NM-404 I-124 ([124I]CLR-1404, LIGHT ) is a phospholipid ether analog labeled with iodine I 124, with a potential imaging property upon positron emission tomography (PET). Upon administration, NM-404 I-124 selectively accumulates in and is retained within tumor cells for a prolonged period of time due to the decreased activity of a phospholipase D (PLD), most likely isoform 1 of PLD, in tumor cells compared to normal cells. As tumor cells are unable to metabolize and eliminate MN404, tumor cells can be visualized upon PET imaging. In addition, NM-404 I-124 may provide a more accurate image of the tumor than imaging with the current standard. PLD is an enzyme found in the cell membrane of normal cells that degrades phospholipids. NM-404 I-124 enters cells predominately via lipid rafts, specialized microdomains of plasma membranes enriched in cholesterol and glycosphingolipids, which are found to be overexpressed 6-10 fold in malignant cells compared to normal cells. Although blood pool uptake in major vascular structures demonstrates high uptake initially, there is no significant CLR1404 uptake in normal brain tissue. Therefore, avid brain tumor uptake results in high tumor to back-ground signal and thus clear identification of viable tumor cells on PET scans. NM-404 I-124 selectively illuminated malignant tumors in 52 of 54 animal models of cancer, demonstrating evidence of broad-spectrum, cancer-selective uptake and retention. FDA granted orphan drug designation in the United States for NM-404 I-124 as a diagnostic for the management of glioma.

Etarfolatide (EC20) is a conjugate of folic acid and peptidic metal-chelating moiety. Technetium Tc99m radiolabeled etarfolatide is a folate-targeted molecular imaging agent that is being developed to non-invasively identify tumors that express folate receptors and that may respond to folate-targeted therapy. It is being co-developed as the companion imaging agent to Vynfinit® (vintafolide) and EC1456 (folate-tubulysin).

OSI-027 is an orally bioavailable mammalian inhibitor of mTOR kinase and has antineoplastic activity. OSI-027 binds to and inhibits of the catalytic site of mTOR, which is a central part of two protein complexes, mTORC1 and mTORC2, which may result in tumor cell apoptosis and a decrease in tumor cell proliferation. OSI-027 is in phase I clinical trial for the investigation on patients with advanced solid tumors or lymphoma.

CNDAC (TAK-109) is an analog of the nucleoside deoxycytidine with potential antineoplastic activity. CNDAC is incorporated into DNA and induces single-strand breaks, which are converted into double-strand breaks (DSBs) when cells go through a second S phase. This results in the cell cycle arrest in the S and G2/M phases, DNA fragmentation, and tumor cell apoptosis. Sapacitabine, a prodrug of CNDAC, is being developed by the US biotechnology company Cyclacel for the treatment of hemalogical cancers and solid tumors.

Nevanimibe (also known as PD-132301 and ATR101) is an acetyl-CoA C-acyltransferase (ACAT1) inhibitor. Millendo Therapeutics is currently advancing the development of nevanimibe for the treatment of two orphan adrenal diseases: classic congenital adrenal hyperplasia (CAH) and endogenous Cushing's syndrome (CS). Both of these diseases are associated with an overactive adrenal cortex causing excess steroid production. Millendo believes that nevanimibe represents an adrenal-specific approach that will address these diseases through the reduction of adrenal steroid production. Millendo is currently conducting Phase 2 clinical trial to assess the safety and efficacy of nevanimibe in subjects with endogenous Cushing’s syndrome and for the treatment of adult CAH.

The labeled 4-IODOPHENYLALANINE I-131 is used for proteomics research. It’s also can be used for the chemoselective modification of proteins.

FILOCICLOVIR, also known as cyclopropavir, is a guanosine nucleoside analog. It is an antiviral drug ready for phase II clinical trials for the treatment of cytomegalovirus infection.

BPR0L075 [6-methoxy-3-(3',4',5'-trimethoxy-benzoyl)-1H-indole] is an anti-microtubule drug and is a promising anticancer compound with antimitotic activity. It has potential for management of various malignancies, particularly for patients with drug resistance. BPR0L075 inhibits tubulin polymerization through binding to the colchicine-binding site of tubulin.

Diazepinomicin is a structurally novel farnesylated dibenzodiazepinone discovered through DECIPHER technology, Thallion's proprietary drug discovery platform. A small-molecule inhibitor of the RAS/RAF/MAPK signaling pathway with potential antineoplastic activity. Diazepinomicin binds to and inhibits Ras kinase, thereby preventing the phosphorylation and activation of proteins downstream of the Ras signal transduction pathway, including serine/threonine kinase RAF (BRAF) and extracellular signal-regulated kinases 1 and 2 (ERK1 and ERK-2). This agent also selectively binds to the peripheral benzodiazepine receptor (PBR), a receptor highly expressed in certain tumor cell types, inducing cell cycle arrest and apoptosis in PBR-expressing cells. The compound was shown to have a broad cytotoxic activity in the low micromolar range, when tested in the NCI 60 cell line panel. Diazepinomicin can cross the blood-brain barrier. Diazepinomicin is in phase II clinical trials for the treatment of Telomeric 22q13 Monosomy Syndrome and phase I for the treatment of Fragile X syndrome.

2,2-DIMETHYLBUTYRIC ACID (HQK-1001) is an orally administered SCFAD (Short Chain Fatty Acid Derivative), which has shown an excellent safety profile and biologic effects on fetal hemoglobin induction and red blood cell production in the laboratory, relevant animal models, and in clinical trials carried out in healthy human subjects as well as patients with sickle cell disease and beta thalassemia. The compound has received Orphan Drug Designation in the United States and Europe for both sickle cell disease and beta thalassemia. HemaQuest Pharmaceuticals was developing HQK-1001 for the oral treatment of sickle cell anaemia and beta thalassaemia. HQK-1001 has been evaluated in phase II trials for beta thalassaemia and sickle cell anaemia.