La Jolla Pharma reports top-line results from Phase II study of chronic kidney disease drug

The trial met its primary efficacy endpoint of a statistically significant improvement in kidney function. Specifically, a dose of 1.5mg/m2 led to a statistically significant (p=0.045) increase in estimated glomerular filtration rate (eGFR) compared to placebo between baseline and end of treatment. At the 30mg/m2 dose, there was no statistically significant difference.

Key secondary endpoints were also met, and the effect on circulating galectin-3 levels was consistent with the effect on eGFR. For the 1.5mg/m2 dose, there was a statistically significant (p=0.067) reduction in circulating levels of galectin-3, while there was no significant difference at the 30 mg/m2 dose level.

Galectin-3 is the molecular target of GCS-100 and has been shown to be a mediator of tissue fibrosis. Potassium, uric acid and blood urea nitrogen (BUN) all improved at the 1.5 mg/m2 dose level.

GCS-100 was well-tolerated. Out of 121 patients enrolled, 117 completed treatment, including all 41 patients treated at the 1.5mg/m2 dose. There were no serious adverse events (SAEs) in the 1.5mg/m2 dose group compared to two in the placebo group and two in the 30 mg/m2 group. All SAEs were deemed by the investigators as not drug-related.

La Jolla Pharmaceutical president and CEO Dr George Tidmarsh noted the company wants to thank the patients who volunteered for this study and our excellent group of investigators for their hard work completing this Phase II trial in a timely fashion.

"We are obviously delighted with the results. We hope to use these results as a platform for further investigation not only in chronic kidney disease but also in other diseases where tissue fibrosis plays a central role," Dr Tidmarsh added.