BridgeBio Pharma (NASDAQ:BBIO) said positive phase 1 data of BBP-711 in healthy volunteers supports developing the therapy for patients with primary hyperoxaluria type 1 (PH1) and recurrent kidney stone formers.
PH1 is a rare disorder that mainly affects kidneys and is caused due to the buildup of a substance called oxalate, which normally is filtered through the kidneys and excreted in the urine. It, when combines with calcium, is also responsible for kidney stone formation.
BBP-711 was evaluated in a two-part, phase 1 trial , which included 92 healthy people.
The company said pharmacokinetic (PK) parameters showed that the drug was rapidly absorbed with a time to maximum concentration of ~2.5 hours and an elimination half-life of ~26 hours, supporting a once-daily dosing.
The results also showed clinically meaningful pharmacodynamic (PD) increases in plasma glycolate of 10-15-fold above baseline levels.
The company noted that the PK-PD data suggests near-complete inhibition of glycolate oxidase (GO) can be sustained throughout the dosing period.
BBP-711 was well-tolerated, according to the company.
Based on this data, BridgeBio plans to start a phase 2/3 study in PH1.
The company also intends to begin a proof-of-concept phase 2 trial in adult recurrent kidney stone formers with elevated urinary oxalate excretion, at the end of 2022, pending discussions with regulators.