Bempedoic acid (also known as ETC-1002) is a novel investigational drug being developed for the treatment of dyslipidemia, hypercholesterolemia and other cardio-metabolic risk factors. The hypolipidemic, anti-atherosclerotic, anti-obesity, and glucose-lowering properties of ETC-1002, characterized in preclinical disease models, are believed to be due to dual inhibition of sterol and fatty acid synthesis and enhanced mitochondrial long-chain fatty acid β-oxidation. Investigations into the mechanism of action revealed that bempedoic acid-free acid activates AMP-activated protein kinase in a Ca(2+)/calmodulin-dependent kinase β-independent and liver kinase β-1-dependent manner, without detectable changes in adenylate energy charge. In the liver, bempedoic acid is also converted to a coenzyme A (CoA) derivative (ETC-1002-CoA )which directly inhibits ATP citrate lyase (ACL), a key enzyme that supplies a substrate for cholesterol and fatty acid synthesis in the liver. Inhibition of ACL by ETC-1002-CoA results in reduced cholesterol synthesis and upregulation of LDL receptor activity in the liver. This promotes the removal of LDL-C from the blood.

Idelalisib is a first-in-class selective inhibitor of adenosine-5'-triphosphate (ATP) binding to PI3Kdelta kinase, resulting in inhibition of the P13K signalling pathway in malignant B cells. The compound is approved for the treatment of several types of blood cancer. Idelalisib is intended to be used in combination with rituximab as second or subsequent line therapy for the treatment of chronic lymphocytic leukaemia. The drug may cause fatal and/or severe diarrhea or colitis, hepatotoxicity, pneumonitis and intestinal perforation.

Ibrutinib is an orally bioavailable Bruton's tyrosine kinase (BTK) inhibitor indicated for the treatment of mantle cell lymphoma (MCL) patients that previously received at least one therapy. The drug was jointly developed by Janssen Biotech and Pharmacyclics. Ibrutinib selectively binds to Cys-481 residue in the allosteric inhibitory segment of BTK (TK/SH1 domain), and irreversibly blocks its enzymatic activity thus preventing B-cell activation and signaling, totally blocking the B-cell receptor and cytokine receptor pathways. This leads to an inhibition of the growth of malignant B cells that overexpress BTK. Apart from mantle cell lymphoma Ibrutinib is approved for the treatment of chronic lymphocytic leukemia and Waldenstrom Macroglobulinemia.

Lamivudine is a reverse transcriptase inhibitor used alone or in combination with other classes of anti-human immunodeficiency virus (HIV) drugs in the treatment of HIV infection. This molecule has two stereo-centers, thus giving rise to four stereoisomers: (+/-)-cis-lamivudine and (+/-)-trans-lamivudine. The latter is considered to be impurity of the pharmaceutically active isomer, (-)-cis-lamivudine.

Corifungin refers to the sodium salt of amphotericin B. Although amphotericin B has become the primary drug of choice for treating primary amoebic meningoencephalitis, its use is associated with multiple side effects, including use-limiting renal toxicity. Initial reports for the in vivo efficacy of corifungin in a mouse model of primary amoebic meningoencephalitis showed activity superior to that of amphotericin B at equivalent dosing. Chemically, corifungin is the sodium salt of amphotericin B with excellent aqueous solubility. The increased solubility of corifungin is likely to account for the described increase in activity. Acea Biotech is developing corifungin for the treatment of fungal infections and amebic diseases. Acea has completed of host of animal studies on corifungin setting the stage to take the drug into the clinic. U.S. FDA has approved orphan drug status for corifungin for the treatment of primary amebic meningoencephalitis.

Ferroheme is formed when iron is inserted into protoporphyrin IX. The active center of hemoglobin and myoglobin consists of iron Ferroheme complex bound through a single, “proximal”, axial histidine (His) to the protein It is an iron containing cofactor that is involved with a wide range of cellular functions including oxygen transport. The antimalarial activity of a number of artemisinin derivatives, both newly synthesized and currently used as drugs, against Plasmodium falciparum in culture shows a correlation with their affinity of binding with Ferroheme.

2-Amino-1-methyl-6-phenylimidazo[4,5-b]pyridine (PhIP) is a mutagenic and carcinogenic heterocyclic amine formed during ordinary cooking, and is subsequently metabolically activated by cytochrome P4501A2 (CYP1A2) and N-acetyltransferase 2 (NAT2). PhIP has been used in trials studying the basic science of Pancreas Cancer.

Cytochalasin B is a cell-permeable alkaloid, isolated from a fungus Helminthosporium dematioideum. Cytochalasin B is an inhibitor of actin polymerization through binding to the fast-growing (barbed) end of F-actin filaments. Cytochalasin is used in studies of actin polymerization, cell division, and cell movement. The compound also inhibits glucose transporters GLUT1,3 and 4 and was investigated in a clinical trial to prevent restenosis after angioplasty surgery.

Echinocandin B is a naturally occurring antibiotic, isolated from Aspergillus nidulans. It acts as an inhibitor of fungal β-1,3-glucan synthase thereby disrupting the formation of the fungal cell wall. The drug itself was not developed in the clinic, but instead, it was used in the production of other echinocandin derivatives.