Rondoraptivon pegol (BT200)

Rondoraptivon pegol (BT200)


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Zhu et al, J Thromb Haemost. 2020 Feb 3.

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

  • BT200 has a cognate aptamer reversal agent (BT101) with in vivo proof of mechanism in non-human primates.

Selected Publications:

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.
    • BT200 Phase 1 clinical proof of mechanism oral presentation at European Hematology Association Virtual Congress, June 9 - 17, 2021
      Title: "HALF-LIFE PROLONGATION OF VON WILLEBRAND FACTOR: A NEW THERAPEUTIC STRATEGY FOR HAEMOPHILIA A AND VON WILLEBRAND DISEASE"
      Session Title: Dilemmas in coagulation and thrombosis
      Final Abstract Code: S302
      Narrated slide presentation video link

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.
    • BT200 Phase 2 clinical proof of concept in Von Willebrand Disease Type 2b oral presentation at International Society for Thrombosis and Haemostasis Virtual Congress, July 17-21, 2021
      Title: BT200 increases von Willebrand factor (VWF), FVIII and platelet counts in patients with von Willebrand disease (VWD) type IIb
      Abstract Number: OC 72.1
      Session Date: 2021-07-21
      Session Time: 13:00 - 14:00 U.S. Eastern Daylight Time
    • BT200 Phase 2 clinical proof of concept in hemophilia A late-breaking clinical trial presentation at International Society for Thrombosis and Haemostasis Virtual Congress, July 17-21, 2021
      Abstract Title: The VWF-A1 domain binding aptamer BT200 prolongs the half-lives of different factor VIII (FVIII) products in patients with severe hemophilia A and increases FVIII levels in non-severe hemophilia A.
      Abstract Presentation Number: LB 02.1
      Session Title: Late-breaking Abstract Session II
      Session Date: Wednesday, July 21, 2021
      Session Time: 10:00:00 AM - 11:00:00 AM U.S. Eastern Daylight Time

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