- In 2019, the U.S. President announced initiatives to increase transplant rate and quality of life for dialysis patients and also reduce expenditure to treat chronic and end-stage renal disease
- About 37 million Americans suffer from some form of kidney disease, which was the nation’s ninth leading cause of death in 2017. When patients reach the final stage, their kidneys fail, requiring either a transplant or frequent dialysis to stay alive
- U.S. Medicare spends more than USD 114bn on kidney care annually, of which USD 79bn covers people with chronic kidney disease and another USD 35bn is on end-stage renal disease (2016)
- Of more than 100,000 Americans who begin dialysis to treat end-stage renal disease each year, one in five will die within a year
Kidney transplantation reduces health care costs in the US
HLA-sensitization is a major barrier to kidney transplantation. Highly sensitized patients have high levels of anti-HLA antibodies, which are likely to target and significantly compromise a transplanted organ. The more antibodies, the lower the likelihood of finding a donor organ that will be a match. Many highly sensitized patients will indefinitely remain in a debilitating disease state on long-term dialysis, which is associated with a high cost, a poor quality of life and an increased mortality rate.
The alternative treatment for patients with failed kidneys is dialysis, a treatment that requires 5 - 6 hours of treatment three to four times per week. Long-term dialysis is associated with risks of cardiovascular complications and premature death. Kidney transplantation enables most patients to return to a normal life, even though all transplanted patients need to be treated with immunosuppressive treatment.
Approximately 9,000 patients die every year on kidney transplant waitlists in the U.S. and Europe. Kidney transplantation is limited by organ availability and donor/recipient matching. An estimated 30% of patients on transplant waitlists are moderately or highly sensitized, 10-15% are highly sensitized
Today there are no approved therapies to desensitize patients.
Acute Autoimmune Conditions
Imlifidase may have additional potential medical applications. These include serious, or even life-threatening, acute autoimmune diseases within neurology, nephrology and haematology.
Acute AMR is one of the most challenging adverse events following a kidney transplantation. It occurs in 10-15% of patients and is the main cause for graft dysfunction. In Europe and the US, there are approximately 40,000 patients who receive kidney transplants annually and approximately 400,000 who currently live with a kidney transplant.
Hansa is conducting a Phase 2 study in AMR, designed to evaluate the safety and efficacy of imlifidase in eliminating donor specific antibodies (DSA´s).
Anti-GBM antibody disease, also known as Goodpasture disease, is a rare severe kidney disease where the immune system mistakenly develops IgG-antibodies against an antigen intrinsic to the glomerular basement membrane (GBM), resulting in an acute immune attack on the kidneys and in some patients also on the lungs. In severe anti-GBM, the disease may progress to renal failure or death. Less than one third of the patients survive with a preserved kidney function after six months follow-up. Anti-GBM antibody disease is affecting one in a million annually. Imlifidase has been granted Orphan Drug Designation for this indication from FDA and the European Commission. Currently, a Phase 2 clinical study is ongoing in anti-GBM to evaluate the safety and tolerability of imlifidase and assess the efficacy based on renal function at 6 months after treatment.
Guillain-Barré Syndrome is a rare, acute, paralyzing disease of the peripheral nervous system, affecting 1-2 in 100,000 people per year. GBS is usually preceded by an infection or other immune stimulation that induces an autoimmune response, targeting the peripheral nervous system. It is the most frequent cause of acute neuromuscular weakness in the Western world and can occur at any age. Mortality rate is 3-7 %. GBS is a rapidly progressive monophasic disorder, often leading to a severe paralysis of the arms and legs. Most GBS patients have sensory disturbances (tingling, numbness or ataxia) and pain, and some patients have double vision or problems with swallowing. GBS may also paralyze the respiratory muscles, leading to intensive care unit admission and mechanical ventilation. Current standard of care is either IVIg or plasmapheresis. There is a significant need for better treatment options. Imlifidase has been granted Orphan Drug Designation for this indication from FDA and the European Commission. Hansa is currently conducting a Phase 2 clinical trial with imlifidase to evaluate the safety, tolerability and efficacy of imlifidase in GBS patients in combination with standard immunoglobulin care.
Hansa Biopharma is dedicated to developing treatments for rare immunological diseases.
In Europe the definition of a rare disease is one that affects five in 10,000 people. According to Eurordis, a non-governmental, patient driven alliance of rare disease patient organizations, approximately 30 million people are affected by rare diseases in Europe.
In the U.S., a rare disease is a condition that affects fewer than 200,000 people. The definition was adopted by the U.S. Congress in the Orphan Drug Act, to create financial incentives for development of new rare disease drugs. Approximately 25-30 million Americans are living with a rare disease.