Ionis Pharmaceuticals Inc IONS
Ionis Pharmaceuticals Inc (Nasdaq: IONS) is a cardio-renal-metabolic drug developer. Its lead program, TRYNGOLZA (olezarsen), is in Phase 3 for severe hypertriglyceridemia (sHTG) and familial chylomicronemia syndrome (FCS).
Upcoming catalysts
No catalyst is dated to the next 30 days or to an FDA action date.
Pipeline
| Program | Indication | Modality | Stage |
|---|---|---|---|
| TRYNGOLZA (olezarsen) | FCS; sHTG (FDA approved June 2026) | RNA (siRNA/ASO/mRNA) | Approved |
| DAWNZERA (donidalorsen) | hereditary angioedema prophylaxis | RNA (siRNA/ASO/mRNA) | Approved |
| WAINUA (eplontersen, with AstraZeneca) | ATTRv-PN | RNA (siRNA/ASO/mRNA) | Approved |
| zilganersen | Alexander disease (NDA under priority review) | RNA (siRNA/ASO/mRNA) | Phase 3 |
| obudanersen | Angelman syndrome (Phase 3 REVEAL fully enrolled) | RNA (siRNA/ASO/mRNA) | Phase 3 |
| bepirovirsen (GSK) | chronic hepatitis B | RNA (siRNA/ASO/mRNA) | Phase 3 |
| eplontersen (ATTR-CM, with AstraZeneca) | ATTR cardiomyopathy | RNA (siRNA/ASO/mRNA) | Phase 3 |
| pelacarsen (Novartis) | Lp(a)-driven cardiovascular disease | RNA (siRNA/ASO/mRNA) | Phase 3 |
| salanersen (Biogen) | spinal muscular atrophy | RNA (siRNA/ASO/mRNA) | Phase 3 |
| sapablursen (Ono) | polycythemia vera | RNA (siRNA/ASO/mRNA) | Phase 3 |
| sefaxersen (Roche) | IgA nephropathy | RNA (siRNA/ASO/mRNA) | Phase 3 |
| ulefnersen (Otsuka) | FUS-ALS | RNA (siRNA/ASO/mRNA) | Phase 3 |
Recent results and decisions
- Oct 23, 2026met Sefaxersen achieved statistically significant 43.1% reduction in proteinuria at 37 weeks in Phase 3 IMAgINATION study in IgA nephropathy.
Investigational sefaxersen achieved a statistically significant and clinically meaningful 43.1% reduction in proteinuria (95% confidence interval [CI]: 32.5 - 52.0, P<0.0001), relative to placebo at 37 weeks, as measured by 24-hour urine protein-to-creatinine ratio (UPCR).
Filing - Oct 20, 2026approval FDA approves Hibsago (bepirovirsen) for chronic hepatitis B treatment in combination with nucleos(t)ide analogue therapy in adult patients without cirrhosis.
The U.S. Food and Drug Administration (FDA) has approved Hibsago (bepirovirsen) for the treatment of chronic hepatitis B (CHB) virus infection in combination with nucleos(t)ide analogue reverse transcriptase inhibitor (NrtI) therapy.
Filing - Oct 20, 2026approval FDA approves Hibsago (bepirovirsen) for treatment of chronic hepatitis B in combination with NrtI therapy in adult patients without cirrhosis.
The U.S. Food and Drug Administration (FDA) has approved Hibsago (bepirovirsen) for the treatment of chronic hepatitis B (CHB) virus infection in combination with nucleos(t)ide analogue reverse transcriptase inhibitor (NrtI) therapy in adult patients without cirrhosis.
Filing
18 more on record.
For subscribers
- Cash runway against each catalyst, with the company's own runway statement
- Track record: every Phase 3 readout, FDA decision and guidance slip since 2014
- Expected move for each dated catalyst, from comparable past events
- Phase 2 evidence behind each Phase 3 readout
- Companies on the same target or class
- Full catalyst list with filing sentences, deals, registered trials, Excel export
Other phase 3 cardio-renal-metabolic companies
The free Monday brief
Last week's readouts and FDA decisions and the dated catalysts in the week ahead, each with its filing sentence.