This week in therapeutics




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Publication and contact information

Endocrine/metabolic disease

Hypercholesterolemia; hyperlipidemia; dyslipidemia

SEC24 family member A (SEC24A); proprotein convertase subtilisin/kexin type 9 (PCSK9); low-density lipoprotein receptor (LDLR)

Mouse studies suggest inhibiting SEC24A could help treat hypercholesterolemia and related metabolic indications. In mice, a Sec24a deficiency decreased hepatic secretion of Pcsk9 and increased Ldlr expression compared with no Sec24a deficiency. In the Sec24a-deficient mice, circulating levels of high-density lipoprotein, low-density lipoprotein and total cholesterol were lower than those in nondeficient controls. Next steps include testing the effects of SEC24A knockdown in human hepatocytes.
AMG 145, a human mAb against PCSK9 from Amgen Inc., is in Phase III testing to treat hypercholesterolemia, hyperlipidemia and dyslipidemia.
REGN727 (SAR236553), a human mAb targeting PCSK9 from Regeneron Pharmaceuticals Inc. and Sanofi, is in Phase III testing to treat hypercholesterolemia.
PF-04950615 (PF-4950615; RN315), a PCSK9 inhibitor from Pfizer Inc., is in Phase II testing to treat hypercholesterolemia and acute coronary syndrome.

SciBX 6(17); doi:10.1038/scibx.2013.415
Published online May 2, 2013

Unpatented; available for partnering or licensing from the University of Michigan
Contact: Robin Rasor, University of Michigan, Ann Arbor, Mich.

Chen, X.-W. et al. eLife;
published online April 9, 2013;
Contact: David Ginsburg, University of Michigan, Ann Arbor, Mich.