Mononine (human plasma derived coagulation factor IX) / CSL Behring - LARVOL DELTA

DETECTION OF FACTOR IX MOLECULES IN LIVER AND PAWS OF HEMOPHILIA-B MICE (EAHAD 2023) - "Introduction: FIX bound to type IV collagen in the endothelial basement membrane may provide haemostatic protection.However, its potential role in the protection against joint bleeds has not been demonstrated yet.Type I collagen is the most common form of collagen into muscle and joints where extravascular FIX might play a key role. Immunohistochemistry,protein engineering Our in-vitro experiments showed that plasma-derived FIX (pdFIX, Mononine®) had 4-fold better binding capacity to type I and IV collagen compared to recombinant FIX (rFIX).Post-translational modifications,mainly present into the activation peptide (AP),may influence FIX binding capacity to collagens and therefore its ability to reach extravascular compartment.To better understand the role of the AP in collagen binding, we have generated a rFIX molecule containing two APs.A bioengineered functional recombinant FIX produced by a human hepatoma cell-line,had full procoagulant activity and bound more..."

Preclinical • Gastrointestinal Cancer • Hematological Disorders • Hemophilia • Hepatocellular Cancer • Liver Cancer • Rare Diseases • Solid Tumor

Product discontinued (Hemophilia News Today) - Hemophilia

Discontinued • Hemophilia

Mononine Soon Will No Longer Be Available to Treat Hemophilia B (Hemophilia News Today) - "Mononine, a long-standing treatment to prevent and control bleeding in hemophilia B, will be discontinued due to declining demand in recent years. CSL Behring, the therapy’s manufacturer, said the decision was based on the emergence of next-generation hemophilia B treatments now in greater use....'Accordingly, we are discontinuing MONONINE'....The medication is expected be available through mid-2021, based on current supply, giving patients time to move to another therapy."

Discontinued • Genetic Disorders • Hemophilia

Edited Transcript of CSL.AX earnings conference call or presentation 19-Aug-20 1:00am GMT (Yahoo Finance) - "The successful evolution of our hemophilia portfolio continues on track with IDELVION up 25% and AFSTYLA sales up 21%....Our hemophilia portfolio has continued to evolve with strong growth, and the recombinant hemophilia products which saw total sales up 8%. IDELVION performed exceptionally well. Sales were up 25% with strong growth profiles in the U.S., Switzerland and Japan....And whilst the plasma-derived coag portfolio declined 3% overall, we did see some modest growth in our von Willebrand sales with Humate/Haemate....Our Factor IX product, MONONINE is also seeing some competition but from our own product, IDELVION....We're waiting on reimbursement approval in about 12 additional entries now for launch of IDELVION."

Launch • Reimbursement • Sales • Hemophilia

Measuring factor IX activity of nonacog beta pegol with commercially-available one-stage clotting and chromogenic assay kits: a two-center study. (PubMed, J Thromb Haemost) - "Background Measurement of factor IX activity (FIX:C) with activated partial thromboplastin time-based one-stage clotting assays is associated with a large degree of interlaboratory variation in samples containing glycoPEGylated recombinant FIX (rFIX), i.e. nonacog beta pegol (N9-GP). Validation and qualification of specific assays and conditions are necessary for the accurate assessment of FIX:C in samples containing N9-GP. Objectives To assess the accuracy of various one-stage clotting and chromogenic assays for measuring FIX:C in samples containing N9-GP as compared with samples containing rFIX or plasma-derived FIX (pdFIX) across two laboratory sites. Methods FIX:C, in severe hemophilia B plasma spiked with a range of concentrations (from very low, i.e. 0.03 IU mL(-1) , to high, i.e. 0.90 IU mL(-1) ) of N9-GP, rFIX (BeneFIX), and pdFIX (Mononine), was determined at two laboratory sites with 10 commercially available one-stage clotting assays and two chromogenic FIX:C assays...

Journal • Biosimilar • Gene Therapies • Hematological Malignancies • Hemophilia • Venous Thromboembolism

Protein aggregates in plasma-derived factor IX (pdFIX) concentrates support platelet activation and microparticle formation in vitro (WFH 2016) - "While we found in BeneFIX only sporadically small singular particles, pdFIX concentrates contained a lot more particles. Size distribution was different in different pdFIX concentrates. PdFIX supported platelet activation and microparticle formation induced by low concentrations of agonists, while rFIX did not. Removal of protein aggregates from pdFIX prevented the activation effect on platelets, without having and influence on FIX activity."

Biomarker • Hemophilia