LaJolla Pharmaceuticals (LJPC) is a biotechnology company focused on developing innovative compounds for unmet medical needs. The lead program for the company is its GCS-100 drug compound that treats patients with CKD – Chronic Kidney Disease. GCS-100 uses a compound known as polysaccharide which is derived from pectin. The reason for the use of pectin is that is has the ability to not only bind to but also block the effects of galectin-3. Galectin-3 is over-expressed in multiple chronic organ failures which makes this compound suitable for treating these patients with Chronic Kidney Disease.
Chronic Kidney Disease is the eventual loss of kidney functions that can be caused by tissue fibrosis being damaged over time. The kidney's functions are to remove wastes and other excess fluids from the body. Without the ability for the Kidney to do these functions wastes build up in the blood of the body which can lead to end-stage renal disease. End-stage renal disease is the final stage of Chronic Kidney Disease and at that point the patient would either need dialysis or a kidney transplant to avoid death. There are about 26 million people in the U.S. that have CKD and a treatment regimen from GCS-100 may be the key to helping these patients.
The company reported positive phase 2a results in CKD and has shown these results at a recent presentation known as the “American Society of Nephrology”. The phase 2a trial recruited 121 patients that received GCS-100 drug doses 1.5 mg/m2 or 30 mg/m2. Another group of patients received a placebo instead and were not given the company's drug at all. One key thing to note is that GCS-100 is an IV drug that is given to patients through an IV infusion. The phase 2a trial was attempting to evaluate the patients' glomerular filtration rate or eFGR. This eFGR was considered to be the primary endpoint of the study while the secondary endpoint was to determine the safety and tolerability of the drug. The eFGR is a test that is used by doctors to determine the level of kidney function in its current state. The end results were that GCS-100 at 1.5 mg/m2 produced a statistically significant eFGR of the kidney as compared to a placebo drug.
