Investigational oral medication fails to meet trial endpoint, does not lower LDL cholesterol

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Patients with hypercholesterolemia who received an oral investigational medication (CAT-2054) and statin therapy did not have a reduction in low-density lipoprotein (LDL) cholesterol, which was the primary endpoint in the phase 2a trial.

Catabasis Pharmaceuticals, Inc., which develops CAT-2054, announced the top-line results in a June 7 news release.

After the announcement, Catabasis’s share price fell more than 25 percent. The company is listed on the NASDAQ exchange.

During the trial, the researchers randomized 153 patients at 31 sites to receive one of four doses of CAT-2054 or placebo. They also received 40 mg of atorvastatin during a four-week run-in period.

At baseline, the patients had hypercholesterolemia while taking statins and had an LDL cholesterol level between 70 mg/dL and 190 mg/dL. The mean LDL level at baseline was 103 mg/dL.

After four weeks of treatment, the maximum LDL decrease was 7.1 percent in the CAT-2054 group and 4.5 percent in the placebo group. There were no statistically significant differences

The researchers found that 9 percent of patients in the CAT-2054 group and 3 percent of patients in the placebo group had nausea. Meanwhile, 8 percent and 0 percent of patients, respectively, had diarrhea and 5 percent and 10 percent, respectively, had urinary tract infection.

Catabasis said that a pre-specified analysis showed that patients more likely to have nonalcoholic steatohepatitis (NASH) may benefit from taking CAT-2054. In that analysis, patients had a larger reduction in ALT from baseline.

“The trial was well designed and efficiently run, enabling us to quickly determine that we do not expect to invest further in CAT-2054 for hypercholesterolemia,” Catabasis CEO Jill C. Milne, PhD, said in a news release. “We plan to complete additional analysis of  the data and determine the best path forward for CAT-2054 in NASH. Our corporate strategy remains focused on our lead program, edasalonexent (CAT-1004) in Duchenne muscular dystrophy.”