MyFatDog        

AAV-miApoB Being developed by Amsterdam Molecular Therapeutics.  AAV-miApoB is a micro RNA that is delivered to its target using an adeno-associated virus as a vehicle.  The drug targets apoB, the major protein on LDL, reducing its production by the liver.  Mice injected with AAV-miApoB has a sustained 60-80% reduction of LDL cholesterol levels.

AEM-18  Being developed by LipimetiX.  AEM-18 is a small piece (peptide) of the apoE protein that can bind to lipoprotein receptors in the liver and promote LDL clearance.  The company also has a second compound, AEM-28, that reportedly has similar effects.  The drug is being developed as a way to reduce LDL levels.

ATI-5261  Being developed by Artery Therapeutics, Inc.  This drug is a apoA-I mimetic peptide that is designed to increase reverse cholesterol transport. 

Bile salt-based treatment  An unnamed treatment is under development by AtheroNova.  The treatment appears to contain the bile salts hyodeoxycholic acid (HDCA) and D-limonene and was shown to reduce artery plaque by 95% in a preclinical animal model.

DGAT1 inhibitors Being developed by Via Pharmaceuticals under license from Roche.  DGAT1 is an enzyme that facilitates triglyceride production in many tissues in the body including liver and fat tissue.  DGAT1 inhibitors are being developed as treatments for obesity, diabetes and dyslipidemia.

LDL receptor gene therapy  Being developed by ReGenX.  Gene therapy with the LDL receptor is designed to insert DNA for the LDL receptor into liver where it will be made normally in people who have absent or defective receptors.  The LDL receptor is responsible for clearing most of LDL from blood so this treatment has the potential to significantly reduce LDL cholesterol levels in patients iwth homozygous familial hypercholesterolemia. 

MAHDL01 (Mazal Plant Pharma).  This is a formulation that consists of a mixture of herbs that is designed to alter plasma lipid levels, particularly HDL.  Although it earlier appears that the company ran out of money, it recently (February 2010) obtained a small amount of funding and would like to move forward towards phase I/II human studies.

PCSK9 RNAi Being developed by Alnylam Pharmaceuticals.  PCSK9 RNAi is a drug that reduces plasma levels of PCSK9, a protein that targets the LDL receptor for degradation.  This drug is being developed as a treatment to lower LDL levels.

PCSK9 RNAi Being developed by Nativis.  PCSK9 RNAi is a drug that reduces plasma levels of PCSK9, a protein that targets the LDL receptor for degradation.  This drug is being developed as a treatment to lower LDL levels.

Recombinant apoA-I and apoA-I(Milano) Being developed by SemBioSys Genetics.  This company is using a novel approach of protein production by using genetic engineering of plants to produce an abundant supply of recombinant apoA-I.  This is being developed as a treatment for low HDL levels.  The company is currently experienceing financial difficulties and its future is questinoable.

SPC5001  Being developed by Santaris Pharma.  SPC5001 is a molecule designed to lower LDL levels by  reducing PCSK9 which prevents LDL from being cleared from the blood.  This is being developed as a treatment to lower LDL levels.

THR-beta agonist  Being developed by Via Pharmaceuticals under license from Roche.  This drug is a thyroid receptor beta activator being developed as a treatment to reduce LDL cholesterol and triglyceride levels in combination with a statin.  The company is also exploring the potential anti-diabetic effects that they have observed in early studies with the drug.

Preclinical –

Discovery is the earliest phase of drug development.  A company may test thousands or millions of drug candidates in the test tube to discover one that has the characteristics they are looking for. Once a drug is found that has the desired result, the company may have their chemists work on it a bit more to improve its characteristics. 

Once a drug has passed the Discovery phase, it moves from testing in the test tube to testing in a whole animal.  Usually this is done in a mouse mode of the disease and then move in to larger animals like dogs and monkeys.  This is done to show that the drug is safe and effective in animals.