As a late-stage data showdown from NASH drug developers Gilead $GILD, Intercept $ICPT and France’s Genfit (EuroNext: $GNFT) looms, the Lille-based drug developer on Thursday reported positive phase II data on its drug elafibranor in patients with primary biliary cholangitis (PBC), a rare and progressive liver disease.
The 12-week phase II trial tested two doses of the dual PPARα/δ agonist elafibranor against a placebo in PBC patients that did not derive adequate benefit from standard treatment, ursodeoxycholic acid. Both doses of the once-daily treatment conferred a statistically significant decrease in serum alkaline phosphatase (ALP) levels. High ALP typically indicates a liver in distress.
Data showed a fall of 48% for patients who got the 80 mg dose (p<0.001), and drop of 41% in those who received the 120 mg dose (p<0.001), while those on placebo saw their ALP levels edge 3% higher. Alongside, the drug met the composite secondary endpoint — which has been previously used for regulatory approval — and induced improvement in other PBC markers, the company said, adding that its drug had been well tolerated.
That’s enough for Genfit chief Jean-François Mouney to declare the drug ready for late-stage development in PBC, noting, “the strength of evidence on the surrogate endpoint for registration as well as the potential benefits on itching qualify the program to rapidly advance.”
Velimir Luketic, a researcher from the Virginia Commonwealth University School of Medicine added: “A substantial number of patients do not benefit from the currently available therapies – UDCA or OCA (Ocaliva) – either because of lack of response or intolerable side effects. The data emerging from this clinical trial are very impressive, particularly the substantial reduction in ALP in just 12 weeks.”
Phase III data on elafibranor for NASH — in what is expected to be a far more lucrative market — is expected in 2019. The biopharma Intercept, whose NASH drug-in-development is already approved for PBC under the name Ocaliva, has brought in nearly $125 million in sales in the first nine months of this year, while CymaBay’s $CBAY PPAR agonist seladelpar entered late-stage development in October for PBC.
Last month, Genfit announced plans to list itself in the United States.
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