A Genfit drug failed in the key study of fatty liver NASH, but still has the potential to treat rare liver diseases and will now make progress in the hands of Ipsen.That company has agreed 120 million euros paid Obtained the global rights to develop and commercialize Genfit’s drug elafibranor in advance.
Elafibranor is currently in Phase 3 testing for primary biliary cholangitis (PBC), a chronic autoimmune disease that can cause inflammation and scarring of the bile ducts of the liver. This rare disease causes the accumulation of bile and other toxins, which can lead to liver damage and eventually liver failure. In severe cases, the patient needs an organ transplant. PBC mainly affects women.
A few drugs are used to treat PBC, but many patients either do not respond to these drugs or cannot tolerate them. Elafibranor is designed to bind 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, Genfit drugs are designed to stimulate anti-inflammatory activity.
Non-alcoholic steatohepatitis or NASH is elafibrinor’s first target, and Genfit advances the drug to phase 3 testing.Last year, a Temporary review of the data found no statistically significant differences Between the treatment group and the placebo group. The failure caused the company to stop developing the drug in NASH. The reorganization splits Genfit into two divisions, one of which continues to develop elafibranor at PBC.
The Phase 3 test of elafibranor is evaluating the drug in 150 PBC patients. These participants were either intolerant to ursodeoxycholic acid, a bile acid currently used to treat PBC, or intolerant. Genfit will continue to be responsible for the drug until the double-blind portion of the ongoing phase 3 clinical trial is completed. The preliminary data of the study is expected to be released in early 2023. According to elafibranor’s progress, Ipsen may pay Genfit a milestone payment of up to 360 million euros, and if the drug enters the market, it will also pay sales royalties. Ipsen also made a 28 million euro equity investment in Genfit, accounting for approximately 8% of the company.
“We are excited about elafibranor’s data package, which demonstrates the potential benefits of this top-notch innovative treatment to help the PBC community,” Ipsen CEO David Loew said in the transaction announcement. “We look forward to the ongoing Phase 3 plan and the results submitted by global regulatory agencies to bring this potential new treatment option to patients.”
The drugs used to treat PBC include Intercept Pharmaceuticals’ Ocaliva; last year, the FDA rejected the company’s application for NASH as a new indication for the drug. Others are seeking to develop new PBC drugs. Similar to Genfit, CymaBay Therapeutics stopped the development of its drug seladelpar in NASH, Shift the focus to the People’s Bank of China. Last month, Ironwood Pharmaceuticals agreed to pay COUR Pharmaceuticals an up-front and near-term milestone fee of US$20 million for Options Granted US rights to the company’s PBC drug candidates prepared in the first phase.
For Ipsen, obtaining the rights to elafibrinor provided the Paris-based company with a late-stage asset that helped alleviate the frustration of palovarotene, a drug used to treat the rare bone disease progressive fibrotic dysplasia. In August, the company Withdraw its application Seeking FDA approval after discussions with regulatory agencies indicates the need for more analysis of the data. The company said it plans to resubmit the application after completing these analyses.
At the same time, a separate deal is bringing a new drug candidate to Genfit’s pipeline.Company owned Licensed Rights to Genoscience Pharma’s drug GNS561, which is in the first phase of testing for cholangiocarcinoma, a rare cholangiocarcinoma with few treatment options. The advance payment was not disclosed, but that it had agreed to pay an unspecified amount to Genoscience to achieve clinical and regulatory milestones. If the drug enters the market, sales royalties will also be paid. Genfit also agreed to make a 3 million euro equity investment in Genoscience Pharma.
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