By Bill Alpert

A triglyceride-cutting drug from Ionis Pharmaceuticals proved so effective in a trial reported this past weekend that the biotech's shares jumped more than 30%.

Other news at the European Society of Cardiology Congress featured an AstraZeneca pill that the company thinks will become the best treatment for hard-to-control high blood pressure. Merck offered a hint about a new cholesterol treatment, saying the long-awaited pill for lowering cholesterol achieved "meaningful" reductions in a Phase 3 trial.

Although those updates didn't noticeably move the big drug stocks, promising research results have been enough to reward investors in smaller pharma companies in the past. Shareholders scored, for example, when Eli Lilly agreed to pay up to $1.3 billion for Verve Therapeutics, a gene editor whose once-and-done treatment cut cholesterol 60% in an early trial.

Now, a bounty of new drugs are arriving through clinical trials that treat causes of heart disease and stroke that aren't tamed by statin drugs.

Among the hard-to-treat troublemakers are a kind of fatty molecule called triglycerides. High levels of triglycerides damages arteries, and very high levels will hospitalize patients with a painfully inflamed pancreas.

Saturday, Ionis reported that monthly injections of its drug Tryngolza cut levels of triglycerides by about 70% in six months, while reducing pancreatitis attacks by 85% in a year. That makes it the first drug proven to significantly stem pancreatitis.

"This is an absolute best-case scenario for Ionis," wrote RBC Capital Markets analyst Luca Issi in a Tuesday note. Investors seemed to agree, lifting Ionis stock 35%, to $57.49, as of the close on Tuesday.

Ionis already markets the drug for the few thousand patients with an inherited form of high triglycerides. On a conference call Tuesday, it said it hopes to file an application by year end to sell it to the more than two million patients with severely high levels of triglycerides. A launch in the second half of 2026 will target about 1.2 million of those patients, the people at the highest risk.

William Blair's Myles Minter said that approval could open a $1.7 billion market for Ionis if the company reaches just 15% of those patients. The triglyceride treatment will be the third product launched by Ionis without a big pharma partner, strengthening his conviction in his Buy rating.

High blood pressure is one of the most common contributors to heart attacks and strokes, but today's medicines fail to control it in some 20 million Americans and another 75 million in other affluent countries.

Saturday, AstraZeneca reported Phase 3 results for baxdrostat, a pill that it thinks will become the leading drug for hard-to-treat blood pressure. Despite taking two or three drugs, the study's patients began with top blood pressure numbers that exceeded 140, compared with the recommended level of below 120.

But after three months on the AstraZeneca drug, that number dropped by about nine points. A New England Journal of Medicine article noted that reductions of five to 10 points are enough to reduce risks of heart disease and death.

Talking to analysts on Sunday, AstraZeneca executives said they hoped to top $5 billion in sales for the drug, if it is approved and launched in 2026. It could be the first to market in a class of drugs that inhibit the body's synthesis of aldosterone, a molecule that plays a big role in hard-to-control blood pressure. A rival drug from the small biotech firm Mineralys Therapeutics showed comparable results in a smaller trial, but AstraZeneca said its pill could prove longer-lasting.

The news didn't move AstraZeneca stock. But Seamus Fernandez of Guggenheim Partners took the results as validating his Buy rating.

The European conference also brought news on one of the most anticipated new heart drugs, but that news was rather skimpy. On Tuesday, Merck announced that its investigational drug enlicitide had hit all endpoints in a large Phase 3 study among patients whose high cholesterol resisted treatment with statins. The drug could be the first pill form to reach market, in a class of drugs that inhibit PCSK9, a key driver of the bad cholesterol that causes artery disease.

There were no numbers in the announcement, however. Merck said reduction in so-called LDL cholesterol after six months was "statistically significant and clinically meaningful." Levels of other harmful fatty molecules fell, too.

In June, Merck reported success in two other Phase 3 studies: one, among patients with inherited high levels of cholesterol, and a second, comparing the pill to other cholesterol treatments. Detailed data on these pill studies will come out at future conferences, said the company.

Write to Bill Alpert at william.alpert@barrons.com

This content was created by Barron's, which is operated by Dow Jones & Co. Barron's is published independently from Dow Jones Newswires and The Wall Street Journal.

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September 02, 2025 16:48 ET (20:48 GMT)

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