Ionis Pharmaceuticals stock surged into a buy zone Monday after the company unveiled promising results for a drug that treats a rare genetic condition.
The biotech company is hoping to treat Angelman syndrome, a nervous system condition that leads to severe physical and learning disabilities. After three doses and six months, 97% of patients had a clinically meaningful improvement on a scale of Angelman symptoms.
Notably, none of the patients experienced weakness in their legs — a side effect Ultragenyx Pharmaceutical reported from its Angelman study four years ago. The firm retooled its dosing and received the Food and Drug Administration's go-ahead to restart testing in 2021.
On the stock market today, Ionis stock jumped 5.8% to 50.61. That put shares back in the 5% chase zone. Ionis Pharmaceuticals stock broke out of a cup-with-handle base with a buy point at 48.61 on July 11, MarketSurge shows. Shares quickly fell below that entry, however.
Ultragenyx stock also rose 2.2% to 43.47.
Ionis Pharmaceuticals Narrowly Trails Ultragenyx
Ionis is now planning to meet with the FDA to discuss the design for a Phase 3 study. The company hopes to begin testing in the first half of 2025.
"This would place Ionis not far behind peer Ultragenyx, which intends to initiate its own Phase 3 study for GTX-102, another (drug with the same mechanism), by year-end following a meeting with the FDA," William Blair analyst Myles Minter said in a report.
He has an outperform rating on Ionis Pharmaceuticals shares.
Ionis tested its Angelman treatment in 51 patients. Patients received three doses of the drug, called ION582, over three months. Researchers evaluated the patients after six months. Patients showed improvements in cognition, communication and motor function compared with the natural course of Angelman syndrome, Ionis said in a news release.
But RBC Capital Markets analyst Luca Issi noted Biogen decided not to opt in to Ionis' program.
"Overall, we are encouraged by the data (and we appreciate a devastating disease where most patients cognitively plateau at age 2), but we ultimately think the program remains high-risk/high-reward and can see why Biogen decided not to opt-in," he said in a report.
He reiterated his outperform rating and 70 price target on Ionis Pharmaceuticals stock.
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