Chinook Receives Rare Pediatric Disease Designation from U.S. Food and Drug Administration for CHK-336 for Treatment of Primary Hyperoxaluria
CHK-336 On Track for Phase 1 Clinical Trial Initiation in the Second Half of 2021
The FDA defines a rare pediatric disease as a serious or life-threatening disease in which the serious or life-threatening manifestations primarily affect individuals aged from birth to 18 years and the disease affects fewer than 200,000 people in
“We are pleased the FDA has granted Chinook rare pediatric disease designation for CHK-336 for the treatment of primary hyperoxaluria, a devastating disease that usually presents in childhood, with life-threatening complications into adulthood,” said
CHK-336, is a first-in-class, liver-targeted oral small molecule LDHA inhibitor for the treatment of PH. LDHA catalyzes the final step in the production of oxalate from glyoxalate in the liver, therefore LDHA inhibition has the potential to treat all forms of PH as well as other disorders arising from excess oxalate. CHK-336 has the potential for robust efficacy by rapidly distributing to the site of oxalate production, while minimizing systemic exposures and potential for off-target activity, to facilitate a favorable tolerability profile required in this chronic disease. In PH1 mouse models, CHK-336 demonstrated significant and dose-dependent reductions in urinary oxalate, with the majority of CHK-336-treated mice reaching the normal range seen in wild-type mice. CHK-336 is currently progressing through IND-enabling studies with phase 1 initiation planned for the second half of 2021.
About Primary Hyperoxaluria (PH)
Hyperoxalurias, including PH, are diseases caused by excess oxalate, a potentially toxic metabolite typically filtered by the kidneys and excreted as a waste product in urine. Symptoms of PH include recurrent kidney stones, severe pain, blood in the urine and urinary tract infections; which when left untreated, can result in kidney failure requiring dialysis or dual kidney/liver transplantation. In patients with hyperoxalurias, excess oxalate combines with calcium to form calcium oxalate crystals that deposit in the kidney, resulting in the formation of painful kidney stones and driving progressive kidney damage over time. PH1, PH2 and PH3 are ultra-rare diseases caused by genetic mutations that result in excess oxalate, and in its most severe forms, can lead to end-stage kidney disease at a young age.
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Source: Chinook Therapeutics, Inc.