IONIS PHARMACEUTICALS INC
IONS evidence brief
What we can confirm
- BioSniper Score:
- 4.7 / 10
- Market Cap:
- $7.85B
- Lead Asset:
- dawnzera (Approved)
What it means
What to watch
Prepared research question
What matters most for IONS—catalysts, financial risk and supporting evidence?
Traceable citations · Unknowns marked
BioSniper AI · IONS
Ionis Pharmaceuticals (IONS) is a biotech company focused on RNA-targeted therapies, with a commercial portfolio including TRYNGOLZA and Dawnzera, and a late-stage pipeline spanning cardiometabolic, neurological, and renal diseases.
Key risk: Net loss persists
BioSniper Score
Based on 4 of 6 signals
Tracking 2 pipeline candidates · 38 clinical trials · 2 catalysts · 2 FDA decisions for IONIS PHARMACEUTICALS INC.
Ionis Pharmaceuticals (IONS) is a biotech company focused on RNA-targeted therapies, with a commercial portfolio including TRYNGOLZA and Dawnzera, and a late-stage pipeline spanning cardiometabolic, neurological, and renal diseases.
- Ionis reported Q3 2025 revenue of US$157 million and a net loss of US$129 million, raising full-year 2025 revenue guidance to US$875–900 million [news · 2025-10-29].
- TRYNGOLZA (olezarsen) received U.S. FDA approval in June 2026 for severe hypertriglyceridemia, and the company presented 1-year Phase 3 extension data at ESC 2026 [news · 2026-08-28].
- Partnered drug bepirovirsen (with GSK) achieved primary endpoints in two Phase 3 trials for chronic hepatitis B, triggering eligibility for up to US$150 million in additional milestone payments [news · 2026-01-08].
- Phase 3 IMAgINATION trial of sefaxersen (with Roche) showed a reduction in proteinuria in IgA nephropathy [news · 2026-09-23].
- Obudanersen for Angelman Syndrome is in Phase 3 with Orphan Drug, Breakthrough Therapy, Fast Track, and Rare Pediatric designations [pipeline].
What works
- Multiple late-stage pipeline candidates have met primary endpoints, including bepirovirsen (hepatitis B) and sefaxersen (IgA nephropathy), demonstrating clinical proof-of-concept [news · 2026-01-08][news · 2026-09-23].
- The company has two independently launched commercial products (TRYNGOLZA, Dawnzera) and a partnered drug (bepirovirsen) approaching regulatory filings, broadening revenue sources [news · 2026-02-05][news · 2026-08-28].
- Ionis raised its full-year 2025 revenue guidance to US$875–900 million, indicating improving financial performance [news · 2025-10-29].
What to weigh
- The company reported a net loss of US$129 million in Q3 2025, reflecting ongoing operational expenses and investment in R&D [news · 2025-10-29].
- Late-stage cardiovascular studies have faced setbacks, which may affect pipeline breadth [news · 2026-09-19].
- Insider share sales have occurred alongside positive news, suggesting potential dilution or insider caution [news · 2026-02-05].
From the filings
Quoted directly from source documents.
TRYNGOLZA (olezarsen) received FDA approval for severe hypertriglyceridemia in June 2026.
“Following its June U.S. FDA approval for severe hypertriglyceridemia (sHTG)”
News · August 28, 2026
Bepirovirsen achieved primary endpoints in two Phase 3 trials for chronic hepatitis B.
“GSK and Ionis Pharmaceuticals reported that their antisense drug bepirovirsen achieved primary endpoints in two Phase 3 trials for chronic hepatitis B”
News · January 8, 2026
Ionis reported Q3 2025 revenue of US$157 million and a net loss of US$129 million.
“Ionis Pharmaceuticals reported third quarter revenue of US$157 million and reduced its net loss to US$129 million”
News · October 29, 2025
Obudanersen for Angelman Syndrome has Orphan Drug, Breakthrough Therapy, Fast Track, and Rare Pediatric designations.
“Regulatory Status: Orphan Drug, Breakthrough Therapy, Fast Track, Rare Pediatric”
Pipeline
Catalyst timeline
- 2034-05-01 · Patent_Expiry · OLEZARSEN SODIUM — Patent expiry for NDA218614 (olezarsen sodium) may open the door for generic competition.
- 2035-05-01 · Patent_Expiry · DONIDALORSEN SODIUM — Patent expiry for NDA219407 (donidalorsen sodium) may affect future revenue exclusivity.
Risks & what to watch (3)›
Key risks
- Net loss persists — Q3 2025 net loss of US$129 million indicates ongoing cash burn [news · 2025-10-29].
- Cardiovascular pipeline setbacks — Late-stage cardiovascular studies have faced setbacks, potentially limiting pipeline diversification [news · 2026-09-19].
- Insider share sales — Insider share sales coincided with positive news, raising potential concerns about insider confidence [news · 2026-02-05].
What to watch
- Regulatory filing outcomes for bepirovirsen (hepatitis B) and olezarsen (sHTG) in global markets [news · 2026-01-08][news · 2026-08-28].
- Commercial uptake of TRYNGOLZA and Dawnzera following recent approvals [news · 2026-07-29][news · 2026-02-05].
- Phase 3 data for obudanersen (Angelman Syndrome) and zilganersen (Alexander disease) [pipeline].
- Milestone payments from GSK for bepirovirsen, up to US$150 million [news · 2026-01-08].
Sources: Catalysts · News · Pipeline · Generated October 9, 2026 · How we verify sources →
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Evidence & data
Score breakdownBased on 4 of 6 signals · derived from public data
Cash runway data unavailable
Financial health data unavailable
Lead: Approved
2 candidates
2 upcoming, next in ~2756d
2 buys · 24 sells (180d)
Catalysts
Pipeline
Regulatory
Financials
Institutional Holdings
Source: SEC Form 13F · as of Mar 2026Institutional holders
491
Total value held
$12.11B
| Holder | Shares | Value |
|---|---|---|
| fmr llc | 24,556,637 | $1.84B |
| capital world investors | 21,186,369 | $1.59B |
| blackrock, inc. | 10,442,476 | $784.1M |
| vanguard portfolio management llc | 7,808,902 | $586.4M |
| vanguard capital management llc | 7,118,330 | $534.5M |
| wellington management group llp | 6,391,816 | $480.0M |
| two sigma investments, lp | 5,857,629 | $439.8M |
| geode capital management, llc | 3,984,332 | $305.2M |
| state street corp | 3,995,564 | $300.0M |
| royal bank of canada | 3,801,815 | $285.5M |
Showing top 10 of 491 institutional holders of IONIS PHARMACEUTICALS INC. Open the full 13F history after sign-in.
Short Interest
as of Apr 30, 2026- % of float
- 11.0%
- Days to cover
- 6.9
- Shares short
- 13,762,159
- Daily short volume
- -
Short percent of float and days-to-cover reflect reported short positioning. Daily short volume is the exchange-reported short share of a single day's trading volume and is noisier. See the most-shorted biotech ranking →
IONS short interestGovernance
Frequently asked questions
What is the AI investment thesis for IONIS PHARMACEUTICALS INC?
Ionis Pharmaceuticals (IONS) is a biotech company focused on RNA-targeted therapies, with a commercial portfolio including TRYNGOLZA and Dawnzera, and a late-stage pipeline spanning cardiometabolic, neurological, and renal diseases.
What is IONIS PHARMACEUTICALS INC's lead drug candidate?
IONIS PHARMACEUTICALS INC's most advanced tracked candidate is dawnzera (Approved).
What is IONIS PHARMACEUTICALS INC's next catalyst?
IONIS PHARMACEUTICALS INC's next tracked catalyst is a Patent_Expiry event — "Patent_Expiry: NDA218614" — expected May 1, 2034.
Who is the CEO of IONIS PHARMACEUTICALS INC?
Dr. Brett P. Monia Ph.D. is the chief executive officer of IONIS PHARMACEUTICALS INC (IONS).
Where is IONIS PHARMACEUTICALS INC headquartered?
IONIS PHARMACEUTICALS INC (IONS) is headquartered in Carlsbad, CA, United States.
The AI overview and BioSniper Score are neutral, data-derived signals built from public SEC filings, ClinicalTrials.gov and FDA records — not investment advice.