GTC Biotherapeutics (fka GTCB)

[DewDiligence]

Musings on GTC’s business model:

Endogenous means “arising from within,” so an endogenous protein is simply a protein that the body makes on its own such as antithrombin or FVIIa or alpha-1 antitrypsin. This is in contrast to the “tweaked” protein in a drug such as the antithrombin fragment from Artisan Pharma (#msg-14524596, #msg-14527640) or the “enhanced” FVIIa from MAXY and Roche (#msg-13763492).

If a recombinant drug based on an endogenous protein is very close structurally to the endogenous protein, then the odds are good that the drug will not incur show-stopping safety problems. ATryn is an example of such a drug. However, when drug developers start tweaking the natural molecular structure of a protein, all bets are off. That’s why I say that the business model of companies that do this (e.g. MAXY) is riskier than GTC’s business model.

You brought up GTC’s CD137 program, and I’m glad you did because this program is fundamentally different than the ATryn and FVIIa programs. In the CD137 program, GTC is seeking to develop a brand new molecule—an antibody directed at the CD137 receptor—rather than simply a recombinant version of an endogenous protein. Thus, the CD137 program is very much riskier than the other programs in GTC’s pipeline (except perhaps the malaria program).

I think GTC’s raison d’etre going forward will be to pursue relatively low-risk programs based on endogenous proteins rather than to be a full-fledged drug-discovery company. GTC has a proprietary production platform that enables the company to produce recombinant drugs based on endogenous proteins more cheaply than anyone else, and this—rather than drug discovery through tweaking—is GTC’s area of greatest competitive advantage. Regards, Dew