Otsuka Pharmaceutical and Ionis Pharmaceuticals said on Tuesday their experimental drug met the main goal of a late-stage study in patients with a rare inherited form of amyotrophic lateral sclerosis, or ALS.

Ionis Pharmaceuticals operates a high-margin, capital-light RNA-targeting platform, with gross margins consistently above 96% and a 99.0% gross margin in 2026Q2. However, the company's financial profile is dominated by extreme revenue volatility—quarterly reve...
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Revenue is highly episodic, with a 40.7% year-over-year decline in 2026Q2 to $267.9M, while gross margins remain exceptionally high at 99.0%, yet operating losses persist at -$102.3M due to R&D and SG&A costs that have outpaced revenue growth.
Ionis Pharmaceuticals specializes in discovering and developing antisense therapy, as well as RNA interference and CRISPR therapeutics, positioning it as a leader in innovative biotechnology.
The company maintains a dedicated Investor Relations website, providing detailed business information for stockholders, potential investors, and financial analysts, indicating transparency and engagement with the investment community.
Latest stock analysis, price targets, earnings estimates, and headlines for Ionis Pharmaceuticals are readily available, facilitating informed investment decisions and market interest.
Trailing total returns as of 9/24/2026, which may include dividends or other distributions. Benchmark is S&P 500 (^GSPC).
Recent results and news deserve attention only when they alter the forward view.
| Quarter | EPS (Act vs Est) | Revenue (Act vs Est) |
|---|---|---|
Q3 2026Latest Jul 29, 2026 | -$0.69+36.7% vs -$1.09 | $268M+41.7% vs $189M |
Q2 2026 Apr 29, 2026 | -$0.56+34.1% vs -$0.85 | $246M+25.8% vs $196M |
Q1 2026 Feb 25, 2026 | -$1.41-14.0% vs -$1.24 | $203M+30.1% vs $156M |
Q4 2025 Oct 29, 2025 | -$0.61+47.0% vs -$1.15 | $157M+2.1% vs $154M |
Otsuka Pharmaceutical and Ionis Pharmaceuticals said on Tuesday their experimental drug met the main goal of a late-stage study in patients with a rare inherited form of amyotrophic lateral sclerosis, or ALS.

PRINCETON, N.J. & TOKYO--(BUSINESS WIRE)--Otsuka Pharmaceutical Development & Commercialization, Inc., Otsuka Pharmaceutical Co., Ltd. (Otsuka) and Ionis Pharmaceuticals, Inc. today announced positive topline results from the Phase 3 FUSION trial evaluating ulefnersen, an investigational RNA-targeted medicine, in people living with a genetic subtype of amyotrophic lateral sclerosis (ALS) known as FUS-ALS, caused by mutations in the fused in sarcoma (FUS) gene1. The study met its primary end.

Ionis has six commercial therapies, mostly focused on rare diseases. Zanvastro treats Alexander disease, a brain disorder.
Ionis Pharmaceuticals NASDAQ: IONS CEO Brett Monia outlined recent regulatory wins, commercial-launch progress and several pipeline updates at the Morgan Stanley Global Healthcare Conference, while acknowledging setbacks in late-stage cardiovascular studies.
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Key metrics vs top competitors for Ionis Pharmaceuticals, Inc. (IONS)
| Company | Price | Market Cap | P/E Ratio | Rev Growth (1Y) | Net Margin | ROE | Div Yield |
|---|---|---|---|---|---|---|---|
| $43.93 | $7.3B | -18.46 | 33.87% | -40.36% | -70.72% | — | |
| $246.51 | $32.91B | 105.80 | 65.19% | 16.82% | 93.61% | — | |
| $18.95 | $2B | -2.66 | 15.58% | -6.93% | -9.96% | — | |
| $62.21 | $12.02B | 34.56 | 12.87% | 2.14% | 1.18% | — | |
| $14.88 | $1.47B | -2.55 | 20.13% | -81.79% | — | — | |
| $54.65 | $4.5B | -14.57 | — | — | -30.58% | — |
Ionis Pharmaceuticals, Inc. (IONS) vs competitors — business, growth, and fundamentals comparison against the closest industry rivals.
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Ionis Pharmaceuticals, Inc. (IONS) SEC filings — annual & quarterly reports (10-K, 10-Q)
Jul 29, 2026·SEC
Jul 10, 2026·SEC
Jul 9, 2026·SEC
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Ionis Pharmaceuticals, Inc. (IONS) stock FAQ — growth, dividends, profitability & financials explained
Ionis Pharmaceuticals, Inc. (IONS) reported $873.8M in revenue for fiscal year 2025. This represents a 3749% increase from $22.7M in 1996.
Ionis Pharmaceuticals, Inc. (IONS) grew revenue by 33.9% over the past year. This is strong growth.
Ionis Pharmaceuticals, Inc. (IONS) reported a net loss of $564.8M for fiscal year 2025.
Ionis Pharmaceuticals, Inc. (IONS) has a return on equity (ROE) of -70.7%. Negative ROE indicates the company is unprofitable.
Ionis Pharmaceuticals, Inc. (IONS) had negative free cash flow of $558.9M in fiscal year 2025, likely due to heavy capital investments.