Ionis Pharmaceuticals Stock Price, News & Analysis

Ionis (Nasdaq: IONS) partner GSK announced positive topline Phase 3 results for bepirovirsen from the B-Well 1 and B-Well 2 trials. The studies enrolled >1,800 patients across 29 countries and met the primary endpoint, with bepirovirsen plus standard of care showing a statistically significant and clinically meaningful functional cure rate versus standard of care alone.

The announcement notes an acceptable safety profile consistent with prior studies, planned global regulatory filings beginning Q1 2026, and existing Fast Track/Breakthrough/SENKU designations. Under the 2019 license, Ionis is eligible for $150M in remaining milestones and 10–12% royalties on net sales.

Ionis (NASDAQ: IONS) will present a company overview at the 44th Annual J.P. Morgan Healthcare Conference. CEO Brett P. Monia, Ph.D., will speak on January 13, 2026 at 8:15 a.m. PT. A live webcast will be available on the company Investors & Media website at https://ir.ionis.com/events, with a replay posted within 48 hours and archived for a limited time.

Ionis (Nasdaq: IONS) announced that the U.S. FDA granted Breakthrough Therapy designation to zilganersen for Alexander disease (AxD), a rare, progressive neurological disorder with no approved disease-modifying treatments.

The FDA decision is supported by pivotal study topline results: zilganersen 50 mg improved 10-Meter Walk Test gait speed by 33.3% versus control (p=0.0412) at week 61, with favorable safety and consistent secondary endpoint benefits. Ionis plans an NDA submission in Q1 2026 and is working to start a U.S. Expanded Access Program.

Ionis said this designation may expedite review and follows a recent Breakthrough Therapy designation for another program, underscoring the company’s late-stage pipeline focus.

Ionis (Nasdaq: IONS) announced that the U.S. FDA granted Breakthrough Therapy designation to olezarsen for use as an adjunct to diet to reduce triglycerides in adults with severe hypertriglyceridemia (sHTG; TG ≥500 mg/dL) on December 1, 2025.

The designation is based on Phase 3 CORE and CORE2 results showing up to 72% placebo‑adjusted triglyceride reduction, an 85% reduction in acute pancreatitis events, and nearly 90% of treated patients achieving TG <500 mg/dL. Data were published in The New England Journal of Medicine and presented at AHA. Ionis said it is on track to submit a supplemental new drug application to the FDA by the end of the year.

Ionis Pharmaceuticals (Nasdaq: IONS) and Otsuka announced the CHMP has adopted a positive opinion for DAWNZERA (donidalorsen) for routine prevention of hereditary angioedema (HAE) in adults and adolescents aged 12 and older.

The opinion is now with the European Commission, with a decision expected in Q1 2026. CHMP based its recommendation on Phase 3 OASIS-HAE and OASISplus results showing a significant, sustained reduction in mean monthly HAE attack rate, including with autoinjector self-administration. DAWNZERA received FDA approval in August 2025.

Ionis (NASDAQ: IONS) priced $700.0 million aggregate principal of 0.00% convertible senior notes due 2030, with an underwriter option for an additional $70.0 million. The offering is expected to close on November 17, 2025, with estimated net proceeds of approximately $682.8 million (or $751.2 million if the option is exercised).

Ionis expects to use about $267.6 million of net proceeds to repurchase $200.0 million aggregate principal of its 0% convertible notes due 2026 via concurrent repurchase transactions and the remainder for additional repurchases, repayment at maturity, and general corporate purposes. Initial conversion rate is 10.1932 shares per $1,000 (approx. $98.10 per share), a ~35.0% premium to the Nov 12, 2025 share price. Notes mature Dec 1, 2030 and are non‑interest bearing.

Ionis (Nasdaq: IONS) announced management will participate in investor fireside chats at two upcoming conferences: the Stifel 2025 Healthcare Conference on Thursday, November 13, 2025 and the Jefferies Global Healthcare Conference on Tuesday, November 18, 2025.

A live webcast of both presentations will be available on the company's Investors & Media website and replays will be posted within 48 hours; replays will be archived for a limited time.

Ionis (NASDAQ: IONS) announced on November 11, 2025 a proposed private placement of $700.0 million aggregate principal amount of Convertible Senior Notes due 2030, with an initial purchaser option for up to an additional $70.0 million. The notes will be unsecured, pay interest semiannually, and on conversion may be settled in cash, shares, or a combination at Ionis’ election. The offering is to qualified institutional buyers under Rule 144A.

Use of proceeds: Ionis expects net proceeds to be used to repurchase or repay its 0% Convertible Senior Notes due 2026, including possible concurrent negotiated repurchases, with any remainder for general corporate purposes. Pricing, interest rate, and conversion rate will be determined at pricing.

Ionis (Nasdaq: IONS) reported positive pivotal Phase 3 CORE and CORE2 results for olezarsen in severe hypertriglyceridemia (sHTG) on November 8, 2025. In nearly 1,100 patients, olezarsen achieved up to a 72% placebo-adjusted mean reduction in fasting triglycerides at six months with sustained reductions to 12 months.

Key outcomes: 86% of treated patients reached TGs <500 mg/dL; adjudicated acute pancreatitis events fell by 85% (p<0.001); NNT=20 overall, NNT=4 in highest-risk subgroup. Data were presented at AHA and published in NEJM. Ionis plans an FDA supplemental NDA submission by year-end and will host a webcast Nov 8 at 3:00 p.m. ET.