This is a Phase II, open-label study designed to evaluate the safety, efficacy, and immunogenicity of NM8074 in PNH patients undergoing complement-inhibitor therapy with Soliris.

Start Date01 Mar 2025

Sponsor / Collaborator

NovelMed Therapeutics, Inc.

NCT06724809 / Not yet recruitingPhase 3

An Open-label, Single-arm, Multi-Center, Interventional Study to Evaluate the Efficacy, Safety, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of Eculizumab in Chinese Adult Participants With Anti-Aquaporin-4 Antibody Positive Neuromyelitis Optica Spectrum Disorders (NMOSD)

The primary objective of this study is to evaluate the efficacy, safety, pharmacokinetics, pharmacodynamics, and immunogenicity of Eculizumab in Chinese Adults with Neuromyelitis Optica Spectrum Disorders (NMOSD).

Start Date24 Dec 2024

Sponsor / Collaborator

Alexion Pharmaceuticals, Inc.

[+1]

NCT06673394 / Not yet recruitingPhase 2IIT

Eculizumab for Acute Attack of Neuromyelitis Optica Spectrum Disorder (NMOSD): a Multi-Center, Phase 2, Open Label Trial (EASE-NMO)

Neuromyelitis optica spectrum disorder (NMOSD) is a relapsing, inflammatory autoimmune disorder of the central nervous system characterized by the pathogenic anti-aquaporin 4 antibody (AQP4-IgG). The objectives of this study are to assess the efficacy and safety of eculizumab for treatment of patients with neuromyelitis optica spectrum disorders during acute phase who are anti-aquaporin-4 (AQP4) antibody-positive. Eculizumab, a humanized monoclonal antibody, inhibits the terminal complement protein C5 and prevents its cleavage into C5a and the formation of C5b-9 (MAC), has approved for preventive treatment of NMOSD. Given the high efficacy of C5 inhibition, eculizumab is proposed to potentially provide rapid relief from astrocyte destruction by reducing MAC formation, which could contribute to the fast alleviation of neurological deficit during NMO acute attack. The potential of eculizumab warrants further investigation as a treatment for acute neuromyelitis optica spectrum disorders attacks.

Start Date01 Dec 2024

Sponsor / Collaborator

Tianjin Medical University General Hospital

100 Clinical Results associated with Eculizumab

100 Translational Medicine associated with Eculizumab

100 Patents (Medical) associated with Eculizumab

2,535

Literatures (Medical) associated with Eculizumab

01 Jan 2025·Current rheumatology reviews

Rituximab Induced Flare of Psoriatic Arthritis in a Patient with Devic's Syndrome: A Case Report

Author: Arkfeld, Daniel G. ; Mortezaei, Kiana ; Gonzales, Sofia Audrey B.

Introduction/Background:::

Devic’s syndrome is a rare autoimmune disorder that occurs when the body's immune system damages and mistakenly attacks the optic nerves and spinal cord, leading to numerous neurological symptoms such as inflammation, weakness, numbness, and vision problems. Rituximab has mainly been utilized as an immunosuppressive therapy for patients with Devic’s syndrome. Although evidence has shown that rituximab is efficient and well tolerated in treating patients with Devic’s syndrome, there is the possibility of rituximab exacerbating severe psoriasis and psoriatic arthritis flare.

Case Presentation:::

In this paper, we describe a case of a 58-year-old female with Devic’s syndrome, blindness, and neurological involvement who responded exceptionally well to rituximab but developed a severe flare of psoriatic arthritis. After withdrawing from the use of rituximab, her psoriatic arthritis symptoms had resolved. However, she did have another episode of blindness, and rituximab was started once again. Although her vision improved, her psoriatic arthritis symptoms had reoccurred. The patient was switched to eculizumab and ustekinumab, which controlled both her psoriatic arthritis and Devic’s syndrome.

Conclusion:::

Very few reports have identified rituximab to induce a flare-up of psoriatic arthritis, raising uncertainty regarding its potential effects on psoriatic symptoms. The precise mechanism underlying the exacerbation of psoriatic arthritis by rituximab remains uncertain. This case report highlights that rituximab can worsen psoriasis and psoriatic arthritis, and that the complexities of Devic’s syndrome may require medication adjustments.

24 Dec 2024·NEUROLOGY

Eculizumab in AQP4-IgG NMOSD

Article

01 Dec 2024·NEUROLOGICAL SCIENCES

Therapeutic challenges and unmet needs in the management of myasthenia gravis: an Italian expert opinion

Review

Author: Liguori, Rocco ; Habetswallner, Francesco ; Rodolico, Carmelo ; Mantegazza, Renato ; Pegoraro, Elena ; Evoli, Amelia ; Sgarzi, Manlio ; Saccà, Francesco ; Bonifati, Domenico Marco ; Pappagallo, Giovanni ; Schenone, Angelo ; Antonini, Giovanni

Myasthenia gravis (MG) is a rare, autoimmune, neurological disorder. Most MG patients have autoantibodies against acetylcholine receptors (AChRs). Some have autoantibodies against muscle-specific tyrosine kinase (MuSK) or lipoprotein-receptor-related protein 4 (LRP4), and some are seronegative. Standard of care, which includes anti-cholinesterase drugs, thymectomy, corticosteroids (CS), and off-label use of non-steroidal immunosuppressive drugs (NSISTs), is bounded by potential side effects and limited efficacy in refractory generalized MG (gMG) patients. This highlights the need for new therapeutic approaches for MG. Eculizumab, a monoclonal antibody that inhibits the complement system, has been recently approved in Italy for refractory gMG. A panel of 11 experts met to discuss unmet therapeutic needs in the acute and chronic phases of the disease, as well as the standard of care for refractory patients. Survival was emphasized as an acute phase outcome. In the chronic phase, persistent remission and early recognition of exacerbations to prevent myasthenic crisis and respiratory failure were considered crucial. Refractory patients require treatments with fast onset of action, improved tolerability, and the ability to slow disease progression and increase life expectancy. The Panel agreed that eculizumab would presumably meet the therapeutic needs of many refractory gMG patients. The panel concluded that the unmet needs of current standard of care treatments for gMG are significant. Evaluating new therapeutic options accurately is essential to find the best balance between efficacy and tolerability for each patient. Collecting real-world data on novel molecules in routine clinical practice is necessary to address unmet needs.

190

News (Medical) associated with Eculizumab

08 Dec 2024

·pipelinereview.com

Novel Combination of Pozelimab and Cemdisiran (Poze-Cemdi) Achieved Greater Control of Intravascular Hemolysis in Patients with Paroxysmal Nocturnal Hemoglobinuria Compared to Ravulizumab

Head-to-head exploratory cohort of a Phase 3 trial showed first-in-class poze-cemdi combination treatment helped patients achieve and maintain greater disease control, as measured by lactate dehydrogenase (LDH) levels, compared to standard-of-care ravulizumab Five patients receiving ravulizumab did not achieve meaningful LDH control compared to one patient receiving poze-cemdi; after switching to the combination, four of the five previously treated with ravulizumab achieved LDH control A separate registrational cohort is ongoing, investigating poze-cemdi against eculizumab TARRYTOWN, NY, USA I December 07, 2024 I Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) today announced positive updated Phase 3 data of an exploratory cohort from the ACCESS-1 trial investigating its first-in-class pozelimab and cemdisiran (poze-cemdi) combination treatment against ravulizumab, a standard-of-care complement factor 5 (C5) inhibitor, in patients with paroxysmal nocturnal hemoglobinuria (PNH). Results were shared during an oral session at the American Society of Hematology (ASH) 2024 Annual Meeting and support continued development of poze-cemdi in PNH, including in a separate registrational cohort, as well as in other complement-mediated diseases. Poze-Cemdi is a first-in-class combination of an antibody and an siRNA targeting C5: pozelimab is a fully human monoclonal antibody designed to block the activity of C5, while cemdisiran is an investigational siRNA therapeutic that reduces circulating levels of C5. PNH is an ultra-rare, chronic, life-threatening complement-mediated blood disorder. People with PNH have an acquired genetic mutation in which red blood cells are destroyed (known as hemolysis) by the complement system, which is part of the innate immune system. The lysed red blood cells release lactate dehydrogenase (LDH), which is a biomarker used to measure the degree of hemolysis. Hemolysis causes a range of symptoms including fatigue, shortness of breath, and life-threatening blood clots. Inhibition of C5, a protein involved in complement system activation, is an established treatment approach to prevent intravascular hemolysis, which occurs inside blood vessels; LDH can be used to determine the effectiveness of C5 inhibition. Addressing intravascular hemolysis is a critical treatment approach to reducing the symptoms and risk of life-threatening complications of PNH. “C5 inhibitors are widely considered the mainstay of PNH treatment, but a proportion of patients still do not achieve adequate control of intravascular hemolysis, may experience residual anemia, and may feel significant treatment burden, as many of these therapies require clinic or home visits for intravenous delivery,” said Christopher Patriquin, M.D., MSc, Assistant Professor of Medicine, Hematology, at the University of Toronto, hematologist at University Health Network and a trial investigator. “In this Phase 3 exploratory cohort, the complementary mechanisms of pozelimab and cemdisiran enabled complete, rapid, uninterrupted and durable inhibition of terminal complement throughout the dosing interval. The combination helped more patients achieve target LDH levels compared to the current standard-of-care C5 inhibitor, with the added benefit of infrequent four-week subcutaneous delivery that has potential for self-administration. These data validate this novel combination approach, and we look forward to results from the registrational cohort, which if repeated, could help transform what may be possible for many people with PNH.” Updated results from an exploratory arm (Cohort A) of the ACCESS-1 trial, as well as interim results from a follow-on, open label extension (OLE) were presented at ASH. Patients were naïve to complement inhibition, with the primary endpoint of Cohort A being percent change in LDH at 26 weeks. LDH is a well-accepted biomarker of intravascular hemolysis that measures how effective a treatment is at inhibiting the destruction of red blood cells and has also demonstrated a correlation to clinical outcomes. 1 Adequate control of hemolysis is defined as LDH levels of ≤1.5 times upper limit of normal (ULN), while normalization is defined as ≤1 times ULN, respectively. In Cohort A, patients were randomized to receive either poze-cemdi or ravulizumab. The ravulizumab arm generally responded as would be expected based on historical clinical trial data, which indicate that 44% of treated patients did not achieve LDH normalization (≤1 x ULN). 2 Results for those treated with poze-cemdi (n=25), compared to ravulizumab (n=23), were as follows: After week 26, all patients who completed ACCESS-1 could enroll in a follow-on OLE trial and receive poze-cemdi, including those who initially received ravulizumab (n=19). At the start of the OLE, 68% (n=13) of patients treated with ravulizumab had adequate LDH control. After switching to poze-cemdi, 95% of patients (n=18) achieved LDH control. This included 4 of 5 patients who had failed to achieve LDH control while on ravulizumab. The safety profile of poze-cemdi was generally consistent with approved C5 inhibitors. During ACCESS-1, treatment-emergent adverse events (TEAEs) occurred in 84% of patients treated with poze-cemdi, compared to 87% treated with ravulizumab. The most common TEAEs (≥10%) for poze-cemdi compared to ravulizumab were headache (28% vs. 17%), upper respiratory tract infection (12% vs. 9%), nausea (12% vs. 4%), anemia (12% vs. 9%), fatigue (8% vs. 13%) and cough (4% vs. 13%). Serious adverse events (SAEs) occurred in two patients receiving poze-cemdi that were considered unrelated to treatment by the investigator. This included one patient who had post-traumatic cellulitis that resolved with treatment while continuing poze-cemdi. The second patient experienced a fever, seizure and hemolytic crisis within one week of the first dose of the combination that also resolved while continuing poze-cemdi; the patient later had a fatal SAE of sepsis and disseminated intravascular coagulation on day 130. In the OLE, among patients who switched to poze-cemdi from ravulizumab, 68% experienced TEAEs, with the most common being non-serious, mild to moderate injection-site reactions. There were no TEAEs consistent with type 3 hypersensitivity reactions due to large drug-target-drug immune complexes after switching from ravulizumab to poze-cemdi, which have been observed when switching between other C5 inhibitors. There were also no fatal TEAEs, and no patients discontinued therapy due to an adverse event. The potential use of pozelimab and cemdisiran for the treatment of PNH is investigational and has not been approved by any regulatory authority. About the Pozelimab and Cemdisiran Clinical Trial Program Pozelimab and cemdisiran are being evaluated in separate Phase 3 trials for several complement-mediated disorders, including PNH, myasthenia gravis (MG) and geographic atrophy (GA). PNH: ACCESS-1 is a randomized, active-controlled study comprised of two cohorts, evaluating poze-cemdi in patients with PNH who are naïve to, or have not recently received, complement inhibitor therapy. The first cohort (Cohort A) is an exploratory cohort examining the combination administered subcutaneously as a maintenance regimen every four weeks compared to ravulizumab delivered as a maintenance regimen every eight weeks by intravenous infusion. Cohort B is a registrational cohort examining poze-cemdi against eculizumab. Patients in both cohorts may participate in a follow-on OLE study ( ACCESS-EXTENSION ) assessing the long-term safety and efficacy of the combination, including in patients who switch from ravulizumab or eculizumab. MG: NIMBLE is a randomized, double-blind placebo controlled trial evaluating poze-cemdi as well as cemdisiran monotherapy in patients with generalized MG. GA: SIENNA is a randomized, double-blind, placebo controlled trial evaluating poze-cemdi as well as cemdisiran monotherapy in patients with GA secondary to age-related macular degeneration. For more information, visit the Regeneron clinical trials website , or contact clinicaltrials@regeneron.com or +1 844-734-6643. The pozelimab and cemdisiran combination is being developed under an agreement with Alnylam Pharmaceuticals, Inc. About Regeneron in Hematology At Regeneron, we’re applying more than three decades of biology expertise with our proprietary VelociSuite ® technologies to develop medicines for patients with diverse blood cancers and rare blood disorders. Our blood cancer research is focused on bispecific antibodies that are being investigated both as monotherapies and in combination with each other and emerging therapeutic modalities. Together, they provide us with unique combinatorial flexibility to develop customized and potentially synergistic cancer treatments. Our research and collaborations to develop potential treatments for rare blood disorders include explorations in antibody medicine, gene editing and gene-knockout technologies, and investigational RNA approaches focused on depleting abnormal proteins or blocking disease-causing cellular signaling. About Regeneron’s VelocImmune Technology Regeneron’s VelocImmune technology utilizes a proprietary genetically engineered mouse platform endowed with a genetically humanized immune system to produce optimized fully human antibodies. When Regeneron’s co-Founder, President and Chief Scientific Officer George D. Yancopoulos was a graduate student with his mentor Frederick W. Alt in 1985, they were the first to envision making such a genetically humanized mouse, and Regeneron has spent decades inventing and developing VelocImmune and related VelociSuite ® technologies. Dr. Yancopoulos and his team have used VelocImmune technology to create a substantial proportion of all original, FDA-approved or authorized fully human monoclonal antibodies. This includes REGEN-COV ® (casirivimab and imdevimab), Dupixent ® (dupilumab), Libtayo ® (cemiplimab-rwlc), Praluent ® (alirocumab), Kevzara ® (sarilumab), Evkeeza ® (evinacumab-dgnb), Inmazeb ® (atoltivimab, maftivimab and odesivimab-ebgn) and Veopoz ® (pozelimab). About Regeneron Regeneron (NASDAQ: REGN) is a leading biotechnology company that invents, develops and commercializes life-transforming medicines for people with serious diseases. Founded and led by physician-scientists, our unique ability to repeatedly and consistently translate science into medicine has led to numerous approved treatments and product candidates in development, most of which were homegrown in our laboratories. Our medicines and pipeline are designed to help patients with eye diseases, allergic and inflammatory diseases, cancer, cardiovascular and metabolic diseases, neurological diseases, hematologic conditions, infectious diseases, and rare diseases. Regeneron pushes the boundaries of scientific discovery and accelerates drug development using our proprietary technologies, such as VelociSuite ® , which produces optimized fully human antibodies and new classes of bispecific antibodies. We are shaping the next frontier of medicine with data-powered insights from the Regeneron Genetics Center ® and pioneering genetic medicine platforms, enabling us to identify innovative targets and complementary approaches to potentially treat or cure diseases. For more information, please visit www.Regeneron.com or follow Regeneron on LinkedIn , Instagram , Facebook or X . 1 Schrezenmeier, H., Kulasekararaj, A., Mitchell, L. et al . Predictors for improvement in patient-reported outcomes: post hoc analysis of a phase 3 randomized, open-label study of eculizumab and ravulizumab in complement inhibitor-naive patients with paroxysmal nocturnal hemoglobinuria. Ann Hematol 103, 5-15 (2024). 2 Ultomiris (ravulizumab) [package insert]. Boston, MA: Alexion Pharmaceuticals, Inc.; 2018. SOURCE: Regeneron Pharmaceuticals

Clinical ResultPhase 3ASH

06 Dec 2024

·endpts.com

Novartis bolsters Fabhalta data in competition with AstraZeneca

Novartis’ Fabhalta improved hemoglobin levels in patients with a rare blood disorder who switched from anti-C5 therapies, bolstering the drug’s case against AstraZeneca’s competing treatments. Fabhalta has been approved for exactly one year in paroxysmal nocturnal hemoglobinuria, a disorder that causes red blood cells to break down and can lead to anemia or blood clots. Some patients require transfusions. AstraZeneca’s C5 inhibitors Soliris and Ultomiris have long been the standard of care, each generating roughly $3 billion in 2023 sales. But on Friday, Novartis said patients in the single-arm Phase 3b APPULSE-PNH study saw their average hemoglobin levels improve from baseline over 24 weeks after switching from Soliris or Ultomiris, meeting the primary endpoint. “These data reinforce our confidence in Fabhalta, the first and only oral monotherapy currently available for the treatment of adults with PNH, to provide meaningful hemoglobin improvement, regardless of previous treatment experience,” David Soergel, global head of Novartis’ cardiovascular, renal and metabolism development unit, said in a news release . The company declined to provide specifics, though a spokesperson told Endpoints News that the data will be presented at a medical meeting in 2025. The results build on data from Novartis’ APPLY-PNH study. At last year’s American Society of Hematology conference, Novartis said that 94% of patients who switched from C5-inhibitors to Fabhalta in the study’s extension period achieved transfusion avoidance, and “mean hemoglobin levels increased to near-normal.” In that group, Novartis said mean hemoglobin levels were 9.1 g/dL at the start of the extension period, which is lower than patients’ average levels at the start of the APPULSE-PNH study. Soliris and Ultomiris are infusions, while Fabhalta is approved as a twice-daily oral pill. Novartis CEO Vas Narasimhan said in January that the company saw “very positive early launch signals” for Fabhalta, but added that he expected a “modest ramp for this brand given the dynamics within the PNH market,” according to an AlphaSense transcript. Fabhalta generated $44 million in third-quarter 2024 sales. In August, it was granted an accelerated approval in adults with primary immunoglobulin A nephropathy (IgAN), a rare autoimmune disease that affects the kidneys.

Clinical ResultDrug ApprovalPhase 3ASHImmunotherapy

06 Dec 2024

·www.globenewswire.com

New Phase IIIB data shows Novartis Fabhalta® improved hemoglobin levels in adult patients with paroxysmal nocturnal hemoglobinuria who switched from anti-C5 therapy

In the Phase IIIB APPULSE-PNH study, oral Fabhalta® (iptacopan) improved the average hemoglobin (Hb) level versus baseline in adult patients with paroxysmal nocturnal hemoglobinuria (PNH) who were switched from anti-C5 therapies (Hb ≥10g/dL following treatment with eculizumab or ravulizumab)1,2PNH is a rare, chronic and serious complement-mediated blood disorder, characterized by hemolysis, anemia, thrombosis and other symptoms, and patients are often treated with anti-C5 therapies3-5Findings from APPULSE-PNH build on the Phase III program showing the efficacy and safety of Fabhalta in adults with PNH1, which is approved in the US, EU, Japan and China6-9 Basel, December 6, 2024 – Novartis today announced positive topline results from APPULSE-PNH, a Phase IIIB study evaluating the efficacy and safety of twice-daily oral monotherapy Fabhalta® (iptacopan) in adult patients with paroxysmal nocturnal hemoglobinuria (PNH) who were switched from anti-C5 therapies (Hb ≥10g/dL following treatment with eculizumab or ravulizumab)1. After 24 weeks of treatment with Fabhalta, the average Hb level improved versus baseline1. “These new results add to the body of evidence reinforcing that Fabhalta can benefit both patients previously treated with anti-C5 therapies studied in the APPULSE-PNH and APPLY-PNH trials and complement-inhibitor naïve patients studied in the APPOINT-PNH trial,” said Antonio Risitano, M.D., Ph.D., Chair of the International PNH Interest Group and Head of the Hematology and Hematopoietic Transplant Unit, Reference Center for Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria at the AORN San Giuseppe Moscati, Avellino, Italy, and APPULSE-PNH trial lead investigator. “Treatment goals for patients with PNH have greatly evolved, and we can now aim to resolve signs and symptoms of disease in most patients. It is promising to see this evolution, and we will continue to make progress to best support these patients.” In the study, the safety profile of Fabhalta monotherapy was consistent with previously reported data1,10. “Across multiple clinical trials, Fabhalta has consistently shown clinically meaningful benefits for patients with PNH, and the APPULSE-PNH trial is a compelling addition to this body of evidence," said David Soergel, M.D., Global Head, Cardiovascular, Renal and Metabolism Development Unit, Novartis. “These data reinforce our confidence in Fabhalta, the first and only oral monotherapy currently available for the treatment of adults with PNH, to provide meaningful hemoglobin improvement, regardless of previous treatment experience.” Data will be presented at an upcoming medical meeting in 2025. Fabhalta was recently granted accelerated approval by the US Food and Drug Administration (FDA) for the reduction of proteinuria in adults with primary immunoglobulin A nephropathy (IgAN) at risk of rapid disease progression, and development is ongoing in multiple complement-mediated diseases6,11. About APPULSE-PNH APPULSE-PNH (NCT05630001) is a Phase IIIB multicenter, single-arm, open-label study to evaluate the efficacy and safety of twice-daily oral Fabhalta® (iptacopan) monotherapy (200mg) in adults with PNH who were switched from anti-C5 therapies (eculizumab or ravulizumab)2. The trial enrolled 52 participants who received Fabhalta for 24 weeks2. Participants enrolled were required to be on a stable regimen with anti-C5 therapies (eculizumab or ravulizumab) for at least 6 months prior to screening with average hemoglobin (Hb) ≥10g/dL and no red blood cell transfusions in this period2,12. The primary endpoint is change from baseline Hb levels after 24 weeks of treatment with Fabhalta2,12. About paroxysmal nocturnal hemoglobinuria (PNH)PNH is a rare, chronic and serious complement-mediated blood disorder13. People with PNH have an acquired mutation in some of their hematopoietic stem cells (which are located in the bone marrow and can grow and develop into red blood cells [RBCs], white blood cells and platelets) that causes them to produce RBCs that are susceptible to premature destruction by the complement system4,13. This leads to intravascular hemolysis (destruction of RBCs within blood vessels) and extravascular hemolysis (destruction of RBCs mostly in the spleen and liver), which cause anemia (low levels of circulating RBCs), thrombosis (formation of blood clots), fatigue and other debilitating symptoms4,13.   It is estimated that approximately 10-20 people per million worldwide live with PNH13. Although PNH can develop at any age, it is often diagnosed in people between 30-40 years old14,15. PNH has a significant unmet need not addressed by anti-C5 therapies (eculizumab or ravulizumab). Anti-C5 therapies (eculizumab or ravulizumab) are commonly administered every 2-8 weeks as intravenous infusions, and treatment visits (including journey, waiting, infusion and recovery time) can take approximately 4 to 5 hours5. Despite treatment with anti-C5 therapies, a large proportion of people with PNH remain anemic, and some dependent on blood transfusions3,4,16-20. About Fabhalta® (iptacopan)  Fabhalta (iptacopan) is an oral, Factor B inhibitor of the alternative complement pathway12.  Fabhalta, discovered at Novartis, received approval by the FDA in December 2023 for the treatment of adults with PNH and by the European Medicines Agency (EMA) in May 2024 for the treatment of adults with PNH with hemolytic anemia6,7. In August 2024, Fabhalta was granted accelerated approval by the US Food and Drug Administration (FDA) for the reduction of proteinuria in adults with primary immunoglobulin A nephropathy (IgAN) at risk of rapid disease progression, and development is ongoing in multiple complement-mediated diseases6,11. Fabhalta is being studied in a broad range of rare kidney diseases, including C3 glomerulopathy (C3G), atypical hemolytic uremic syndrome (aHUS), immune complex membranoproliferative glomerulonephritis (IC-MPGN) and lupus nephritis (LN). Studies are ongoing to evaluate the safety and efficacy profiles in these investigational indications and support potential regulatory submissions. DisclaimerThis media update contains forward-looking statements within the meaning of the United States Private Securities Litigation Reform Act of 1995. Forward-looking statements can generally be identified by words such as “potential,” “can,” “will,” “plan,” “may,” “could,” “would,” “expect,” “anticipate,” “look forward,” “believe,” “committed,” “investigational,” “pipeline,” “launch,” or similar terms, or by express or implied discussions regarding potential marketing approvals, new indications or labeling for the investigational or approved products described in this media update, or regarding potential future revenues from such products. You should not place undue reliance on these statements. Such forward-looking statements are based on our current beliefs and expectations regarding future events, and are subject to significant known and unknown risks and uncertainties. Should one or more of these risks or uncertainties materialize, or should underlying assumptions prove incorrect, actual results may vary materially from those set forth in the forward-looking statements. There can be no guarantee that the investigational or approved products described in this media update will be submitted or approved for sale or for any additional indications or labeling in any market, or at any particular time. Nor can there be any guarantee that such products will be commercially successful in the future. In particular, our expectations regarding such products could be affected by, among other things, the uncertainties inherent in research and development, including clinical trial results and additional analysis of existing clinical data; regulatory actions or delays or government regulation generally; global trends toward health care cost containment, including government, payor and general public pricing and reimbursement pressures and requirements for increased pricing transparency; our ability to obtain or maintain proprietary intellectual property protection; the particular prescribing preferences of physicians and patients; general political, economic and business conditions, including the effects of and efforts to mitigate pandemic diseases; safety, quality, data integrity or manufacturing issues; potential or actual data security and data privacy breaches, or disruptions of our information technology systems, and other risks and factors referred to in Novartis AG’s current Form 20-F on file with the US Securities and Exchange Commission. Novartis is providing the information in this media update as of this date and does not undertake any obligation to update any forward-looking statements contained in this media update as a result of new information, future events or otherwise. About Novartis Novartis is an innovative medicines company. Every day, we work to reimagine medicine to improve and extend people’s lives so that patients, healthcare professionals and societies are empowered in the face of serious disease. Our medicines reach more than 250 million people worldwide. Reimagine medicine with us: Visit us at https://www.novartis.com and connect with us on LinkedIn, Facebook, X/Twitter and Instagram. References Novartis. Data on file.Clinicaltrials.gov. NCT05630001. Single Arm, Open Label Trial With Iptacopan Treatment for 24 Weeks, in Patients on Stable Regimen of Anti-C5 Who Switch to Iptacopan. (APPULSE). Available from: https://clinicaltrials.gov/study/NCT05630001 Accessed October, 2024.McKinley CE, Richards SJ, Munir T, et al. Extravascular Hemolysis Due to C3-Loading in Patients with PNH Treated with Eculizumab: Defining the Clinical Syndrome. Blood. 2017;130(Supplement 1):3471. Dingli D, Matos JE, Lehrhaupt K, et al. The burden of illness in patients with paroxysmal nocturnal hemoglobinuria receiving treatment with the C5-inhibitors eculizumab or ravulizumab: results from a US patient survey. Ann Hematol. 2022;101(2):251-263.Levy AR, Dysart L, Patel Y, et al. Comparison of Lost Productivity Due to Eculizumab and Ravulizumab Treatments for Paroxysmal Nocturnal Hemoglobinuria in France, Germany, Italy, Russia, Spain, the United Kingdom, and the United States. Blood. 2019;134(Supplement_1):4803.Fabhalta®. US FDA Prescribing information. East Hanover, NJ:Novartis Pharmaceuticals Corp; 2024. Available from: https://www.accessdata.fda.gov/drugsatfda_docs/label/2024/218276s001lbl.pdf Accessed November 2024.Fabhalta®. EMA Summary of Product Characteristics. Novartis Europharm Limited; 2024. Available from: https://www.ema.europa.eu/en/documents/product-information/fabhalta-epar-product-information_en.pdf Accessed November 2024.Fabhalta®. PMDA Drug Information Sheet. Novartis Pharma KK; 2024. Available from: https://www.pmda.go.jp/files/000271929.pdf Accessed November 2024.Fabhalta®. NMPA drug insert. Novartis Pharma Schweiz AG; 2024. Available from: https://www.ccfdie.org/en/gzdt/webinfo/2024/05/1716632195327630.html Accessed November 2024.Dighriri IM, Al-Qahtani RM, Almutairi AO, et al. Iptacopan Efficacy and Safety to Treat Paroxysmal Nocturnal Hemoglobinuria (PNH): A Systematic Review and Meta-Analysis. Cureus. 2024;16(8):e67830. Novartis. Press release. Novartis receives FDA accelerated approval for Fabhalta® (iptacopan), the first and only complement inhibitor for the reduction of proteinuria in primary IgA nephropathy (IgAN). Available from: https://www.novartis.com/news/media-releases/novartis-receives-fda-accelerated-approval-fabhalta-iptacopan-first-and-only-complement-inhibitor-reduction-proteinuria-primary-iga-nephropathy-igan Accessed October, 2024.Risitano AM, Araten DJ, Kuter D, et al. Pb2063: APPULSE-PNH: A phase IIIB trial to evaluate the efficacy and safety of switching to iptacopan in patients with paroxysmal nocturnal hemoglobinuria (PNH) on anti-c5 therapy with hemoglobin >10g/dl. Hemasphere. 2023;7(Suppl):e08587b7.Cançado RD, Araújo ADS, Sandes AF, et al. Consensus statement for diagnosis and treatment of paroxysmal nocturnal haemoglobinuria. Hematol Transfus Cell Ther. 2021;43(3):341-348.Hill A, DeZern AE, Kinoshita T, Brodsky RA. Paroxysmal nocturnal haemoglobinuria. Nat Rev Dis Primers. 2017;3:17028.Röth A, Maciejewski J, Nishimura JI, Jain D, Weitz JI. Screening and diagnostic clinical algorithm for paroxysmal nocturnal hemoglobinuria: Expert consensus. Eur J Haematol. 2018;101(1):3-11.Risitano AM, Marotta S, Ricci P, et al. Anti-complement Treatment for Paroxysmal Nocturnal Hemoglobinuria: Time for Proximal Complement Inhibition? A Position Paper From the SAAWP of the EBMT. Front Immunol. 2019;10:1157.Shammo et al. HemaSphere 2023 Shammo J, Kim J, Georget M, et al. P796: Hospitalization in patients with paroxysmal nocturnal hemoglobinuria: a retrospective analysis of observational study data from the United States. Hemasphere. 2023;7(Suppl):e22585a2.Debureaux PE, Kulasekararaj AG, Cacace F, et al. Categorizing hematological response to eculizumab in paroxysmal nocturnal hemoglobinuria: a multicenter real-life study. Bone Marrow Transplant. 2021;56(10):2600-2602.Schrezenmeier H, Kulasekararaj A, Mitchell L, et al. One-year efficacy and safety of ravulizumab in adults with paroxysmal nocturnal hemoglobinuria naïve to complement inhibitor therapy: open-label extension of a randomized study. Ther Adv Hematol. 2020;11:2040620720966137.Young NS, Meyers G, Schrezenmeier H, Hillmen P, Hill A. The management of paroxysmal nocturnal hemoglobinuria: recent advances in diagnosis and treatment and new hope for patients. Semin Hematol. 2009;46(1 Suppl 1):S1-S16. # # # Novartis Media RelationsE-mail: media.relations@novartis.com Central North America Anja von Treskow +41 79 392 9697 Michael Meo +1 862 274 5414 Anna Schäfers +41 79 801 7267 SwitzerlandSatoshi Sugimoto +41 79 619 2035 Novartis Investor RelationsCentral investor relations line: +41 61 324 7944E-mail: investor.relations@novartis.com Central North America Isabella Zinck +41 61 324 7188 Sloan Simpson +1 862 345 4440 Nicole Zinsli-Somm +41 61 324 3809 Jonathan Graham +1 201 602 9921 Imke Kappes +41 61 324 8269 Parag Mahanti +1 973 876 4912

Phase 3Clinical ResultDrug ApprovalAccelerated ApprovalNDA

100 Deals associated with Eculizumab

External Link

KEGG	Wiki	ATC	Drug Bank
D03940	Eculizumab	L04AA25	DB01257

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
AQP4-IgG positive Neuromyelitis optica spectrum disorder	AU	Alexion Pharmaceuticals, Inc.	20 Mar 2009
Atypical Hemolytic Uremic Syndrome	EU	Alexion Europe SAS	20 Jun 2007
Atypical Hemolytic Uremic Syndrome	IS	Alexion Europe SAS	20 Jun 2007
Atypical Hemolytic Uremic Syndrome	LI	Alexion Europe SAS	20 Jun 2007
Atypical Hemolytic Uremic Syndrome	NO	Alexion Europe SAS	20 Jun 2007
Myasthenia Gravis	EU	Alexion Europe SAS	20 Jun 2007
Myasthenia Gravis	IS	Alexion Europe SAS	20 Jun 2007
Myasthenia Gravis	LI	Alexion Europe SAS	20 Jun 2007
Myasthenia Gravis	NO	Alexion Europe SAS	20 Jun 2007
Neuromyelitis Optica	EU	Alexion Europe SAS	20 Jun 2007
Neuromyelitis Optica	IS	Alexion Europe SAS	20 Jun 2007
Neuromyelitis Optica	LI	Alexion Europe SAS	20 Jun 2007
Neuromyelitis Optica	NO	Alexion Europe SAS	20 Jun 2007
Hemoglobinuria, Paroxysmal	US	Alexion Pharmaceuticals, Inc.	16 Mar 2007

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Delayed Graft Function	Phase 3	US	Alexion Pharmaceuticals, Inc.	21 Aug 2014
Delayed Graft Function	Phase 3	AU	Alexion Pharmaceuticals, Inc.	21 Aug 2014
Delayed Graft Function	Phase 3	BR	Alexion Pharmaceuticals, Inc.	21 Aug 2014
Delayed Graft Function	Phase 3	CA	Alexion Pharmaceuticals, Inc.	21 Aug 2014
Delayed Graft Function	Phase 3	CZ	Alexion Pharmaceuticals, Inc.	21 Aug 2014
Delayed Graft Function	Phase 3	FR	Alexion Pharmaceuticals, Inc.	21 Aug 2014
Delayed Graft Function	Phase 3	DE	Alexion Pharmaceuticals, Inc.	21 Aug 2014
Delayed Graft Function	Phase 3	IT	Alexion Pharmaceuticals, Inc.	21 Aug 2014
Delayed Graft Function	Phase 3	ES	Alexion Pharmaceuticals, Inc.	21 Aug 2014
AQP4-IgG positive Optic Neuritis	Phase 3	US	Alexion Pharmaceuticals, Inc.	11 Apr 2014

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT04320602 (CTgov)	Phase 4	Hemoglobinuria, Paroxysmal	18	Eculizumab+Ravulizumab	mokgcbkvum(jyadjubauc) = deknygjbup zvjtrkzdix (ijodallowm, leoujdszlz - zlzqvnbgug) View more	-	12 Aug 2024
NCT00122330 (FDA_CDER) Manual	Phase 3	Hemoglobinuria, Paroxysmal	87	Placebo	gzlsuidayb(svwmhfskzh) = dktkhqgoxw ijaftdcibd (mitizihyjr ) View more	Positive	19 Jul 2024
				Eculizumab	gzlsuidayb(svwmhfskzh) = iqibdshpzj ijaftdcibd (mitizihyjr ) View more
NCT00844844 \| NCT00844545 (C08-002B, FDA_CDER) Manual	Phase 2	Atypical Hemolytic Uremic Syndrome	17	Eculizumab (at 26 wks)	nylhpqfitj(iysyteeseq) = fpyfjnqrig ajyommfbqg (gleukahzyp ) View more	Positive	19 Jul 2024
				Eculizumab (at 2 yrs)	nylhpqfitj(iysyteeseq) = qkstfollwb ajyommfbqg (gleukahzyp ) View more
NCT00844428 \| NCT00838513 (C08-003B, FDA_CDER) Manual	Phase 2	Atypical Hemolytic Uremic Syndrome	20	Eculizumab (at 26 wks)	aqbbrfsobd(ahtazmybcy) = pvidnnilwv vzanwempjm (jqjyfcgjjj ) View more	Positive	19 Jul 2024
				Eculizumab (at 2 yrs)	aqbbrfsobd(ahtazmybcy) = zzbpdyyfis vzanwempjm (jqjyfcgjjj ) View more
NCT01193348 (C10-003, FDA_CDER) Manual	Phase 2	Atypical Hemolytic Uremic Syndrome	22	Eculizumab (Patients 1 month to <12 years)	fsdftqtatg(pqahkavghc) = epctidrabg wrdpjekxlr (btsamkoqhz, 36 - 83) View more	Positive	19 Jul 2024
				Eculizumab (All Patients)	fsdftqtatg(pqahkavghc) = xmlgqlnfsd wrdpjekxlr (btsamkoqhz, 41 - 83) View more
NCT01194973 (C10-004, FDA_CDER) Manual	Phase 2	Atypical Hemolytic Uremic Syndrome	41	Eculizumab	aycxeosstw(wwzudylvnl) = xwgwjszrma mvhewhmmwe (qvcvikwkjm, 40 - 72) View more	Positive	19 Jul 2024
NCT01770951 (C09-001r, FDA_CDER) Manual	Not Applicable	Atypical Hemolytic Uremic Syndrome	19	Eculizumab (<2 yrs)	pnxpykkjho(ysjwgqpwqd) = ifxeqdaqiw wxaruxwilx (hcyrwrsbio ) View more	Positive	19 Jul 2024
				Eculizumab (2 to <12 yrs)	pnxpykkjho(ysjwgqpwqd) = lipevcssgy wxaruxwilx (hcyrwrsbio ) View more
NCT03500549 (PEGASUS, EHA2024) Manual	Phase 3	Hemoglobinuria, Paroxysmal	68	pegcetacoplaneculizumab	-	Positive	14 May 2024
				pegcetacoplan +eculizumab (patients with baseline IO)	wjyryxhjxz(zrfscyevmb) = vluoplcwnz ouyzwfmcsj (aqzrzzpgxs, 8.0) View more
EHA2024	Not Applicable	Hemoglobinuria, Paroxysmal	200	Eculizumab	ntsmfojwye(yftoytfhyb) = egsgjudgpc vxgwprodir (ytyqxkdqcz ) View more	Positive	14 May 2024
				Ravulizumab	ntsmfojwye(yftoytfhyb) = selubyodnk vxgwprodir (ytyqxkdqcz ) View more
ISN WCN2024	Not Applicable	Atypical Hemolytic Uremic Syndrome genetic mutations in the alternate complement pathway	6	Eculizumab sparing protocols	qdwpxswmla(qbclespboh) = None had BK virus infection qhbgmypjxp (phmopiaruc ) View more	Positive	01 Apr 2024

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