LRP4

First FcRn blocker to demonstrate sustained disease control over 24 weeks in antibody positive adolescents aged 12 – 17 years, broadening the population in which nipocalimab has been studied SAVANNAH, Ga., Oct. 15, 2024 /PRNewswire/ -- Johnson & Johnson (NYSE: JNJ) today announced positive results from the Phase 2/3 Vibrance-MG study of nipocalimab in anti-AChRa positive adolescents (aged 12 – 17 years) living with generalized myasthenia gravis (gMG). Study participants who were treated with nipocalimab plus standard of care (SOC) achieved sustained disease control as measured by the primary endpoint of immunoglobulin G (IgG) reduction from baseline over 24 weeks, and secondary endpoints of improvement in MG-ADLb and QMGc scores. These Phase 2/3 data will be featured in an oral presentation (Abstract #MG100) at the Myasthenia Gravis Foundation of America (MGFA) Scientific Session during the American Association of Neuromuscular & Electrodiagnostic Medicine (AANEM) Annual Meeting, where Johnson & Johnson will present 25 abstracts. Experience the full interactive Multichannel News Release here: "Findings from the Vibrance-MG study underscore the potential of this investigational therapy for young individuals aged 12 – 17 living with gMG. Results show a significant reduction in IgG of approximately 70% in adolescents and a clinical benefit that is consistent with the Vivacity-MG3 study in adults," said Jonathan Strober, M.D., Director of Clinical Services for Child Neurology and Director of the Muscular Dystrophy Clinic at UCSF Benioff Children's Hospital.d "It is encouraging to see these positive results as there are currently no approved advanced treatment options for this adolescent population in the United States." About 10% of new cases of myasthenia gravis are diagnosed in adolescents (12 – 17 years of age) and the severity of gMG in pediatric patients is heightened with 43% having experienced over five hospitalizations in their lifetime, 46% having at least one intensive care unit stay and 68% having periods of exacerbated disease.1,2,3,4 Treatment with nipocalimab plus SOC met the study's primary endpoint of reduction in total serum IgG (-69%), and the two secondary endpoints of MG-ADL and QMG, which are measures of disease activity.5,e Four of five patients achieved minimum symptom expression (MG-ADL score 0-1) by the end of their treatment phase.f,g Nipocalimab was well-tolerated over the six-month period, similar to tolerability seen in adult participants in the Vivacity-MG3 study.5 There were no serious adverse events and no discontinuations due to an adverse event. Presented for the first time, these open-label Phase 2/3 results in adolescents are consistent with findings from the pivotal study of nipocalimab in adult patients with gMG. Nipocalimab when added to SOC is the first FcRn blocker to demonstrate sustained disease control in a registrational trial as measured by improvement in MG-ADL over placebo plus SOC over a period of six months of consistent dosing (Q2 week) among adults living with gMG. "The Vibrance-MG data add to the expanding clinical profile of nipocalimab and highlight its potential for adolescents living with gMG who are in need of new treatments," said Sindhu Ramchandren, M.D., Executive Medical Director, Neuroscience, Johnson & Johnson Innovative Medicine. "We are committed to developing innovations for autoantibody-driven neurological diseases, like gMG, with the aim of transforming the lives of people living with these conditions." Earlier this year, Johnson & Johnson announced the submission of applications to the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) seeking approval for nipocalimab for the treatment of gMG. Editor's notes: a. Patients with a positive blood test for acetylcholine receptor (anti-AChR) antibodies or muscle-specific tyrosine kinase (anti-MuSK) antibodies are eligible for the study. b. 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. c. QMG (Quantitative Myasthenia Gravis) is a 13-item assessment by a clinician that quantifies MG disease severity through muscle weakness. The total QMG score ranges from 0 to 39, where higher scores indicated greater disease severity. d. Dr. Jonathan Strober is a paid consultant for Johnson & Johnson. He has not been compensated for any media work. e. Treatment with nipocalimab showed a mean percentage change from baseline to week 24 for total serum IgG of -68.98% (standard error [SE] = 7.561). f. Adolescents who received nipocalimab plus current SOC had a mean baseline score of 4.29 (SE = 2.430) on the MG-ADL scale and a mean baseline score of 12.50 (SE = 3.708) on the QMG scale. g. Adolescents who received nipocalimab plus current SOC had a mean change at week 24 of -2.40 (SE = 0.187) on the MG-ADL scale and -3.80 (SE = 2.683) on the QMG scale. 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. 6,7 The disease impacts an estimated 700,000 people worldwide.6 Approximately 10 to 15% of new cases of MG are diagnosed in adolescents (12 – 17 years of age).1,2,3 Among juvenile MG patients, girls are affected more often than boys with over 65% of pediatric MG cases in the US diagnosed in girls.8,9,10 Initial disease manifestations are usually ocular but in 85% or more cases, the disease generalizes (gMG), which is characterized by fluctuating weakness of the skeletal muscles leading to symptoms like limb weakness, drooping eyelids, double vision and difficulties with chewing, swallowing, speech, and breathing.6,11,12,13,14 Approximately 100,000 individuals in the U.S. are living with gMG.15 Vulnerable gMG populations, such as pediatric patients, have more limited therapeutic options.3 Currently, SOC treatments for adolescents with gMG are extrapolated from adult trials.3 Other than symptomatic treatments, there are no approved FcRn blockers that may address the root cause of the disease for adolescents with gMG in the United States.3 About the Phase 2/3 Vibrance-MG Study The Phase 2/3 Vibrance-MG study (NCT05265273) is an on-going open-label study to determine the effect of nipocalimab in pediatric participants with gMG.16 Seven participants aged 12 – 17 years with a diagnosis of gMG as reflected by a Myasthenia Gravis Foundation of America (MGFA) Class of II through IV at screening, and an insufficient clinical response to ongoing, stable SOC therapy, have been enrolled in the trial.5 Participants must have a positive blood test for either anti-AChR or anti-MUSK autoantibodies. The study consists of a screening period of up to four weeks, a 24-week open-label Active Treatment Phase during which participants receive nipocalimab intravenously every two weeks, and a Long-term Extension Phase; a safety follow-up assessment will be conducted at eight weeks after last dose.16 The primary outcome of the study is the effect of nipocalimab on total serum IgG, safety and tolerability, and pharmacokinetics in pediatric participants with gMG at 24 weeks. Secondary endpoints include change in MG-ADL and QMG scores at 24 weeks.5,16 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 Prevalent Rheumatology.17,18,19,20,21,22,23,24,25 Blockade of IgG binding to FcRn in the placenta is also believed to limit transplacental transfer of maternal alloantibodies to the fetus.26,27 The U.S. Food and Drug Administration (FDA) and European Medicines Agency (EMA) have granted several key designations to nipocalimab including: 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 2024 U.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 2023 U.S. FDA Breakthrough Therapy designation for HDFN in February 2024 by the FDA 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 or at Follow us at @JanssenUS and @JNJInnovMed. Janssen Research & Development, LLC and Janssen Biotech, Inc. are both 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 , 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 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. 2 Evoli A. Acquired myasthenia gravis in childhood. Curr Opin Neurol. 2010 Oct;23(5):536-40. doi: 10.1097/WCO.0b013e32833c32af. 3 Finnis MF, Jayawant S. Juvenile myasthenia gravis: a paediatric perspective. Autoimmune Dis. 2011;2011:404101. doi: 10.4061/2011/404101. 4 Barraud C, Desguerre I, Barnerias C, Gitiaux C, Boulay C, Chabrol B. Clinical features and evolution of juvenile myasthenia gravis in a French cohort. Muscle Nerve. 2018 Apr;57(4):603-609. doi: 10.1002/mus.25965. 5 Strober J et al. Safety and effectiveness of nipocalimab in adolescent participants in the open label Phase 2/3 Vibrance-MG clinical study. Presentation at American Association of Neuromuscular & Electrodiagnostic Medicine (AANEM) Annual Meeting. October 2024. 6 Chen J, Tian D-C, Zhang C, et al. Incidence, mortality, and economic burden of myasthenia gravis in China: A nationwide population-based study. The Lancet Regional Health - Western Pacific. 7 Wiendl, H., et al., Guideline for the management of myasthenic syndromes. Therapeutic advances in neurological disorders, 16, 17562864231213240. . Last Accessed: October 2024. 8 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. 9 Parr JR, Andrew MJ, Finnis M, Beeson D, Vincent A, Jayawant S. How common is childhood myasthenia? The UK incidence and prevalence of autoimmune and congenital myasthenia. Arch Dis Child. 2014 Jun;99(6):539-42. doi: 10.1136/archdischild-2013-304788. 10 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. 11 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. 12 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. 13 Myasthenia gravis fact sheet. Retrieved April 2024 from . 14 Myasthenia Gravis: Treatment & Symptoms. (2021, April 7). Retrieved April 2024 from . 15 DRG EPI (2021) & Optum Claims Analysis Jan 2012-December 2020. 16 ClinicalTrials.gov. NCT05265273. Available at: . Last accessed: October 2024 17 ClinicalTrials.gov Identifier: NCT04951622. Available at: . Last accessed: October 2024. 18 ClinicalTrials.gov. NCT03842189. Available at: . Last accessed: October 2024 19 ClinicalTrials.gov Identifier: NCT05327114. Available at: . Last accessed: October 2024 20 ClinicalTrials.gov Identifier: NCT04119050. Available at: . Last accessed: October 2024. 21 ClinicalTrials.gov Identifier: NCT05379634. Available at: . Last accessed: October 2024. 22 ClinicalTrials.gov Identifier: NCT05912517. Available at: . Last accessed: October 2024 23 ClinicalTrials.gov Identifier: NCT06028438. Available at: . Last accessed: October 2024. 24 ClinicalTrials.gov Identifier: NCT04968912. Available at: . Last accessed: October 2024. 25 ClinicalTrials.gov Identifier: NCT04882878. Available at: . Last accessed: October 2024. 26 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. Last accessed: October 2024. 27 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. SOURCE Johnson & Johnson WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Marks first EMA submission for nipocalimab, an investigational treatment that binds with high affinity and specificity to block FcRn and reduce levels of autoantibodies Filing based on the Phase 3 Vivacity-MG3 programme, the first registrational study results in the class demonstrating sustained disease control over 24 weeks in antibody-positive adult patients: anti-AChR+, anti-MuSK+, anti-LRP4+ BEERSE, Belgium I September 12, 2024 I Janssen-Cilag International NV, a Johnson & Johnson company, today announced the submission of the Marketing Authorisation Application (MAA) to the European Medicines Agency (EMA) seeking first approval of nipocalimab for the treatment of people living with generalised myasthenia gravis (gMG). The application included data from the Phase 3 Vivacity-MG3 study which showed that outcomes for a broad population of antibody-positive participants who received nipocalimab plus standard of care (SOC) were superior compared to those who received placebo plus SOC. 1 The primary endpoint of the study measured improvement in the Myasthenia Gravis – Activities of Daily Living (MG-ADL) a score from baseline over 24 weeks and study participants included anti-AChR+, anti-MuSK+, and anti-LRP4+ b antibody-positive adults, which account for approximately 95 percent of the gMG patient population, making Vivacity-MG3 the first registrational study to demonstrate sustained disease control in these subtypes. 1,2 Safety and tolerability were consistent with other nipocalimab studies. 3,4,5,c “We are encouraged by the potential of nipocalimab to provide sustained disease control for people living with generalised myasthenia gravis, a chronic, life-long disease,” said Bill Martin, Ph.D., Global Therapeutic Area Head, Neuroscience, Johnson & Johnson Innovative Medicine. “The filing for approval of nipocalimab represents an important step forward as Johnson & Johnson continues to push the boundaries of research to develop innovative solutions to treat autoantibody-driven diseases, building on decades of expertise in neuroscience and immunology. We look forward to working with the EMA in their review of the data supporting the submission.” Nipocalimab is the first FcRn blocker to demonstrate sustained disease control measured by improvement in MG-ADL when added to background SOC compared with placebo plus SOC over a period of six months of consistent dosing (every other week). 1,6,d Editor’s notes: About Generalised Myasthenia Gravis (gMG) Myasthenia gravis (MG) is an autoantibody disease in which autoantibodies target proteins at the neuromuscular junction, disrupt neuromuscular signalling, and impair or prevent muscle contraction. 8,9 In MG, the immune system mistakenly attacks proteins at the neuromuscular junction, e.g., anti-acetylcholine receptor [AChR], anti-muscle-specific tyrosine kinase [MuSK] or anti-lipoprotein-related protein 4 [LRP4], that can block or disrupt normal functioning, preventing signals from transferring from nerves to muscles. 10 The disease impacts between 56,000 and 123,000 people in Europe and an estimated 700,000 people worldwide. 8,11 The disease affects both men and women and occurs across all ages, racial and ethnic groups, but most frequently impacts young women and older men. 12 Roughly 50 percent of individuals diagnosed with MG are women, and about one in five of those women are of child-bearing potential. 13,14,15 Initial disease manifestations are usually ocular but in 85 percent or more the disease generalises (gMG), which is characterised by fluctuating weakness of the skeletal muscles leading to symptoms like limb weakness, drooping eyelids, double vision and difficulties with chewing, swallowing, speech, and breathing. 9,16,17 Although gMG may be managed with current SOC therapies, research is needed to develop new treatments for those who may not respond well enough to or tolerate these options. 9 About the Phase 3 Vivacity-MG3 Study The Phase 3 Vivacity-MG3 study 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 standard of care (SOC) therapy were identified and 199 patients, 153 of which were antibody positive, enrolled in the 24-week double-blind placebo-controlled trial. 18,19 Randomisation 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. 19 Baseline demographics were balanced across arms (77 nipocalimab, 76 placebo). 19 The primary endpoint of the study was mean change in MG-ADL b score from baseline over Weeks 22, 23 and 24 in antibody-positive patients. A key secondary endpoint included change in Quantitative Myasthenia Gravis (QMG) score, which is a 13-item assessment by a clinician that quantifies MG disease severity. Long-term safety and efficacy were further assessed in an ongoing OLE phase. 18 About Nipocalimab Nipocalimab is an investigational monoclonal antibody, purposefully designed to bind with high affinity to block FcRn and reduce levels of circulating immunoglobulin G (IgG) antibodies, while preserving immune function without causing broad immunosuppression. 20 This includes autoantibodies and alloantibodies that underlie multiple conditions across three key segments in the autoantibody space including Rare Autoantibody diseases, Maternal Foetal diseases mediated by maternal alloantibodies and Prevalent Rheumatology. 18,21,22,23,24,25,26,27,28 Blockade of IgG binding to FcRn in the placenta is also believed to prevent transplacental transfer of maternal alloantibodies to the foetus. 29 The European Medicines Agency (EMA) and U.S. Food and Drug Administration (FDA) have granted several key designations to nipocalimab including: 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 www.janssen.com/emea . Follow us at www.linkedin.com/jnj-innovative-medicine-emea . References 1 Antozzi, C et al., Efficacy and Safety of Nipocalimab in patients with Generalized Myasthenia Gravis- Top Line Results from the Double-Blind, Placebo-Controlled, Randomized Phase 3 Vivacity-MG3 study. 2024 European Academy of Neurology Congress. June 2024. 2 J&J Data on file (RF-421587). Approximate incidence of seronegativity in generalised myasthenia gravis (gMG). Date of preparation: June 2024. 3 Kenneth J. Moise Jr., et al. Nipocalimab in Early-onset Severe Hemolytic Disease of the Fetus & Newborn. N Engl J Med. 2024; DOI: 10.1056/NEJMoa2314466. 4 Efficacy and safety of nipocalimab, an anti-FcRn monoclonal antibody, in primary Sjogren’s disease: results from a Phase 2, multicenter, randomized, placebo-controlled, double-blind study (DAHLIAS). Late-breaking presentation at European Alliance of Associations for Rheumatology (EULAR) Annual Meeting; June 12–15, 2924. LBA0010 5 Guptill et.al. Vivacity-MG: A Phase 2, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety, Tolerability, Efficacy, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of Nipocalimab Administered to Adults with Generalized Myasthenia Gravis (2157). Neurology Journals. April 13, 2021. https://doi.org/10.1212/WNL.96.15_supplement.2157 6 Johnson & Johnson EMEA. Nipocalimab pivotal Phase 3 trial demonstrates sustained disease control in FcRn class for a broad population of myasthenia gravis patients. Available at: https://www.janssen.com/sites/www_janssen_com_emea/files/jj_emea_ean_nipocalimab_press_release_final.pdf . Last accessed: September 2024. 7 Wolfe GI Myasthenia gravis activities of daily living profile. Neurology. 1999;22;52(7):1487-9. doi: 10.1212/wnl.52.7.1487. 8 Chen J, Tian D-C, Zhang C, et al. Incidence, mortality, and economic burden of myasthenia gravis in China: A nationwide population-based study. The Lancet Regional Health – Western Pacific. 2020;5(100063). https://doi.org/10.1016/j.lanwpc.2020.100063. 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: September 2024. 11 Bubuioc A, et al. The epidemiology of myasthenia gravis. Journal of Medicine & Life (2021). Jan-Mar;14(1):7-16. doi: 10.25122/jml-2020-0145 12 National Institute of Neurological Disorders and Stroke. Myasthenia Gravis. Available at: https://www.ninds.nih.gov/health-information/disorders/myasthenia-gravis . Last accessed: September 2024. 13 Ye, Yun et al. Epidemiology of myasthenia gravis in the United States. Frontiers in neurology vol. 15 1339167. 16 Feb. 2024, doi:10.3389/fneur.2024.1339167 14 Dresser, Laura et al. Myasthenia Gravis: Epidemiology, Pathophysiology and Clinical Manifestations. Journal of clinical medicine vol. 10,11 2235. 21 May. 2021, doi:10.3390/jcm10112235. 15 Johnson & Johnson Data on file. (RF-430993). MG: Proportion of Diagnosed Patients who are People of Child-bearing Potential (POCBP). Date of preparation: May 2024. 16 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 17 Gilhus N. E, et al. Myasthenia Gravis. Nat Rev Dis Primers. (2019) 5(30): DOI: https://doi.org/10.1038/s41572-019-0079-y 18 ClinicalTrials.gov Identifier: NCT04951622. Available at: https://clinicaltrials.gov/ct2/show/NCT04951622 . Last accessed: September 2024. 19 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. 20 Ling LE., et al. M281, an anti‐fcrn antibody: Pharmacodynamics, pharmacokinetics, and safety across the full range of IGG reduction in a first‐in‐human study. Clinical Pharmacology & Therapeutics., 2018;105;4:1031–1039. https://doi.org/10.1002/cpt.1276. 21 ClinicalTrials.gov. NCT03842189. Available at: https://clinicaltrials.gov/ct2/show/NCT03842189. Last accessed: September 2024 22 ClinicalTrials.gov Identifier: NCT05327114. Available at: https://www.clinicaltrials.gov/study/NCT05327114 . Last accessed: September 2024 23 ClinicalTrials.gov Identifier: NCT04119050. Available at: https://clinicaltrials.gov/study/NCT04119050 . Last accessed: September 2024. 24 ClinicalTrials.gov Identifier: NCT05379634. Available at: https://clinicaltrials.gov/study/NCT05379634 . Last accessed: September 2024. 25 ClinicalTrials.gov Identifier: NCT05912517. Available at: https://www.clinicaltrials.gov/study/NCT05912517 . Last accessed: September 2024 26 ClinicalTrials.gov Identifier: NCT06028438. Available at: https://clinicaltrials.gov/study/NCT06028438 . Last accessed: September 2024. 27 ClinicalTrials.gov Identifier: NCT04968912. Available at: https://clinicaltrials.gov/study/NCT04968912 . Last accessed: September 2024. 28 ClinicalTrials.gov Identifier: NCT04882878. Available at: https://clinicaltrials.gov/study/NCT04882878 . Last accessed: September 2024. 29 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. SOURCE: Johnson & Johnson

On August 29, 2024, Johnson & Johnson formally filed a Biologics License Application (BLA) with the U.S. Food and Drug Administration (FDA) seeking the world's first approval of the FcRn monoclonal antibody Nipocalimab for the treatment of patients with generalized myasthenia gravis (gMG).   In June, the Phase III VIVACITY-MG3 study of Nipocalimab for gMG published positive results, showing that the combination of Nipocalimab with standard of care (SOC) significantly improved MG-ADL scores over 24 weeks compared with placebo-combined SOC, meeting the study's primary endpoint.   The study was a double-blind, placebo-controlled trial that enrolled a large number of patients with AChR+, MuSK+, and/or LRP4+, who accounted for approximately 95% of the total number of patients with gMG. At weeks 22, 23, and 24, patients receiving Nipocalimab+SOC improved MG-ADL scores by 4.70 points from baseline, which was significantly higher than 3.25 points in the placebo +SOC group (P=0.002). For people with gMG, a 1 to 2 point change in the MG-ADL score can mean the difference from a normal diet to frequent food choking, or from shortness of breath at rest to needing to use a ventilator.   In addition, the study achieved several key secondary endpoints: at week 22 and 24, the Nipocalimab+SOC group showed significant improvements in strength and function across different muscle groups assessed by QMG compared to the placebo +SOC group (P<0.001); At weeks 22, 23, and 24, the MG-ADL response was significantly higher in the Nipocalimab+SOC group (≥2 points improvement from baseline) than in the placebo +SOC group (P=0.021), further highlighting the potential of Nipocalimab to mitigate the impact of gMG on patients' daily lives.   Nipocalimab is a high-affinity, fully human, glycated, non-effective monoclonal antibody that selectively blocks FcRn, thereby reducing circulating immunoglobulin G (IgG) antibodies, including autoantibodies and alloantibodies that cause a variety of diseases.   Nipocalimab was originally developed by AnaptysBi and subsequently transferred to Momenta. In August 2020, Johnson & Johnson acquired Momenta for $6.5 billion, giving it the rights to the drug.   gMG is a chronic, lifelong, rare, and highly disabling autoantibody-mediated neuromuscular disease characterized by fluctuating myasthenia. Two FcRn monoclonal antibodies have previously been approved by the FDA, Argenx/ Re-ting's efgartigimod and UXbi's rozanolixizumab, both approved for gMG indications. Egamod has shown good market prospects, 2022 is its first full year of sales in overseas markets, sales exceeded 400 million US dollars, and 2023 reached 1.19 billion US dollars, among the "blockbuster" drugs.