Ionis Pharmaceuticals (IONS) investor relations material

Key business milestones and pipeline updates

• 2026 is positioned as a major year with multiple pipeline and commercial events, building on momentum from 2025’s transformation into a fully integrated commercial-stage biotech.

• Two successful product launches: TRYNGOLZA for FCS (first FDA-approved for this indication) and DAWNZERA for HAE prophylaxis, both off to strong starts.

• Anticipates two additional FDA approvals and launches in 2026, including TRYNGOLZA for severe hypertriglyceridemia (sHTG) and a neurology launch for Alexander disease.

• Five phase III readouts expected from partnered pipeline in 2026, including bepirovirsen (chronic HBV), pelacarsen (Lp(a) CVD), eplontersen (ATTR cardiomyopathy), an ALS drug, and IONIS-FB-LRx (IgA nephropathy).

• Plans to initiate at least two new phase III trials this year: salanersen for SMA and sapablursen for polycythemia.

Commercial strategy and pricing insights

• Priority review for TRYNGOLZA in sHTG with a PDUFA date of June 30, 2026, expected to positively impact revenue guidance, which will be updated at the Q1 earnings call.

• No significant impact from new competitors in the FCS space; pricing strategy aims to maximize value while ensuring broad patient access and minimizing payer resistance.

• Net price guidance for TRYNGOLZA in sHTG is trending toward the high end of the $10K–$20K range, with flexibility for negotiation and rebates.

• DAWNZERA’s launch is supported by a free drug program to facilitate rapid patient access while reimbursement is secured.

Clinical data and product performance

• TRYNGOLZA’s phase III data show over 70% triglyceride reduction and 85% reduction in acute pancreatitis attacks in sHTG, with broad label expected for patients with triglycerides above 500 mg/dL.

• Ongoing data presentations planned at major medical congresses; two-year liver fat data indicate adaptation and return to baseline, with no toxicity observed.

• DAWNZERA’s commercial uptake is primarily among switch patients from other prophylactic treatments, with most starting on every four-week dosing and some transitioning to every eight weeks.