Medicines Co., Alnylam prepare to throw the dice on a late-stage effort to leapfrog PCSK9 giants

While Amgen and its rivals at Regeneron/Sanofi struggle to establish a market for their pioneering PCSK9 therapies — while grappling over a nasty lawsuit over the IP — Alnylam $ALNY and The Medicines Company $MDCO are hatching plans for a come-from-behind attempt to leapfrog the squabbling frontrunners.

Clive Meanwell, The Medicines Company

This morning the companies announced that the FDA has signed off on their Phase III program, which will enroll a total of 3,000 subjects with atherosclerotic cardiovascular disease and familial hypercholesterolemia to test the ability of inclisiran to slash LDL over 18 months. And they aren’t waiting for the results to begin their cardio outcomes study.

Researchers plan to sweep up 14,000 patients for the outcomes study, looking to see if this new drug can impress payers with data on a composite of coronary heart disease death, non-fatal myocardial infarction and fatal and non-fatal ischemic stroke.

That’s a tricky proposition, though.

Amgen hit marks for several outcomes with their cardio follow-up trial earlier this year, but was slapped with a backlash of skepticism from analysts and payers who wanted to see better data to justify the price of the drug — particularly on reducing mortality. Amgen’s money-back guarantee for patients on their drug who go on to experience a heart attack failed to win over the doubtful, who were quick to do the math on costs and savings.

Learning from the pioneers, The Medicines Company and Alnylam — which developed the RNA drug — want to nail down the right data and then angle in with a drug that may only have to be given two or three times each year. After that, the whole global battle for market share could get down to price.

Last fall researchers in the Phase II study reported that a single 300 mg injection of their RNAi drug — now dubbed inclisiran — registered a mean 43% reduction in bad LDL cholesterol after 180 days, triggering some excited buzz among the analysts following this program.

Noted Medicines Co. CEO Clive Meanwell:

Although not required for the NDA, we will also perform an aggressive cardiovascular outcomes trial in high-risk subjects with ASCVD and/or risk-equivalents, such as diabetes. We believe that positive outcomes data, with primary outcome clinical effects greater than those reported for anti-PCSK9 monoclonal antibodies, will drive a high level of competitiveness in the worldwide market – which we expect to become very large.

Sanofi and Regeneron, meanwhile, expect to have their cardio data available later this year, hoping to trump Amgen as the companies fight it out on the patent front.

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