IONIS PHARMACEUTICALS Q2 FY26 earnings call IONS

Ionis achieved a landmark milestone with the FDA approval of TRYNGOLZA for severe hypertriglyceridemia (SHTG), making it the first and only approved medicine to reduce triglycerides and the risk of acute pancreatitis. The launch has shown encouraging early momentum, with prescriptions received on day one and both 50mg and 80mg doses available in the channel within a week. The label includes acute pancreatitis risk reduction, supported by Phase III CORE and CORE II studies showing up to 72% triglyceride reduction and 91% reduction in acute pancreatitis events. The company estimates 3 million people in the U.S. have SHTG, with 1 million at high risk, and expects payer coverage to expand through 2026 and into 2027, with an anticipated 60% commercial and 40% government payer mix.

DAWNZERA for hereditary angioedema (HAE) prophylaxis continues to gain momentum, with Q2 sales reaching $26 million, a 63% increase over Q1. In less than a year, DAWNZERA has captured a meaningful share of the U.S. HAE prophylaxis market, which is largely a switch market. Growth is driven by adoption across all patient segments, including switches from existing therapies, patients previously on on-demand treatment, and treatment-naive patients. Physicians and patients provide positive feedback on its efficacy, safety, differentiated RNA-targeted mechanism, and patient-friendly self-administered auto-injector.

Ionis is well-prepared for the upcoming launch of Zilganerse for Alexander's disease, with an FDA PDUFA date of September 22. This represents the first independent launch from their neurology pipeline and the first disease-modifying treatment for the rare condition. Following Zilganerse, Olezarsen for Angelman Syndrome completed enrollment in its Phase III REVEAL study last month, with data expected next year. The company also initiated clinical development for ION337 in Dravet syndrome, expanding its wholly-owned neurology pipeline, leveraging proprietary NMA chemistry for enhanced potency and longer dosing intervals.

The partnered pipeline continues to advance, with Bepirovirsen (chronic hepatitis B) on track for global launch this year, including a PDUFA date of October 26 in the U.S. and Japan. Key catalysts in the second half of 2026 include data from the Phase III pelacarsen LPA horizon study for Lp(a)-driven cardiovascular disease. However, the Phase III Cardio Transform study for eplontersen in ATTR cardiomyopathy did not meet its primary efficacy endpoint in the overall population, though it showed substantial TTR reductions. Biogen also advanced Zilganerse into Phase III for SMA and plans Phase III for derinersen in early Alzheimer's disease based on positive Phase II data.

Ionis delivered strong financial results in the first half of 2026, with Q2 revenues of $268 million (up 56% YoY) and H1 revenues of $540 million (up 69% YoY). Commercial revenue increased by 15% in Q2 and 27% in H1, driven primarily by DAWNZERA. The company ended Q2 with $2.1 billion in cash, cash equivalents, and short-term investments. Ionis reaffirmed its full-year 2026 revenue guidance of $875 million to $900 million and expects a non-GAAP operating loss between $425 million and $475 million, remaining on track for cash flow breakeven in 2028.

The company emphasized its strong R&D engine, commercial capabilities, and financial discipline to execute on strategic opportunities and achieve its goals. Ionis is focused on building momentum across its commercial medicines and strengthening its wholly-owned pipeline for future growth. The call also recognized Frank Bennett, Chief Scientific Officer, for his planned retirement, highlighting his foundational contributions to RNA-targeted medicines and the neurology field, including SPINRAZA and Kaldi.