Drug Development

Medicines Co, Alnylam generate positive buzz with evidence of a durable, 6-month LDL response to PCSK9 rival

Clive Meanwell, The Medicines Company

Clive Meanwell, CEO

The Medicines Company $MDCO and its partner Alnylam $ALNY posted an impressive set of 6-month results for the first batch of patients to reach the annual halfway mark on their new and perhaps much easier PCSK9 drug regimen. 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.

The Medicines Company has attracted some considerable enthusiasm for a cholesterol drug that might only be needed to be dosed two or three times a year—highlighting a drug that could well slap down approved therapies from Amgen and Sanofi/Regeneron. So when they turned up with a late-breaker at the American Heart Association Scientific Sessions 2016 in New Orleans, analysts were ready to clap at the first solid sign of durability from the first 189 subjects in the 497-patient study.

Two 300 mg injections of the drug triggered a 57% mean drop in LDL on day 120 and 52% by day 180.

“Together these data support feasibility for tri-annual and potentially bi-annual dosing,” wrote Gena Wang, an analyst at Jefferies.

Umer Raffat from Evercore ISI noted:

Ph 2 PCSk9 data presented at AHA just now looks very consistent and durable through day 180 (pts getting single injection had -43% LDL reduction at day 180).

I am getting several questions on 1 death in the 500 mg arm:  it was in a patient with extensive CAD history and multiple CV events in the past (note that exclusion criteria only selected out pts with MACE event within last 6 months).  Also, 1 case of ALT>3ULN occurred in a patient escalating dose of statin.”

Both Amgen as well as the team from Sanofi/Regeneron have been forced to lay out disappointing launches for their PCSK9 offerings, both tapped as likely blockbusters in the making. The cost, as well as questions of the underlying health benefits, have conspired to limit their initial use.

The dream scenario from Clive Meanwell, the CEO:

“We will focus our resources on inclisiran for aggressive Phase 3 development to ensure that this promising agent is investigated thoroughly and rapidly in Phase III, submitted to worldwide regulatory agencies and, if approved, made available to millions of at-risk, often non-adherent, patients worldwide who continue to grapple with the realities and risks of high LDL-C.”


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