Near the end of September, Hansa Biopharma (“Hansa”) announced promising data from a Phase 2 clinical trial on imlifidase. This therapy is designed to treat patients with severe anti-GBM antibody disease, or Goodpasture’s disease. This rare autoimmune disorder causes severe kidney damage. However, imlifidase was found to be fairly safe, effective, and well-tolerated, possibly filling an unmet therapeutic need for patients.

Phase 2 Trial

Within the Phase 2 clinical trial, researchers evaluated imlifidase as a treatment in the 15 patients enrolled. Previously, treatments for this condition included invasive steps such as dialysis and transplants. Now, imlifidase offers a less invasive – but still effective – solution to preventing renal damage.

The trial participants ranged in age from 19 to 79. The median age was 61. Patients came from various European countries. 40% were females and 60% were males. At the start of the trial, 10 patients (66%) required frequent dialysis (also known as “dialysis dependent”) and 5 did not. By the end of the trial, one patient had died (unrelated to the study). Of the remaining participants, only 4 required dialysis, while 10 were now dialysis independent. Additionally, imlifidase lowered anti-GBM antibodies to a normal and safe range.

Imlifidase received Orphan Drug status for the treatment of Goodpasture’s disease from both the United States and the European Union.

Goodpasture’s Disease

In some cases, Goodpasture’s disease is also called anti-glomerular basement membrane disease, or anti-GBM disease. This rare autoimmune disorder occurs when the body’s immune system creates anti-GBM antibodies. The glomerular basement membrane is located in your kidneys. So those with Goodpasture’s disease experience severe kidney damage. In some cases, the condition may also affect the lungs. Generally, Goodpasture’s disease affects males in their 20s, but normally affects females in their 60s. Doctors are not yet sure what causes Goodpasture’s disease. However, it is believed to be a combination of genetics and environmental factors, such as chemical exposure, drug use, smoking, and certain types of infections.

As described by the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDKD), there are serious complications of this condition, which include:

severe kidney inflammation, which can quickly lead to kidney failure; [and] severe bleeding in the lungs, which can cause respiratory failure.

Symptoms include:

• Fatigue
• Muscle weakness
• Anemia (low red blood cell count)
• Nausea and vomiting
• Foamy urine
• Proteinuria (excess protein in the urine)
• High blood pressure
• Dark or pink urine
• Swelling in the legs and feed
• Chest pain
• Difficulty breathing
• A persistent, dry cough
• Coughing up blood
• High blood pressure

Learn more about Goodpasture’s disease.