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Preclinical Pipeline

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

APA-01 Being developed by Phosphagenics.  APA-01 is an antiinflammatory drug that is being developed to be used in combination with statins to lower LDL levels and reduce inflammation. 

Cerenis HDL  Being developed by Cerenis Therapeutics.  This drug is a apoA-I mimetic peptide that is designed to increase reverse cholesterol transport. 

CSL112 Being developed by CSL Limited.  CSL112 is a reconstituted HDL that is being developed as a treatment to increase HDL levels and stimulate cholesterol removal from tissues (reverse cholesterol transport). The percursor to CSL112 was a similar compound, CSL111 that had progressed to phase II studies. CSL111 appears to have been discontinued following  a study that showed that the drug was no better than placebo in reducing atherosclerosis.  CSL112 is reported to be more potent than CSL111.

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.

IMO-3100  Being developed by Idera Pharmaceuticals.   IMO-3100 is a compound that blocks the action of two receptors, Toll-like receptors (TLR) 7 and 9, that turn on inflammatory pathways in cells.  Although intially developed to treat inflammation, IMO-3100 has been shown to effectively reduce LDL in preclinical studies in mice and may prove to be of benefit in improving diseases with generalized inflammation such as the metabolic syndrome.

NCX6560 Being developed by NicOx.  NCX6560 is a new statin drug that also has vasodilatory properties.  This drug is being developed to lower LDL levels and to enhance vasodilation.

PCSK9 Antisense Being co-developed by Bristol-Myers Squibb and Isis Pharmaceuticals. PCSK9 antisense 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 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.

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.

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.

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