Haemostasis November & December 2024 Industry News

Our round-up of the latest developments from across the haemostasis industry for November & December 2024, including key readouts at ASH2024

REGULATORY

• Vaderis Therapeutics’ VAD044 (AKT inhibitor) granted FDA Fast Track designation for the treatment of Hereditary Haemorrhagic Telangiectasia (HHT) (18 Nov)
• Kedrion Biopharma announced that the FDA approved the company’s manufacturing facility in Bolognana, Italy, to produce Ryplazim (plasma-derived human plasminogen), the first and only FDA-approved therapy for plasminogen deficiency type 1, PLGD-1 (19 Nov)
• Pfizer’s Hympavzi (marstacimab-hncq, TFPI mAb) approved by the EMA for the treatment of adults and adolescents with severe hemophilia A or B without inhibitors; closely following the FDA approval on 11 Oct and making it the first once-weekly subcutaneous treatment authorised in the EU (20 Nov)
• Novo Nordisk’s  Alhemo (concizumab-mtci, TFPI mAb) approved by the FDA as a once-daily prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and paediatric patients 12 years of age and older with hemophilia A or B with inhibitors, based on explorer7 data (23 Dec)

CLINICAL

• Anthos Therapeutics presented new data from AZALEA-TIMI 71 Ph2 trial, demonstrating that abelacimab (Factor XI inhibitor) led to consistent and substantial reductions in bleeding for patients on or off antiplatelet (APT) therapy, as compared to rivaroxaban (Factor Xa inhibitor) at AHA2024 (18 Nov)
• TAHO Pharmaceuticals announced the dosing of the first subjects in its US Ph3 trial evaluating TAH3311, an antithrombotic oral dissolving film formulation of apixaban (19 Nov)
• Novo Nordisk presented interim efficacy and safety data from FRONTIER4 Ph 3 trial of Mim8 (Factor VIIIa mimetic bispecific Ab) prophylaxis in patients living with haemophilia A with and without inhibitors (HA/HAwI) at ASH2024 (3 Dec)
• Novo Nordisk also presented data from the Ph 3 explorer7 study assessing the efficacy of concizumab-mtci (TFPI mAb) in people with haemophilia A or B with inhibitors (HAwI/HBwI), with or without recurring bleeds into the same joint (or target joints) at baseline (3 Dec)
• Sirius Therapeutics presented preliminary Ph1 data for RSD107, a Factor XI siRNA therapeutic, under clinical development for the prevention and treatment of thromboembolic disorders (9 Dec)
• Star Therapeutics presented interim clinical data from VIVID 2, a Ph1 single ascending dose trial of VGA039 (Protein S mAb) in VWD patients at ASH2024, outlining plans to begin a multi-dose study with VGA039 in early 2025 (9 Dec)
• Metagenomi presented updated preclinical data on (B domain deleted bioengineered FVIII gene therapy) for treatment of haemophilia A at ASH2024, outlining plans for IND filing in 2026 (10 Dec)
• Expression Therapeutics presented initial data from the Ph1 trial evaluating ET3 (CD68-ET3-LV-CD34+, Factor VIII lentiviral vector gene therapy) for the treatment of haemophilia A (11 Dec)
• Regeneron Pharmaceuticals announced positive Ph2 data for its mAbs targeting distinct domains of Factor XI: REGN7508 (catalytic domain) and REGN9933 (A2 domain), outlining plans begin Ph3 program in 2025 (19 Dec)

COMMERCIAL

• Alveron Pharma raised €5.0M to advance the development of OKL-1111, a procoagulant cyclodextrin, for the treatment of intracranial haemorrhage (ICH) (6 Dec)
• Sangamo Therapeutics regained full rights to haemophilia A gene therapy programme, following Pfizer’s decision to terminate global license and collaboration agreement and cease the development of giroctocogene fitelparvovec (30 Dec)