Journal: Nature reviews. Drug discovery

Article Title: Therapeutic targeting of the complement system

doi: 10.1038/s41573-019-0055-y

Figure Lengend Snippet: Selected inhibitors targeting complement a

Article Snippet: ​ Drug (Company) Target Indication Status Targeting pathway initiation C1-Inhibitor: Cinryze (Shire), Berinert (CSL), Cetor (Sanquin), Ruconest (Pharming) b C1r, C1s, MASPs, other proteases Hereditary angioedoema Kidney IRI Sepsis Kidney transplantation Approved Phase II Phase III Phase II Sutimlimab (Sanofi) C1s Cold agglutinin disease Immune thrombocytopenic purpura Phase III Phase I Narsoplimab (Omeros) MASP-2 aHUS, IgA nephropathy, HSCT-TMA Lupus nephritis Phase III Phase II Targeting the alternative pathway and amplification loop APL-2/pegcetacoplan (Apellis) C3 AMD (GA), PNH, AIHA Nephropathies Phase III Phase II AMY-101 (Amyndas) C3 Periodontal disease Stroke, transplantation Phase II Phase I Mirococept Convertase Kidney transplantation Phase II LNP023 (Novartis) FB C3G PNH IgA nephropathy, glomerulonephritis Phase II Phase II Phase II IONIS-FB-LRx (lonis/Roche) FB AMD (GA) Phase II Danicopan (Achillion) FD PNH C3G Phase II Phase II CLG561 (Alcon) FP AMD (GA) Phase II Targeting the terminal pathway effectors Soliris, Eculizumab (Alexion) c C5 PNH, aHUS Generalized MG Neuromyelitis optica Approved Approved Approved Ultomiris, Ravulizumab (Alexion) C5 PNH aHUS Generalized MG Approved Phase III Phase III ABP 959 (Amgen) C5 PNH Phase III Nomacopan (Akari) C5 PNH BP, AKC Phase III Phase II Pozelimab (Regeneron) C5 PNH Phase I Crovalimab (Roche) C5 PNH Phase I Tesidolumab (Novartis) C5 PNH Phase II Zilucoplan (Ra) C5 Generalized MG INMN, renal disorders Phase II Phase I Cemdisiran (Alnylam) C5 IgA nephropathy Phase II Zimura (lveric) C5 AMD (GA), Stargardt Phase II IFX-1 (lnflaRx) C5a HS, AAV Phase II Avacopan (ChemoCentryx) C5aR1 AAV C3G, HS Phase III Phase II HMR59 (Hemera) MAC AMD (GA, CNV) Phase I Open in a separate window For a comprehensive overview of drug candidates and clinical trials, the authors refer to Refs. 2 and 3 . a For each company, the approved drugs or the candidate in furthest clinical development is shown. b Distinct C1-INH preparations are involved in the listed clinical trials. c Alongside the improved indications, eculizumab is also clinically evaluated in a series of other indications.

Techniques: Transplantation Assay, Amplification

Journal: Journal of Managed Care & Specialty Pharmacy

Article Title: Iptacopan and danicopan for paroxysmal nocturnal hemoglobinuria

doi: 10.18553/jmcp.2024.30.6.618

Figure Lengend Snippet: Base-Case Results and Incremental Cost-Effectiveness Ratios for Danicopan Add-On Therapy Compared With Ravulizumab Alone, Health Care Sector Perspective

Article Snippet: The Institute for Clinical and Economic Review (ICER) conducted a systematic literature review and cost-effectiveness analysis to evaluate the clinical and economic outcomes of the newer agents, iptacopan and danicopan, for patients with PNH.

Techniques:

Journal: Journal of Managed Care & Specialty Pharmacy

Article Title: Iptacopan and danicopan for paroxysmal nocturnal hemoglobinuria

doi: 10.18553/jmcp.2024.30.6.618

Figure Lengend Snippet: California Technology Assessment Forum Votes on Potential Other Benefits or Disadvantages Questions

Article Snippet: The Institute for Clinical and Economic Review (ICER) conducted a systematic literature review and cost-effectiveness analysis to evaluate the clinical and economic outcomes of the newer agents, iptacopan and danicopan, for patients with PNH.

Techniques:

Journal: Frontiers in Immunology

Article Title: Complement Mediated Hemolytic Anemias in the COVID-19 Era: Case Series and Review of the Literature

doi: 10.3389/fimmu.2021.791429

Figure Lengend Snippet: Hemolytic flares in patients with paroxysmal nocturnal hemoglobinuria after SARS-CoV-2 vaccine.

Article Snippet: 1)Classic PNH 2)AA PNH 3)AA 4)Classic PNH 5)Classic PNH 6)Classic PNH , 1)treatment naïve 2)Ravulizumab 3)Ravulizumab and Danicopan (oral complement factor D inhibitor) 4)Ravulizumab 5)Ravulizumab 6)Ravulizumab and Danicopan , 6 , 1)Pfizer 2)Pfizer 3)Moderna 4)Moderna 5)Pfizer 6)Pfizer , 1)5 days after second dose 2)same day of first dose 3)same day of second dose 4)same day of second dose 5)No BTH 6)No BTH , 1)Ravulizumab, LMWH 2)Ravulizumab 3)Ravulizumab, RBC transfusion 4)Ravulizumab 5)Ravulizumab 6)Ravulizumab , 1) microvascular bowel thrombosis 2)Resolved 3)Resolved 4)Resolved 5) resolved 6) resolved , Gerber et al., Blood 2021 ( ) .

Techniques: