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CARLSBAD, Calif. - Ionis Pharmaceuticals (NASDAQ:IONS), whose stock has surged over 80% in the past six months and currently trades near its 52-week high of $62.08, announced Tuesday that its experimental drug ION582 has received Breakthrough Therapy designation from the U.S. Food and Drug Administration for the treatment of Angelman syndrome, a rare neurological disorder with no approved disease-modifying treatments.
The designation, which aims to expedite the review process for medicines that may offer substantial improvement over existing therapies, was based on results from the Phase 1/2 HALOS study. According to the company’s press release, the trial showed "consistent and encouraging clinical improvement" across all Angelman syndrome functional domains, including communication, cognition and motor function. The $9.72 billion market cap company appears overvalued according to InvestingPro Fair Value metrics, though it maintains strong liquidity with a current ratio of 2.87.
Angelman syndrome affects approximately 1 in 21,000 people worldwide and typically presents in infancy. The condition is characterized by profound intellectual disability, motor impairment, balance issues, and debilitating seizures. Most patients are unable to speak and require lifelong caregiver support.
"With no approved disease-modifying treatments available for people living with Angelman syndrome, receiving this Breakthrough Therapy designation for ION582 underscores both the severity of this disease and the significant unmet need for treatment," said Holly Kordasiewicz, senior vice president of neurology at Ionis.
ION582 is an antisense medicine designed to inhibit the UBE3A antisense transcript and increase production of UBE3A protein. The drug has previously received Orphan Drug designation from both U.S. and European regulators, as well as Fast Track and Rare Pediatric designations from the FDA.
Ionis has already initiated the global Phase 3 REVEAL study, which is expected to enroll children and adults with Angelman syndrome who have a maternal UBE3A gene deletion or mutation. The company reports that enrollment is on track to complete in 2026. With revenue growth of 16.05% over the last twelve months, investors can access detailed analysis and 12 additional ProTips through InvestingPro’s comprehensive research reports.
In other recent news, Ionis Pharmaceuticals reported positive topline results from its Phase 3 CORE and CORE2 trials for severe hypertriglyceridemia, which led to a significant 32% stock increase. These trials showed notable benefits in triglyceride lowering and reducing acute pancreatitis risk. Following these developments, H.C. Wainwright raised its price target for Ionis Pharmaceuticals to $95, citing strong triglyceride reductions in pivotal studies. Similarly, RBC Capital increased its price target to $80, highlighting the promising data for the company’s acute pancreatitis treatment. Oppenheimer also adjusted its price target to $81, maintaining an Outperform rating after the positive trial results for Tryngolza. BMO Capital upgraded Ionis Pharmaceuticals from Market Perform to Outperform, seeing blockbuster potential in the drug Olezarsen. This series of upgrades and positive trial outcomes reflect the growing confidence in Ionis Pharmaceuticals’ recent advancements.
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