Setanaxib is a NOX enzyme inhibitor initially developed by Calliditas Therapeutics AB (“Calliditas”) for the treatment of primary biliary cholangitis. NOX enzymes produce reactive oxygen species (ROS) in the body that regulate processes like immune modulation, inflammation, fibrosis, and cell proliferation and differentiation. But when someone has excess ROS, researchers believe that it can cause oxidative stress and drive a variety of diseases. Since developing setanaxib, Calliditas has expanded its focus to include squamous cell carcinoma of the head and neck and idiopathic pulmonary fibrosis; the drug is currently being evaluated in studies for all three of the above indications.

Now, Calliditas has begun exploring setanaxib as a potential therapeutic intervention for another rare condition: Alport syndrome. In fact, reports European Pharmaceutical Manufacturer, the U.S. FDA recently granted Orphan Drug designation to setanaxib for Alport syndrome. Orphan drug designation is granted to drugs or biologics designed to treat, diagnose, or prevent rare conditions affecting fewer than 200,000 people nationwide. Traditionally, it can be difficult to mobilize drug development in the rare disease space. That is why Orphan drug designation comes with incentives such as fee waivers, tax credits, increased company communication with and guidance from the FDA, and seven years of market exclusivity if/when the drug is approved. 

Currently, there are no FDA-approved treatments for Alport syndrome. Available treatments are symptomatic and designed to slow the progression of kidney damage. Therefore, identifying a new and effective treatment could have significant and meaningful impact on this patient community. Moving forward, Calliditas plans to launch a Phase 2 clinical trial to evaluate setanaxib in 20 individuals with Alport syndrome by the end of this year.

Learn More: Alport Syndrome

Alport syndrome is a rare kidney disease which damages the small blood vessels in your kidneys. As the kidneys become increasingly damaged, kidney function worsens. Caused by COL4A3, COL4A4, or COL4A5 gene mutations, Alport syndrome may be inherited in an X-linked, autosomal dominant, or autosomal recessive manner. About 80% of those affected inherit it in the X-linked fashion, with 90% of affected males developing kidney failure by age 40. Kidney failure is considered less common in affected females. Beyond the kidneys, people with Alport syndrome may experience inner ear and eye abnormalities; again, this is more common in males. 1:50,000 people is born with Alport syndrome.

Symptoms can include:

• Proteinuria (excess protein in the urine)
• Hematuria (blood in the urine)
• Sensorineural hearing loss
• Misshapen lenses in the eyes (anterior lenticonus)
• High blood pressure
• Swelling of the ankles, legs, feet, or around the eyes
• Chest aneurysm
• Abnormal coloring of the retina
• End stage renal disease and associated symptoms
  • Weakness and fatigue
  • Changes in appetite
  • Poor digestion
  • Excessive thirst
  • Frequent urination