Naltalimide (TRK-130), a mu opioid receptor partial agonist, is being developed by Toray Industries for the treatment of overactive bladder. A phase IIb trial is underway in Japan. In radioligand-binding assays with cells expressing human µ-opioid receptors (MORs), δ-opioid receptors (DORs), or κ-opioid receptors (KORs), TRK-130 showed high selectivity for MORs (Ki for MORs, DORs, and KORs = 0.268, 121, and 8.97 nM, respectively). TRK-130 is a potent and selective human MOR partial agonist without undesirable opioid adverse effects such as constipation and enhances the storage function by suppressing the afferent limb of the micturition reflex pathway.

Sapanisertib is an oral dual inhibitor of mTORC1/mTORC2, discovered by Intellikine for the treatment of cancer. The drug is being tested in phase II of clinical trials for different cancers among which are sarcoma, hepatocellular carcinoma, etc. The drug is currently developed by Takeda with breast cancer, renal cancer and endometrial cancer being the main target indications.

MK-2048, diketo acid derivative, is a second-generation integrase strand transfer inhibitor (INSTI) developed to retain activity against HIV containing mutations associated with resistance to first-generation INSTIs, raltegravir (RAL) and elvitegravir (EVG). MK-2048 implements its inhibitory mechanism by modifying viral integrase-DNA interactions, the important step of the linear HIV-1 cDNA integration into the host genome. It binds to and inactivates the synaptic complex, an intermediate in the concerted integration pathway in vitro thereby preventing target DNA binding and concerted integration. MK-2048 is active against viruses resistant to RAL and EVG. MK-2048 is equally potent against wild-type IN and raltegravir-resistant IN mutant N155H with a low IC50 value of 42 nM for inhibiting concerted integration. It inhibits R263K mutants slightly more effectively than G118R mutants. MK-2048 exposure leads to the selection of G118R as a possible novel resistance mutation.

Giminabant is a cannabinoid receptor antagonist for the treatment of obesity.

Oprozomib (PR-047) is an orally bioavailable derivative of carfilzomib, with similar biological activity, i.e. inhibition of the chymotrypsin-like activity of the proteasome. It inhibits the activity of the proteasome, thereby blocking the targeted proteolysis normally performed by the proteasome; this may result in an accumulation of unwanted or misfolded proteins. Disruption of various cell signaling pathways may follow, eventually leading to the induction of apoptosis and inhibition of tumor growth. Oprozomib (PR-047) is being investigated for the treatment of hematologic malignancies, specifically, multiple myeloma, with Phase I/II trial ongoing.

Mafodotin (mc-MMAF) is a hydrophilic non-cleavable antimicrotubule and antimitotic agent, monomethyl auristatin F (MMAF) derivative, having a maleimidocaproyl linker (mc linker), which is ready to conjugate to an antibody or other proteins or biopolymers. Antibody-drug conjugates having mafodotin such as Depatuxizumab mafodotin and Belantamab mafodotin have undergone clinical trials for the treatment of neoplasms.

TT-301 is a small molecule compound that has demonstrated efficacy in preclinical models of rheumatoid arthritis, intracerebral haemorrhage and traumatic brain injury. In preclinical studies, TT-301 has been shown to act by inhibiting the overproduction of inflammatory cytokines, such as interleukin-1 (IL-1Beta), tumour necrosis factor-alpha (TNF-Alpha), a key disease process associated with many inflammatory and degenerative diseases. TT-301 was designed to cross the blood-brain-barrier and has the flexibility to be administered by injection or orally. In preclinical studies to date, TT-301 has shown a favorable safety profile and a significant therapeutic benefit in animal models of disease. TT-301 has been used in trials studying the treatment of traumatic brain injury.