Odevixibat

GSK plc (LSE/NYSE: GSK), headquartered in London, and Bologna-based Alfasigma S.p.A. have entered into a licence agreement under which Alfasigma will acquire worldwide exclusive rights to develop, manufacture, and commercialise linerixibat, an oral ileal bile acid transporter (IBAT) inhibitor being developed for cholestatic pruritus in patients with primary biliary cholangitis (PBC). Linerixibat is currently under approval review by the US FDA for cholestatic pruritus in primary biliary cholangitis (PBC), with a PDUFA target date of 24 March 2026, while filings have also been made in all major markets. GSK will receive an upfront payment of USD 300 million, with additional regulatory and sales-based milestones bringing the total potential value to approximately USD 690 million, excluding royalties. The financial structure includes a USD 100 million milestone tied to US FDA approval, which GSK expects prior to transaction closing based on a PDUFA target date of 24 March, 2026. A further USD 20 million is payable upon EU and UK approval, with up to USD 270 million in sales-based milestone payments. GSK will also receive tiered double-digit royalties on worldwide net sales. The transaction is subject to customary conditions. Linerixibat (GSK2330672) is a small molecule that selectively inhibits the ileal bile acid transporter (IBAT/SLC10A2) in the gut, reducing reabsorption of bile acids into the enterohepatic circulation. In PBC, impaired bile flow leads to accumulation of bile acids systemically, which is thought to drive cholestatic pruritus — a persistent internal itch that can cause sleep disturbance, fatigue, and reduced quality of life. By lowering circulating bile acid levels, linerixibat addresses a mechanistic link between bile acid excess and pruritus severity. Marketing applications are under regulatory review in the US, EU, UK, China, and Canada, based on data from the Phase III GLISTEN trial, which met its primary and key secondary endpoints. Linerixibat holds Orphan Drug Designation in the US, EU, and Japan, and priority review status in China. For GSK, the out-licensing represents a strategic narrowing of its hepatology portfolio toward chronic hepatitis B, metabolic dysfunction-associated steatohepatitis (MASH), and alcohol-associated liver disease (ALD). For Alfasigma, the deal adds a late-stage PBC asset to a portfolio already anchored in hepatology following the company’s approximately USD 750 million acquisition of Intercept Pharmaceuticals in 2023, which brought obeticholic acid (Ocaliva), a farnesoid X receptor (FXR) agonist approved for primary biliary cholangitis treatment. The linerixibat PBC drug licence gives Alfasigma a second distinct mechanism in the same disease. The IBAT class has also seen major deal activity: Ipsen announced its acquisition of Albireo in January 2023 and completed it in March 2023 for around USD 952 million, and Gilead acquired CymaBay for USD 4.3 billion in March 2024. Other molecules targeting IBAT or cholestatic pruritus in PBC under active development include: Odevixibat (Ipsen; originally developed by Albireo) — an IBAT inhibitor approved for progressive familial intrahepatic cholestasis (PFIC) and Alagille syndrome, with additional clinical studies evaluating its use in other cholestatic liver diseases. Maralixibat (Mirum Pharmaceuticals; originally developed by Shire/Takeda) — an IBAT inhibitor approved for Alagille syndrome and PFIC, with ongoing studies in additional pediatric cholestatic disorders. Volixibat (developed by Shire, now Mirum Pharmaceuticals) — an IBAT inhibitor being evaluated in clinical studies for cholestatic pruritus, including in patients with primary biliary cholangitis and primary sclerosing cholangitis. Your email address will not be published. Required fields are marked * Comment * Name * Email * Website

iStock, tomozina Ipsen will withdraw Tazverik’s follicular lymphoma and epithelioid sarcoma indications as emerging data point to an elevated safety risk in patients undergoing treatment. Ipsen is voluntarily withdrawing the methyltransferase inhibitor Tazverik from the market after an ongoing clinical study detected secondary hematologic malignancies in patients treated with a regimen that included the drug. In the Phase 1b/3 SYMPHONY-1 trial, patients treated with Tazverik, lenalidomide and Roche’s Rituxan showed higher rates of such malignancies than those on lenalidomide plus Rituxan alone. Because of this safety signal, an independent data board found that the risks of taking Tazverik may now outweigh its benefits, according to a company release on Monday. First approved in January 2020 for epithelioid sarcoma, Tazverik is a small-molecule drug that works by blocking the methyltransferase enzyme, which in cancer has been linked to a tumor’s growth, metastasis and ability to evade the immune system. In June 2020, just months after its initial approval, Tazverik picked up another indication , this time for follicular lymphoma. SYMPHONY-1 was conducted in patients with follicular lymphoma. Monday’s withdrawal will affect both of Tazverik’s indications “effective immediately,” the biotech said in its press announcement. Ipsen is also terminating all ongoing studies of Tazverik, including SYMPHONY-1, in which the biotech has started discontinuing dosing. All patients in the trial will receive standard of care and will be followed long-term to assess safety. Christelle Huguet, the company’s head of R&D, called SYMPHONY-1’s outcome “extremely disappointing,” having painted a “safety pro is unfavorable compared to that previously observed.” Nevertheless, Tazverik’s exit from the market will not affect Ipsen’s financial guidance. In its full-year 2025 report last month, Ipsen announced that it aims to hit 13% sales growth this year, with core operating margin above 35%. To meet this target, the biotech is relying on five major milestones this year, including a potential European approval for its oral kinase inhibitor Ojemda for pediatric low-grade glioma. Other key 2026 catalysts for the Ipsen include Phase 3 readouts for Bylvay, indicated for itching in progressive familiar intrahepatic cholestasis, and Dysport, approved for frown lines. Ipsen is also looking ahead to late-stage data for the primary biliary cholangitis drug Iqirvo. Cancer continued to be Ipsen’s biggest cash-maker in 2025, with sales growing 4.1% year-on-year to hit €2.545 billion (around $2.94 billion), while its rare disease portfolio surged 102.5% to reach €384 million (roughly $445 million).