On September 30, 2020, BioCryst Pharmaceuticals (“BioCryst”) announced promising data on their drug candidate BCX9930. The therapy, an orally administered Factor D inhibitor, is designed to treat patients with previously untreated paroxysmal nocturnal hemoglobinuria (PNH).
BCX9930
Discovered and then developed by BioCryst, BCX9930 is a Factor D inhibitor. According to ScienceDirect, Complement Factor D:
is a serine protease that cleaves a lysine-arginine bond in factor B and thereby participates in the activation of the alternative pathway.
In simpler terms, Factor D is an enzyme in the alternative pathway, which activates and amplifies the complement system. Since the complement system plays a role in immune function and spurs inflammation, an over-active complement system leads to severe inflammatory disorders. BCX9930 is designed to inhibit immune response in PNH.
Currently, BCX9930 is still in Phase 1 clinical development. However, BioCryst has explored the safety, efficacy, and tolerability of the therapy. Patients with severe but previously untreated (treatment-naive) PNH received a monotherapy dose of 400mg for 6+ weeks. The therapy reduced LDH and increased hemoglobin levels. This offers an effective way to increase hemoglobin levels without requiring transfusions.
Further, BCX9930 was found to be safe, effective, and well-tolerated. Side effects included a headache and mild rash. However, no moderate-to-severe adverse reactions were found. Based on its promise, BCX9930 received both Fast Track and Orphan Drug designations.
Paroxysmal Nocturnal Hemoglobinuria (PNH)
Caused by PIGA gene mutations, which cause PNH cells to quickly multiple, paroxysmal nocturnal hemoglobinuria (PNH) is a hematopoietic stem cell (HSC) disorder which affects platelets, as well as red and white blood cells. PNH causes the immune system to mistakenly attack stem cells. It also destroys blood cells. Around 30% of PNH cases result from aplastic anemia treatment. Generally, patients are diagnosed between ages 35 and 40. In mild cases, patients may survive for a while. However, general overall survival rate is around 10 years following diagnosis. Symptoms include:
- Shortness of breath or difficulty breathing
- Fatigue
- Dark, bloody urine
- Chest pain
- Headaches
- High heart rate
- Difficulty swallowing
- Kidney damage
Learn more about PNH.
