Ionis reports positive results for olezarsen in study

CARLSBAD, Calif. - Ionis Pharmaceuticals, Inc. (NASDAQ:IONS), a $5.3 billion market cap biotechnology company, today revealed positive topline results from a study of olezarsen, a drug aimed at treating moderate hypertriglyceridemia. The Essence study showed a significant reduction in triglycerides compared to placebo at both 80 mg and 50 mg monthly doses over six months. According to InvestingPro data, seven analysts have recently revised their earnings estimates upward, suggesting growing confidence in the company’s pipeline potential.

The Phase 3 trial met its primary endpoint, achieving a 61% reduction in triglyceride levels at the 80 mg dose and a 58% reduction at the 50 mg dose, with a p-value of less than 0.0001, indicating strong statistical significance. Additionally, olezarsen met all key secondary endpoints, including the proportion of patients achieving normal triglyceride levels of less than 150 mg/dL.

According to the press release, the study enrolled 1,478 participants with moderate hypertriglyceridemia who were at risk for atherosclerotic cardiovascular disease. The participants were already on existing lipid-lowering medications before the trial. The favorable safety and tolerability profile of olezarsen was noted, with injection site reactions being the most common adverse event, mostly mild in severity. While the company maintains strong liquidity with a current ratio of 9.66 and operates with moderate debt levels, InvestingPro analysis indicates the company is not yet profitable, with an EBITDA of -$460.85 million in the last twelve months.

Ionis plans to present detailed results at an upcoming scientific conference and is looking forward to the outcomes of the pivotal Phase 3 CORE and CORE2 studies, expected in Q3 2025. These studies will examine olezarsen’s efficacy in treating severe hypertriglyceridemia and potentially support a supplemental New Drug Application filing by the year’s end.

Olezarsen, an investigational RNA-targeted medicine, is designed to reduce apoC-III protein production, which plays a key role in triglyceride metabolism. It has recently been approved in the U.S. under the brand name TRYNGOLZA for reducing triglycerides in adults with familial chylomicronemia syndrome, a rare genetic condition causing severely elevated triglycerides.

This announcement follows Ionis’ consistent efforts to innovate in RNA therapies and its commitment to addressing high patient needs in cardiology and neurology. The company emphasizes that the press release contains forward-looking statements and that actual results may vary due to various factors and uncertainties. Ionis Pharmaceuticals has been a pioneer in RNA-targeted medicines for over three decades, with a pipeline that includes six marketed medicines. Analyst targets suggest significant upside potential, with price targets ranging from $37 to $83 per share. For comprehensive analysis of Ionis’ financial health, growth prospects, and more detailed metrics, investors can access the full Pro Research Report available on InvestingPro, which provides deep-dive analysis of this and 1,400+ other US stocks.

In other recent news, Ionis Pharmaceuticals reported its first-quarter 2025 earnings, surpassing analyst expectations with an earnings per share (EPS) of -0.93, compared to the forecasted -1.08. The company recorded revenues of $132 million, slightly below the projected $136.69 million, but still reflecting a 10% year-over-year increase. Ionis has raised its 2025 revenue guidance by over 20%, projecting between $650 million and $750 million for the year. This increase is attributed to strong commercial product revenue growth, including a 28% rise in SPINRAZA royalties. Additionally, H.C. Wainwright raised its price target for Ionis to $50 from $45, maintaining a Buy rating, following the successful launch of Tryngolza for familial chylomicronemia syndrome (FCS). The launch capitalized on pent-up patient demand and transitions from an open-label extension study, with expectations of further growth as insurance coverage policies are established. The potential approval of Tryngolza for severe hypertriglyceridemia is also anticipated to boost demand. Ionis is preparing for upcoming product launches, including Donadaluirsen for Hereditary Angioedema and olazarsen for Severe Hypertriglyceridemia, which are expected to drive future growth.

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