Belgium's Opperbas Holding has started a mid-stage clinical trial of a new formulation of factor VIII - used to treat haemophilia A - that should cut the number of injections that sufferers of the bleeding disease need to endure.
Haemophilia A is currently treated by up to three weekly infusions of FVIII, a protein that is lacking or defective in the affected patients. But combining the protein with Opperbas' delivery platform could reduce this down to one a week.
This could lead to a major improvement in patient convenience and treatment, enabling more patients to use prophylactic treatment and preventing painful and disabling bleeding episodes, according to the company.
The platform is based on the use of pegylation - a technique involving the attachment of a polymer (polyethylene glycol or PEG) to a protein that is already widely used to extend the activity of protein-based drugs in the body - alongside small lipid particles called liposomes.
Liposome encapsulation is used to improve the delivery and reduce the side effects of some drugs. But Opperbas says its technology differs in one crucial respect - instead of encapsulating the protein, the liposomes are pegylated and the protein is bound to the PEG side chains, leaving it free to exert its effects.
Importantly, the protein does not bind covalently to the liposome, so there is no need for a change in the manufacturing process for the peptide or protein that is to be delivered.
"Since the protein/peptide is formulated with the liposomes simply by hydrating the dry protein concentrate with the liposome solution, there is no need to change the production process," said Opperbas. And this could also mean that the regulatory route for the new peptide is simplified.
Israel-based Omri Laboratories developed the delivery technology for Opperbas, and the factor VIII formulation showed encouraging results in the Phase I/II trial completed last year.
Robert Taub, Opperbas' founder, said the goal is to out-license use of the delivery technology in this indication to one of the major haemophilia pharmaceutical companies. The leading players in this sector include Bayer, Novo Nordisk, Aventis Behring, Wyeth and Baxter International.