Lumateperone (ITI-722/ITI-007) is a dual 5HT2A receptor antagonist/dopamine phosphoprotein modulator (DPPM) for the treatment of schizophrenia. It is an orally available compound which combines potent 5HT2A receptor antagonism with cell-type-specific modulation of phosphoprotein pathways downstream of dopamine receptors. Lumateperone was developed by Intra-Cellular Therapies, Inc., and is being evaluated for the treatment of schizophrenia and bipolar depression. In 3 efficacy studies in patients with acute schizophrenia, lumateperone was well-tolerated with a favorable safety profile, and in 2 studies of 3 demonstrated significantly superior efficacy over placebo.

Selinexor (KPT-330) is a first in class XPO1 antagonist being evaluated in multiple later stage clinical trials in patients with relapsed and/or refractory hematological and solid tumor malignancies.

LEFAMULIN is a pleuromutilin antibiotic under development for the treatment of community-acquired bacterial pneumonia, as well as acute bacterial skin and skin structure infections. It inhibits bacterial protein synthesis by binding to the peptidyl transferase center of the 50S ribosome, resulting in the cessation of bacterial growth.

Entrectinib (previously known as RXDX-101, NMS-E628) is an investigational drug, potent inhibitor of ALK, ROS1, and, importantly, of TRK family kinases, which shows promise for therapy of tumors bearing oncogenic forms of these proteins. Entrectinib (RXDX-101) is a selective inhibitor for all three Trk receptor tyrosine kinases encoded by the three NTRK genes, as well as the ROS1 and ALKreceptor tyrosine kinases.This investigational drug is active at low nanomolar concentrations, allowing for once-daily oral administration to patients whose tumors have been shown to have gene rearrangements in NTRK, ROS1, or ALK. Nerviano Medical Sciences, the original sponsor for entrectinib (formerly referred to as NMS-1191372), initiated the first-in-human Phase 1 study ALKA-372-001 in Italy in October 2012. The study is currently ongoing in Italy. Entrectinib is currently being tested in a global phase 2 basket clinical trial called STARTRK-2. In the U.S., entrectinib has orphan drug designation and rare pediatric disease designation for the treatment of neuroblastoma and orphan drug designation for treatment of TrkA-, TrkB-, TrkC-, ROS1- and ALK-positive non-small cell lung cancer (NSCLC) and metastatic colorectal cancer (mCRC).

ADX-N05, originally discovered by SK Holdings, is a selective dopamine and norepinephrine reuptake inhibitor (DNRI). ADX-N05 (Solriamfetol, sold under the brand name Sunosi) is approved in the US and is under regulatory review in the EU to improve wakefulness in adult patients with hypersomnia associated with narcolepsy or obstructive sleep apnoea.The US FDA has approved solriamfetol (Sunosi, Jazz Pharmaceuticals) for the treatment of excessive daytime sleepiness in adults with narcolepsy or obstructive sleep apnea.The dual-acting dopamine and norepinephrine reuptake inhibitor is approved for narcolepsy in once-daily 75 mg and 150 mg doses, and in obstructive sleep apnea in once-daily 37.5 mg, 75 mg, and 150 mg doses.

Alpelisib (BYL719) is a PI3Kα-selective inhibitor. PI3K-AKT-mTOR pathway is frequently activated in cancer, therefore investigational PI3K inhibitor alpelisib is considered to be effective as an anticancer agent and has been in clinical development by Novartis. Alpelisib have demonstrated activity in preclinical models of solid tumors and had favorable tolerability profiles, with the most common adverse events consistent with “on-target” inhibition of PI3K in early clinical studies. There are ongoing clinical trials of alpelisib in a range of cancer types, including breast cancer, head and neck squamous cell carcinoma, non-small cell lung carcinoma, lymphoma, and glioblastoma multiforme. Combination therapy with other chemo therapeutics may be preferable.

Cantharidin is a toxic compound, isolated from the Spanish fly or blistering beetle (Lytta (Cantharis) vesicatoria) and other insects. It is a potent and specific inhibitor of protein phosphatases 1 (PP1) and 2A (PP2A). Cantharidin is a medication used to remove warts and a viral skin infection called molluscum contagiosum. It is made from the secretions that come from the green blister beetle in combination with salicylic acid. It works by creating a blister just below the wart, which pushes the wart up and away from the underlying tissue, cutting of the blood supply to the wart. As the blister and the wart dry out, they both slough off, leaving fresh, unmarred skin behind. It is also used as an experimental anti-tumor agent. Several studies also show potential novel applications of cantharidin in acquired perforating dermatosis, acute herpes zoster, and leishmaniasis. In 1962, cantharidin lost Food and Drug Administration (FDA) approval owing to the failure of its manufacturers to submit data attesting to cantharidin's efficacy. However, in 1999, the FDA included cantharidin on its “Bulk Substances List” of drugs which although not available as commercial products, were approved for compounding on a customized basis for individual patients.

Ubrogepant, a small molecule drug, is being developed by Merck & Co for the treatment of migraine. The calcitonin gene-related peptide receptor (CGRP) antagonist is administered orally as a film coated tablet. Ubrogepant is a competitive antagonist with high affinity, potency, and selectivity for the human CGRP receptor. In the four clinical studies (ACHIEVE I, ACHIEVE II, UBR-MD-04 and 3110-105-002) ubrogepant demonstrated efficacy, safety and tolerability in the acute treatment of migraine among a broad patient population, including those who had an insufficient response to a triptan or those patients in whom triptans were contraindicated, as well as in patients who had moderate to severe CV risk profile.

Tenapanor is an inhibitor of the sodium-proton (Na(+)/H(+)) exchanger NHE3 and reduces sodium absorption in the GI tract, thus increasing intestinal fluid. Ardelyx has completed Phase 3 development of tenapanor for the treatment of irritable bowel syndrome with constipation (IBS-C) and submitted a new drug application to the U.S. Food and Drug Administration for the treatment of patients with IBS-C. In addition, tenapanor successfully completed phase III clinical trial for the treatment of hyperphosphatemia in people with end-stage renal disease who are on dialysis and RDX013, a potassium secretagogue program for the potential treatment of high potassium, or hyperkalemia, a problem among certain patients with kidney and/or heart disease.