U.S. Department of Health & Human Services Divider Arrow National Institutes of Health Divider Arrow NCATS

    {{facet.count}}
    {{facet.count}}

    {{facet.count}}
    {{facet.count}}

    {{facet.count}}
    {{facet.count}}

    {{facet.count}}
    {{facet.count}}

    {{facet.count}}
    {{facet.count}}

    {{facet.count}}
    {{facet.count}}

    {{facet.count}}
    {{facet.count}}

    {{facet.count}}
    {{facet.count}}

    {{facet.count}}
    {{facet.count}}

    {{facet.count}}
    {{facet.count}}

    {{facet.count}}
    {{facet.count}}

    {{facet.count}}
    {{facet.count}}

    {{facet.count}}
    {{facet.count}}

    {{facet.count}}
    {{facet.count}}

    {{facet.count}}
    {{facet.count}}

    {{facet.count}}
    {{facet.count}}

    {{facet.count}}
    {{facet.count}}

    {{facet.count}}
    {{facet.count}}

Class (Stereo):
CHEMICAL (ACHIRAL)



Tepotinib is an investigational small molecule inhibitor of the c-Met receptor tyrosine kinase. Alterations of the c-Met signaling pathway are found in various cancer types and correlate with aggressive tumor behavior and poor clinical prognosis. Tepotinib is a potent and selective c-Met inhibitor, >200-fold selective for c-Met than IRAK4, TrkA, Axl, IRAK1, and Mer. Tepotinib is currently in Phase I/II trials in liver cancer and lung cancer.

Class (Stereo):
CHEMICAL (ABSOLUTE)

Voclosporin (trans-ISA247) is a Cyclosporin A derivative and immunosuppressive compound currently being investigated for the treatment of psoriasis, lupus nephritis and for the prevention of organ rejection in kidney transplant patients. An animal study showed that a lower blood level of Voclosporin was able to produce a greater or similar inhibition of lymphocyte proliferation, expression of T-cell activation surface antigens, and T-cell cytokine production compared to Cyclosporin A. Voclosporin has been shown to be an efficacious and safe immunosuppressant in phase IIb and phase III trials in renal transplant recipients and in plaque psoriasis patients. In clinical trials, Voclosporin added to standard-of-care induction therapy for lupus nephritis increases complete renal remission (CRR) rates, but higher rates of adverse events including death were observed.
Umbralisib (TGR-1202) is an orally available PI3K delta inhibitor, targeting the delta isoform with nanomolar potency and several fold selectivity over the alpha, beta, and gamma isoforms of PI3K. The delta isoform of PI3K is strongly expressed in cells of hematopoietic origin and is believed to be important in the proliferation and survival of B-cell lymphocytes. Inhibition of PI3K delta signaling with umbralisib has demonstrated robust activity in numerous pre-clinical models and primary cells from patients with hematologic malignancies. Umbralisib is currently in Phase 3 clinical development in combination with Ublituximab for patients with hematologic malignancies.

Class (Stereo):
CHEMICAL (ABSOLUTE)

Cyclic pyranopterin monophosphate is a precursor to molybdenum cofactor, which is required for the enzyme activity of sulfite oxidase, xanthine dehydrogenase/oxidase, and aldehyde oxidase. The drug is being investigated in severe molybdenum cofactor deficiency type A. In a clinical study, intravenous administration of the drug resulted in greatly improved neurodevelopmental outcome when started sufficiently early.
Estetrol is the natural human fetal selective estrogen receptor modulator. It is synthesized exclusively by the human fetal liver during pregnancy. Estetrol has a moderate affinity for human estrogen A receptor (ERa) and estrogen B receptor (ERb). Estetrol may be suitable as a potential drug for human use in applications such as hormone replacement therapy (vaginal atrophy, hot flushes), contraception and osteoporosis. The most common drug-related adverse events were lower abdominal pain, nausea, headache, dysmenorrhoea, breast enlargement and acne. Estetrol had been in clinical trials for the treatment of breast and prostate cancers.

Class (Stereo):
CHEMICAL (ACHIRAL)


Conditions:

Vericiguat, discovered at Bayer, is the first soluble guanylate cyclase (sGC) stimulator. Vericiguat is currently being studied in a Phase III clinical program for the treatment of heart failure with reduced ejection fraction (HFrEF).
Cabotegravir is an investigational drug that is being studied for the treatment and prevention of HIV infection. Cabotegravir belongs to a class (group) of HIV drugs called integrase inhibitors. Integrase inhibitors block an HIV enzyme called integrase. (An enzyme is a protein that starts or increases the speed of a chemical reaction.) By blocking integrase, integrase inhibitors prevent HIV from multiplying and can reduce the amount of HIV in the body. Cabotegravir does not require boosting with an additional drug. Two forms of cabotegravir are being studied: tablets that are taken by mouth (known as oral cabotegravir or oral CAB) and a long-acting injectable form that is injected into the muscle (known as cabotegravir LA or CAB LA; LA stands for "long-acting"). (A long-acting drug formulation works over a long period of time. Using this type of drug might mean that the drug could be taken less often, making a treatment or prevention regimen simpler to take.) Cabotegravir is in Phase-III clinical trials for HIV infections.

Class (Stereo):
CHEMICAL (ABSOLUTE)

Melphalan flufenamide ethyl ester (melflufen, previously denoted J1) is a peptidase-potentiated dipeptide prodrug of alkylating agent melphalan. Melflufen readily penetrates membranes and an equilibrium is rapidly achieved, followed by enzymatic cleavage in aminopeptidase positive cells, which results in trapping of less lipophilic metabolites. This targeting effect results in very high intracellular concentrations of its metabolite melphalan and subsequent apoptotic cell death. This results in a potency increase (melflufen vs melphalan) ranging from 10- to several 100-fold in different in vitro models. Melflufen triggers a rapid, robust, and an irreversible DNA damage which may account for its ability to overcome melphalan-resistance in multiple myeloma cells. Melflufen has been granted orphan drug designation for the treatment of multiple myeloma.
Viloxazine is an antidepressant drug was used to treat patients with depression. Viloxazine inhibits noradrenaline uptake. This drug was approved in some Europe countries, but not in the USA, but then it was discontinued because of competition from other drugs. In the frame of drug repositioning, Viloxazine participated in clinical trials for the treatment of attention deficit hyperactivity disorder. Phase II of trials was successfully passed.