Real-life Treatment Outcomes of Ravulizumab in Polish Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH). Prospective and Retrospective, Multicenter, Non-interventional Study.

The PNH-RECORD study, a Polish multicenter observational (non-interventional), open-label, retrospective with prospective follow-up.

Start Date31 Jan 2025

Sponsor / Collaborator

AstraZeneca PLC

NCT06333652 / RecruitingPhase 2IIT

Clinical Trial on the Use of Ravulizumab in Pregnancies Complicated by Severe Hypertensive Disorders

The researchers are testing a medication named ravulizumab for the treatment of severe preeclampsia and Hemolysis, Elevated Liver enzymes, Low Platelets (HELLP) syndrome.

Start Date01 Dec 2024

Sponsor / Collaborator

Mayo Clinic

CTR20242703 / Active, not recruitingPhase 3

一项在中国阵发性睡眠性血红蛋白尿症（PNH）补体抑制剂初治成人受试者中评价ravulizumab的有效性、安全性、药代动力学、药效学和免疫原性的III期、单臂、开放标签、多中心研究

[Translation] A phase III, single-arm, open-label, multicenter study to evaluate the efficacy, safety, pharmacokinetics, pharmacodynamics, and immunogenicity of ravulizumab in Chinese adults with paroxysmal nocturnal hemoglobinuria (PNH) who are complement inhibitor-naive

评价ravulizumab在PNH成人受试者中的有效性

[Translation]

To evaluate the efficacy of ravulizumab in adult subjects with PNH

Start Date10 Oct 2024

Sponsor / Collaborator

AstraZeneca Global R&D (China) Co., Ltd.

[+2]

100 Clinical Results associated with Ravulizumab-CWVZ

100 Translational Medicine associated with Ravulizumab-CWVZ

100 Patents (Medical) associated with Ravulizumab-CWVZ

236

Literatures (Medical) associated with Ravulizumab-CWVZ

01 Jan 2025·Neurology-Neuroimmunology & Neuroinflammation

Ravulizumab and Efgartigimod in Myasthenia Gravis

Article

Author: Lünemann, Jan D ; Herdick, Meret ; Aigner, Annette ; Stein, Maike ; Lehnerer, Sophie ; Gerischer, Lea M ; Sousa, Carla Daiane Ferreira de ; Stascheit, Frauke ; Tzartos, John ; Keller, Christian W ; Doksani, Paolo ; Lazaridis, Konstantinos ; Behrens, Malina ; Hoffmann, Sarah ; Wiendl, Heinz ; Meisel, Andreas ; Klotz, Luisa

BACKGROUND AND OBJECTIVES:

Biologics that target pathogenic antibodies (Abs) and their effector functions such as the complement inhibitor ravulizumab and the neonatal Fc receptor agonist efgartigimod have recently been approved for the treatment of acetylcholine receptor (AChR)-Ab-positive myasthenia gravis (MG), but comparative studies are lacking.

METHODS:

In a prospective, exploratory real-world study, we assessed clinical efficacy, safety, and biological effects of ravulizumab and efgartigimod treatment initiation. Myasthenia Gravis-Activities of Daily Living and Quantitative Myasthenia Gravis scores were used as clinical endpoints. Ab effector functions were determined by AChR-Ab-dependent complement activation and phagocytosis assays and systemic complement activation profiling.

RESULTS:

We observed similar moderate short-term efficacy of ravulizumab and efgartigimod in achieving clinical improvement. Ravulizumab reduced systemic terminal complement activation, but neither treatment showed significant effects on complement pathways proximal to C5 or functional capacities of AChR-Abs. Both treatment modalities were well tolerated with no serious adverse events reported.

DISCUSSION:

Clinical benefits obtained with ravulizumab and efgartigimod can be remarkably heterogeneous in daily clinical practice. Neither treatment relevantly changed effector functions of pathogenic AChR-Abs, supporting the concept that durable disease control in MG requires continuous administration of both fast-acting agents.

CLASSIFICATION OF EVIDENCE:

This study provides Class III evidence that in AChR-Ab-positive patients with generalized MG, ravulizumab and efgartigimod provide comparable modest improvement in MG functional scales.

01 Dec 2024·EUROPEAN JOURNAL OF NEUROLOGY

Time to response with ravulizumab, a long‐acting terminal complement inhibitor, in adults with anti‐acetylcholine receptor antibody‐positive generalized myasthenia gravis

Article

Author: Meisel, Andreas ; Benatar, Michael ; Katsuno, Masahisa ; Vu, Tuan ; Howard, James F. ; Attarian, Shahram ; Beasley, Kathleen N. ; Habib, Ali A. ; Liao, Serena

Abstract:

Background and Purpose:

The efficacy and safety of ravulizumab, a terminal complement C5 inhibitor, in adults with anti‐acetylcholine receptor antibody‐positive (AChR Ab+) generalized myasthenia gravis (gMG) were demonstrated in the CHAMPION MG study (NCT03920293). This analysis aimed to characterize the latency to onset of a clinically meaningful therapeutic effect for ravulizumab.

Methods:

Post hoc analysis of data collected for up to 60 weeks from CHAMPION MG was performed to assess the timing of response to ravulizumab. Response was analyzed based on reductions of ≥2 and ≥3 points (minimal clinically important differences [MCIDs]) in Myasthenia Gravis–Activities of Daily Living (MG‐ADL) and Quantitative Myasthenia Gravis (QMG) total scores, respectively, and on more rigorous reductions of ≥3 and ≥5 points, respectively. Time to first response was assessed using the Kaplan–Meier product‐limit method.

Results:

The median (95% confidence interval) time to first response was 2.1 (2.1–2.6) and 4.1 (2.3–10.0) weeks for reductions of ≥2 and ≥3 points in MG‐ADL total score, respectively (n = 139), and 4.1 (2.1–10.0) and 18.3 (11.0–33.4) weeks for reductions of ≥3 and ≥5 points in QMG total score, respectively (n = 134). Cumulative response rates at Week 60 (data cut‐off) were 88% and 82% for ≥2‐ and ≥3‐point MG‐ADL score reductions, respectively, and 86% and 59% for ≥3‐ and ≥5‐point QMG score reductions, respectively.

Conclusions:

The median times to MCID with ravulizumab treatment in patients with AChR Ab+ gMG were ~2 weeks and ~4 weeks based on MCID MG‐ADL and QMG total score reductions, respectively.

01 Dec 2024·RHEUMATOLOGY

Eculizumab and ravulizumab in the treatment of refractory antiphospholipid syndrome—a first case report

Article

Author: Karandyszowska, Natali ; Antovic, Aleksandra ; Yilmaz, Esin Beste ; Faustini, Francesca ; Svenungsson, Elisabet ; Gunnarsson, Iva ; Bruzelius, Maria

193

News (Medical) associated with Ravulizumab-CWVZ

12 Dec 2024

·medcitynews.com

ASH 2024 Recap: Movement in Multiple Myeloma, Cell Therapy, Sickle Cell Disease & More - MedCity News

The annual meeting of the American Society of Clinical Oncology drew more than 30,000 attendees to San Diego, and more who participated in the conference virtually, all of them looking to see and hear the latest developments in blood cancers and hematological disorders. Multiple myeloma is always a big topic of discussion at the annual ASH meeting, and developments this year include clinical data supporting the use of certain therapies in even earlier lines of treatment. On the cell therapy front, there is an ongoing effort to improve the manufacturability of these treatments. Data were presented demonstrating shorter “vein-to-vein times,” the time from when cells are harvested to when therapeutic cells are infused into the patient. And in sickle cell disease, encouraging early data were presented showing promise for a gene-editing therapy that could offer patients a one-time treatment. We couldn’t catch everything at what is one of the biggest medical meetings of the year. Here’s a recap of some highlights from the ASH 2024 meeting: Sponsored Post The Funding Model for Cancer Innovation is Broken — We Can Fix It Closing cancer health equity gaps require medical breakthroughs made possible by new funding approaches. By Eve McDavid - CEO of Mission-Driven Tech Movement in Multiple Myeloma —GSK reported Phase 3 data that support bringing the multiple myeloma drug Blenrep back to the market. The latest results show a Blenrep drug regimen, as a second-line of treatment, helped patients live longer compared against a drug regimen that includes the blockbuster Johnson & Johnson drug Darzalex. These results at ASH follow a Phase 3 data readout earlier this year showing Blenrep beat a drug combination that includes the Bristol Myers Squibb cancer drug Pomalyst. Blenrep won accelerated approval in 2020. GSK withdrew Blenrep from the market after it failed a confirmatory Phase 3 study. But that test evaluated the drug as a monotherapy and as a fifth line of treatment. The Phase 3 studies that are part of regulatory submissions currently under review tested the drug in combination with other multiple myeloma therapies and as an earlier line of treatment. GSK’s plans for the drug include a study underway that could support expanding the drug’s use to first-line multiple myeloma treatment, where its competition will include Darzalex. —Speaking of Darzalex, Johnson & Johnson reported data for an injectable formulation of the drug, Darzalex Faspro, as a treatment for smoldering multiple myeloma, which is an asymptomatic precursor to this type of blood cancer. In smoldering multiple myeloma, patients have high levels of abnormal plasma cells and elevated levels of monoclonal protein in the blood. presented by Market Trends to Watch for Health Systems and Their Specialty Pharmacies The future looks bright for the integrated specialty pharmacy model – for health systems, providers, and most importantly, for patients. By Jake Gaffey, MBA, Chief Strategy Officer at Shields Health Solutions In a Phase 3 test that enrolled 390 participants, J&J’s Darzalex Faspro significantly delayed high-risk smoldering multiple myeloma from progressing to active disease. The drug also extended overall survival compared to active monitoring, which is the current standard of care. Darzalex Faspro currently has nine approvals in multiple myeloma. Last month, the company submitted applications in the U.S. and Europe seeking to add smoldering multiple myeloma to the product’s list of approved indications. —Arcellx’s BCMA-targeting cell therapy, anito-cel, posted Phase 2 results in multiple myeloma that analysts say are comparable to Carvykti from partners Legend Biotech and Johnson & Johnson. It’s the safety results that reinforce their view of the Arcellx cell therapy as potentially best in class. Carvykti’s trials showed some cases of delayed neurological toxicities, including parkinsonism, a movement disorder. Arcellx is engineered to offer better safety and the results so far show no signs of parkinsonism. Analysts say Arcellx also has advantages through its partnership with Gilead Sciences, which has a global cell therapy manufacturing infrastructure and experience commercializing such products. —J&J and Legend added to the body of evidence showing that Carvykti is helping multiple myeloma patients live longer compared to standard therapies. The Phase 3 study, which tested the cell therapy in patients who had received one to three prior lines of therapy, showed that after a median 33.6 months of follow-up, Carvykti led to significantly increased rates of rates of minimal residual disease negativity, a marker of survival outcomes for multiple myeloma patients. A Look at Leukemia & Lymphoma Drugs —Kura Oncology reported more detailed data for ziftomenib, a drug candidate now partnered with Kyowa Kirin. The small molecule menin inhibitor is being developed to treat leukemias driven by certain genetic mutations. Results from the Phase 1 dose-escalation study showed a 100% complete response rate in those with NPM1 mutations. In patients whose disease harbored KMT2A mutations, the complete response rate was 83%. The companies said the drug was safe and well tolerated in combination with standard of care therapies. Last month, Kyowa Kirin paid $330 million to begin the alliance on ziftomenib. If the drug reaches the market, it will compete against Revuforj, a Syndax Pharmaceuticals menin inhibitor that won FDA approval in November. —Eli Lilly drug Jaypirca landed accelerated FDA approval last year as a treatment for advanced cases of mantle cell lymphoma following at least two prior lines of therapy. At the ASH conference, Lilly reported Phase 3 data that could support full approval for the drug, a small molecule inhibitor of a protein called BTK. Results show Jaypirca reduced the risk of disease progression or death by 46% compared to standard treatments. Treatment with the Lilly drug also prolonged the time to the next treatment or death by a median 23.9 months. Lilly said the safety profile of Jaypirca in Phase 3 was consistent with the Phase 1/2 test used to support the drug’s accelerated approval. —In a pre-planned analysis of a Phase 2 clinical trial in diffuse large B-cell lymphoma, Merck’s experimental drug zilovertamab vedotin led to a 97.2% complete response rate. What kept the study from achieving the 100% mark was discontinuation of two participants by physician decision, one patient each in the middle- and high-dose cohorts. All participants in the low-dose group showed a complete response. Based on the clinical trial results for the drug candidate, an antibody drug conjugate (ADC), Merck has selected the low dose to advance to Phase 3 testing. The results so far are encouraging for this therapy and others developed to seek out ROR1, a novel target currently unaddressed by any approved drugs. Other companies developing ROR1-targeting ADCs include Ipsen, which licensed its candidate from Sutro Biopharma earlier this year, and CStone Pharmaceuticals. Sickle Cell Disease Developments —Last month, Beam Therapeutics teased encouraging early clinical results for six patients dosed with BEAM-101, a gene-editing therapy for sickle cell disease. Updated data at ASH put some figures to the results as of an Oct. 28 data cutoff. Results showed these patients achieved fetal hemoglobin levels exceeding 60% and a reduction in sickle-shaped hemoglobin below 40% at month 1. Those levels were sustained to the last time point available, indicating that the effects of the one-time treatment are durable so far. The therapy’s safety profile was consistent with that of busulfan, the conditioning therapy that is used to prepare a patient to receive BEAM-101. As previously reported, there was one fatality from respiratory failure, which investigators attributed to busulfan. In all six patients dosed, there have been no serious adverse events attributed to BEAM 101. —Novo Nordisk presented Phase 2 data for etavopivat showing the once-daily oral drug led to a reduction in vaso-occlusive crises, a complication of sickle cell disease. The one-year, proof-of-concept study also showed an increase in hemoglobin levels. With these results, Novo Nordisk said it has selected the dose to test in a Phase 3 clinical trial. Etavopivat came to Novo Nordisk from the $1.1 billion acquisition of Forma Therapeutics in 2022. Catching Up on Cell Therapies —The cancer target GPRC5D is currently addressed by only one FDA-approved drug, the Johnson & Johnson bispecific antibody Talvey, a treatment for multiple myeloma. Bristol Myers Squibb aims to be the first to drug GPRC5D with a cell therapy. At the ASH meeting, BMS presented Phase 1 data showing its candidate, arlocabtagene autoleucel (arlo-cel), led to durable responses that deepened over time. Bristol says these results support arlo-cel as a potential first-in-class treatment for relapsed or refractory multiple myeloma in heavily pretreated patients. A Phase 2 test of the cell therapy is ongoing. —Galapagos’s non-Hodgkin lymphoma cell therapy candidate GLPG5101 posted additional Phase 1/2 results showing encouraging efficacy and safety. The company also pointed out that the vein-to-vein time was a median seven days. By comparison, the multi-step manufacturing process for currently available cell therapies can take a month or more. Galapagos’s process leverages Cocoon, a point-of-care manufacturing system from contract manufacturing giant Lonza. —Orca Bio posted data for Orca-T, an allogeneic T-cell immunotherapy in testing as a treatment for three types of blood cancer: acute myeloid leukemia, acute lymphoblastic leukemia, and high-risk myelodysplastic syndrome. Results from a three-year follow-up analysis from Phase 1b showed an overall survival rate of 86% for the study drug compared to 67% in a non-randomized historical group that received an allogeneic stem cell transplant. Orca-T is comprised of highly purified immune cells from donors. In the Phase 1b test, Orca Bio said this allogeneic therapy was manufactured reliably with vein-to-vein time of 72 hours or less across the U.S. An ongoing Phase 3 test comparing Orca-T to conventional allogeneic stem cell transplants is expected to report preliminary data in the first half of 2025. A Recap in Rare Blood Disease —Sanofi revealed details of its Phase 3 test of rilzabrutinib in persistent or chronic immune thrombocytopenia, a rare immune disorder leading to low platelet levels. Results show the small molecule BTK inhibitor led to platelet response in 65% of patients in the study drug arm compared to 33% of those who received a placebo. Furthermore, a durable platelet response was observed in 23% of patients who received rilzabrutinib versus zero in the placebo group. The drug, which came from the $3.7 billion acquisition of Principia Biopharma in 2020, is currently under regulatory review in the U.S. and Europe. —Standard of care for the rare blood disorder paroxysmal nocturnal hemoglobinuria (PNH) includes two blockbuster AstraZeneca drugs, Soliris and Ultomiris, though Apellis Pharmaceuticals and Novartis are trying to take market share with their FDA-approved therapies. Regeneron Pharmaceuticals aims to compete with those complement inhibitors with a drug combination: the approved antibody pozelimab (brand name Veopoz) and the experimental RNA-interference therapy cemdisiran. A Phase 3 test is underway testing the combination against Soliris. At ASH, Regeneron reported data from an exploratory cohort testing the two drugs against Ultomiris. Results showed the Regeneron drug combo helped patients achieve and maintain greater control over their disease compared to Ultomiris. Public domain image by Flickr user SciTechTrend

Clinical ResultPhase 3ASCOPhase 1Cell Therapy

09 Dec 2024

·pmlive.com

Regeneron shares positive late-stage results for poze-cemdi in rare blood disorder PNH

Regeneron Pharmaceuticals has shared positive data from a phase 3 trial of pozelimab plus cemdisiran (poze-cemdi) in patients with the rare blood disorder paroxysmal nocturnal haemoglobinuria (PNH). Results from an exploratory cohort of the ACCESS-1 trial were presented at this year’s American Society of Hematology (ASH) annual meeting and support the continued development of the combination treatment in PNH and other complement-mediated diseases, Regeneron said. Affecting up to 1.5 people per million in the US, PNH is caused by a genetic mutation that results in the body’s complement system attacking its red blood cells. Patients can experience a range of symptoms, including fatigue, shortness of breath and life-threatening blood clots. Pozelimab is a fully human monoclonal antibody designed to block the activity of the C5 protein, which is involved in complement system activation, while cemdisiran is an investigational siRNA therapeutic that reduces circulating levels of C5. Data from ACCESS-1 showed that 96% of poze-cemdi-treated patients achieved adequate lactate dehydrogenase (LDH), a biomarker used to measure the degree of haemolysis, control across study visits (weeks eight to 26) on average, compared to 80% of those randomised to receive AstraZeneca’s standard-of-care C5 inhibitor ravulizumab, marketed under the brand name Ultomiris. Of the patients in the poze-cemdi group, 93% achieved LDH normalisation across study visits on average, compared to 65% of those in the ravulizumab arm, and an 84% decrease in LDH from baseline was observed for Regeneron’s combination compared to 74% with ravulizumab. After 26 weeks, all patients who completed ACCESS-1 could enrol in a follow-on open label extension trial (OLE) and receive poze-cemdi. At the start of the OLE, 68% of patients treated with ravulizumab had adequate LDH control, with this rising to 95% after switching to poze-cemdi, including four of five patients who had failed to achieve LDH control while on ravulizumab. A separate registrational cohort investigating poze-cemdi against another of AstraZeneca’s C5 inhibitors Soliris (eculizumab) is ongoing. ACCESS-1 trial investigator, Christopher Patriquin, University of Toronto and University Health Network, said: “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.” “This data validates 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,” he added.

Clinical ResultPhase 3ASH

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

100 Deals associated with Ravulizumab-CWVZ

External Link

KEGG	Wiki	ATC	Drug Bank
-	Ravulizumab-CWVZ	L04AA43	DB11580

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Neuromyelitis Optica	EU	Alexion Europe SAS	12 Jul 2023
Neuromyelitis Optica	IS	Alexion Europe SAS	12 Jul 2023
Neuromyelitis Optica	LI	Alexion Europe SAS	12 Jul 2023
Neuromyelitis Optica	NO	Alexion Europe SAS	12 Jul 2023
Thrombotic Microangiopathies	KR	AstraZeneca Korea Ltd.	21 May 2020
AQP4-IgG positive Neuromyelitis optica spectrum disorder	AU	Alexion Pharmaceuticals Australasia Pty Ltd.	17 Oct 2019
Myasthenia Gravis	AU	Alexion Pharmaceuticals Australasia Pty Ltd.	17 Oct 2019
Atypical Hemolytic Uremic Syndrome	EU	Alexion Europe SAS	02 Jul 2019
Atypical Hemolytic Uremic Syndrome	IS	Alexion Europe SAS	02 Jul 2019
Atypical Hemolytic Uremic Syndrome	LI	Alexion Europe SAS	02 Jul 2019
Atypical Hemolytic Uremic Syndrome	NO	Alexion Europe SAS	02 Jul 2019
Hemoglobinuria, Paroxysmal	US	Alexion Pharmaceuticals, Inc.	21 Dec 2018

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Glomerulonephritis, IGA	Phase 3	US	AstraZeneca Global R&D (China) Co., Ltd.	29 Mar 2024
Glomerulonephritis, IGA	Phase 3	US	Alexion Pharmaceuticals, Inc.	29 Mar 2024
Glomerulonephritis, IGA	Phase 3	CN	Alexion Pharmaceuticals, Inc.	29 Mar 2024
Glomerulonephritis, IGA	Phase 3	JP	AstraZeneca Global R&D (China) Co., Ltd.	29 Mar 2024
Glomerulonephritis, IGA	Phase 3	JP	Alexion Pharmaceuticals, Inc.	29 Mar 2024
Glomerulonephritis, IGA	Phase 3	AR	AstraZeneca Global R&D (China) Co., Ltd.	29 Mar 2024
Glomerulonephritis, IGA	Phase 3	AR	Alexion Pharmaceuticals, Inc.	29 Mar 2024
Glomerulonephritis, IGA	Phase 3	AU	AstraZeneca Global R&D (China) Co., Ltd.	29 Mar 2024
Glomerulonephritis, IGA	Phase 3	AU	Alexion Pharmaceuticals, Inc.	29 Mar 2024
Glomerulonephritis, IGA	Phase 3	AT	Alexion Pharmaceuticals, Inc.	29 Mar 2024

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


ASH2024	Not Applicable	Atypical Hemolytic Uremic Syndrome	-	Eculizumab	xodyobeylr(udhdlvcgxa) = fdgoktvzzb kklprtjqfb (lrolvixwgg, 230.0 - 362.0) View more	-	08 Dec 2024
				Ravulizumab	lecmgktnce(vmuzutyihe) = ricpabepoq lfiohgkqeb (ttldqsrdll, 11.5% - 36.4%)
NCT02949128 (ALXN1210-aHUS-311, Pubmed \| Thromb J)	Phase 3	Atypical Hemolytic Uremic Syndrome platelet count \| lactate dehydrogenase (LDH) \| serum creatinine (sCr)	56	Ravulizumab	qzibnbrbws(ycnxabivyw) = kvmfbdplsv plgqoolylj (etqfpuksvz )	Positive	28 Oct 2024
NCT04320602 (CTgov)	Phase 4	Hemoglobinuria, Paroxysmal	18	Eculizumab+Ravulizumab	ungthvbmrg(hrkzsvhfzk) = hrmvyuemlv dmkpsgdbza (cgmnkrbcex, fnipkkkghr - ynrjlrqqgc) View more	-	12 Aug 2024
MG-SPOTLIGHT Registry (EAN2024)	Not Applicable	Myasthenia Gravis	70	Ravulizumab	tkabzegqhi(colsfpsdkg) = xklwevbdhg ejtajrmtzx (rcjpyyxmsv, 88.9)	Positive	28 Jun 2024
EHA2024	Not Applicable	Hemoglobinuria, Paroxysmal	200	Eculizumab	vlhhlbifkj(yfcjliscmx) = pwzlrivich wfvmlwajml (frvlovtsah ) View more	Positive	14 May 2024
				Ravulizumab	vlhhlbifkj(yfcjliscmx) = koxjpdhlyk wfvmlwajml (frvlovtsah ) View more
P10-11.002 (AAN2024)	Not Applicable	Myasthenia Gravis acetylcholine receptor-positive (AChR+)	204	Eculizumab	zdnbcgkzru(gmuexpwlms) = psfsejzfoa imvekjvusw (ytillvtxzb, 3.7)	Positive	09 Apr 2024
				Ravulizumab	zdnbcgkzru(gmuexpwlms) = qktajditdz imvekjvusw (ytillvtxzb, 4.1)
NCT04564339 (WCN24-1197, ISN WCN2024)	Phase 2	Glomerulonephritis, IGA	66	Ravulizumab	fdinkgobak(kxwfkiywgc) = oicnysueyv qhddefzdys (zrgxeooqas )	Positive	01 Apr 2024
				Placebo	fdinkgobak(kxwfkiywgc) = gjqsqtkvbf qhddefzdys (zrgxeooqas )
ACTRIMS2024	Not Applicable	Neuromyelitis Optica	-	Ravulizumab with or without immunosuppressive therapy	horhddvhlt(stlzrcqnoy) = pdyjfygcpr tuzmawtocl (dvcbarfqeg ) View more	-	01 Mar 2024
				Placebo with or without immunosuppressive therapy	psdqotclcx(tjqecqjvig) = hphnwucolt jcczyretkb (kuuiubfohy )
NCT05288816 (CTgov)	Phase 1	-	16	ALXN1210 (Cohort 1: ALXN1210 400 mg Single Dose)	pjzgyylhlk(dygpwvrnxu) = iwakgqvolr vqfakstgcz (ubqjfehpfh, hqgqlufwtv - drrlxsgols) View more	-	05 Feb 2024
				ALXN1210 (Cohort 2: ALXN1210 800 mg Single Dose)	pjzgyylhlk(dygpwvrnxu) = iovrnsmror vqfakstgcz (ubqjfehpfh, rswmxjtwzl - edbvuhpkyq) View more
NCT04201262 (CHAMPION-NMOSD, Pubmed \| Front Neurol)	Phase 3	AQP4-IgG positive Neuromyelitis optica spectrum disorder anti-aquaporin-4 antibody-positive	58	Ravulizumab	ypieyihgvw(jjhwukxsjh) = nhaxpxmrcy kfawtdetee (xtuwtosklh, 217.68)	Positive	31 Jan 2024

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