As a new therapeutic agent with a novel mechanism of action, it has the potential to offer significant improvement in treatment for transplant recipients and patients suffering from autoimmune related diseases.
BCX-4208 is a second-generation transition-state analog inhibitor of the enzyme purine nucleoside phosphorylase (PNP), which may have the potential to offer greater efficacy and activity in the treatment of autoimmune disease and transplant rejection than currently available therapies.
It is believed to have a potent ability to modulate T- cell activity. T-cells help the body determine when to initiate immune responses and when to accept or reject newly transplanted organs.
By specifically modulating T-cell activity, BCX-4208 may offer transplant and autoimmune patients a more efficacious and tolerable treatment option.
Under the terms if the agreement, Roche will obtain worldwide rights to BCX-4208 in exchange for a $25 million up-front payment and a $5 million payment as reimbursement for supply of material during the first 24 months of the collaboration.
Future event payments could reach $530 million in addition to royalties on product sales of BCX-4208.
In addition, Roche will have a right of first negotiation on existing back-up PNP inhibitors in transplant rejection or autoimmune diseases for a total of five years.
BioCryst retains the right to co-promote BCX-4208 in the US for several indications. Any new PNP inhibitor discovered subsequent to this agreement will be exempt from this agreement and BioCryst will retain all rights to such compounds.
"This collaboration not only produces a substantial strategic and economic benefit to BioCryst, it also provides all of the essential elements for the comprehensive and competitive development of BCX-4208," said Charles Bugg, BioCryst's Chairman and CEO.
The greatest threat to transplant patients is rejection of the transplanted organ by the body's own immune system. For this reason, transplant recipients must take drugs to suppress the immune response and prevent rejection usually for the rest of their lives.
A regimen combining several drugs is usually given and this treatment has to be continued indefinitely. Rejection of the new kidney by the patient's immune system can lead to loss of the transplanted organ and a return to dialysis for kidney transplant recipients.
For heart, lung and liver transplant patients, loss of the transplanted organ presents an immediate threat to life.
Autoimmune diseases occur when the immune system attacks the body's own cells rather than invading microorganisms.
There are more than 80 clinically distinct autoimmune diseases (i.e. multiple sclerosis, rheumatoid arthritis and some types of diabetes), each affecting the body in different ways.
Presentation of these diseases can also vary from patient to patient with the same condition, and can lead to organ failure requiring transplantation.
Corticosteroids are still the mainstay of treatment for many autoimmune diseases and physicians have to constantly balance the requirement for best possible disease control with the drug related morbidities associated with long term steroid exposure.