Closing a tough year: An opinionated take on NEJM highlights for December 2020

Cardiovascular health – go big or go narrow?

A follow up on the development of evinacumab (Regeneron), an inhibitor of ANGPLT3 (see this opinionated take from 2017), confirming the safety and LDL reduction effect in a population of individuals with cholesterol levels refractory to other therapies (including PCSK9 inhibitors).  Given the history of PCSK9 inhibitors, the development path of this asset will be interesting to follow. Of the 6 trials listed on not one has anything close to a clinical endpoint – it’s all biomarker based. That will probably be sufficient for use in patients with familial hypercholesterolemia because their risk is so high and so well correlated with cholesterol levels, but for your run of the mill cardiovascular patients, regulators and especially payers will likely want to see something more concrete than a cholesterol reduction. This clinical development path would then essentially represent a rare disease strategy which is a paradox for a disease state which is the top killer in the US. Evinacumab in Patients with Refractory Hypercholesterolemia; Factoring in ANGPTL3 When LDL Is Refractory


The story of a triumph: the first fully validated Covid-19 vaccine

Results from the Pfizer/BioNTech vaccine have been well reported in the media but the actual papers describing the phase 1 and phase 3 studies are worth a read if only to find out what side effect profile awaits you as you join the vaccination line. The one element that I would point out is that from the way the curves between placebo and active treatment diverge before the second dose of the vaccine, one would guess that a single dose of the vaccine is protective – but perhaps not for as long.  Safety and Immunogenicity of Two RNA-Based Covid-19 Vaccine Candidates; Safety and Efficacy of the BNT162b2 mRNA Covid-19 Vaccine (free access)


The New England Journal of Medicine is a premier weekly medical journal covering many topics of interest to the health sector. In this monthly series we offer an opinionated perspective on selected highlights that might be of interest to our clients and others.

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