Indication

Target/marker/pathway

Summary

Licensing status

Publication and contact information

Endocrine/metabolic disease

Hypercholesterolemia; hyperlipidemia

ATP-binding cassette sub-family A member 1
(ABCA1); microsomal triglyceride transfer protein (MTTP; MTP)

Mouse studies suggest inhibiting MTP and ABCA1 in the intestines could help treat hypercholesterolemia and hyperlipidemia. In mice, combined, intestine-specific knockout of Mtp and Abca1 decreased acute cholesterol absorption and plasma cholesterol concentrations compared with single knockouts or no knockout. Next steps include designing an agent that inhibits both MTP and ABCA1.
Aegerion Pharmaceuticals Inc. and Catalent Pharma Solutions Inc. market the MTP inhibitor Juxtapid lomitapide to treat hypercholesterolemia.
Nano Terra Inc. and Kadmon Corp. LLC have KD026, an enterocyte-selective MTP inhibitor, in Phase II testing to treat diabetes and obesity.

SciBX 6(39); doi:10.1038/scibx.2013.1097
Published online Oct. 10, 2013

Findings unpatented; unavailable for licensing

Iqbal, J. et al. J. Biol. Chem.; published online Sept. 9, 2013;
doi:10.1074/jbc.M113.501247
Contact: M. Mahmood Hussain, SUNY Downstate Medical Center,
Brooklyn, N.Y.
e-mail:

mahmood.hussain@downstate.edu