Cethromycin is a ketolide antibiotic derived from erythromycin A being investigated for use in community-acquired pneumonia and other respiratory tract infections. Cethromycin possesses reliable activity against the bacteria most commonly associated with community-acquired pneumonia including S. pneumoniae, H. influenzae, M. catarrhalis, M. pneumoniae, C. pneumoniae, and L. pneumophila. Unlike fluoroquinolones, cethromycin has a narrower spectrum of activity against gram-negative bacteria, which may reduce the risk of collateral damage and the incidence of Clostridium difficile infection. It offers an advantage over telithromycin in that hepatotoxicity does not seem to be a concern. The FDA denied approval of cethromycin for the treatment of CAP in 2009, requesting more efficacy data.

Galunisertib is a potent inhibitor of TGF beta type 1 receptor. The drug is under clinical development for the treatment of different cancers: pancreatic, hepatocellular, breast, rectal, prostate etc. and reached phase 2/3 in patients with myelodysplastic syndromes.

Vedaclidine [LY 297802, NNC 101053, NNC 111053, butylthio[2.2.2]], a quinuclidine, is a muscarinic receptor agonist being developed by Novo Nordisk and Eli Lilly as an analgesic drug. Vedaclidine is a muscarinic receptor ligand which is equiefficacious to morphine in producing antinociception. In vitro, Vedaclidine had high affinity for muscarinic receptors in brain homogenates, but had substantially less or no affinity for several other neurotransmiter receptors and uptake sites. Its pharmacological profile suggests that it may have clinical utility in the management of pain as an alternative to opioids.

CX 516, a compound synthesized by Cortex Pharmaceuticals using Ampakine® technology licensed from the University of California, was in clinical investigations for the treatment of Alzheimer's disease, schizophrenia, fragile X syndrome and autism, and sleep disorders, however, development of this drug candidate has been discontinued. CX 516 had an extremely short halflife in humans, very low potency and failed to show any benefits in phase II studies. The compound did have a good safety profile, reducing concerns about the toxicity of excess glutamate. Cortex subsequently terminated clinical development of CX 516.

Formebolone is an orally active anabolic-androgenic steroid that is included in the list of prohibited substances by the World Anti-Doping Agency. Formebolone was used in Italy and Spain for infants with malnutrition.

Tigloidine is a tropane alkaloid and a naturally occurring analog of atropine, found in small quantities in Duboisia myoporoides. Tigloidine has been found to be beneficial in the treatment of Parkinsonism, Huntington’s Chorea and spastic paraplegia. Tigloidine may provide relief in parkinsonian patients by increasing the gamma-efferent activity and reducing alpha motoneurone activity. In preclinical models, Tigloidine failed to reverse sedation and ptosis in rats induced by reserpine and tetrabenazine. In mice, amphetamine response was not significantly affected by Tigloidine or atropine. However, in the cat and dog, it was markedly facilitated by Tigloidine but not by atropine.

Flindokalner (BMS 204352; MaxiPost™) is a neuroprotective agent with potential in the treatment of stroke developed by Bristol-Myers Squibb. Flindokalner is a potent and effective opener of two important subtypes of neuronal potassium channels, the calcium-activated, big-conductance potassium channels (K(Ca) channels) and voltage-dependent, non-inactivating potassium channels known as KCNQ channels. Flindokalner significantly reduced cortical infarct volume in a animal models of stroke. Flindokalner failed to show superior efficacy in acute stroke patients compared to placebo in a Phase III study.