Medicines Co drug shows long-term cholesterol lowering in study

WASHINGTON (Reuters) - A long-acting new type of drug being developed by Medicines Co cut levels of “bad” LDL cholesterol in half in high-risk heart patients with no serious safety issues, according to data from a Phase II trial presented on Friday.

The company said it plans to begin enrolling patients in a pivotal Phase III study for the drug, inclisiran, later this year.

Despite the positive results, Medicine Co shares fell 14.6 percent after highly-anticipated data from Amgen Inc’s rival drug Repatha failed to reach investor expectations for how much it cut the risk of serious adverse heart events in data presented earlier on Friday at the American College of Cardiology (ACC) scientific meeting in Washington.

Inclisiran belongs to a new class of medicines that block a protein known as PCSK9, which prevents the removal of LDL from the blood. But the Medicines Co drug works in a different way than the two already on the market, utilizing a technology known as RNA interference, or RNAi, that blocks PCSK9 production at its source in the liver.

After an initial injection of 300 milligrams of inclisiran and another three months later, the drug is designed to be taken just twice a year. Repatha and Praluent from Regeneron Pharmaceuticals Inc and Sanofi are injected either every two or four weeks.

Aside from the convenience of far fewer injections, Medicines Co is planning to make the drug significantly less expensive than its rivals, if approved.

Chief Executive Clive Meanwell said the company will seek a price based on the value of the drug and risk level of the patient, suggesting it would fall between $2,000 and $8,500 a year. Repatha and Praluent cost more than $14,000 a year before discounts and rebates.

Medicines Co licensed inclisiran from AlnylamPharmaceuticals, which would be entitled to royalty payments on future sales.

In the 9-month study of 497 patients, most already on cholesterol-lowering statins, two doses of inclisiran reduced LDL by up to 53 per cent. Nearly half the patients saw LDL levels fall below 50, well below the common target of 70 for patients at high risk of a heart attack or stroke.

“This is a game changer,” said Dr. Kausik Ray, who presented the data at the ACC meeting.

“You have a treatment regimen that gives you on average, with an initial starting dosing regimen, a 50 percent (LDL) reduction through to nine months,” said Ray. “That’s unheard of.”

Reporting by Bill Berkrot; Editing by Nick Zieminski