La Jolla's GCS-100 Improves Kidney Function In Study

La Jolla Pharmaceutical Co announced positive top-line results from its Phase II trial evaluating GCS-100 for treatment in chronic kidney disease (CKD). The company’s shares rose about 40 percent in extended trading following the news.

The trial titled GCS-100 Phase 2 CKD study met its primary efficacy endpoint of statistically significant increase in approximated glomerular filtration rate (eGFR) against placebo between base line and end of treatment based on a dose of 1.5mg/m. The Phase II trial was a blinded, multi-center, randomized clinical trial which involved 121 patients with Stage 3b or 4 chronic kidney disease.

“Chronic kidney disease is an enormous and growing medical problem worldwide. A disease-modifying agent that could slow and potentially reverse the tissue fibrosis that is a hallmark of this disease would be a welcome advance in the field and could have a major impact on patients’ lives. Our experience with GCS-100 in this trial was very encouraging, and my patients tolerated the therapy well,” said Pablo E. Pergola, Director of the Clinical Advancement Center subsidiary of Renal Associates P.A. and Clinical Associate Professor of Medicine at the University of Texas Health Science Center at San Antonio.

CKD currently affects 49 million people in the U.S. alone, including 19 million with Stage 3 and 4 disease. GCS-100 is a high molecular weight, complex polysaccharide that works to inhibit galectin-3. Galectin-3 is a molecule thought to play a central role in tissue fibrosis.

George Tidmarsh, President and Chief Executive Officer of La Jolla Pharmaceutical, said “We want to thank the patients who volunteered for this study and our excellent group of investigators for their hard work completing this Phase 2 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.”

If approved, GCS-100 could be a potential blockbuster drug in the CKD market where no approved treatments currently exist.