Pro Bono Bio is using its experience developing nano-lipid based devices and products with PEG to create a SQ version, PBB-8-SQ.

Alongside treating patients with inhibitors, one of the biggest drawbacks has been the need for intravenous administration of blood factors. Subcutaneous delivery is much more easily administered, encouraging self-medication and improved compliance.

This product is suitable for severe haemophilia A patients who make up approximately 40% of the haemophilia A population and require prophylactic treatment to manage their condition effectively.

Pro Bono Bio has focused on the benefits of Chaperoned FVIII because of its unique attributes:

  • PEG covalently bonded to the liposome not protein
  • No alteration required to the base protein so no new biologic or chemical entity is formed
  • Highly polarised sites on blood factors (two on FVIII near/on epitopes) attractive to highly polarised sites on PEGylated Liposomes
  • The product is a device that improves the performance of plasma derived and recombinant blood factors alike
  • It can be used when reconstituting ANY version of FVIII. A similar Pro Bono Bio Chaperone, PBB-7-IV, for FVII has completed Phase II in Russia.
  • This means it applies to ALL haemophiliacs, whether on plasma derived FVIII or rFVIII, providing Pro Bono Bio with the largest market by meeting the broadest unmet medical need

The unique capability to work with ALL versions of FVIII (and in due course FVII) and to deliver a subcutaneous solution makes PBB-8-SQ a highly differentiated, market disruptive (and therefore valuable) product.

PBB is planning a Phase I/II study to begin in 2017.