Treatment & Symptoms. (2021, April 7). Retrieved April 2024 from https://my.clevelandclinic.org/health/diseases/17252-myasthenia-gravis-mg. |
21 DRG EPI (2021) & Optum Claims Analysis Jan 2012-December 2020. |
22 O'Connell K, Ramdas S, Palace J. Management of Juvenile Myasthenia Gravis. Front Neurol. 2020 Jul 24;11:743. doi: 10.3389/fneur.2020.00743. PMID: 32793107; PMCID: PMC7393473. |
23 Antozzi, C et al., Nipocalimab in Generalized Myasthenia Gravis: Results from a Double-Blind, Placebo-Controlled, Randomized Phase 3 Study. Abstract at 2024 European Academy of Neurology Congress. June 2024. |
24 ClinicalTrials.gov Identifier: NCT04951622. Available at: https://clinicaltrials.gov/ct2/show/NCT04951622. Last accessed: January 2025 |
25 ClinicalTrials.gov Identifier: NCT04951622. Available at: https://clinicaltrials.gov/ct2/show/NCT04951622. Last accessed: January 2025 |
26 ClinicalTrials.gov. NCT03842189. Available at: https://clinicaltrials.gov/ct2/show/NCT03842189. Last accessed: January 2025 |
27 ClinicalTrials.gov Identifier: NCT05327114. Available at: https://www.clinicaltrials.gov/study/NCT05327114. Last accessed: January 2025 |
28 ClinicalTrials.gov Identifier: NCT04119050. Available at: https://clinicaltrials.gov/study/NCT04119050. Last accessed: January 2025 |
29 ClinicalTrials.gov Identifier: NCT05379634. Available at: https://clinicaltrials.gov/study/NCT05379634. Last accessed: January 2025. |
30 ClinicalTrials.gov Identifier: NCT05912517. Available at: https://www.clinicaltrials.gov/study/NCT05912517. Last accessed: January 2025 |
31 ClinicalTrials.gov Identifier: NCT06028438. Available at: https://clinicaltrials.gov/study/NCT06028438. Last accessed: January 2025 |
32 ClinicalTrials.gov Identifier: NCT04968912. Available at: https://clinicaltrials.gov/study/NCT04968912. Last accessed: January 2025 |
33 ClinicalTrials.gov Identifier: NCT04882878. Available at: https://clinicaltrials.gov/study/NCT04882878. Last accessed: January 2025. |
34 Lobato G, Soncini CS. Relationship between obstetric history and Rh(D) alloimmunization severity. Arch Gynecol Obstet. 2008 Mar;277(3):245-8. DOI: 10.1007/s00404-007-0446-x. |
35 Roy S, Nanovskaya T, Patrikeeva S, et al. M281, an anti-FcRn antibody, inhibits IgG transfer in a human ex vivo placental perfusion model. Am J Obstet Gynecol. 2019;220(5):498 e491-498 e499. |
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