Drug companies with products related to Familial Hypercholesterolemia

Some of the companies on Wall Street who are
interested in the
FH population (past or present)

  • NVLN      Novelion
    Therapeutics Inc. (Aegerion Pharmaceuticals)
  • MDGL     Madrigal
    Pharmaceuticals Inc.
  • GEMP     Gemphire
    Therapeutics Inc.
  • ESPR       Esperion
    Therapeutics Inc.
  • AMGN    Amgen
  • REGN      Regeneron
  • MRK        Merck
    & Co. (Had hopes for Anacetrapib
    2011 ~ 2017)
  • Companies which market statins also have an

Big players like Amgen, Regeneron, Sanofi, Merck, and others are financially interested in FH prevalence. However, Novelion (NVLN), Gemphire (GEMP), Madrigal (MDGL) and Esperion (ESPR) are also valued according to how large or small the FH population might be. These four companies go the extra mile and actually claim prevalence estimates in their annual reports filed with the SEC. Below are screenshots I took last year of the 2017 10-K’s. The same claims are made in the 2018 10-Ks.  All four claims depend upon “consensus” reports, which actually conducted no prevalence studies of their own. After chasing the literary references down to their sources, the stones upon which these prevalence claims are built are not scientific. They are linguistic and depend upon preserving asymmetry between what the authors and their readers know.  The 2014 EAS HoFH prevelance estimates – 1 in 300,000 and 1 in 160,000 – are derived from citation kiting. This results in the the equivocation of the (1) genetic definition of the disease and (2) diagnosed FH. Of these two, we will first look into the former, the linguistic equivocation of the geneticdefintion of FH. In a separate presentation, I will provide evidence of the equivocation of “FH” through the removal of key elements/steps in the diagnostic procedure.

four pharmaceutical companies with inflated markets due to 2014 EAS consensus report

For prevalence of homozygous FH, the difference between the Danish result of 1/160,000 and the Dutch result of 1/300,000 is large, but we must also remember that the original estimate by the Nobel Prize winner was 1/1,000,000. Even if we just consider Novelion, and not the entire industry, we can see what is at stake. What will shares of Novelion be worth with this or that prevalence figure? When raising money from investors, what value will investors place on such a company after the Dutch report? The annual price of Aegerion/Novelion’s drug, Juxtapid, has ranged from $290,000 to well over $300,000 during the last 5 years. To create a rough sketch, I used $300,000 to represent the price over these years and 320 million for the U.S. population. Then I calculated the annual addressable market per prevalence estimate according to the Danish and Dutch reports.  I set these two estimates between Aegerion/Novelion’s actual Juxtapid U.S. sales during the last 5 years and an addressable market in accordance with the Nobel Prize winners’ estimate.

prevalence of HoFH, Novelion's actual U.S. Juxtapid sales

The two pillars to the tripled and sextupled HoFH prevalence are the Danish and the Dutch reports. It is clear that these new “scientific” numbers are used for and by investors, enabling fund raising. Here are just two examples. Below, in a 10-K for investors, prevalence is in a text devoted to Madrigal’s “market opportunity.”  

2017 Madrigal Pharmaceuticals, market opportunity

Novelion uses these numbers, not in a scientific context, but in an investor presentation.

Novelion Therapeutics investor presentation - 2014 EAS Consensus Panel