Rondoraptivon pegol (BT200)

BT200 is a PEGylated aptamer that binds A1 domain of human VWF

Basic pharmacology of BT200

• Basic pharmacology of BT200 and BT101 (Zhu et al 2020a and 2020b)
• https://onlinelibrary.wiley.com/doi/full/10.1111/jth.14755

BT200 Phase 1 program (NCT04103034)

• 112 healthy volunteers in SAD, MAD, relative bioavailability, and pharmacologic challenge studies.
• Overall safety & tolerability profile is excellent.
• PK profile compatible with once weekly, small volume subcutaneous dosing regimen.
• Headline Clinical Proof of Mechanism results presented at European Hematology Association Congress 2021.

• ⚯ Kovacevic KD, Grafeneder J, Schörgenhofer C, et al. The von Willebrand Factor A-1 domain binding aptamer BT200 elevates plasma levels of VWF and Factor VIII: a first-in-human trial. Haematologica. 2022; 107(9): September, 2022.

• ⚯ Cécile V. Denis and Peter J. Lenting. How to keep the FVIII/VWF complex in the circulation. Haematologica. 2022; 107(9): 2011–2013.

BT200 Phase 2 pilot study in hereditary bleeding disorders (NCT04677803)

• “Basket design” trial included patients with either Von Willebrand Disease or Hemophilia A.
• Headline clinical proof of concept results presented at ISTH meeting in July 2021.

• ⚯ Ay C, Pabinger IP, Kovacevic KD, et al. The VWF binding aptamer rondoraptivon pegol increases platelet counts and VWF/FVIII in type 2B von Willebrand disease. Blood Adv. 2022; 6(18): 5467-5476.

• ⚯ Ay C, Kovacevic KD, Kraemmer D, et al. von Willebrand Factor-binding aptamer rondoraptivon pegol as treatment for severe and non-severe hemophilia A. Blood. 2022.