This is an open-label study designed to evaluate the long-term safety and efficacy of evinacumab, a fully human ANGPTL3 antibody, in patients with homozygous familial hypercholesterolemia (HoFH), in a real-life setting in Canada.
Eligible patients for this study are male and female adult patients with HoFH. Evinacumab will be added on top of the patient's background lipid-modifying therapy (LMT), including statins, ezetimibe, PCSK9 inhibitors, lomitapide or other lipid lowering therapies. This study will be conducted using an hybrid (on-site, foldable sites) approach. Patients will enter the current study, in an open-label treatment period, following their screening. This study will continue until reimbursement of evinacumab in Canada or for a maximum of 24 months. The end of study (EoS) visit will be scheduled 4 weeks after the last dose has been injected and will be followed by a 52-week follow-up.

Start Date21 Feb 2023

Sponsor / Collaborator

Ultragenyx Pharmaceutical, Inc.

NCT04863014 / TerminatedPhase 2

A Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Evinacumab in Patients With Severe Hypertriglyceridemia for the Prevention of Recurrent Acute Pancreatitis

The primary objective of the study is to determine the proportion of patients with elevated triglycerides (TG), without familial chylomicronemia syndrome (FCS) due to loss of function (LoF) mutations in lipoprotein lipase (LPL), and a history of hypertriglyceridemia (HTG)-associated acute pancreatitis (AP) who experience a recurrent episode of AP after treatment with evinacumab versus placebo.
The secondary objectives of the study are:
To determine the change in the standard lipid profile after therapy with evinacumab versus placebo
To determine the changes in specialty lipoprotein parameters (ApoC3, ApoB48, ApoB100, and nuclear magnetic resonance [NMR] lipid profile) after therapy with evinacumab versus placebo
To measure the number of AP episodes per patient
To assess the safety and tolerability of evinacumab
To assess the potential immunogenicity of evinacumab
To assess the concentrations of total evinacumab and total angiopoietin-like 3 (ANGPTL3)

Start Date12 Jul 2021

Sponsor / Collaborator

Regeneron Pharmaceuticals, Inc.

100 Clinical Results associated with Evinacumab-dgnb

100 Translational Medicine associated with Evinacumab-dgnb

100 Patents (Medical) associated with Evinacumab-dgnb

Literatures (Medical) associated with Evinacumab-dgnb

11 Nov 2024·European journal of preventive cardiology

Current treatments for the management of homozygous familial hypercholesterolaemia: a systematic review and commentary

Review

Author: Gupta, Rupal N ; Xu, Yingxin ; Gu, Jing ; Cheng, Henry K ; Raal, Frederick J

Abstract:

Aims:

Homozygous familial hypercholesterolaemia (HoFH) is a rare disorder characterized by markedly elevated circulating low-density lipoprotein cholesterol (LDL-C) from birth. This review aimed to critically evaluate treatments for HoFH with respect to their efficacy, safety, accessibility, overall context and position within the treatment pathway.

Methods and results:

A mixed-methods review was undertaken to systematically identify and characterize primary interventional studies on HoFH, with a focus on LDL-C reduction as the primary outcome. Interventions assessed were ezetimibe, proprotein convertase subtilisin/kexin type 9 inhibitors (PCSK9i), lomitapide, evinacumab, with or without LDL apheresis. Twenty-six seminal studies reporting unique patient data were identified. Four studies were randomized controlled trials (RCTs) with the remainder being single-arm trials or observational registries. Data extracted were heterogeneous and not suitable for meta-analyses. Two RCTs, assessed at being low risk of bias, demonstrated PCSK9i were safe and moderately effective. A randomized controlled trial (RCT) demonstrated evinacumab was safe and effective in all HoFH subgroups. Lomitapide was reported to be efficacious in a single-arm trial, but issues with adverse events, tolerability, and adherence were identified. An RCT on ezetimibe showed it was moderately effective when combined with a statin. LDL apheresis was reported as effective, but its evidence base was at very high risk of bias. All interventions lowered LDL-C, but the magnitude of this, and certainty in the supporting evidence, varied.

Conclusion:

In practice, multiple treatments are required to treat HoFH. The sequencing of these should be made on an individualized basis, with consideration made to the benefits of each intervention.

21 Oct 2024·EUROPEAN HEART JOURNAL

Correction to: Evinacumab in homozygous familial hypercholesterolaemia: long-term safety and efficacy

01 Sep 2024·Journal of Clinical Lipidology

Real-world experience of long-term efficacy and safety of evinacumab in patients with homozygous familial hypercholesterolemia treated and untreated with lipoprotein apheresis

Article

Author: Zeppa, Giovanna ; Chan, Dick C ; Watts, Gerald F ; Stefanutti, Claudia

BACKGROUND:

Evinacumab is an inhibitor of angiopoietin-like 3 protein (ANGPTL3) that offers a new approach for correcting high low-density lipoprotein-cholesterol (LDL-C) and may reduce the need or frequency for lipoprotein apheresis (LA) in patients with homozygous familial hypercholesterolemia (HoFH).

OBJECTIVE:

We aimed to investigate the long-term efficacy and safety of evinacumab in patients with HoFH aged between 14 and 63 years on and off LA in real-world clinical practice.

METHODS:

Evinacumab was administrated intravenously (15 mg /kg every 4 weeks) for the first 24 months in 7 patients with genetically confirmed HoFH, receiving best standard of lipid-lowering treatment and LA, followed by a subsequent compassionate extension period of approximately 12-month treatment with evinacumab without LA. Patient experience of evinacumab and health-related EuroQol (EQ-5D-3L) quality of life questionnaire were also assessed.

RESULTS:

Compared with baseline, evinacumab resulted in sustained reductions in plasma LDL-C concentration of -43.4% and -54.2% at 30 and 36 months, respectively. All 7 HoFH patients achieved an LDL-C reduction >30% with 3 patients having on-treatment LDL-C level < 2.5 mmol/L (96 mg/dL). Evinacumab was well-tolerated, with no major adverse events reported or significant changes in liver enzyme concentrations. All FH patients agreed that evinacumab was acceptable and less physically demanding than LA. The mean EQ- utility score and visual analogue score were 0.966 and 78.6, respectively, which are comparable to the Italian general population.

CONCLUSIONS:

Our findings suggest that evinacumab is a safe and effective treatment for high LDL-C that is acceptable to HoFH patients receiving and not receiving LA.

113

News (Medical) associated with Evinacumab-dgnb

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

18 Nov 2024

·www.globenewswire.com

Dupixent® (dupilumab) sBLA Accepted for FDA Review for the Treatment of Chronic Spontaneous Urticaria (CSU)

Nov. 15, 2024 -- Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) and Sanofi today announced that the U.S. Food and Drug Administration (FDA) has accepted for review the resubmission of the supplemental Biologics License Application (sBLA) for Dupixent® (dupilumab) to treat adults and pediatric patients aged 12 years and older with chronic spontaneous urticaria (CSU) whose disease is not adequately controlled with H1 antihistamine treatment. The target action date for the FDA decision is April 18, 2025. The resubmitted sBLA is supported by data from the multi-trial, LIBERTY-CUPID Phase 3 clinical program (Study A, Study B, and Study C) for Dupixent in CSU. The sBLA adds results from Study C, which was conducted in patients with uncontrolled CSU who were on standard-of-care antihistamines. Study C, the second LIBERTY-CUPID pivotal trial in biologic-naïve patients, met its primary and key secondary endpoints confirming results seen in the previous Study A. Results showed Dupixent significantly reduced itch and urticaria activity (itch and hives). Safety results in all LIBERTY-CUPID Phase 3 trials were generally consistent with the known safety profile of Dupixent in its approved indications. Adverse events more commonly observed with Dupixent (≥5%) compared to placebo were injection site reactions and COVID-19 infection. CSU is a chronic inflammatory skin disease driven in part by type 2 inflammation, which causes sudden and debilitating hives and recurring itch. CSU is typically treated with H1 antihistamines, medicines that target H1 receptors on cells to control symptoms of urticaria. However, the disease remains uncontrolled despite antihistamine treatment in many patients, some of whom are left with limited alternative treatment options. These individuals continue to experience symptoms that can be debilitating and significantly impact their quality of life. More than 300,000 people in the U.S. suffer from CSU that is inadequately controlled by antihistamines. The LIBERTY-CUPID Phase 3 study program evaluating Dupixent for CSU consists of Study A, Study B, and Study C. Study A and Study C were conducted in CSU patients who were uncontrolled on standard-of-care antihistamines while Study B was conducted in CSU patients who were uncontrolled on standard-of-care antihistamines and refractory or intolerant to omalizumab. Dupixent has been approved for CSU in Japan and the United Arab Emirates (UAE) and is also under regulatory review in the European Union based on earlier trial readouts. Outside of Japan and the UAE, the safety and efficacy of Dupixent for CSU has not been fully evaluated by any regulatory authority. Dupixent, which was invented using Regeneron’s proprietary VelocImmune® technology, is a fully human monoclonal antibody that inhibits the signaling of the IL-4 and IL-13 pathways and is not an immunosuppressant. The Dupixent development program has shown significant clinical benefit and a decrease in type 2 inflammation in Phase 3 trials, establishing that IL-4 and IL-13 are two of the key and central drivers of type 2 inflammation that play a major role in multiple related and often co-morbid diseases. Dupixent has received regulatory approvals in more than 60 countries in one or more indications including certain patients with atopic dermatitis, asthma, chronic rhinosinusitis with nasal polyps, eosinophilic esophagitis, prurigo nodularis, CSU, and chronic obstructive pulmonary disease in different age populations. More than 1,000,000 patients are currently being treated with Dupixent globally.1 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. This includes REGEN-COV® (casirivimab and imdevimab), Dupixent, Libtayo® (cemiplimab-rwlc), Praluent® (alirocumab), Kevzara® (sarilumab), Evkeeza® (evinacumab-dgnb), Inmazeb® (atoltivimab, maftivimab and odesivimab-ebgn) and Veopoz® (pozelimab-bbfg). Dupilumab is being jointly developed by Regeneron and Sanofi under a global collaboration agreement. To date, dupilumab has been studied across more than 60 clinical trials involving more than 10,000 patients with various chronic diseases driven in part by type 2 inflammation. In addition to the currently approved indications, Regeneron and Sanofi are studying dupilumab in a broad range of diseases driven by type 2 inflammation or other allergic processes in Phase 3 trials, including chronic pruritus of unknown origin and bullous pemphigoid. These potential uses of dupilumab are currently under clinical investigation, and the safety and efficacy in these conditions have not been fully evaluated by any regulatory authority. 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. We are an innovative global healthcare company, driven by one purpose: we chase the miracles of science to improve people's lives. Our team, across the world, is dedicated to transforming the practice of medicine by working to turn the impossible into the possible. We provide potentially life-changing treatment options and life-saving vaccine protection to millions of people globally, while putting sustainability and social responsibility at the center of our ambitions. The content above comes from the network. if any infringement, please contact us to modify.

Clinical ResultDrug ApprovalPriority Review

13 Nov 2024

·www.globenewswire.com

Regeneron to Highlight Pioneering Pipeline Progress Across Multiple Modalities Spanning 10 Types of Blood Cancers and Disorders at ASH

Oral presentation shares head-to-head results for investigational combination pozelimab plus cemdisiran vs. ravulizumab in paroxysmal nocturnal hemoglobinuria Initial results for odronextamab in first-line follicular lymphoma showcase compelling monotherapy potential and progress in the confirmatory trial Additional oral presentations explore odronextamab in areas of high unmet need, including the primary analysis in diffuse large B-cell lymphoma progressing after CAR-T therapy and first results in relapsed/refractory marginal zone lymphoma TARRYTOWN, N.Y., Nov. 13, 2024 (GLOBE NEWSWIRE) -- Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) today announced new and updated data from its hematology pipeline will be shared across 23 abstracts at the American Society of Hematology (ASH) 2024 Annual Meeting, taking place from December 7-10 in San Diego, CA. The latest research advances demonstrate the potential of Regeneron’s diverse pipeline, which aims to address unmet needs in ten types of blood cancers and disorders. These innovative and differentiated approaches include CD3 bispecific antibodies, costimulatory bispecific antibodies, and a pioneering combination of a monoclonal antibody and small interfering RNA (siRNA). “Our presentations at ASH demonstrate the progress we are making toward transforming care for a range of blood cancers and disorders where advancements are desperately needed,” said L. Andres Sirulnik, M.D., Ph.D., Senior Vice President and Hematology Clinical Development Unit Head at Regeneron. “These include new head-to-head data exploring a novel combination that aims to maximize disease control in paroxysmal nocturnal hemoglobinuria compared to a standard-of-care treatment, as well as initial results from our confirmatory odronextamab trial in first-line follicular lymphoma. Together, our presentations underscore our commitment to work towards translating scientific breakthroughs to differentiated medicines for hematology patients.” At ASH, abstracts in blood disorders include an oral presentation from an exploratory cohort of a Phase 3 trial investigating a novel combination of pozelimab with cemdisiran compared to ravulizumab in patients with paroxysmal nocturnal hemoglobinuria (PNH) who were naïve to complement inhibition at baseline. Notably, the presentation will detail interim results from patients who were initially randomized to ravulizumab but crossed over to the combination in an open label extension portion of the trial. Progress on the odronextamab development program will be featured in twelve abstracts. Among them are initial results in patients with previously untreated follicular lymphoma (FL) from Part 1 of the Phase 3 OLYMPIA-1 confirmatory trial, which consists of a non-randomized safety run-in (Part 1) followed by a randomized efficacy portion (Part 2) comparing odronextamab monotherapy to rituximab plus standard-of-care chemotherapies. Two oral presentations on the ELM-1 and pivotal ELM-2 trials will feature new analyses in settings with significant unmet needs: one in patients with diffuse large B-cell lymphoma (DLBCL) who progressed after CAR-T therapy and the other in relapsed/refractory (R/R) marginal zone lymphoma (MZL). Other notable abstracts include the latest linvoseltamab efficacy and safety results from the pivotal LINKER-MM1 study in R/R multiple myeloma (MM), preclinical data evaluating a CD38xCD28 costimulatory bispecific antibody in combination with linvoseltamab, and preclinical data on TMPRSS6 inhibition in beta-thalassemia. The full list of Regeneron presentations at ASH includes: Abstract TitleAbstractPresenterSession Date/Time (PT)Odronextamab Efficacy and Safety of Odronextamab Monotherapy in Patients (Pts) with Diffuse Large B-Cell Lymphoma (DLBCL) Progressing after CAR T-Cell Therapy: Primary Analysis from the ELM-1 StudyAbstract #866Oral PresentationSessionMatthew MatasarMonday, December 9, at 2:45-4:15 pmMarriott Marquis San Diego Marina, Pacific Ballroom Salons 15-17Efficacy and Safety of Odronextamab in Relapsed/Refractory Marginal Zone Lymphoma (R/R MZL): Data from the R/R MZL Cohort in the ELM-2 StudyAbstract #862Oral PresentationSessionTae Min KimMonday, December 9, at 2:45-4:15 pmMarriott Marquis San Diego Marina, Marriott Grand Ballroom 11-13Odronextamab Monotherapy in Previously Untreated Patients with High-Risk Follicular Lymphoma (FL): Results of the Safety Lead-in of the Phase 3 OLYMPIA-1 StudyAbstract #4411Poster PresentationSessionElizabeth BremMonday, December 9, at 6:00-8:00 pmSan Diego Convention Center, Halls G-HEfficacy and Safety of Odronextamab in Rare Subtypes of Relapsed/Refractory Aggressive B-Cell Non-Hodgkin Lymphoma (B-NHL): Data from a Dedicated Cohort of Other B-NHLs in the ELM-2 StudyAbstract #4502Poster PresentationSessionEmmanuel BachyMonday, December 9, at 6:00-8:00 pmSan Diego Convention Center, Halls G-HTreatment Duration and Risk of Fatal Infections in Patients with B-Cell Non-Hodgkin Lymphoma Achieving Complete Response with OdronextamabAbstract #3080Poster PresentationGottfried von KeudelMonday, December 9, at 6:00-8:00 pmSan Diego Convention Center, Halls G-HDynamics of Complete Responses in Patients with Relapsed or Refractory Diffuse Large B Cell Lymphoma Treated with Odronextamab in the ELM-2 StudyAbstract #4486Poster PresentationSessionSabarish AyyappanMonday, December 9, at 6:00-8:00 pmSan Diego Convention Center, Halls G-HLong-Term Efficacy and Safety of Odronextamab in Relapsed/Refractory Diffuse Large B-Cell Lymphoma (DLBCL): Pooled Analysis from ELM-1 and ELM-2 StudiesAbstract #3118Poster PresentationSessionJohn N. AllanSunday, December 8, at 6:00-8:00 pmSan Diego Convention Center, Halls G-HEvaluation of CAR-HEMATOTOX Scoring as a Predictor of Infection Risk following Treatment with Odronextamab (a CD20×CD3 Bispecific Antibody) in Relapsed/Refractory Diffuse Large B-Cell LymphomaAbstract #3076Poster PresentationSessionMathew MatasarSunday, December 8, at 6:00-8:00 pmSan Diego Convention Center, Halls G-HEvaluation of Baseline CAR-HEMATOTOX Scores to Predict Increased Severe Infection Risk in Patients with Relapsed/Refractory Follicular Lymphoma Treated with OdronextamabAbstract #1650Poster PresentationSessionMatthew MatasarSaturday, December 7, at 5:30-7:30 pmSan Diego Convention Center, Halls G-HDynamics of Complete Responses in Patients with Relapsed or Refractory Follicular Lymphoma Treated with Odronextamab in the ELM-2 StudyAbstract #1628Poster PresentationSessionStefano LuminariSaturday, December 7, at 5:30-7:30 pmSan Diego Convention Center, Halls G-HTrial in Progress: Odronextamab for the Treatment of Patients with Relapsed/Refractory Mantle Cell Lymphoma following Prior BTK Inhibitor Therapy - A Cohort of the ELM-2 StudyAbstract Publication OnlySrikanth AmbatiN/AMatching-Adjusted Indirect Comparisons (MAICs) of Odronextamab Versus Mosunetuzumab and Epcoritamab for the Treatment of Patients with Relapsed/Refractory Follicular Lymphoma (R/R FL) after Two or More Lines of Systemic TherapyAbstract Publication OnlyDeepa JagadeeshN/ALinvoseltamabLinvoseltamab in Patients with Relapsed/Refractory Multiple Myeloma: Longer Follow-Up and Selected High-Risk Subgroup Analyses of the LINKER-MM1 StudyAbstract #3369Poster PresentationMansi R. ShahSunday, December 8, at 6:00-8:00 pmSan Diego Convention Center, Halls G-HSoluble BCMA Dynamics in Patients with Relapsed/Refractory Multiple Myeloma (RRMM) Treated with Linvoseltamab in LINKER-MM1Abstract #3310Poster PresentationAnasuya HazraSunday, December 8, at 6:00-8:00 pm San Diego Convention Center, Halls G-HCharacterization of Linvoseltamab’s BCMA Binding Epitope and Efficacy Against BCMA Mutations in Relapsed/Refractory Multiple MyelomaAbstract #3265Poster PresentationKen Lee & Yi ZhouSunday, December 8, at 6:00-8:00 pm San Diego Convention Center, Halls G-HA CD38xCD28 Costimulatory Bispecific Antibody Demonstrates Potent Preclinical Combinatorial Activity with a BCMAxCD3 T Cell-EngagerAbstract #3283Poster PresentationKara Olson & David J. DiLilloSunday, December 8, at 6:00-8:00 pm San Diego Convention Center, Halls G-HReducing Time Toxicity for Anti-B-Cell Maturation Antigen (BCMA) Bispecific Treatment: Evidence from Pivotal Single-Arm Trial Data on Teclistamab, Elranatamab, and Linvoseltamab for Triple-Class Exposed (TCE) Relapsed/Refractory Multiple Myeloma (RRMM)Abstract #2271Poster Presentation SessionSikander AilawadhSaturday, December 7, at 5:30-7:30 pmSan Diego Convention, Halls G-HExposure-Response Analyses of Various Efficacy and Safety Endpoints in Support of Registrational Dose Selection of Linvoseltamab in Patients with Relapsed/Refractory Multiple MyelomaAbstract Publication OnlyOleg MilbergN/AComparative Effectiveness of Linvoseltamab Versus Current Real-World Standard-of-Care Therapies in Triple-Class Exposed Relapsed/Refractory Multiple Myeloma Treated at IMWG SitesAbstract Publication OnlyShaji KumarN/AComparative Effectiveness of Linvoseltamab Versus Current Real-World (RW) Standard-of-Care (SOC) Therapies in Triple-Class Exposed Relapsed/Refractory Multiple Myeloma (RRMM): Key Subgroups AnalysisAbstract Publication OnlyShaji KumarN/AAdditional PresentationsEfficacy and Safety of Pozelimab Plus Cemdisiran vs Ravulizumab in Patients with Paroxysmal Nocturnal Hemoglobinuria who are Naïve to Complement Inhibition* Abstract #306Oral Presentation SessionChristopher PatriquinSaturday, December 7, at 4 – 5:30 pmSan Diego Convention Center, Room 11TMPRSS6 Inhibition Rapidly Reverses Liver Iron Overload and Prevents an Increase of Splenic Pro-Inflammatory Macrophages in a Mouse Model of Beta-Thalassemia Abstract #2473Poster Presentation SessionHeinrich E. LobSunday, December 8, at 6:00-8:00 pm San Diego Convention Center, Halls G-HThe Reality of Beta Thalassemia and Iron Chelation Therapy – A Qualitative Study Unveiling the Patient BurdenAbstract Publication OnlyChris Hartford *Agreement with Alnylam Pharmaceuticals, Inc. Linvoseltamab as well as the combination of pozelimab and cemdisiran are investigational, and the potential uses of odronextamab in R/R MZL and rare subtypes of R/R aggressive B-cell non-Hodgkin lymphoma are also investigational and have not been approved by any regulatory authority. Odronextamab is approved in the European Union as Ordspono™ to treat R/R FL or DLBCL after two or more lines of systemic therapy, but the safety and efficacy of odronextamab have not been fully evaluated by any other regulatory authority. 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 various combinations 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®, Praluent® (alirocumab), Kevzara® (sarilumab), Evkeeza® (evinacumab-dgnb), Inmazeb® (atoltivimab, maftivimab and odesivimab-ebgn) and Veopoz® (pozelimab-bbfg). 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. Forward-Looking Statements and Use of Digital Media This press release includes forward-looking statements that involve risks and uncertainties relating to future events and the future performance of Regeneron Pharmaceuticals, Inc. (“Regeneron” or the “Company”), and actual events or results may differ materially from these forward-looking statements. Words such as “anticipate,” “expect,” “intend,” “plan,” “believe,” “seek,” “estimate,” variations of such words, and similar expressions are intended to identify such forward-looking statements, although not all forward-looking statements contain these identifying words. These statements concern, and these risks and uncertainties include, among others, the nature, timing, and possible success and therapeutic applications of products marketed or otherwise commercialized by Regeneron and/or its collaborators or licensees (collectively, “Regeneron’s Products”) and product candidates being developed by Regeneron and/or its collaborators or licensees (collectively, “Regeneron’s Product Candidates”) and research and clinical programs now underway or planned, including without limitation pozelimab in combination with cemdisiran, odronextamab, linvoseltamab, and the other programs discussed or referenced in this press release; the likelihood, timing, and scope of possible regulatory approval and commercial launch of Regeneron’s Product Candidates and new indications for Regeneron’s Products, such as odronextamab for the treatment of follicular lymphoma in the United States, linvoseltamab for the treatment of relapsed/refractory multiple myeloma in the United States and/or European Union, and the other programs discussed or referenced in this press release; uncertainty of the utilization, market acceptance, and commercial success of Regeneron’s Products and Regeneron’s Product Candidates (such as those referenced above) and the impact of studies (whether conducted by Regeneron or others and whether mandated or voluntary), including the studies discussed or referenced in this press release, on any of the foregoing or any potential regulatory approval of Regeneron’s Products and Regeneron’s Product Candidates; the ability of Regeneron’s collaborators, licensees, suppliers, or other third parties (as applicable) to perform manufacturing, filling, finishing, packaging, labeling, distribution, and other steps related to Regeneron’s Products and Regeneron’s Product Candidates; the ability of Regeneron to manage supply chains for multiple products and product candidates; safety issues resulting from the administration of Regeneron’s Products and Regeneron’s Product Candidates (such as those referenced above) in patients, including serious complications or side effects in connection with the use of Regeneron’s Products and Regeneron’s Product Candidates in clinical trials; determinations by regulatory and administrative governmental authorities which may delay or restrict Regeneron’s ability to continue to develop or commercialize Regeneron’s Products and Regeneron’s Product Candidates; ongoing regulatory obligations and oversight impacting Regeneron’s Products, research and clinical programs, and business, including those relating to patient privacy; the availability and extent of reimbursement of Regeneron’s Products from third-party payers, including private payer healthcare and insurance programs, health maintenance organizations, pharmacy benefit management companies, and government programs such as Medicare and Medicaid; coverage and reimbursement determinations by such payers and new policies and procedures adopted by such payers; competing drugs and product candidates that may be superior to, or more cost effective than, Regeneron’s Products and Regeneron’s Product Candidates (including biosimilar versions of Regeneron’s Products); the extent to which the results from the research and development programs conducted by Regeneron and/or its collaborators or licensees may be replicated in other studies and/or lead to advancement of product candidates to clinical trials, therapeutic applications, or regulatory approval; unanticipated expenses; the costs of developing, producing, and selling products; the ability of Regeneron to meet any of its financial projections or guidance and changes to the assumptions underlying those projections or guidance; the potential for any license, collaboration, or supply agreement, including Regeneron’s agreements with Sanofi and Bayer (or their respective affiliated companies, as applicable), to be cancelled or terminated; the impact of public health outbreaks, epidemics, or pandemics (such as the COVID-19 pandemic) on Regeneron's business; and risks associated with intellectual property of other parties and pending or future litigation relating thereto (including without limitation the patent litigation and other related proceedings relating to EYLEA® (aflibercept) Injection), other litigation and other proceedings and government investigations relating to the Company and/or its operations (including the pending civil proceedings initiated or joined by the U.S. Department of Justice and the U.S. Attorney's Office for the District of Massachusetts), the ultimate outcome of any such proceedings and investigations, and the impact any of the foregoing may have on Regeneron’s business, prospects, operating results, and financial condition. A more complete description of these and other material risks can be found in Regeneron’s filings with the U.S. Securities and Exchange Commission, including its Form 10-K for the year ended December 31, 2023 and its Form 10-Q for the quarterly period ended September 30, 2024. Any forward-looking statements are made based on management’s current beliefs and judgment, and the reader is cautioned not to rely on any forward-looking statements made by Regeneron. Regeneron does not undertake any obligation to update (publicly or otherwise) any forward-looking statement, including without limitation any financial projection or guidance, whether as a result of new information, future events, or otherwise. Regeneron uses its media and investor relations website and social media outlets to publish important information about the Company, including information that may be deemed material to investors. Financial and other information about Regeneron is routinely posted and is accessible on Regeneron's media and investor relations website (https://investor.regeneron.com) and its LinkedIn page (https://www.linkedin.com/company/regeneron-pharmaceuticals). Contacts:Media RelationsTammy AllenTel: +1 914-306-2698tammy.allen@regeneron.comInvestor RelationsMark HudsonTel: +1 914-847-3482mark.hudson@regeneron.com

Phase 3Clinical ResultDrug ApprovalASHImmunotherapy

100 Deals associated with Evinacumab-dgnb

External Link

KEGG	Wiki	ATC	Drug Bank
D11753	Evinacumab-dgnb	C10	DB15354

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Homozygous familial hypercholesterolemia	US	Regeneron Pharmaceuticals, Inc.	11 Feb 2021

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Hypertriglyceridemia	Phase 2	US	Regeneron Pharmaceuticals, Inc.	07 Jun 2018
Hypertriglyceridemia	Phase 2	CA	Regeneron Pharmaceuticals, Inc.	07 Jun 2018
Hypertriglyceridemia	Phase 2	IT	Regeneron Pharmaceuticals, Inc.	07 Jun 2018
Hypertriglyceridemia	Phase 2	GB	Regeneron Pharmaceuticals, Inc.	07 Jun 2018
Pancreatitis	Phase 2	US	Regeneron Pharmaceuticals, Inc.	07 Jun 2018
Pancreatitis	Phase 2	CA	Regeneron Pharmaceuticals, Inc.	07 Jun 2018
Pancreatitis	Phase 2	IT	Regeneron Pharmaceuticals, Inc.	07 Jun 2018
Pancreatitis	Phase 2	GB	Regeneron Pharmaceuticals, Inc.	07 Jun 2018
Hypercholesterolemia	Phase 2	US	Regeneron Pharmaceuticals, Inc.	10 Nov 2017
Hypercholesterolemia	Phase 2	JP	Regeneron Pharmaceuticals, Inc.	10 Nov 2017

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT04863014 (CTgov)	Phase 2	Hypertriglyceridemia \| Pancreatitis	21	Placebo (Placebo)	zyhqcwhcnm(maxjhdozjl) = fjlxzvzpyl ahjcvelzsx (crhtkseuvm, jwscttqhls - rrsaezxogu) View more	-	22 May 2024
				evinacumab (Evinacumab)	zyhqcwhcnm(maxjhdozjl) = cufmtaxgdq ahjcvelzsx (crhtkseuvm, ffaipvborq - lqbdrrxmmc) View more
NCT04233918 (Pubmed) Manual	Phase 3	Homozygous familial hypercholesterolemia	14	evinacumab 15 mg/kg	fwpimranrk(kaeyrdtyph) = uepfxsssmx uzunugwwjy (jghzrdhzoy ) View more	Positive	30 Jan 2024
NCT04233918 (phase 3, part B, CTgov)	Phase 3	Homozygous familial hypercholesterolemia	20	Evinacumab (Part A: Evinacumab 15mg/Kg IV)	bmljcsyzwp(pgortayois) = jjhhsuwefb avomjkwddc (qqiortnmlp, ohmzshekhz - oabzblnxyh) View more	-	07 Jun 2023
				Evinacumab (Part B: Evinacumab 15mg/Kg IV Q4W)	orcwssitax(mlnyitvcgn) = zkbkbdxgxp owwhmottkh (ztxuuwbhye, hhmdmcdmbo - xzlcaijmyr) View more
NCT04233918 (Corporate Publications) Manual	Phase 3	Hypercholesterolemia	14	EVINACUMAB	vzbuficcyj(iyowfnjqlo) = nnostqeovj kjqzawqpzz (rzvsmnaism ) View more	Positive	21 May 2022
NCT03399786 (CTgov)	Phase 3	Homozygous familial hypercholesterolemia	65	Placebo (Placebo IV Q4W)	fxszgqzgyf(gjeboqfehh) = iegqmabdjs xvgaktsnfc (qiyzdnerac, qphqycdzsb - ddghpvznbm) View more	-	18 May 2021
				Evinacumab (Evinacumab 15 mg/kg IV Q4W)	fxszgqzgyf(gjeboqfehh) = pyhntuvitd xvgaktsnfc (qiyzdnerac, ecbzpbfeku - ysmdtffofk) View more
NCT03399786 (Pubmed \| Br Dent J) Manual	Phase 3	Homozygous familial hypercholesterolemia	65	Evinacumab	rzbzybtgzg(krgnvgbeqn) = rytpubxoix plnutgrnfl (aplaorywhl )	Positive	20 Aug 2020
				Placebo	rzbzybtgzg(krgnvgbeqn) = bcgtsvkbgy plnutgrnfl (aplaorywhl )
NCT02265952 (CTgov)	Phase 2	Homozygous familial hypercholesterolemia	9	REGN1500 (REGN1500 250 mg SC/15 mg/kg IV/450 mg SC)	xikfflelqw(dgnurcnhln) = ynqsiotxrc fsytntimew (hanjuafvtl, obvvgegxli - szpvrwycyx) View more	-	09 Dec 2019
				REGN1500 (REGN1500 300 mg SC/20 mg/kg IV)	qocznsjhso(fgrqcwxnok) = ppevndeltc vphxtjzxgf (lnkfyjcmay, qxlsipddmk - jwkezpvebg) View more
NCT02265952 (Pubmed) Manual	Phase 2	Homozygous familial hypercholesterolemia	9	evinacumab	pitfuuluix(yfflztmnlp) = lchsklvtgq xxoguvzrvf (drwvcxhryu, 18)	Positive	01 Nov 2019
NCT02265952 (ESC2017)	Phase 2	Homozygous familial hypercholesterolemia LDL-receptor mutations \| ANGPTL3	9	Evinacumab	qewhnnkhtl(smghhminbj) = fegkinotnl mkhcavoith (wdibnaoutm ) View more	-	29 Aug 2017

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