Catabasis shares decimated after LDL drug fails PhIIa trial

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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.

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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

Related Articles:
Catabasis pulls off a $60M IPO as biotech's Wall Street window stays open
Catabasis raises $9M for PhII-ready drug development program
Prepping for a Duchenne study, Catabasis banks $12.4M in new fundraising

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