Pivoting Again, Catabasis Looks to NASH After Cholesterol Drug Flops

Fibrotic Liver

Catabasis Pharmaceuticals has done its share of strategic shuffling over the past few years, and some more changes have come today.

A drug known as CAT-2054, one of two prospects Catabasis (NASDSAQ: [[ticker:CATB]]) has in clinical testing, failed a mid-stage trial in people with high cholesterol. The drug didn’t do a good enough job lowering cholesterol or triglycerides compared to a placebo, leading the Cambridge, MA-based company to say it’ll no longer advance CAT-2054 as a cholesterol treatment. The news is a setback for Catabasis, which according to a conference call last month, had been hoping to license out CAT-2054 before a Phase 3 trial.

Instead, Catabasis will pivot and look to assess the drug’s potential as a treatment for nonalcoholic steatohepatitis (NASH), an increasingly prevalent liver disease that has become the focus of a number of drugmakers. Catabasis has been evaluating CAT-2054 in NASH, and said it has seen signs of potential impact on liver function with CAT-2054 that “need further investigation.” CEO Jill Milne said in the statement that the company plans to “determine the best path forward for CAT-2054 in NASH.” The drug blocks sterol regulatory element-binding protein, a molecule involved in lipid metabolism; NASH is caused by a buildup of fat in the liver that leads to scarring and often liver failure.

Catabasis has shifted its strategy a few times. Several years ago, for instance, the company talked up another candidate, CAT-1004, as a potential treatment for type 2 diabetes before shifting to inflammatory bowel disease and Duchenne muscular dystrophy. Another drug, CAT-2003, was developed through mid-stage trials for “niche” disorders associated with high triglyceride levels, like multifactorial chylomicronemia syndrome, but then shelved in favor of a next-gen version, CAT-2054, that is now in limbo. The focus of the company is now CAT-1004, which is in a mid-stage study for Duchenne, a deadly, progressive genetic disease with no effective treatments. That trial is expected to produce data later this year.

Stockholders aren’t waiting around to see whether CAT-2054 does in fact have a future in NASH, meanwhile. Shares plummeted more than 31 percent in after-hours trading Tuesday, down to $4.50 apiece. The company went public at $12 per share in June 2015, but hasn’t closed higher than its IPO price since last August.

Here’s more on Catabasis, which was was formed in 2008 and built around a technology called  “SMART-Linker” used to attach two compounds together.

Author: Ben Fidler

Ben is former Xconomy Deputy Editor, Biotechnology. He is a seasoned business journalist that comes to Xconomy after a nine-year stint at The Deal, where he covered corporate transactions in industries ranging from biotech to auto parts and gaming. Most recently, Ben was The Deal’s senior healthcare writer, focusing on acquisitions, venture financings, IPOs, partnerships and industry trends in the pharmaceutical, biotech, diagnostics and med tech spaces. Ben wrote features on creative biotech financing models, analyses of middle market and large cap buyouts, spin-offs and restructurings, and enterprise pieces on legal issues such as pay-for-delay agreements and the Affordable Care Act. Before switching to the healthcare beat, Ben was The Deal's senior bankruptcy reporter, covering the restructurings of the Texas Rangers, Phoenix Coyotes, GM, Delphi, Trump Entertainment Resorts and Blockbuster, among others. Ben has a bachelor’s degree in English from Binghamton University.