Ipsen and Genfit Enter Global Partnership in Rare Liver Diseases, Including Primary Biliary Cholangitis

Ipsen and Genfit have entered a long-term strategic partnership in which Genfit has given Ipsen exclusive worldwide license to develop, manufacture, and commercialize its experimental late-stage treatment elafibranor, for people living with primary biliary cholangitis.

Photo: Pascal Prigent, CEO of Genfit

The partnership also gives Ipsen access to future clinical programs led by Genfit and combines Genfit’s scientific expertise and proprietary technologies in liver disease with Ipsen’s development and commercialization capabilities. To underscore the long-term commitment represented by this partnership, Ipsen will make a $32 million (€28 million) equity investment in Genfit, becoming one of the largest shareholders.

Under the terms of their agreement, Ipsen will pay Genfit up to $543 million (€480 million), comprising an upfront cash payment of $136 million (€120 million), as well as regulatory, commercial, and sales-based milestone payments up to $407 million (€360 million), plus tiered double-digit royalties of up to 20 percent. Ipsen also becomes a shareholder of Genfit through the purchase of 3,985,239 newly issued shares representing 8 percent of the company after issuance. The new shares will be issued pursuant to Genfit’s June 2021 shareholders’ meeting and will be subject, upon issuance, to a lock-up period ending, in the event of positive phase 3 results, on the earlier of the date on which the EMA makes a formal recommendation to the European Commission for the marketing authorization of elafibranor in PBC or the date on which the U.S. FDA grants approval of elafibranor in PBC. In addition, the Board of Directors of Genfit will propose at the next shareholders’ meeting that Ipsen becomes a board member.

Genfit remains responsible for the phase 3 trial until the completion of the double-blind period. Ipsen will assume responsibility for all additional clinical development, including completion of the long-term extension period of the trial, and global commercialization. This newly established strategic partnership will also provide Ipsen with access to Genfit’s research capabilities and other clinical programs through rights to first negotiation.

Primary biliary cholangitis (PBC) is a rare, progressive, chronic autoimmune disease of the liver. Bile is a liquid produced inside the liver that is used to help digest fats and remove waste products from the body. PBC leads to a slow, progressive destruction of the small bile ducts of the liver, causing bile and other toxins to build up in the liver (known as cholestasis). Further damage can lead to scarring, fibrosis and eventually cirrhosis of the liver. Common symptoms of PBC include fatigue and pruritus (itching), which can be debilitating, and in more advanced cases, jaundice. Untreated, PBC can lead to liver failure, or in some cases death. PBC is more common in women with nine women diagnosed for every man; it is also a leading cause of liver transplantation.

The ongoing pivotal phase 3 global trial is evaluating the safety and efficacy of elafibranor, a dual agonist of PPAR-alpha and PPAR-delta, in 150 people living with PBC who have an inadequate response or intolerance to ursodeoxycholic acid (UDCA). Global recruitment is well underway. There is significant unmet medical need for people with PBC and, following positive Phase II data, elafibranor was granted Orphan and Breakthrough Therapy designations by the U.S. Food and Drug Administration and Orphan Drug designation by the European Medicines Agency.  Results from the Phase 2 randomized double-blind, placebo-controlled trial found that after 12 weeks of dosing with elafibranor, patients with PBC unresponsive to UDCA experienced significantly reduced levels of disease-activity markers including alkaline phosphatase (ALP) and composite endpoints with bilirubin as well as other markers of disease activity when compared to placebo.

“Today’s landmark agreement demonstrates our ability to advance highly promising assets into late-stage development in-house and derive significant value,” said Pascal Prigent, CEO of Genfit. “While we hope, above all, that this partnership with Ipsen will be a significant step towards having a positive impact on the lives of millions of patients suffering from life-threatening liver diseases, we also believe our shareholders will recognize the benefit offered by this collaboration model. The transaction proceeds indeed reinforce Genfit’s long-term financial visibility, including further funding to expand its pipeline, and opportunities for targeted business development.”

Author: Rare Daily Staff