Lifirafenib (BGB-283 or BEIGENE-283), a dual inhibitor targeting BRAF kinase and EGFR, was studied for the treatment of different cancer types harboring mutations in BRAF, KRAS, and NRAS. Lifirafenib participates in Phase II clinical trial in combination with PD-0325901 to treat the patients with solid tumor.

CUDC-907 is a small molecule inhibitor of histone deacetylase and PI3 kinase developed by Curis. It is investigated in clinical trials for the treatment of relapsed or refractory lymphomas, thyroid cancer, multiple myeloma, breast cancer and other malignancies.

Tomivosertib (eFT-508) is a dual inhibitor of MAP kinase-interacting kinases (MNK1 and MNK2) thereby controlling translation. It has potent in vivo antitumor activity in models of diffuse large cell B-cell lymphoma and solid tumors. Tomivosertib acts on multiple points in the cancer immunity cycle simultaneously, underscoring its therapeutic potential as both a monotherapy and in combination with existing checkpoint inhibitor treatments. Preclinical studies suggest combining tomivosertib with a checkpoint inhibitor can overcome mechanisms of resistance to checkpoint inhibitors, resulting in enhanced sensitivity to checkpoint inhibitors and a higher response rate. In addition, preclinical data demonstrate that tomivosertib as a single agent promotes antitumor immunity that persists after stopping drug treatment. Tomivosertib is currently being evaluated in Phase 2 clinical trials in solid tumors and lymphoma. Additionally, tomivosertib may be a new therapeutic for neuropathic pain.

PQR-309 is an orally bioavailable pan inhibitor of phosphoinositide-3-kinases (PI3K) and inhibitor of the mammalian target of rapamycin (mTOR), with potential antineoplastic activity. PI3K/mTOR kinase inhibitor PQR-309 inhibits the PI3K kinase isoforms alpha, beta, gamma and delta and, to a lesser extent, mTOR kinase, which may result in tumor cell apoptosis and growth inhibition in cells overexpressing PI3K/mTOR. By inhibiting mTOR to a lesser extent than PI3K, PQR-309 does not interfere with the mTOR-mediated negative feedback loop on PI3K signaling. Blocking the negative feedback loop would potentially increase PI3K signaling and decrease therapeutic efficacy. PQR-309 is in phase II clinical trial for the treatment of glioblastoma, head and neck cancer, lymphoma and breast cancer. Common adverse events included fatigue, hyperglycemia, nausea, diarrhea, constipation, rash, anorexia and vomiting.

AV608, a 4-aminopiperidine derivative, is a selective, specific, long-acting, orally active and potent nonpeptidic antagonist of the NK-1 receptor. AV-608 had been in phase II clinical trials for the treatment of social phobia and overactive bladder (OAB). This compound was originally discovered by Novartis, and then licensed to Areva Pharmaceuticals in October 2003. Addition this drug was in phase I clinical trial for the treatment of Irritable Bowel Syndrome (IBS), this disease is characterized by chronic abdominal pain and frequent comorbid anxiety. The substance P ⁄ neurokinin-1 receptor system is implicated in the regulation of both pain and anxiety, suggesting a potential therapeutic target in IBS. However, the researches on this drug candidate were discontinued in 2010.

KOSN-1724, also known as iso-fludelone, is the third-generation epothilone B analogue. It binds to tubulin and induces microtubule polymerization/reduces microtubule depolymerization, resulting in the inhibition of cell division and induction of apoptosis. KOSN-1724 is in phase I clinical trials for the treatment of solid tumors.

Vintiamol was developed as a new form of vitamin B1. Information about the current use of this agent is not available.

Acetiamine (syn. thianeurone or diacetamine) is a thiamine derivative which is rarely used in pharmaceuticals. It is lipid-soluble. It has been studied for the treatment of rheumatic diseases.