Ionis begins phase 3 trial of Angelman syndrome treatment

CARLSBAD, Calif. - Ionis Pharmaceuticals (NASDAQ:IONS), a $5.86 billion market cap biotechnology company, announced Wednesday that the first participant has been dosed in its global Phase 3 REVEAL study evaluating ION582, an investigational treatment for Angelman syndrome, a rare neurodevelopmental disorder. According to InvestingPro data, seven analysts have recently revised their earnings expectations upward for the upcoming period, suggesting growing confidence in the company’s pipeline.

The placebo-controlled study will enroll approximately 200 children and adults with Angelman syndrome who have a maternal UBE3A gene deletion or mutation. Participants will be randomized to receive either ION582 or placebo during the 52-week treatment period, with those in active treatment groups receiving quarterly doses of 40 mg or 80 mg.

The primary endpoint is improvement in expressive communication as assessed by the Bayley Scales for Infant and Toddler Development-4. Secondary endpoints include measures of overall disease severity, cognition, communication, sleep, motor functioning and daily living skills.

"Dosing the first person with Angelman syndrome in our pivotal REVEAL study marks an important milestone for this underserved community, who currently have no approved disease modifying treatments," said Holly Kordasiewicz, senior vice president of neurology at Ionis.

Angelman syndrome affects an estimated 1 in 21,000 people worldwide and is characterized by intellectual disability, balance issues, motor impairment and seizures. Most people with the condition are unable to speak and require lifelong care.

The company expects to complete enrollment in 2026. The study follows the Phase 1/2 HALOS trial, which showed clinical improvements in communication, cognition and motor function with favorable safety and tolerability, according to the company’s press release statement. While the company maintains a strong liquidity position with a current ratio of 9.66, InvestingPro analysis indicates the stock is currently trading above its Fair Value. Investors seeking deeper insights can access comprehensive analysis of IONS and 1,400+ other stocks through InvestingPro’s detailed research reports.

In other recent news, Ionis Pharmaceuticals reported its first-quarter 2025 earnings, surpassing analyst expectations with an earnings per share of -0.93, compared to the forecasted -1.08. The company achieved revenues of $132 million, slightly below the forecast of $136.69 million, yet still marking a 10% year-over-year increase. Ionis has raised its 2025 revenue guidance by over 20%, projecting between $650 million and $750 million, bolstered by robust sales of its drug Tryngolza for familial chylomicronemia syndrome. H.C. Wainwright responded to these developments by raising its price target for Ionis shares to $50, maintaining a Buy rating, while Stifel analysts held a Hold rating with a $38 target. Additionally, Ionis announced positive Phase 3 trial results for olezarsen, showing significant triglyceride reductions, and plans to present more detailed findings at an upcoming scientific conference. The company also shared updates on its pipeline, including the expected launch of donidalorsen for Hereditary Angioedema and pivotal readouts for its LP(a) program. Ionis recently held its annual stockholders meeting, where key proposals, including director elections and amendments to its equity incentive plan, were approved.

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