Ionis posts Q2 2026 results, flags second-half pipeline milestones

Ionis Pharmaceuticals reported second quarter 2026 results on July 29, 2026, and said it expects multiple important milestones in the second half of the year, including approval of zilganersen for Alexander disease, which would mark its first independent launch from its neurology portfolio¹. The company also said it anticipates results from the Phase 3 pelacarsen Lp(a) cardiovascular outcomes trial known as HORIZON, along with a global launch of bepirovirsen for chronic hepatitis B¹.

On the Angelman syndrome program, Ionis said it completed enrollment in the Phase 3 REVEAL study of obudanersen, with data anticipated in the second half of 2027¹. Zilganersen's New Drug Application is under FDA Priority Review with a PDUFA target action date of September 22, 2026, and the company has a license agreement with Recordati to develop and commercialize the drug outside the U.S.¹ Bepirovirsen is under regulatory review in the U.S., EU, Japan, and China, with U.S. Priority Review and a PDUFA date of October 26, 2026, following positive Phase 3 B-Well data on functional cure rates presented at the EASL Congress 2026¹.

On the partnered eplontersen program, Ionis disclosed that the Phase 3 CARDIO-TTRansform study in transthyretin-mediated amyloid cardiomyopathy missed its primary composite endpoint in the overall population, though it showed nominal significance in the pre-specified monotherapy population and a favorable safety profile¹, with full results to be presented at the ESC Congress in August 2026.

Ionis said its cash position at quarter end was $2.1 billion, down from $2.7 billion at the end of 2025, mainly reflecting repayment of the 0% convertible notes on April 1, 2026¹. CFO Elizabeth Hougen said the company remains on track to achieve its 2026 financial guidance and its goal of cashflow breakeven in 2028¹, despite the CARDIO-TTRansform outcome.