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.

Apitolisib, a dual inhibitor of mTOR and phosphatidylinositol 3-kinase (PI3K), was being developed by Roche and Genentech as an orally administered therapy of cancer. Apitolisib is a selective, potent, orally bioavailable inhibitor of Class I PI3 kinase (PI3K) and mTOR kinase (TORC1/2) with excellent pharmacokinetic and pharmaceutical properties. Apitolisib displayed excellent potency against class I PI3K isoforms (IC50 PI3K-α, β, δ and γ = 4.8, 27, 6.7 and 14 nM) and mTOR kinase (IC50 = 17 nM) and selectivity against a large panel of other kinases. Apitolisib is in phase II trials by Genentech for the treatment of breast cancer, prostate cancer, endometrium cancer, kidney cancer. However, no recent development has been reported. It is also in phase I trials by Genentech for the treatment of non-Hodgkin's lymphoma.

FERMAGATE is a phosphate binder with phosphate plasma levels lowering activity. It is in Phase 3 development in the US and in Europe for the treatment of hyperphosphatemia in dialysis patients. It is made up of magnesium and ferric iron atoms held in an insoluble, rigid, crystalline-layered structure, with carbonate groups lying between the layers. Upon oral administration, the carbonate ions in FERMAGATE are exchanged for free phosphate ions released from food in the gastrointestinal tract; thereby, strongly binding phosphate. This inhibits phosphate uptake and preventing hyperphosphatemia.

Rebastinib (DCC-20) is a TIE2 kinase inhibitor currently in Phase 1 clinical development to treat breast cancer and Chronic Myeloid Leukemia. Rebastinib potently inhibited TIE2 kinase in cellular assays and blocked primary tumor growth by 75% as a single agent and by 90% in combination with the standard chemotherapeutic agent paclitaxel. Furthermore, rebastinib therapy significantly reduced the presence of tumor-promoting macrophages in tumor biopsies by 80%. This blockade of tumor macrophages correlated with inhibition of breast cancer lung metastases.

Trepirium is a ganglion-blocking agent. During clinical testing of the administration of ganglion-blocking agent by inhalation it was established that drug aerosol of trepirium possesses high effectiveness, acts rapidly, and is relatively safe. Trepirium does not cause any local irritating effect. It blocks the n-cholinergic receptors of the autonomic ganglia (it inhibits the transmission of excitation from the preganglionic to the postganglionic fibers of the autonomic nerves) of the adrenal medulla and the sinocarotid region. Reduces the flow of vasoconstrictor impulses to the vessels and adrenaline secretion by the adrenal glands, weakens reflex pressor reactions, and lowers blood pressure. Trepirium is used for the treatment of cerebral or pulmonary edema, hypertensive crisis, nephropathy and eclampsia (in obstetric practice), for the hypotension control in anesthesia practice.

PF-04531083 is selective Nav1.8 blocker. It has been investigated for the treatment of chronic pain, heart pain, post-surgical dental pain. However, these trials were discontinued.