US-based La Jolla Pharmaceutical has completed dosing in its Phase II clinical study of GCS-100 for the treatment of chronic kidney disease (CKD) and has also collected the final data for assessment of the primary endpoint.
Primary efficacy endpoint of the trial is the change in estimated glomerular filtration rate (eGFR) from baseline to the average at day 50 and 57 in each GCS-100 dose group compared to placebo.
According to the company, the trial's primary endpoint will be reached if the difference in eGFR between both GCS-100 dose group and placebo has a significance of less than 10%.
La Jolla president and CEO George Tidmarsh said: "We look forward to analysing the data, which we expect to release in March."
The trial included a total of 121 subjects with Stage 3b or 4 CKD, with 117 subjects having completed it with no discontinuation due to an adverse event.
In the trial, subjects were randomly assigned 1:1:1 to treatment with placebo, 1.5 or 30mg/m2 of GCS-100.
In addition, randomisation was stratified by baseline renal function, defined by eGFR values of 15-29 and 30-44 mL/min/1.73m2.
Once randomised, the subjects were given eight consecutive weekly doses of their assigned treatment with a follow-up four weeks after treatment.
La Jolla's lead product candidate, GCS-100 is a modified pectin and is currently being tested in a Phase II clinical trial and extension study in CKD patients.
It is a first-in-class inhibitor of galectin-3, a novel molecular target implicated in chronic organ failure and cancer.
Pectins are high molecular weight, complex polysaccharides that can be secured from various sources, all sharing a common basic structure including a block co-polymer of 1,4-linked galacturonic acid and 1-2-linked rhamnose with side branches of either 1,4-linked ß-D-galactose or 1,5-a-linked L-arabinose.
Image: La Jolla's lead product candidate, GCS-100 is currently being tested in a Phase II clinical trial and extension study in CKD patients. Photo: courtesy of cooldesign/ freedigitalphotos.net.