Pivoting Again, Catabasis Looks to NASH After Cholesterol Drug Flops

Xconomy Boston — 

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: 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.