One of the hottest competitions in drug development has spurred Amgen ($AMGN), Sanofi ($SNY) and Pfizer ($PFE) to invest heavily in PCSK9 drug programs aimed at lowering bad cholesterol in a way that could prove a game-changer in the field of heart disease. And the New York Times' Gina Kolata carefully reviews the sleuthing that pointed investigators to a rare genetic mutation that affected both copies of a gene inherited from parents.
The potential impact on public health "was so hot it sizzled," said Dr. Steven Nissen, the chairman of the department of cardiovascular medicine at the Cleveland Clinic and leader of an Amgen trial.
The impact of these drugs is also so profound that Pfizer has set a ground floor on the LDL levels it will permit in its studies. Amgen is already setting up factories at three locations so it can churn out the quantities it believes it will need--an extraordinary upfront investment for a company still engaged in a lengthy clinical trial program.
The most optimistic analysts say these biologics--REGN727 by Sanofi and Regeneron ($REGN), AMG 145 at Amgen and Pfizer's RN316 (PF-04950615)--all have megablockbuster potential stretching as high as $10 billion. That's usually the kind of revenue potential reserved for a new Alzheimer's drug. But there are also plenty of analysts who say that figure is wildly inflated. And Novartis ($NVS) as well as a few biotechs like Alnylam ($ALNY) have been working in this field as well, raising the prospect of multiple drugs aiming for the same market.
Patients with stubbornly high levels of LDL continue to sign up for these studies, desperate to find something that will finally blunt their risk of heart disease. David Mayse told Kolata that he had had two heart attacks by the time doctors pointed him to one of the studies.
"I was willing to try anything at that point," Mayse remarked.
- here's the feature from The New York Times