New Drug Performs in Chronic Kidney Disease Clinical Trial

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La Jolla Pharmaceutical Company announced on March 10 that its lead experimental drug, which treats chronic kidney disease, met its primary goal of improving kidney function as measured by blood filtering through the kidneys.

The results from the phase 2a study lent the company’s shares a 40 percent bump in trading upon the announcement, which reported results with two tested doses of the drug, known now as GCS-100.

The lower dose was more favorable than the higher. The lower amount of drug showed an increase in the rate of blood filtering, whereas the higher dose did not. The lower dose also reduced the levels of galectin-3, a protein associated with tissue scarring, which causes organ damage.

The higher dose did not show a statistically significant increase in either the rate of blood filtering or the decrease of galectin-3 levels in comparison with placebo. The researchers speculated that a higher dose of the drug might stop the production of galectin-3 so much and so effectively that the body might start producing the protein again, in a feedback loop effect.

The doses were well tolerated, with no adverse effects in the lower-dose group; side effects in the higher-dose group were not related to the drug, the company reported.

Reuters wrote that analysts were bullish on the results. “Even with a modest penetration, we estimate the drug could have more than $2 billion in peak sales in 2024, and that is a conservative estimate,” said Ling Wang of Chardan Capital Markets.

Before the company announced the results, Wedbush analyst Liana Moussatos gave an “outperform rating,” StreetInsider reported.

“GCS-100 is the lead drug candidate which reduces elevated galectin-3 associated with chronic kidney disease and nonalcoholic steatohepatitis (NASH)—both large market opportunities for which the standard-of-care can cause life-threatening side effects and/or has no approved therapies,” Moussatos said.