Enanta plots a future in NASH, chasing another blockbuster

SAN FRANCISCO--Enanta Pharmaceuticals ($ENTA), perhaps best known as AbbVie's ($ABBV) partner in hepatitis C, has blueprinted its next big solo step, joining the race to develop treatments for a pervasive liver disease with blockbuster potential.

The Watertown, MA, biotech is diving into nonalcoholic steatohepatitis (NASH), a liver-scarring disease that affects as many as 5% of Americans but has no approved therapies, and its commercial prospects have spurred a frenzy of activity in R&D around the industry.

Enanta CEO Jay Luly

Enanta is in the early stages of development with its NASH program, CEO Jay Luly announced at the JP Morgan Healthcare Conference in San Francisco. The company's approach works by activating the body's farnesoid X receptors (FXRs), which regulate bile and lipids in the liver. Targeting FXR could help prevent the accumulation of fat and occurrence of inflammation, preventing the fibrosis that marks NASH and, secondarily, the orphan ailment primary biliary cirrhosis.

The biotech is now in the lead-optimization phase, Luly said, and hopes to pick a NASH candidate for preclinical studies later this year.

Enanta bills its move into NASH as a natural outgrowth of its core competency in hep C. The liver disease leads to fibrosis, cirrhosis and hepatic failure--all hallmarks of the virus Enanta has spent the last 10 years dissecting and attacking.

"It's the same pathophysiology as HCV," Luly said. "It just happens to be caused by a fatty liver instead of a virus."

And, as Luly points out, Enanta has a healthy cash flow uncommon for a biotech at its stage. Thanks to the FDA's December approval of the AbbVie-partnered Viekira Pak, the company picked up a $75 million milestone payment, bringing its total hep C haul to $227 million so far. Enanta is in line for another $50 million payout if and when the combination treatment wins European approval, and the biotech is due a double-digit royalty on global sales.

And NASH looks like it could create a similar commercial opportunity, Luly said, an opinion shared by more and more drug developers.

Gilead Sciences ($GILD) splashed into the field this month with a $470 million deal with Phenex Pharmaceuticals that gives it a Phase II treatment for the disease. Leading the way in NASH is Intercept Pharmaceuticals ($ICPT), whose obeticholic acid has charted stellar efficacy and some troubling side effects in its ongoing Phase II program. Shire ($SHPG) is also toiling in the field after buying Lumena Pharmaceuticals for up to $260 million and getting its hands on a Phase I NASH treatment, trailed by La Jolla Pharmaceuticals ($LJPC), Raptor Pharmaceuticals ($RPTP) and others.

Enanta's embrace of NASH won't distract from its ongoing work in hep C, the company said, which includes a next-generation, AbbVie-partnered cocktail treatment on track to win approval in 2017. The biotech also has three wholly owned hep C treatments in Phase I and preclinical development.

- listen to Enanta's presentation

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