Biopharmaceutical company La Jolla Pharmaceutical Company, a leading developer of therapeutics that target galectin proteins, has announced the first patient has been dosed in a randomized, single blind Phase 2 clinical trial of GCS-100 for chronic kidney disease (CKD).
“Based on the favorable safety profile and encouraging Phase 1 results, we are pleased to continue the development of GCS-100 with the initiation of this Phase 2 trial in patients with CKD,” said La Jolla President and CEO George Tidmarsh, M.D., Ph.D. “The reduction in serum galectin-3 concentration we observed after a single dose of GCS-100 in the Phase 1 trial suggests an effect on a significant biomarker of disease and indicates that GCS-100 may have clinical activity. Millions of people in the world suffer from CKD without curative therapy, and we hope to one day provide help to those in need.”
The Phase 2 study of GCS-100 has been designed to enroll around 117 subjects that are at least 18 years old and have stage 3b or 4 CKD. These subjects will be randomly assigned 1:1:1 to treatment with placebo, 1.5 mg/m2GCS-100, or 30 mg/m2GCS 100. Randomization will additionally be stratified into two groups, according to baseline renal functions as defined by eGFR values of 15-29 and 30-44 mL/min/1.73m2, respectively. Subjects, once randomized, will receive eight consecutive weekly doses of their assigned treatment and then a follow-up four weeks after treatment.
The study’s primary objective is to compare the change in eGFR from baseline to week eight between placebo and GCS-100 treatment. The study’s secondary objective is to determine the safety and tolerability of GCS-100 administered for eight weeks relative to placebo. The study will additionally measure the effect of GCS-100 on circulating galectin-3 and other disease activity markers.
Recently, LaJolla completed and announced the results of a Phase 1 study of GCS-100 in patients with stage 3b and 4 CKD. It was found that the product candidate was safe with no serious adverse events observed and no adverse changes in laboratory measures. Baseline serum galectin-3 levels inversely correlated with renal function, as measured by eGFR, with higher galectin-3 levels found in patients who had worse renal function (lower eGFR). That correlation strengthens the belief that high levels of galectin-3 cause organ fibrosis, resulting in reduced kidney function. A post-dose reduction of serum galectin-3 that is statistically significant was also observed at the higher doses of GCS-100. This reduction in serum galectin-3 was sustained until day seven, when a rebound to levels above baseline was observed. This rebound could be due to release of galectin-3 from cellular receptors, including those found in kidney tissue.
For more information, visit www.ljpc.com
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