Catabasis has suffered its first big setback in the clinic, writing off its midstage program for CAT-2054 in hypercholesterolemia after its trial flopped and sent its shares into a tailspin.
The oral drug inhibits sterol regulatory element-binding protein (SREBP), which is described as a master regulator of lipid metabolism in the body. What it didn’t do, though, was influence LDL-C levels in patients when provided with high-intensity statins.
That doesn’t mean that Catabasis ($CATB), run by CEO Jill Milne, is finished with the drug, though. Researchers flagged an unspecified change in liver function tests among a subgroup of patients more likely to have NASH, or fatty liver disease, and want to pursue that angle more.
That strategy, though, didn’t have much influence on investor sentiment. The biotech’s shares plunged by 35% as the news hit after the markets closed.
The biotech’s lead drug is CAT-1004 (edasalonexent), which is designed to hit NF-kB, a fairly well-known inflammatory pathway that may play a role in hampering muscle regeneration. Catabasis raised venture cash and pushed through a $60 million IPO a year ago--during the waning days of the biotech boom--largely on the promise that it could develop a drug that would be helpful for the entire Duchenne muscular dystrophy population, rather than just a slice of it based on dystrophin mutation, as envisioned by Sarepta ($SRPT).
The Duchenne drug passed a safety hurdle early this year and still has a long way to go in the clinic.
“We plan to complete additional analysis of the data and determine the best path forward for CAT-2054 in NASH,” noted Milne in a statement. "Our corporate strategy remains focused on our lead program, edasalonexent (CAT-1004) in Duchenne muscular dystrophy.”
- here's the release
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