ADL5859 (or ADL-5859) is Adolor Corporation developed a novel, oral compound that targets the delta opioid receptor. Delta receptor agonists are thought to offer benefits over other approaches to the management of pain. ADL-5859 was in the phase II of clinical trial for the treatment of neuropathic pain, acute pain, and pain due to osteoarthritis of the knee and for patients with osteoarthritis. Further development of this drug as potential pain treatments was discontinued.

5-(hydroxymethyl)-2-furaldehyde (5-Hydroxymethyl-2-furancarboxaldehyde, 5‐hydroxymethyl‐2‐furfural) is found in garden onion. It is obtainable from various carbohydrates. 5-Hydroxymethyl-2-furancarboxaldehyde is present in tomatoes, tobacco oil etc. It is a constituent of numerous plant species. 5-Hydroxymethyl-2-furancarboxaldehyde is used as an index of heat treatment and deterioration in food such as tomato paste, honey and fruit juices. Also an indicator of adulteration with acid-converted invert sugars. The heterocyclic aldehyde 5‐hydroxymethyl‐2‐furfural (5HMF, Aes‐103, BAX 555) interacts allosterically with the abnormal form of haemoglobin (Hb), HbS, in red blood cells (RBCs) from patients with sickle cell disease (SCD), thereby increasing oxygen affinity and decreasing HbS polymerization and RBC sickling during hypoxia. 5HMF markedly reduced the deoxygenation-induced dehydration of RBCs whether in response to maintained deoxygenation or to cyclical deoxygenation/re-oxygenation. 5HMF was found to inhibit Psickle, an effect which correlated with its effects on sickling. Deoxygenation-induced activation of the Gardos channel and exposure of phosphatidylserine were also inhibited, probably indirectly via reduced entry of Ca(2+) through the Psickle pathway. Effects of 5HMF on KCC were more modest with a slight inhibition in N-ethylmaleimide (NEM, 1 mm)-treated RBCs and stimulation in RBCs untreated with NEM. These findings support the hypothesis that 5HMF may also be beneficial through effects on RBC ion and water homeostasis. AesRx is developing Aes-103 as an orally bioavailable, chronic therapy for SCD. Aes‐103 is currently in phase II clinical trials in SCD patients in the USA and UK. Aes-103 has received orphan designation from the US Food and Drug Administration and is eligible for orphan status in Europe.

Novartis Oncology (previously Novartis) is developing WNT-974 (formerly LGK 974), a first-in-class selective small molecule inhibitor of O-acyltransferase. WNT-974 is under development for the treatment of cancers that are driven by the Wnt pathway in a Wnt ligand-dependent manner. Upon oral administration, WNT-974 binds to and inhibits PORCN in the endoplasmic reticulum (ER), which blocks post-translational acylation of Wnt ligands and inhibits their secretion. This prevents the activation of Wnt ligands, interferes with Wnt-mediated signaling, and inhibits cell growth in Wnt-driven tumors. Porcupine, a membrane-bound O-acyltransferase (MBOAT), is required for the palmitoylation of Wnt ligands, and plays a key role in Wnt ligand secretion and activity. Wnt signaling is dysregulated in a variety of cancers

NB-001, an oil-in-water emulsion containing high energy nanometer-sized droplets stabilized by surfactants is designed for topical treatment of herpes labialis infections. NB-001 diffuses through the stratum corneum via the follicular route to accumulate in the epidermal and dermal tissues, without disrupting the normal epithelial matrix. The concentrations achieved in the epidermis and dermis are well above the concentrations required for antiviral activity.

Inspyr Therapeutics (formerly GenSpera) developed mipsagargin (previously known as G-202), as a novel thapsigargin-based targeted prodrug that is activated by prostate-specific membrane antigen (PSMA)-mediated cleavage of an inert masking peptide. The active moiety is an inhibitor of the sarcoplasmic/endoplasmic reticulum calcium adenosine triphosphatase (SERCA) pump protein that is necessary for cellular viability. Mipsagargin was granted Orphan Drug designation by the U.S. Food and Drug Administration (FDA) in 2013 for evaluation in patients with hepatocellular carcinoma. In addition, mipsagargin has been studied in phase 2 clinical trial in patients with recurrent or progressive glioblastoma, in patients with clear cell renal cell carcinoma that expresses PSMA. Mipsagargin is expected to be launched on the market in the coming years.