Busulipo™ is one of Pharmalink's lead products and has been developed to provide a best-in-class conditioning agent for use prior to hematopoietic stem cell transplantation (HSCT), also often known as bone marrow transplantation. While a niche indication, HSCT has become a treatment of choice for many diseases in adults and children and a conditioning regimen is an important part of a successful outcome. There are more than 50,000 HSCTs carried out per year.
The basis of the Busulipo™ concept is a liposome/lipid complex formulation that improves the safety and stability of the chemotherapy agent busulfan that is currently the gold standard for use in conditioning prior to HSCT. Busulipo™ eliminates the need for the toxic solvent DMA and has increased stability after reconstitution.
The concept behind Busulipo™ was originally developed by Prof. Moustapha Hassan and Dr. Zuzana Hassan, at Karolinska Institute (Sweden) for use in children suffering from malignancies or metabolic disorders and in need of bone marrow transplantation. In 2006, Pharmalink acquired the project with the aim of bringing it to the market.
Busulipo™ is patented in the US and EU, and pending in Japan. It was granted orphan drug designation by the US FDA in 2013.
An early formulation of Busulipo™ has successfully undergone clinical trials with more than 90 patients treated. Extensive data on efficacy, safety and pharmacokinetics have been collected and these compare positively with historical controls. Pharmalink has optimised this product formulation to provide a two-year shelf life.
The Company is currently preparing for the pivotal clinical trials with this formulation, which are expected to start in 2014 and are-needed for registration and introduction of Busulipo™ into key global markets in discussion with regulatory authorities.
Pharmalink’s strategy is to partner Busulipo™ for all territories outside the Nordic region.