Riding a PhII success, a small French biotech lands a rare ‘breakthrough’ status in NASH

For all the hype NASH drugs have received, the FDA has not been quick to roll out the red carpet for the most talked-about compounds. The agency gave breakthrough designation to Intercept’s obeticholic acid in 2015 but, since then, have declined to offer that seal to other late-stage drugs, including those from Gilead or Genfit.

On Monday afternoon, though, the French biotech Inventiva announced that they had won breakthrough status, becoming — by their count — the first company to receive the designation for a NASH drug since Intercept did 5 years ago. It was enough to send the company’s stock up 6%, from $13.65 to $14.50 pre-market.

The designation comes in the wake of a series of setbacks across NASH pipelines, including the FDA’s decision earlier this year to reject Intercept’s obeticholic acid on the apparent grounds that it did not show strong enough evidence for efficacy. The surprise CRL capped a year and a half of disappointments that shifted industry focus to a host of newer, smaller biotechs and their earlier-stage compounds.

In June, Inventiva announced that its lead drug, lanifibranor, was successful in a Phase II trial. Across nearly 250 volunteers, 49% of those who received the high dose of the drug saw their liver cell inflammation and ballooning decline by at least two points on a standard scale and didn’t see their fibrosis worsen, compared to 27% on placebo. The trial also hit secondary endpoints at low and high dose for NASH resolution and improvement of fibrosis and NASH.

The readout surprised not only because of other companies’ struggles in NASH, but also because of the history of the drug itself. It had already failed the year prior in the rare fibrosis disease systemic sclerosis. And a drug with a similar mechanism at Genfit had just failed one of the largest ever NASH studies, triggering significant layoffs and prompting the fellow French biotech to exit the field entirely.

It’s difficult to discern what the difference was yet, though Inventiva notes that their drug activated all of a receptor known as PPAR, while Genfit’s compound only activated PPAR alpha and delta.

The win was enough to vault Inventiva to a $108 million IPO the following month, amid a run of pandemic biotech public offerings — a raise that valued the company at over $500 million. The company still has to meet with the FDA to discuss the Phase II results before they can launch what they want to be a pivotal Phase III trial in the first half of 2021, but the breakthrough designation has given CEO and co-founder Frédéric Cren confidence ahead of the discussions.

“Ahead of our end of Phase IIb meeting with the FDA later this year, the Breakthrough Therapy designation for lanifibranor is a fantastic achievement and a regulatory recognition of the significant clinical benefits lanifibranor could provide to NASH patients as shown in our recent NATIVE Phase IIb clinical trial,” he said.

Social: Frédéric Cren, Inventiva CEO (Inventiva via YouTube)

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