Ascension is using its experience developing nano-lipid based 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.
Ascension 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
- It improves the performance of plasma derived and recombinant blood factors alike
- Applies to all haemophiliacs, whether on plasma derived FVIII or rFVIII, providing Ascension with the largest market by meeting the broadest unmet medical need
The unique capability to work with all versions of FVIII and to deliver a subcutaneous solution makes PBB-8-SQ a highly differentiated, market disruptive (and therefore valuable) product.
Ascension is planning a Phase II study to begin in 2019.