Small Molecule Drug Candidate Shows Promise for Rare Kidney Stone Disease with No Current Treatment

Scientists at the Buck Institute for Research on Aging have demonstrated that N-propargylglycine (N-PPG), an orally administered small molecule, completely prevents calcium oxalate kidney stone formation and protects against kidney failure in mouse models of primary hyperoxaluria type 2. The rare genetic disorder affects an estimated 1,700 people in the United States, though experts believe many more cases remain undiagnosed, and currently has no available treatment. Primary hyperoxaluria type 2 causes progressive kidney failure in infants and young adults. The preclinical study showed N-PPG fully restored normal survival in the mouse model, offering potential hope for patients facing this life-threatening condition.

N-PPG Drug Candidate and Its Effects

• ▪N-propargylglycine (N-PPG) can completely prevent the formation of calcium oxalate kidney stones in a mouse model of primary hyperoxaluria type 2
• ▪N-propargylglycine (N-PPG) protects against kidney failure in a mouse model of primary hyperoxaluria type 2
• ▪N-propargylglycine (N-PPG) is an orally administered small molecule drug candidate
• ▪Scientists at the Buck Institute for Research on Aging demonstrated the effects of N-propargylglycine (N-PPG) in a mouse model of primary hyperoxaluria type 2
• ▪N-propargylglycine (N-PPG) fully restores normal survival in a mouse model of primary hyperoxaluria type 2

Primary Hyperoxaluria Type 2 Disease Characteristics

• ▪Primary hyperoxaluria type 2 (PH2) currently has no treatment available
• ▪Experts believe many more cases of primary hyperoxaluria type 2 (PH2) go undiagnosed beyond the estimated 1,700 cases in the United States
• ▪An estimated 1,700 people in the United States suffer from primary hyperoxaluria type 2 (PH2)
• ▪Primary hyperoxaluria type 2 (PH2) is a rare genetic disorder
• ▪Primary hyperoxaluria type 2 (PH2) causes progressive kidney failure in infants and young adults

Perspective of Buck Institute for Research on Aging researchers

• ▪The preclinical results for N-propargylglycine demonstrate complete prevention of kidney stone formation, suggesting strong therapeutic potential
• ▪N-propargylglycine represents a potential breakthrough for primary hyperoxaluria type 2, which currently lacks any approved treatment options

Perspective of Primary hyperoxaluria type 2 patients and families

• ▪An orally administered treatment for primary hyperoxaluria type 2 would offer a non-invasive option for managing the disease
• ▪Primary hyperoxaluria type 2 patients currently face progressive kidney failure without any available therapeutic interventions