Ionis Pharmaceuticals stock tumbled Friday after partner AstraZeneca decided against sending the companies' high cholesterol treatment into Phase 3 testing.
The decision comes despite apparent success in a Phase 2 study. In that study, a 60-milligram dose given each month lowered "bad" LDL cholesterol by 62.3% after 28 weeks compared with a placebo. But the results didn't meet AstraZeneca's pre-specified effectiveness criteria, Ionis said in a news release.
"While the LDL-C reductions seen in high-risk (high cholesterol) patients on maximum statin therapy were both statistically significant and robust, these results did not meet AstraZeneca's target product profile criteria to invest in a broad Phase 3 development program," Ionis Chief Clinical Development Officer Eugene Schneider said in a written statement.
On today's stock market, Ionis stock toppled 3.9% to 43.23. AstraZeneca stock also fell 3.1% to 54.58.
Ionis Stock: Moving Into An Under-Penetrated Market
The drug is based on Ionis' bread-and-butter technology, known as antisense.
It aims to reduce levels of a key protein called PCSK9 in the blood plasma. Further, PCSK9 plays a role in regulating LDL cholesterol. Already marketed drugs from Amgen, Novartis and Regeneron Pharmaceuticals use a different mechanism to control the same protein.
But these already marketed drugs aren't gaining as much traction as the companies had hoped. In the second quarter, sales of Amgen's Repatha grew 14%, but that came as more patients piled onto a lower net selling price. Sales of Praluent, jointly made by Regeneron and Sanofi, tumbled 26% globally. Novartis is still in the early days of its Leqvio launch.
Ionis stock investors are looking for the company to expand its portfolio of approved drugs. The company has two drugs approved in the U.S. and a third in Europe.
More Options For High Cholesterol Patients
Now, companies like Ionis and Verve Therapeutics are trying to move into the market, offering patients on maximum statins more options for lowering their cholesterol.
In the Phase 2 study, Ionis said its AstraZeneca-partnered high cholesterol drug was generally safe and tolerable. AstraZeneca is now reviewing its next steps for the program.
"AstraZeneca continues to be a valued collaborator and we look forward to working with them to advance multiple important programs," Schneider said.
The news put Ionis stock on track for its fourth straight day in the red. Still, shares have a strong Relative Strength Rating of 95, which puts them in the top 5% of all stocks in terms of 12-month performance, according to IBD Digital.
Follow Allison Gatlin on Twitter at @IBD_AGatlin.
