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

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Momelotinib (CYT387) is an ATP-competitive small molecule that potently inhibits JAK1/JAK2 kinases. Momelotinib is developing by Gilead Sciences for the oral treatment of pancreatic and non-small cell lung cancers, and myeloproliferative disorders (including myelofibrosis, essential thrombocythaemia and polycythaemia vera).
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.
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.
Selpercatinib (LOXO-292, ARRY-192) is a potent and specific RET (c-RET) inhibitor that was granted accelerated FDA approval on May 8, 2020, for specific RET-driven cancer indications. It is currently marketed under the brand name RETEVMO™ by Loxo Oncology Inc.
Tazemetostat (EPZ-6438) is a selective inhibitor of histone-lysine N-methyltransferase EZH2. The drug is under clinical development (phase II) for the treatment of Diffuse Large B Cell Lymphoma, Malignant Mesothelioma and Synovial Sarcoma.

Class (Stereo):
CHEMICAL (ABSOLUTE)

Setmelanotide (RM-493), is an investigational, first-in-class melanocortin-4 receptor (MC4R) agonist in development for the treatment of rare genetic disorders of obesity. Setmelanotide is thought to activate the MC4R, part of a key biological pathway in humans that regulates weight by increasing energy expenditure and reducing appetite. Variants in genes within the MC4 pathway are associated with unrelenting hunger, known as hyperphagia, and severe, early-onset obesity. Setmelanotide is a potential replacement therapy that may restore lost activity in the MC4 pathway, reestablishing weight and appetite control in patients with these rare genetic disorders.
KX-01 is a dual inhibitor of Src kinase and tubulin polymerization. KX01 promotes the induction of p53, G2/M arrest of proliferating cell populations and subsequent apoptosis via the stimulation of Caspase-3 and PARP cleavage. The drug was developed by Kinex Pharmaceuticals and reached phase II of clinical trials for the treatment of Castration-Resistant Prostate Cancer and Actinic Keratosis. KX-01 demonstrated good in vitro pofile against different cancer cell lines with IC50 in nanomolar range.

Class (Stereo):
CHEMICAL (ABSOLUTE)


Relebactum sodium (MK-7655) is a piperidine analog 3 that inhibits class A and C β-lactamases (in vitro). It is being investigated for use in treatment of infectious diseases, such as treatment of gram-negative bacterial infections. Its potential as an alternative to existing medicines in the treatment of drug-resistant bacterial infections is being studied. Clinical trials have been conducted and are still ongoing to evaluate the efficacy and safety of relebactum sodium in treatment of intra-abdominal infections, urinary tract infections (such as pyelonephritis), hospital-acquired and ventilator-associated bacterial pneumonias, and gram-negative bacterial infections.
ODM-201 (also known as BAY-1841788) is a non-steroidal antiandrogen, specifically, a full and high-affinity antagonist of the androgen receptor (AR), that is under development by Orion and Bayer HealthCare for the treatment of advanced, castration-resistant prostate cancer (CRPC). ODM-201 appears to negligibly cross the blood-brain-barrier. This is beneficial due to the reduced risk of seizures and other central side effects from off-target GABAA receptor inhibition that tends to occur in non-steroidal antiandrogens that are structurally similar to enzalutamide. Moreover, in accordance with its lack of central penetration, ODM-201 does not seem to increase testosterone levels in mice or humans, unlike other non-steroidal antiandrogens. Another advantage is that ODM-201 has been found to block the activity of all tested/well-known mutant ARs in prostate cancer, including the recently-identified clinically-relevant F876L mutation. ODM-201 has been studied in phase I and phase II clinical trials and has thus far been found to be effective and well-tolerated, with the most commonly reported side effects including fatigue, nausea, and diarrhea. No seizures have been observed.