Drug Development

No early success for Regeneron, Sanofi in PhIII outcomes study for PCSK9 drug Praluent

Christmas is not coming early for Regeneron and Sanofi.

The companies noted early today that they will have to go all the way to the end of their outcomes trial to see if their PCSK9 drug Praluent can deliver the goods in reducing the risk of a major cardio event after failing to find overwhelming evidence of success in its second preliminary review of the trial.

Brian Skorney, Baird analyst

Brian Skorney, Baird analyst

Investigators recruited 18,000 patients with uncontrolled LDL for the big trial, which will need to be positive if they ever expect to open up the market for their cholesterol drug. Payers have dug their heels in on this drug, along with the rival Repatha from Amgen, which just delivered fresh biomarker data to indicate the likelihood of bending the curve on efficacy for an at-risk group of patients.

Payers, though, are holding out for clear data showing that these drugs are effective on key disease measures.

“REGN had guided the interim bar was high and would need to meet several criteria in addition to overwhelming CV outcome reduction over the control group,” noted Jefferies’ Biren Amin today. “We expect final data in 2017.”

That will give The Medicines Company and Alnylam more time to advance their new and improved PCSK9 drug, which may only need to be dosed two or three times a year. In the meantime, some of the skeptics say the need to finish up indicates that the data are likely to be somewhat underwhelming.

Notes Baird’s Brian Skorney:

A hazard ratio of 0.802 is not a high bar, in our opinion. While we concede that trends on secondaries could be holding the study back, if the drug were truly “transformational,” the study mostly likely would have been stopped early.


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RAPS Regulatory Convergence 2017