MedCo Preps for FDA Filing as Cholesterol Drug Clears Last Two Tests

Xconomy New York — 

The Medicines Co. this morning inched closer to bringing a twice-yearly cholesterol-lowering drug to market, touting a positive result in two more Phase 3 studies.

Without providing details, MedCo (NASDAQ: MDCO) said that the experimental medicine, inclisiran, hit all of its main and secondary goals in two Phase 3 studies called ORION-9 and ORION-10.

Like the similarly sized ORION-11, which MedCo aced earlier this month, ORION-10 enrolled 1,561 patients who had high cholesterol despite treatment with statins. But ORION-10 enrolled only US patients; ORION-11 took patients from Europe and South Africa. ORION-9 tested inclisiran in 482 patients with an inherited form of high cholesterol called Heterozygous familial hypercholesterolemia.

Importantly, MedCo didn’t report any unexpected side effects. There were no treatment-related liver or kidney problems in either study, MedCo said. The company will provide detailed results from both trials at a medical meeting in November. Shares ticked up about 2 percent in pre-market trading Wednesday.

Inclisiran is an RNA-based medicine that is injected twice a year. Like two FDA-approved drugs—known as evolocumab (Repatha), from Amgen (NASDAQ: AMGN), and alirocumab (Praluent), from Regeneron Pharmaceuticals (NASDAQ: REGN) and Sanofi (NYSE: SNY)—the drug interferes with a cholesterol-boosting protein called PCSK9. Repatha and Praluent bind to excess PCSK9 so it can be cleared out of the body. Inclisiran gets into liver cells and stops them from making PCSK9 in the first place. MedCo licensed inclisiran from RNA drug developer Alnylam Pharmaceuticals (NASDAQ: ALNY) in 2013.

The two approved PCSK9 inhibitors were supposed to change medical practice, but have struggled mightily because insurers balked at their high prices. Despite price concessions, evidence of long-term health benefits, and more, the two drugs have yet to generate $1 billion in annual sales combined, well short of once-lofty sales projections.

MedCo believes convenience—and what its executives have called a “flexible” pricing strategy—could be the difference. Inclisiran is injected twice a year, compared with the other two drugs that are self-injected once or twice a month. Speaking with Xconomy earlier this month, CEO Mark Timney noted that patients at risk of a heart attack or stroke may already be seeing a doctor every six months, which could make insurers more willing to ante up for a treatment that’s timed for those regular visits. He added that the “cost of goods” for inclisiran is different than for Repatha and Praluent. Inclisiran will be a high-volume, lower-cost product to make, Timney said, so MedCo could “reduce the price relatively quickly” as more patients get access.

We could know soon if MedCo’s strategy will pay off, or if—as with Repatha and Praluent—insurers instead wait for the results from a large outcomes study to prove the drug’s ability to reduce the risk of heart attacks and strokes. Given today’s results, the company plans to file for approval of inclisiran in the US later this year and Europe in 2020. MedCo’s 15,000-patient outcomes study, ORION-4, should produce results in 2024. MedCo is also enrolling patients in a long-term study called ORION-8.

Here’s more on MedCo, inclisiran, and the commercial struggles of PCSK9 inhibitors.