AUBURN — Dr. Robert Weiss knew early on that the drug he was testing had promise.

The first in a new class of medication — a PCSK9 inhibitor — it was supposed to lower cholesterol. 

“The results were so dramatic, even in small numbers of patients,” said Weiss, director and CEO of Maine Research Associates in Auburn. “Lowering cholesterol 50, 60 percent was very exciting.”

Four years later, the medication, Praluent, has received FDA approval and is the first drug in its new class to hit the market. Doctors say PCSK9 inhibitors have the potential to be the biggest breakthrough in lowering cholesterol since statins were introduced in the late 1980s

And some of the drug’s testing was done — and is still being done — in Auburn with local patients.

“The effectiveness is very exciting,” Weiss said.

According to the FDA, Praluent works by targeting proprotein convertase subtilisin kexin type 9, or PCSK9, a protein that reduces the number of liver receptors that remove LDL cholesterol. Praluent blocks PCSK9’s ability to work, making more receptors available to get rid of LDL cholesterol.

LDL cholesterol is often referred to as “bad cholesterol” and has been linked to heart disease. About 610,000 Americans die of heart disease every year, according to the U.S. Centers for Disease Control and Prevention.

Statins are effective in lowering cholesterol in a different way, but some patients can’t tolerate the side effects, including muscle pain. Other patients have found that statins don’t reduce their cholesterol numbers quite enough, even at high dosages.

Researchers have been seeking new cholesterol-lowering drugs for years.

Sanofi, a pharmaceutical company based in France, and Regeneron Pharmaceuticals, in New York, began working on a PCSK9 inhibitor. The work included 14 clinical trials evaluating more than 23,000 patients.

Lewiston-Auburn can be attractive to companies looking to test cholesterol-lowering drugs because the area has high cholesterol rates and a large population with familial hypercholesterolemia, a genetic disorder that causes extremely high cholesterol levels at very young ages. 

Weiss and his Maine Research Associates got involved in Praluent’s testing four years ago. The 30-year-old Auburn-based research group began working with a small number of Androscoggin County patients as part of phase 2 trials, testing the drug’s dosage and how it should be given. Later, it worked on phase 3, testing a larger group of local patients to gauge how the drug worked in a specific form and dose. 

In all, about 60 to 70 patients were involved in the drug trials. 

Maine Research Associates, which has a relationship with Central Maine Medical Center in Lewiston, found patients by looking through their medical records. It contacted people who met the criteria, including those who had problems with cholesterol. About 10 to 15 percent agreed to participate, about average for Maine Research Associates.

“For this one, people realized their cholesterol needed to be lower and they were already on the maximum dose (of statins) they could buy or the maximum dose that they could tolerate,” Weiss said.

His trials found LDL cholesterol sometimes dropped 50 to 60 percent in people who used Praluent. On average, the drug lowered LDL cholesterol 36 to 59 percent, according to the FDA. 

The FDA approved Praluent in July. It was expected Thursday to decide the fate of a second PCSK9 inhibitor, Repatha, proposed by California-based pharmaceutical company Amgen. 

Although Praluent has caused excitement, there are drawbacks. It is expensive, with a wholesale cost of $40 a day or about $1,200 a month. And patients must inject themselves with the drug every two weeks.

Stains, in comparison, cost a fraction of that and come in pill form.

It’s unclear how much of a problem the cost and injection will be for patients. A spokesperson for Sanofi and Regeneron Pharmaceuticals said some insurers are covering the cost of Praluent for patients. Weiss said the injection didn’t seem to bother the people involved in his trial. 

“The patients had no problem with that, so that was another very positive thing. It’s a very simple injection, like insulin,” Weiss said.

Although the drug has received FDA approval and is on the market, its trials continue. Praluent has been shown to lower cholesterol, but it has not yet proven to reduce heart attacks or deaths from heart disease. 

Lower cholesterol numbers do not automatically mean fewer heart attacks. Niacin, for example, has long been taken to lower cholesterol, but some studies have shown it does not lower heart attack risk and may actually cause harm.

Weiss said about 15,000 patients are testing Praluent now to see whether it reduces the risk of heart attack and death. Six or seven of those patients are from this area.

That research has been going on for several years, Weiss said, and will likely continue for about another year.

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