Canadian-based Revive Therapeutics has initiated a Phase II clinical trial of REV-004 (Bucillamine) for the treatment of patients with cystinuria in the US.
REV-004 is a potent thiol donor with two thiol groups designed to undergo a thiol-disulfide exchange with cystine to form a soluble bucillamine-cysteine compound.
The multicentre, open-label, sequential dose escalation Phase II trial will evaluate the safety and tolerability of the drug in approximately 30 patients over a period of seven days with follow-up for safety of an additional seven days.
Revive Therapeutics president Fabio Chianelli said: "The initiation of the Phase II study marks another important clinical milestone for Revive.
"I am very excited to advance our cystinuria treatment into human clinical trials in the US, and we look forward to updating the investment and medical communities on our developments as they arise."
Cystinuria is an inherited autosomal recessive disease, which causes the formation of cystine stones in the kidneys, ureter, and bladder due to high concentrations of the amino acid cystine in the urine.
The bucillamine-cysteine compound formed by the drug can be excreted through urine, leading to a decrease in the concentration of cystine to below the solubility limit, preventing stone formation.
The primary objective of the trial is to measure the incidence of treatment-emergent adverse events, while the secondary objectives include measurement of 24-hour urine cystine excretion and capacity.
The firm has recently received the investigational new drug application (IND) approval and the orphan designation status for REV-004 in cystinuria from the US Food and Drug Administration (FDA).