Ionis Pharmaceuticals (NASDAQ: IONS) CEO Brett Monia participated in a fireside chat Thursday September 10, 2026 at the Wells Fargo 21st Annual Healthcare Conference in Boston, disclosing TRYNGALZA (olezarsen, an antisense oligonucleotide that reduces apolipoprotein C-III production and lowers triglycerides) H1 2026 U.S. net product sales of $32 million (Q2 alone was $5 million) with full-year 2026 guidance of $100-110 million. The severe hypertriglyceridemia (sHTG) launch followed June 2026 FDA approval and represents the first indication expansion from the initial familial chylomicronemia syndrome (FCS) indication. Ionis raised peak sales guidance in the sHTG indication from $1 billion-plus to $2 billion-plus. The sHTG marketing application is under review in the European Union with potential launch in 2027. Ionis is also commercializing the September 3, 2026 FDA-approved ZANVASTRO (zilganersen, an antisense oligonucleotide) for Alexander disease, a first-in-class disease-modifying therapy for a rare progressive neurodegenerative disorder with no other approved therapies. The company acknowledged parallel setbacks in cardiovascular studies including the Novartis pelacarsen Phase 3 Lp(a)HORIZON miss reported September 4.
Arrowhead Pharmaceuticals (NASDAQ: ARWR) presented Sunday August 30, 2026 the full Phase 3 SHASTA-3 and SHASTA-4 12-month data of plozasiran (an RNAi therapeutic targeting APOC3, administered as quarterly subcutaneous injection) in adults with severe hypertriglyceridemia at the European Society of Cardiology Congress 2026 in Munich, Germany, in a Hot Line Late-Breaking Science session. SHASTA-3 and SHASTA-4 met their primary and all prespecified secondary endpoints with median triglyceride reductions from baseline of 79% and 81%, respectively, at Month 12 (p<0.0001 in both studies). More than 90% of plozasiran-treated patients achieved triglycerides below 500 mg/dL at Month 12, and more than half achieved TG below 150 mg/dL. In a prespecified pooled analysis, plozasiran significantly reduced acute pancreatitis events across the broad sHTG study population, with greater absolute benefit among higher-AP-risk patients. Three fatal events in the plozasiran arm (two cardiovascular deaths, one CMML death) were assessed as unrelated to study treatment. Arrowhead plans to file a supplemental NDA before end of 2026 for the broader sHTG population, building on the earlier familial chylomicronemia syndrome approval.
Arrowhead Pharmaceuticals (NASDAQ: ARWR) disclosed the full terms of its Priority Review Voucher (PRV) acquisition first mentioned on the August 4, 2026 fiscal Q2 2026 conference call. Terms: $215 million paid to an undisclosed seller under an asset purchase agreement expected to close in fiscal Q4 2026. Applied to: the plozasiran (Redemplo) supplemental new drug application (sNDA) for severe hypertriglyceridemia (sHTG), planned for submission before end of 2026 following the July 23, 2026 Phase 3 SHASTA-3 and SHASTA-4 positive readouts (79% SHASTA-3 and 81% SHASTA-4 median triglyceride reductions at Month 12 versus approximately 27% for placebo, plus statistically significant reductions in acute pancreatitis events). Return projection: Arrowhead management projects a 3x return on the $215 million PRV investment by shifting the plozasiran sHTG commercial uptake curve forward by approximately four months (the PRV compresses FDA new drug application review from the standard 10-month timeline to a 6-month priority review timeline). PRVs are transferable FDA-issued regulatory instruments awarded to sponsors that develop drugs for rare pediatric diseases, tropical diseases, or specific medical countermeasures; recent secondary-market transactions have priced PRVs in the $100-250 million range depending on demand and pipeline urgency. Redemplo (plozasiran) was FDA-approved November 2025 for familial chylomicronemia syndrome; the sHTG indication would substantially expand the addressable patient population.
Arrowhead Pharmaceuticals (NASDAQ: ARWR) confirmed on the Fiscal Q2 2026 conference call Tuesday August 4, 2026 that the company has acquired a Priority Review Voucher (PRV), a transferable FDA-issued voucher that provides the option to shorten a future FDA new drug application review from the standard 10-month timeline to a 6-month timeline. PRVs are issued to sponsors that develop drugs for rare pediatric diseases, tropical diseases, or specific medical countermeasures under FDA statutory authority; they are transferable and can be sold on the secondary market where recent transactions have priced PRVs in the $100-200 million range. Arrowhead did not disclose the specific candidate to which the PRV will be applied but the timing (following the July 23, 2026 Phase 3 SHASTA-3 and SHASTA-4 positive readouts for plozasiran/Redemplo in severe hypertriglyceridemia) suggests the voucher may support the sNDA filing planned before end of 2026 for the severe hypertriglyceridemia indication. Separately, REDEMPLO (plozasiran) prescription volume has approximately doubled over fiscal Q3 following the November 2025 FDA approval for familial chylomicronemia syndrome, with continued momentum into the current quarter. Arrowhead management expressed continued satisfaction with the launch trajectory approximately 8.5 months after initial approval.
Arrowhead Pharmaceuticals (NASDAQ: ARWR) reported positive Phase 3 topline results Thursday July 23, 2026 from the SHASTA-3 and SHASTA-4 studies of plozasiran, an ApoC-III-targeting siRNA administered as a 25 mg subcutaneous injection once every three months, in adults with severe hypertriglyceridemia (sHTG). Both trials met their primary endpoint: median triglyceride reductions of 79% (SHASTA-3) and 81% (SHASTA-4) at Month 12 versus approximately 27% for placebo. All prespecified secondary endpoints were met, including a statistically significant reduction in the rate of acute pancreatitis events compared with placebo. Arrowhead shares rose approximately 19% on the readout. The company plans to file a supplemental New Drug Application (sNDA) with the US FDA before the end of 2026 for the sHTG indication (which extends the current Redemplo label from familial chylomicronemia syndrome), and Arrowhead intends to use the SHASTA-3, SHASTA-4, and MUIR-3 program data for marketing authorization filings across multiple global geographies. Plozasiran is a nucleic-acid therapeutic (siRNA), an adjacent-modality to peptides that operates through RNA interference at the ApoC-III gene expression level to lower circulating triglycerides.