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Cardio drug at The Medicines Co gets the heave-ho. (But they prefer PCSK9 anyway.)

Clive Meanwell, The Medicines Company

Clive Meanwell, The Medicines Company

The Medicines Company’s cardio pipeline is being pared back to its showcase PCSK9 project following a Phase II failure of their atherosclerosis drug MDCO-216.

MDCO-216 was tested in a 120-patient study to see how effective it would be in spurring cholesterol efflux. It’s built from the ApoA-1 Milano protein — discovered in a town in northern Italy where residents had low levels of “good” HDL but with little or no atherosclerotic plaque buildup associated with coronary artery disease — and a phospholipid.

Investigators found that it didn’t have the desired effect, so they’re killing it and releasing the data at ASH in a few days. And their stock slid 6% in after-market trading on Monday.

The Parsippany, NJ-based biotech $MDCO, though, positioned the setback as a positive, asserting that they can now put more resources behind a closely-watched PCSK9 drug partnered with Alnylam $ALNY. Those two companies have enjoyed considerable buzz that their PCSK9si program can effectively kick back LDL with a two- or three-time per year dosing regimen, which would make it potentially much more competitive than the pioneering rival cholesterol meds advanced by Amgen and the Sanofi/Regeneron team.

The giants in the field, though, have found it tough going in the early days of making a market with initial sales falling well behind early forecasts. For The Medicines Company, which is now lining up expensive registration studies, the timing for a superior player to come along could be spot on.

“We deliberately focused our initial development investment in MDCO-216 on clinical proof of concept. Unfortunately, the efficacy data from MILANO-PILOT do not support a prudent decision to make the significant, near-term investment required to move MDCO-216 forward,” stated Clive Meanwell, the CEO of The Medicines Company. “In spite of promising earlier research findings, and impressive progress with manufacturing development and safety, in the light of these efficacy data and in view of the potentially enormous opportunity and highly-favorable risk-reward profile presented by our PSCK9 synthesis inhibitor, we have been decisive in immediately terminating the MDCO-216 development program.”


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RAPS Regulatory Convergence 2017