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85 Diseases |  |
26 Trials |
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26 Trials |  |
4609 News |  |
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- | Spikevax (elasomeran) / Moderna
Journal: End stage renal disease in patient with microscopic polyangiitis and atypical hemolytic-uremic syndrome arose 3 weeks after the third dose of anti-SARS-CoV2 vaccine mRNA-1273: A case report with literature revision. (Pubmed Central) - Dec 24, 2023
This systematic review offers additional perspectives. It is plausible to hypothesize that the vaccine was the trigger for the development of these 2 diseases.Solid evidence on the mechanisms of interaction between vaccine and immune system, the role of genetic predisposition, and other variables, will shed additional light on the controversial link between anti-SARS-CoV2 vaccine and autoimmunity.
- | Soliris (eculizumab) / AstraZeneca
Journal: Severe proteinuria (but not being on dialysis) may be associated with initial inadequate complement inhibition and delayed hematological response to eculizumab therapy. (Pubmed Central) - Dec 22, 2023
It is plausible to hypothesize that the vaccine was the trigger for the development of these 2 diseases.Solid evidence on the mechanisms of interaction between vaccine and immune system, the role of genetic predisposition, and other variables, will shed additional light on the controversial link between anti-SARS-CoV2 vaccine and autoimmunity. No abstract available
- | Soliris (eculizumab) / AstraZeneca
Journal: Plain language summary on long-term effectiveness of eculizumab: Data from the International PNH Registry. (Pubmed Central) - Dec 19, 2023
No abstract available No abstract available
- | Soliris (eculizumab) / AstraZeneca
Journal, Checkpoint inhibition: Eculizumab for myasthenic exacerbation during treatment with immune-checkpoint inhibitors. (Pubmed Central) - Dec 18, 2023
Eculizumab potentially offers a mechanism-based treatment of MG in patients under anti-programmed cell death protein 1 (PD-1) agents, without interfering with their mechanism of action and avoiding their discontinuation. Larger case series deserve to be evaluated.
- | Soliris (eculizumab) / AstraZeneca, Rituxan (rituximab) / Biogen, Zenyaku Holdings, Roche
Retrospective data, Journal: Clinical characteristics, treatments, and outcomes of interferon-beta-induced thrombotic microangiopathy: a literature-based retrospective analysis. (Pubmed Central) - Dec 18, 2023
therapy, and patients taking IFN-? should be monitored for symptoms such as headache and hypertension.
- || Empaveli (pegcetacoplan SC) / Apellis, SOBI
Retrospective data, Review, Journal: Efficacy of complement inhibitors for patients with paroxysmal nocturnal hemoglobinuria: a systematic review and meta-analysis. (Pubmed Central) - Dec 18, 2023
Clinically meaningful improvements in FACIT-F were observed both before and after 26?weeks, with a pooled estimate of 6.8 (95% CI: 6.0-7.6) and 9.5 (95% CI: 7.0-12.0), respectively. Our findings suggest that complement inhibitors can result in positive treatment outcomes and sustained benefits for patients with PNH.
- || Soliris (eculizumab) / AstraZeneca, Rituxan (rituximab) / Biogen, Zenyaku Holdings, Roche
Review, Journal: Studying the Role of C5-Inhibition Therapy in Scleroderma Renal Crisis-Induced Thrombotic Microangiopathy - A Review of Literature. (Pubmed Central) - Dec 17, 2023
These findings should prompt testing on a larger cohort of SRC-TMA patients. This would help us determine whether aggressive treatment combining ACE-I and Eculizumab can target the various underlying endothelial, inflammatory, and immunologic mechanisms involved in SRC-TMA, and improve patient outcomes.
- Soliris (eculizumab) / AstraZeneca
Is Crossing DSA Detected in the Dilution Assay Safe for Patients Undergoing Heart Transplant? (Ninth Avenue & Dolores St.) - Dec 13, 2023 - Abstract #WRMC2024WRMC_217;
Crossing DSA at the time of heart transplant appears safe with customized immunosuppression and has acceptable clinical outcomes. DSA detected in the dilution assay at the time of heart transplant may lead to more AMR which does not impact clinical outcome.
- Soliris (eculizumab) / AstraZeneca
Does Crossing DSA and Its Binding Levels at the Time of Transplant Lead to Post-Op Complications? (Promenade Lobby (Sunset Center)) - Dec 13, 2023 - Abstract #WRMC2024WRMC_29;
Crossing DSA at the time of heart transplant can be done safely (with customized immunosuppression) but is it associated with an increased risk for first-year AMR. However, this did not impact 3-year survival or other outcomes.
- Soliris (eculizumab) / AstraZeneca
Phase classification: Soliris: Eculizumab in Pediatric and Adult Participants With Atypical Hemolytic Uremic Syndrome (aHUS) in China (clinicaltrials.gov) - Dec 12, 2023
P3, N=25, Recruiting, Sponsor: Alexion Pharmaceuticals, Inc.
However, this did not impact 3-year survival or other outcomes. Phase classification: P=N/A --> P3
- Gamifant (emapalumab-lzsg) / SOBI
Emapalumab to Prevent Graft Failure in High-Risk Patients: The Experience of Stanford Children's Health () - Dec 11, 2023 - Abstract #TCTASTCTCIBMTR2024TCT_ASTCT_CIBMTR_1313;
The successful outcomes observed in 2 patients who received emapalumab prior to their 1st HSCT suggests that administering emapalumab earlier - potentially before the GF process initiates, may yield the greatest benefits. In this high-risk population, a synergistic approach combining emapalumab and eculizumab may hold promise in mitigating the risk of GF.
- Soliris (eculizumab) / AstraZeneca
Assay Panel for Diagnosis and Monitoring Treatment of TA-TMA in HSCT Recipients () - Dec 11, 2023 - Abstract #TCTASTCTCIBMTR2024TCT_ASTCT_CIBMTR_831;
Assays measuring sC5b-9 and CH50 with same day turnaround time have been validated or verified so that timely results can be provided to physicians with patients at risk of organ damage due to TA-TMA. An assay to measure Eculizumab levels within 24 hours is currently under development.
- narsoplimab (OMS721) / Omeros
Real-World Experience with Narsoplimab (OMS721) for the Treatment of Transplant-Associated Thrombotic Microangiopathy () - Dec 11, 2023 - Abstract #TCTASTCTCIBMTR2024TCT_ASTCT_CIBMTR_828;
Six patients in this cohort had major ABO incompatibility making transfusion independence a poor predictor of response. Haptoglobin was paradoxically increased, likely as an acute phase reactant, in patients with acute GVHD.
- Soliris (eculizumab) / AstraZeneca
Eculizumab Treatment Outcomes in Patients with Allogeneic Hematopoietic Cell Transplant (alloHCT)-Associated Thrombotic Microangiopathy (TA-TMA): A Retrospective Single-Center Experience () - Dec 11, 2023 - Abstract #TCTASTCTCIBMTR2024TCT_ASTCT_CIBMTR_814;
TA-TMA with preceding GvHD appeared to be more resistant to complement inhibition. Future efforts may include early disease detection/intervention, finding a better complement inhibition, and a better understanding of other modifiable mechanisms of action for TA-TMA.
- Soliris (eculizumab) / AstraZeneca
Thrombotic Microangiopathy Following CAR-T Cell Therapy Successfully Treated with Eculizumab in a Pediatric Patient () - Dec 11, 2023 - Abstract #TCTASTCTCIBMTR2024TCT_ASTCT_CIBMTR_802;
More data is needed to understand the exact pathophysiology of TMA post-CAR in association with CRS, ICANS, and IEC-HS. More data is also needed to describe the potential risk factors associated with developing TMA post CAR-T, such as prior SCT, type of CAR and presence of other post CAR-T toxicities.
- Soliris (eculizumab) / AstraZeneca
Elevated C3 Is Predictive, Diagnostic and Prognostic in Pediatric Transplant Associated Thrombotic Microangiopathy- a Prospective Study (221 (Meeting Level, Henry B. Gonzalez Convention Center); in-person) - Dec 11, 2023 - Abstract #TCTASTCTCIBMTR2024TCT_ASTCT_CIBMTR_745;
Of those with TA-TMA, 14 (52%) developed MOD; 3 prior to TA-TMA diagnosis and 11 after TA-TMA diagnosis (median 13 days). Of the 18 patients treated with eculizumab, 14 (78%) had no response by organ function or microangiopathy markers and/or died with active TA-TMA.
- Soliris (eculizumab) / AstraZeneca
TA-TMA Is Frequent, but Not All TA-TMA Is Actionable in Adult Allogeneic HCT Patients: A Report from the MIDAS (Microangiopathy, Endothelial Damage in Adults undergoing Stem cell transplantation) Consortium. (Stars at Night B4 (Ballroom Level, Henry B. Gonzalez Convention Center); in-person) - Dec 5, 2023 - Abstract #TCTASTCTCIBMTR2024TCT_ASTCT_CIBMTR_660;
Of 17 patients adjudicated as severe TA-TMA by MIDAS, 10 were reported as having TA-TMA by the center, with 8 receiving TA-TMA-directed therapy (6 eculizumab, 2 other) and only 2 of 8 survived (both received eculizumab)...Actionable cases of severe TA-TMA can be identified by end-organ damage and high transfusion requirements. Weekly biospecimens have been collected up to day+100 and will be analyzed for biomarkers associated with development and prognosis of TA-TMA.
- | Soliris (eculizumab) / AstraZeneca
Journal: CD55 Deficiency With Budd-Chiari Syndrome Treated by Liver Transplantation and Eculizumab. (Pubmed Central) - Dec 4, 2023
We recommend testing for CD55 deficiency in patients with protein-losing enteropathy. In addition, CD55 deficiency should be considered in the differential diagnosis of patients with Budd-Chiari syndrome in whom an underlying cause is uncertain.
- ||| danicopan (ALXN2040) / AstraZeneca, Soliris (eculizumab) / AstraZeneca, Ultomiris (ravulizumab-cwvz) / AstraZeneca
P3 data, Journal: Addition of danicopan to ravulizumab or eculizumab in patients with paroxysmal nocturnal haemoglobinuria and clinically significant extravascular haemolysis (ALPHA): a double-blind, randomised, phase 3 trial. (Pubmed Central) - Nov 30, 2023
P3
In addition, CD55 deficiency should be considered in the differential diagnosis of patients with Budd-Chiari syndrome in whom an underlying cause is uncertain. These primary efficacy and safety results show that danicopan as add-on treatment to ravulizumab or eculizumab significantly improved haemoglobin concentrations at week 12 with no new safety concerns, suggesting an improved benefit-risk profile in patients with PNH and clinically significant extravascular haemolysis.
- | Soliris (eculizumab) / AstraZeneca
Journal: Early Mortality in Paroxysmal Nocturnal Hemoglobinuria. (Pubmed Central) - Nov 30, 2023
These primary efficacy and safety results show that danicopan as add-on treatment to ravulizumab or eculizumab significantly improved haemoglobin concentrations at week 12 with no new safety concerns, suggesting an improved benefit-risk profile in patients with PNH and clinically significant extravascular haemolysis. We conclude that early mortality in PNH is associated with older age, cardiovascular comorbidity, and hematological malignancies.
- | Soliris (eculizumab) / AstraZeneca
Journal: Warm autoimmune hemolytic anemia associated with SARS-CoV-2 infection responsive to eculizumab. (Pubmed Central) - Nov 28, 2023
We conclude that early mortality in PNH is associated with older age, cardiovascular comorbidity, and hematological malignancies. No abstract available
- | Soliris (eculizumab) / AstraZeneca
Journal: Eculizumab in the treatment of neuromyelitis optica spectrum disorder. (Pubmed Central) - Nov 28, 2023
No abstract available No abstract available
- || Soliris (eculizumab) / AstraZeneca
Clinical, P2a data, PK/PD data, Journal: Safety and pharmacodynamic efficacy of eculizumab in aneurysmal subarachnoid hemorrhage (CLASH): A phase 2a randomized clinical trial. (Pubmed Central) - Nov 27, 2023
P2
The study did not reveal new safety concerns, except for a C. albicans drain-related infection prior to antifungal monitoring and treatment. EudraCT 2017-004307-51, https://www.clinicaltrialsregister.eu/.
- | pioglitazone / Generic mfg.
Journal: Pioglitazone, a PPAR-y agonist, as one of the new therapeutic candidates for C3 glomerulopathy. (Pubmed Central) - Nov 27, 2023
EudraCT 2017-004307-51, https://www.clinicaltrialsregister.eu/. In this patient affected by C3G, pioglitazone proved effective in reducing proteinuria levels.
- | Soliris (eculizumab) / AstraZeneca
Journal: Eculizumab in STEC-HUS: a paradigm shift in the management of pediatric patients with neurological involvement. (Pubmed Central) - Nov 27, 2023
We present a case series of four children with STEC-HUS and CNS involvement, managed with eculizumab only, in lieu of plasma exchange (as per our previous policy). The marked improvement in symptoms in our cohort supports the use of eculizumab, rather than plasma exchange in the CNS involvement of STEC-HUS.
- | Soliris (eculizumab) / AstraZeneca
Observational data, Journal: Atypical haemolytic uraemic syndrome in the era of terminal complement inhibition- An observational cohort study. (Pubmed Central) - Nov 27, 2023
We demonstrate that biallelic pathogenic mutations in RNA processing genes, including EXOSC3, encoding an essential part of the RNA exosome, cause eculizumab non-responsive aHUS. Recessive HSD11B2 mutations causing apparent mineralocorticoid excess may also present with a thrombotic microangiopathy.
- || Soliris (eculizumab) / AstraZeneca
Trial completion date, Trial primary completion date: Comparative PK, Safety, Tolerability, Immunogenicity, and PD Profile Study of TUR03 and Soliris in Healthy Participants (clinicaltrials.gov) - Nov 26, 2023
P1, N=120, Recruiting, Sponsor: Turgut Ardika PTY LTD
Recessive HSD11B2 mutations causing apparent mineralocorticoid excess may also present with a thrombotic microangiopathy. Trial completion date: Apr 2024 --> Oct 2024 | Trial primary completion date: Sep 2023 --> Jul 2024
- | Ultomiris (ravulizumab-cwvz) / AstraZeneca
Retrospective data, Journal: Dosing Patterns of Patients with Paroxysmal Nocturnal Hemoglobinuria Treated with Ravulizumab in the United States: A Retrospective Claims-Based Analysis. (Pubmed Central) - Nov 24, 2023
Trial completion date: Apr 2024 --> Oct 2024 | Trial primary completion date: Sep 2023 --> Jul 2024 Although limited by missing clinical characteristics including body weight, this study of ravulizumab dosing patterns in patients with PNH identified potential deviations from label-recommended dosing, warranting further investigations of treatment response to complement inhibitors in PNH.
- | Soliris (eculizumab) / AstraZeneca
Journal: Low C3 in a 4-month-old baby: is it a problem? (Pubmed Central) - Nov 24, 2023
The same mutation was found homozygous in the uncle. In genetically inherited diseases, findings may occur sequentially; early screening of at-risk individuals contributes to kidney survival.
- || Soliris (eculizumab) / AstraZeneca, Rituxan (rituximab) / Biogen, Zenyaku Holdings, Roche
Review, Journal: Thrombotic Microangiopathy After Hematopoietic Stem Cell and Solid Organ Transplantation: A Review for Intensive Care Physicians. (Pubmed Central) - Nov 22, 2023
However, the relatively larger studies using eculizumab have been mostly conducted in the pediatric population with limited data on the adult population. This review is focused on the role of intensive care physicians to emphasize the clinical approach to patients with suspected TA-TMA and to discuss diagnosis and treatment strategies.
- | Soliris (eculizumab) / AstraZeneca, Ultomiris (ravulizumab-cwvz) / AstraZeneca
Retrospective data, Journal: Complement-mediated thrombotic microangiopathy treated with anticomplement protein 5 therapy, a retrospective study. (Pubmed Central) - Nov 21, 2023
Our findings validate that CM-TMA is an aggressive disease with significant morbidity and mortality, and confirm that aC5-mab is a relatively effective therapy for CM-TMA. Our study adds practical, real-world experience to the literature, but future research remains imperative.
- | Soliris (eculizumab) / AstraZeneca
Journal: Modeling complement activation on human glomerular microvascular endothelial cells. (Pubmed Central) - Nov 17, 2023
The increased C5b-9 deposition on control ciGMVECs by zymosan-activated serum could be dose-dependently inhibited by adding the C5 inhibitor eculizumab...In conclusion, we demonstrate a robust and reliable model to adequately measure C5b-9-based complement deposition on human control and patient ciGMVECs. This model can be used to study the pathophysiological mechanisms of aHUS or other diseases associated with endothelial complement activation ex vivo.
- | Soliris (eculizumab) / AstraZeneca
Journal: Newly emerging type B insulin resistance (TBIR) during treatment with eculizumab for AQP4-IgG-positive neuromyelitis optica spectrum disorder (NMOSD): fatal outcome. (Pubmed Central) - Nov 14, 2023
More real-world data are needed on the risk/benefit ratio of ECU treatment in patients who have co-existing autoimmune comorbidities that may compromise immune function. Strategies to mitigate the risk of serious infection in patients treated with ECU are discussed.
- || Soliris (eculizumab) / AstraZeneca
Clinical, Journal: Prospective Multi-Institutional Study of Eculizumab to Treat High-Risk Stem Cell Transplant Associated TMA. (Pubmed Central) - Nov 10, 2023
P2
This study serves as a benchmark for planning future studies that should focus on preventative measures or targeted therapy that can be initiated prior to organ injury. ClinicalTrials.gov (NCT03518203).
- | Soliris (eculizumab) / AstraZeneca, Gohibic (vilobelimab) / Staidson Biopharma, InflaRx
Journal: Rational design of antibody-like peptides for targeting the human complement fragment protein C5a. (Pubmed Central) - Nov 7, 2023
The computational data further supports the potential of designer peptides for mimicking the function of antibodies targeting C5a. However, further experimental studies will be required to establish the structure-function relationship of the designer peptides and also to establish the hypothesis of antibody-like peptides targeting C5a.
- | Tavneos (avacopan) / Kissei, Amgen, Otsuka, Soliris (eculizumab) / AstraZeneca
Journal: Editorial: Role of complement activation in kidney diseases. (Pubmed Central) - Nov 6, 2023
However, further experimental studies will be required to establish the structure-function relationship of the designer peptides and also to establish the hypothesis of antibody-like peptides targeting C5a. No abstract available
- Soliris (eculizumab) / AstraZeneca
Systemic AAV9-mediated Gene Addition for Canavan Disease (CANaspire Trial): Lowering N-acetylaspartate Levels, Impacting Disease Severity, and Managing Immune Responses (WCC Halls A-C) - Nov 3, 2023 - Abstract #Neuroscience2023NEUROSCIENCE_12980;
P=N/A, P1/
These responses have been managed with high-dose steroids in all 6 patients as well as complement inhibition (eculizumab) in the last 2 participants dosed...Most adverse events (AEs) have been well-managed with steroids and complement inhibition, with no treatment-related serious AEs to date. While the literature and the ongoing natural history study suggest an association between NAA levels and phenotype, longer follow-up is needed to assess whether the intervention translates to clinical efficacy.
- Empaveli (pegcetacoplan SC) / Apellis, SOBI
Health-Related Quality of Life and Symptom-Specific Functional Impairment Among Patients Treated with Parenterally Administered Complement Inhibitors for Paroxysmal Nocturnal Hemoglobinuria (Halls G-H (San Diego Convention Center)) - Nov 3, 2023 - Abstract #ASH2023ASH_6597;
Despite receiving treatment for PNH, patients experienced deficits in areas of HRQoL related to fatigue, participation in social roles, cognitive function, and functional limitations due to dyspnea, relative to a sample of GP adults. During follow-up interviews, patients emphasized the deep and broad impacts that fatigue and cognitive functioning had, making daily activities and their social lives very difficult.
- Soliris (eculizumab) / AstraZeneca, Ultomiris (ravulizumab-cwvz) / AstraZeneca
Incremental Burden Associated with Dose Escalation in Patients with Paroxysmal Nocturnal Hemoglobinuria Treated with Complement Inhibitors (Halls G-H (San Diego Convention Center)) - Nov 3, 2023 - Abstract #ASH2023ASH_6590;
In this study of PNH patients treated with C5 inhibitors, dose escalation was associated with significant incremental costs for both ECU and RAVU. Further research is warranted to clarify the reasons for dose escalation, which may potentially indicate an unmet clinical need for more efficacious treatment options to help prevent breakthrough symptoms and reduce the burden of PNH.
- Fabhalta (iptacopan) / Novartis
Setting Cost-Effective Price Thresholds before FDA Approval: Cost-Effectiveness of Iptacopan Monotherapy Versus Standard-of-Care Anti-C5 Therapy in Transfusion-Dependent, Treatment-Experienced Adult Patients with Paroxysmal Nocturnal Hemoglobinuria in the United States (Halls G-H (San Diego Convention Center)) - Nov 3, 2023 - Abstract #ASH2023ASH_6510;
If the monthly price is no more than 149% of SOC, iptacopan monotherapy can be a cost-effective therapeutic option for transfusion-dependent, treatment-experienced patients with PNH. This is achieved by increasing the proportion of transfusion-independence in this patient population and, in turn, decreasing the burden of both the risks and costs associated with health resource utilization for patients with PNH.
- Clinical Characteristics and Treatment Outcomes of Idiopathic Multicentric Castleman Disease: Data from a Pediatric Cohort (Halls G-H (San Diego Convention Center)) - Nov 3, 2023 - Abstract #ASH2023ASH_5855;
This is achieved by increasing the proportion of transfusion-independence in this patient population and, in turn, decreasing the burden of both the risks and costs associated with health resource utilization for patients with PNH. Nine patients received three unique siltuximab regimens: siltuximab
- Soliris (eculizumab) / AstraZeneca
Paroxysmal Nocturnal Haemoglobinuria (PNH) Arising from Non-Canonical Mutations (SDCC - Halls G-H) - Nov 3, 2023 - Abstract #ASH2023ASH_5522;
Symptoms improved on eculizumab but he remained transfusion dependent and the disease later transformed to acute myeloid leukaemia...Similar to the previously reported PIGB mutated case, the patients with PIGT mutations only had partially deficient cells. It is important to fully characterise these emerging non-canonical variants in order to improve our understanding of the pathophysiology of this disorder and to provide more individualised care.
- Soliris (eculizumab) / AstraZeneca, Bekemv (eculizumab biosimilar) / Amgen
Efficacy of Parallel and Crossover Analysis As Well As Pharmacokinetic Similarity Were Confirmed between ABP 959 and Eculizumab Reference Product in Patients with PNH (SDCC - Halls G-H) - Nov 3, 2023 - Abstract #ASH2023ASH_5517;
Additionally, analyses of serum total and unbound PK concentrations for patients with PNH further demonstrated PK similarity between ABP 959 and eculizumab RP. The results of this study, along with previously demonstrated analytical, non-clinical, clinical PK/PD in healthy adults, and efficacy and safety evaluations in PNH patients, further support a demonstration of no clinically meaningful differences between ABP 959 and eculizumab RP.
- Soliris (eculizumab) / AstraZeneca, crovalimab (RG6107) / Roche
Patient-Reported Outcomes (PROs) in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Treated with Crovalimab and Eculizumab: Results from the Phase III Randomized COMMODORE 2 and COMM... (SDCC - Halls G-H) - Nov 3, 2023 - Abstract #ASH2023ASH_5515;
P3
These trends were maintained after pts were switched from ecu to crova. These data provide supportive evidence for the treatment benefit of crovalimab for self-reported fatigue and quality of life.
- Soliris (eculizumab) / AstraZeneca, crovalimab (RG6107) / Roche
Biomarker Analyses in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Treated with Crovalimab and Eculizumab: Results from the Phase III Randomized COMMODORE 2 Trial (SDCC - Halls G-H) - Nov 3, 2023 - Abstract #ASH2023ASH_5513;
P3
Overall, biomarker data from COMMODORE 2 support the non-inferior efficacy of crova vs ecu in patients with PNH. Further analyses correlating additional biomarkers with clinical outcomes is warranted.
- Fabhalta (iptacopan) / Novartis
Categorization of Hematological Responses to Oral Iptacopan Monotherapy in Anti-C5-Treated Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) and Persistent Anemia in the Phase III APPLY-PNH Trial and Complement Inhibitor-Na (Halls G-H (San Diego Convention Center)) - Nov 3, 2023 - Abstract #ASH2023ASH_5509;
P3
APPLY-PNH enrolled adult PNH patients with a mean Hb level <10 g/dL who had been receiving eculizumab or ravulizumab for ?6 months. This post hoc analysis of the APPLY-PNH and APPOINT-PNH trial data demonstrates that the majority of patients achieved complete or major hematological responses during treatment with iptacopan monotherapy, highlighting the ability of both complement inhibitor-na
- Enjaymo (sutimlimab) / Sanofi, Soliris (eculizumab) / AstraZeneca
The Bioluminescent Modified Ham Test Identifies the Classical Pathway As the Major Driver of Complement Activation in Atypical Hemolytic Uremic Syndrome and Primary Antiphospholipid Syndrome (SDCC - Halls G-H) - Nov 3, 2023 - Abstract #ASH2023ASH_5362;
It is easy to perform, more closely approximates the complement regulators on renal endothelium, can be utilized for therapeutic monitoring, and has already offered novel disease insights through the use of specific pathway inhibitors. Although aHUS is traditionally categorized as a disorder of the AP, these results suggest the
- SB12 (eculizumab biosimilar) / AffaMed Therap, Samsung, Soliris (eculizumab) / AstraZeneca
Efficacy of SB12 (Eculizumab Biosimilar) in Asian and Non-Asian Patients with Paroxysmal Nocturnal Hemoglobinuria: Subgroup Analysis of a Global Phase III Randomized Controlled Trial (SDCC - Halls G-H) - Nov 3, 2023 - Abstract #ASH2023ASH_3670;
Although aHUS is traditionally categorized as a disorder of the AP, these results suggest the Conclusion SB12 showed comparable efficacy to ECU in complement-inhibitor na
- Ultomiris (ravulizumab-cwvz) / AstraZeneca
Ravulizumab Effectiveness in the Real World: Evidence from the International PNH Registry (Halls G-H (San Diego Convention Center)) - Nov 3, 2023 - Abstract #ASH2023ASH_3665;
P=N/A
Transfusion rates were lowest with Rav treatment. Pts treated with Rav experienced no MAVEs, TE, meningococcal infections, or deaths.
- cemdisiran (ALN-CC5) / Alnylam, Regeneron, Soliris (eculizumab) / AstraZeneca, Veopoz (pozelimab-bbfg) / Regeneron, Sanofi
52-Week Open-Label Extension Data from a Phase 2 Study Evaluating the Safety and Efficacy of Pozelimab and Cemdisiran Combination Therapy in Patients with Paroxysmal Nocturnal Hemoglobinuria Who Switched from Eculizumab (Halls G-H (San Diego Convention Center)) - Nov 3, 2023 - Abstract #ASH2023ASH_3659;
P2
Results suggest that, in patients with PNH transitioning from eculizumab treatment, the combination of pozelimab and cemdisiran was generally well tolerated and provided long term sustained control of intravascular hemolysis without any breakthrough hemolysis events. Findings support the ongoing development of pozelimab and cemdisiran combination therapy.