Betrixaban is an anticoagulant drug which acts as a direct factor Xa inhibitor. Betrixaban is now being developed by Portola Pharmaceuticals. Oral, once-daily Factor Xa inhibitor anticoagulant that directly inhibits the activity of Factor Xa, an important validated target in the blood coagulation pathway, to prevent life-threatening thrombosis. U.S. Food and Drug Administration granted Fast Track designation to betrixaban for extended-duration prevention of venous thromboembolism (VTE; blood clots) in acute medically ill patients (i.e., those who are hospitalized for serious medical conditions, such as heart failure, stroke, infection and pulmonary disease). Has the potential to become the first oral Factor Xa inhibitor anticoagulant approved for hospital-to-home prevention of VTE in acute medically ill patients.

Copanlisib, developed by Bayer, is a phosphoinositide 3-kinase (PI3K) inhibitor with potential antineoplastic activity. Copanlisib inhibits the activation of the PI3K signaling pathway, which may result in inhibition of tumor cell growth and survival in susceptible tumor cell populations. Activation of the PI3K signaling pathway is frequently associated with tumorigenesis and dysregulated PI3K signaling may contribute to tumor resistance to a variety of antineoplastic agents. Copanlisib is currently under Phase II/III clinical trials for the treatment of non-Hodgkin lymphoma and chronic lymphocytic leukemia.

Macimorelin (AEZS 130) is an orally active, small-molecule, peptidomimetic growth hormone secretagogue receptor (GHSR1A) agonist (ghrelin analogue), being developed by AEterna Zentaris for the diagnosis of adult growth hormone deficiency (AGHD; somatotropin deficiency), and for the treatment of cachexia associated with chronic disease such as AIDS and cancer. Macimorelin was approved by the FDA in December 2017 under the market name Macrilen for oral solution. Macimorelin stimulates GH release by activating growth hormone secretagogue receptors present in the pituitary and hypothalamus. Macimorelin has been granted orphan drug designation by the FDA for diagnosis of AGHD.

Copanlisib, developed by Bayer, is a phosphoinositide 3-kinase (PI3K) inhibitor with potential antineoplastic activity. Copanlisib inhibits the activation of the PI3K signaling pathway, which may result in inhibition of tumor cell growth and survival in susceptible tumor cell populations. Activation of the PI3K signaling pathway is frequently associated with tumorigenesis and dysregulated PI3K signaling may contribute to tumor resistance to a variety of antineoplastic agents. Copanlisib is currently under Phase II/III clinical trials for the treatment of non-Hodgkin lymphoma and chronic lymphocytic leukemia.

Rabacfosadine was approved in 2017 under the brand name Tanovea-CA1 for the treatment of canine lymphoma. In addition, this drug has demonstrated effectiveness against non-Hodgkin's lymphoma in dogs, as well as canine cutaneous T-cell lymphoma, and relapsed canine B-cell lymphoma. Rabacfosadine a prodrug, which is hydrolyzed intracellularly to the metabolites, 9-(2-phosphonylmethoxyethyl)-N6-cyclopropyl-2,6-diaminopurine (cPrPMEDAP) and 9-(2-phosphonylmethoxyethyl) guanine (PMEG). PMEG is then converted to its active phosphorylated form, which is a chain-terminating inhibitor of the replicative deoxyribonucleic acid (DNA) polymerases.

QS-21 is a purified soapbark tree (Quillaja saponaria) extract that enhances the ability of the immune system to respond to vaccine antigens. QS-21 is a promising adjuvant candidate for use in humans due to the ease of purification, its improved safety profile, and its ability to enhance cellular and humoral immunogenicity. The mechanism of action of QS-21 was speculated to be similar to QA, forming complexes with cholesterol that intercalate into the cell membrane lipids. This intercalation creates pores in the membrane to accelerate the uptake of a co-delivered antigen by the antigen presenting cells. Multiple clinical trials using QS-21 as an adjuvant, demonstrated satisfactory safety profiles and enhanced immunogenicity in immunocompromised volunteers

Rabacfosadine was approved in 2017 under the brand name Tanovea-CA1 for the treatment of canine lymphoma. In addition, this drug has demonstrated effectiveness against non-Hodgkin's lymphoma in dogs, as well as canine cutaneous T-cell lymphoma, and relapsed canine B-cell lymphoma. Rabacfosadine a prodrug, which is hydrolyzed intracellularly to the metabolites, 9-(2-phosphonylmethoxyethyl)-N6-cyclopropyl-2,6-diaminopurine (cPrPMEDAP) and 9-(2-phosphonylmethoxyethyl) guanine (PMEG). PMEG is then converted to its active phosphorylated form, which is a chain-terminating inhibitor of the replicative deoxyribonucleic acid (DNA) polymerases.

Trimethylamine (or TMA) a tertiary amine, is synthesized by the action of microbial enzymes in humans. The decrease of TMA metabolism and excessive TMA excretion cause the disease trimethylaminuria and some other diseases associated with the abnormal level of TMA, e.g., obesity, diabetes, cardiovascular diseases. It was shown, that TMS is a full agonist of human trace amine-associated receptor 5, TAAR5. In addition, TMA is a precursor of N-oxide form, an emergent biomarker of human health that can lead to renal diseases, neurological disorders, and cancer.