Ionis Pharmaceut

At the Wells Fargo Healthcare Conference Ionis highlighted commercial progress: two in-house FDA approvals in 2024 (TRYNGALZA, ZANVASTRO) and an upcoming GSK approval for chronic hepatitis B. The TRYNGALZA launch is outperforming expectations with strong prescription momentum, payer acceptance and a U.S. revenue potential of over $3 billion. At the same time, Pelacarsen (Lp(a)) and Eplontersen (ATTR cardiomyopathy) missed primary endpoints, increasing pipeline risk; however, Eplontersen monotherapy subgroups still show positive signals. Ionis is advancing next‑gen candidates such as ION775 and remains committed to long‑term revenue targets for TRYNGALZA and DAWNZERA. » More on de.investing.com

Ionis Pharmaceuticals' stock fell about 13% premarket after the Phase‑3 Lp(a)HORIZON trial of Pelacarsen, developed with Novartis, failed. Although Pelacarsen lowered Lipoprotein(a) levels, it did not significantly reduce the combined risk of cardiovascular death, heart attack, stroke or urgent revascularization. As a result, expected future licensing and milestone payments to Ionis will likely not materialize and the stock is approaching its 52‑week low; analysts say the downside is partly limited by the rest of the pipeline and recent approvals. » More on de.investing.com

The FDA approved Zanvastro 19 days ahead of the PDUFA date, opening an unserved market for a rare neurological disease. H.C. Wainwright reiterates a buy rating with a $115 price target, implying roughly 98% upside. The potential sale of a Priority Review Voucher for about $200 million would strengthen the financial base for further development. » More on finanzen.net