In a late March 2022 news release from biopharmaceutical company CANbridge Pharmaceuticals, Inc. (“CANbridge”), the company noted that the first patient was dosed in a Phase 1b/2 clinical trial evaluating CAN106 for paroxysmal nocturnal hemoglobinuria (PNH). This trial, centered in China, has the potential to significantly change patient outcomes. Ultimately, this is due to the fact that there are few C5 inhibitors available in China for those with PNH; currently, only one approved option exists.
C5, in the complement system, has been associated with a number of different complement-mediated disorders. CAN106 is a human monoclonal antibody, given via intravenous administration, which inhibits C5, prevents red blood cell destruction, and helps to preserve immunity. Prior studies have shown that the treatment is relatively safe and well-tolerated. This burgeoning Phase 1b/2 trial will further this understanding by evaluating the therapy’s pharmacokinetics, pharmacodynamics, safety, efficacy, and tolerability. Patients in the study have not been previously treated. Learn more about CAN106.
What is Paroxysmal Nocturnal Hemoglobinuria (PNH)?
PIGA gene mutations cause paroxysmal nocturnal hemoglobinuria (PNH), an acquired hematopoietic stem cell disorder. These mutations cause rapidly multiplying PNH cells. As the immune system attacks the stem cells, it mistakenly attacks the healthy cells, killing them off and allowing PNH cells to survive. PIGA mutations occur spontaneously. PNH affects all of the different blood cells. It is a variable disorder, meaning some patients may experience mild symptoms and a longer lifespan, while others may experience more severe symptoms and a lowered lifespan. An estimated 30% of PNH cases occur as a result of aplastic anemia treatment.
PNH causes the death and destruction of red blood cells, as well as abnormalities in platelets and white blood cells. An estimated 1 to 2 people per million in Western countries, and 10 per million in China, have PNH. It affects males and females equally. Typically, symptoms manifest between ages 30-40, though symptoms may appear outside of these bounds. These symptoms include:
- Red blood cell hemolysis (destruction)
- Back and chest pain
- Difficulty swallowing
- Fatigue and general weakness
- Abdominal contractions
- High heart rate
- Headache
- Dark or bloody urine, which may come and go over time
- Shortness of breath and/or difficulty breathing
- Easy bruising and bleeding
- Blood clots
- Male sexual dysfunction
- Kidney disease
