A Genfit drug that failed a pivotal study in the fatty liver disease NASH but still holds potential as a treatment for a rarer liver disorder will now move forward in the hands of Ipsen. That company has agreed to pay €120 million up front to acquire global rights to develop and commercialize the Genfit drug, elafibranor.
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Elafibranor is currently in Phase 3 testing in primary biliary cholangitis (PBC), a chronic autoimmune disease that leads to inflammation and scarring of the liver’s bile ducts. The rare disease causes the buildup of bile and other toxins, which leads to liver damage that can eventually progress to liver failure. In severe cases, patients require an organ transplant. PBC primarily affects women.
A handful of drugs are used to treat PBC, but many patients either don’t respond to those medicines or they can’t tolerate them. Elafibranor is designed to bind to and activate PPAR alpha and PPAR delta, two receptors that control the expression of genes involved in inflammation, lipid metabolism, glucose metabolism, insulin sensitivity, oxidative stress, and fibrosis. By activating PPAR alpha and PPAR delta, the Genfit drug is intended to spark anti-inflammatory activity.
Nonalcoholic steatohepatitis, or NASH, was the first target of elafibrinor, and Genfit advanced the drug to Phase 3 testing. Last year, an interim look at the data found no statistically significant differences between the treatment group and the placebo arm. The failure led the company to cease development of the drug in in NASH. A restructuring split Genfit into two divisions, one of which continued development of elafibranor in PBC.
The Phase 3 test of elafibranor is evaluating the drug in 150 PBC patients. Those participants are either intolerant of ursodeoxycholic acid, a bile acid currently used to treat PBC, or they cannot tolerate it. Genfit remains responsible for the drug until completion of the double-blind portion of the ongoing Phase 3 clinical trial. Preliminary data from the study are expected in early 2023. Depending on elafibranor’s progress, Ipsen could pay Genfit up to €360 million in milestone payments, plus royalties from sales if the drug reaches the market. Ipsen is also making a €28 million equity investment in Genfit, representing about 8% of the company.
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“We are excited by elafibranor’s data package, demonstrating the potential benefit of this first-in-class, innovative treatment option to help the PBC community,” Ipsen CEO David Loew said in the announcement of the deal. “We look forward to the results of the ongoing Phase 3 program and regulatory submissions around the world to bring this potential new treatment option to patients.”
Among the treatments for PBC is the Intercept Pharmaceuticals drug Ocaliva; last year, the FDA rejected that company’s application seeking to add NASH as a new indication for that drug. Others are pursuing development of new PBC drugs. Similar to Genfit, CymaBay Therapeutics stopped development of its drug seladelpar, in NASH, shifting its focus to PBC. Last month, Ironwood Pharmaceuticals agreed to pay COUR Pharmaceuticals $20 million in upfront and near-term milestones for an option to license U.S. rights to that company’s Phase 1-ready PBC drug candidate.
For Ipsen, getting elafibrinor’s rights gives the Paris-based company a late-stage asset that helps ease the setbacks to palovarotene, a drug developed to treat the rare bone disorder fibrodysplasia ossificans progressiva. In August, the company withdrew its application seeking FDA approval after discussions with the regulator revealed that more analyses of the data were needed. The company said it plans to resubmit the application after completing those analyses.
Meanwhile, a separate transaction is bringing a new drug candidate to Genfit’s pipeline. The company has licensed rights to Genoscience Pharma drug GNS561, which is in Phase 1 testing for cholangiocarcinoma, a rare bile duct cancer that has few treatment options. No upfront payment was disclosed but Genfit said it has agreed to pay Genoscience unspecified amounts for the achievement of clinical and regulatory milestones, plus royalties from sales if the drug reaches the market. Genfit also agreed to a make a €3 million equity investment in Genoscience Pharma.
Photo: Sakramir, Getty Images
