La Jolla Pharmaceutical Shares Surge on Positive Trial Results

The company said the randomized, placebo-controlled Phase II trial of GCS-100 in chronic kidney disease met its primary efficacy endpoint of a statistically significant improvement in kidney function. Key secondary endpoints were also met.

La Jolla Pharmaceutical CEO George Tidmarsh 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.

Chronic kidney disease reportedly affects 14% of Americans, or around 49 million people. GCS-100 is a high molecular weight, complex polysaccharide that inhibits galectin-3, a molecule suspected of playing a central role in tissue fibrosis. GCS-100 also has been shown to be therapeutically active in preclinical models of other diseases where tissue fibrosis plays a central role, such as nonalcoholic steatohepatitis.

La Jolla Pharmaceutical shares were up more than 58% in premarket trading to $17.27. The 52-week range had been $3.00 to $12.00.