Emideltide or delta-sleep-inducing peptide (Trp-Ala-Gly-Gly-Asp-Ala-Ser-Gly-Glu) is a nonapeptide originally isolated from rabbit cerebral venous blood. It promotes sleep and this has been demonstrated in rabbits, mice, rats, cats and human beings. Emideltide showed significant and specific enhancement/induction of delta and spindle electroencephalogram patterns. In addition to sleep, the peptide also has been observed to affect electrophysiological activity, neurotransmitter levels in the brain, circadian and locomotor patterns, hormonal levels, psychological performance, and the activity of neuropharmacological drugs including their withdrawal.

IRL-1620 is an endothelin-B receptor agonist with the potential neuroprotective effect. A Phase II clinical study with IRL-1620 has been initiated in patients with cerebral ischemia and mild to moderate Alzheimer’s disease.

Chlorotoxin is a disulfide-rich stable peptide from the venom of the Israeli scorpion Leiurus quinquestriatus, which has potential therapeutic applications in the treatment of cancer. Chlorotoxin is the first reported high-affinity peptide blocker for small conductance chloride channels. Chlorotoxin is able to penetrate blood-brain-barrier and binds preferentially to tumor cells, making the design of therapeutics using chlorotoxin as a scaffold to treat glioma and other types of cancer of neuroectodermal origins such as melanoma, neuroblastoma, and medulloblastoma possible. To date, no obvious toxicities nor immunogenic responses after being administered to humans have been reported

Paclitaxel trevatide (formerly known as ANG 1005 or GRN 1005) is a paclitaxel-peptide drug conjugate, where three paclitaxel molecules linked by a cleavable succinyl ester linkage to a brain peptide vector, Angiopep-2. Paclitaxel trevatide is an oncology product to leverage the low-density lipoprotein receptor-related protein 1 (LRP-1) pathway to cross the blood-brain barrier (BBB) and enter cancer cells. This drug successfully completed phase II clinical trials in breast cancer patients with recurrent brain metastases and in patients with high-grade glioma. 30-May-2014 Angiochem, the company that developed this drug, announced that the Food & Drug Association (FDA) had granted both orphan drug and fast track designation to paclitaxel trevatide for the treatment of glioblastoma multiforme (GBM). In addition, paclitaxel trevatide is going to be involved in phase III clinical trial to see if this drug can prolong survival compared to a Physician Best Choice control in HER2-negative breast cancer patients with the newly diagnosed leptomeningeal disease and previously treated brain metastases.

Orphanin FQ (OFQ, Nociceptin) is a recently discovered 17-amino acid neuropeptide that is structurally related to the opioid peptides but does not bind opioid receptors. OFQ selectively binds its own receptor (OFQR), which is also sequentially related to the opioid receptors, yet does not bind opioid ligands. OFQ has been proposed to act as an anti-opioid peptide, but its widespread sites of action in the brain suggest that it may have more general functions. Nociceptin acts as an anxiolytic to attenuate behavioral responses to stress. In vivo experiments have demonstrated that Nociceptin modulates a variety of biological functions ranging from nociception to food intake, from memory processes to cardiovascular and renal functions, from spontaneous locomotor activity to gastrointestinal motility, from anxiety to the control of neurotransmitter release at peripheral and central sites. Nociceptin has being shown to directly inhibit an adaptive immune response, i.e., antibody formation, both in vitro and in vivo.

Angiotensin III (Ang III) is a bioactive heptapeptide that is formed from the degradation of the Angiotensin II peptide by aminopeptidase A. In peripheral Angiotensin systems, Angiotensin II is the main effector peptide in the systemic circulation, although exogenous Angiotensin III can be as potent as Angiotensin II in, for example, stimulating aldosterone secretion or inhibiting renin release. In the rat brain, Angiotensin III was found to be equipotent with Angiotensin II as a pressor agent or dipsogen and was bound as avidly to the nervous system as Angiotensin II. Angiotensin receptor subtype AT1 has the greater affinity towards Angiotensin II and is also responsive to Angiotensin III, while the AT2 receptor subtype appears to be more sensitive to Angiotensin III but less responsive to Angiotensin II. Angiotensin III enhances blood pressure, vasopressin release and thirst when it is centrally administrated. Angiotensin III infusion increases blood pressure in healthy volunteers and hypertensive patients as well as augments aldosterone release. Although Angiotensin III does not change renal function in humans, it induces natriuresis in AT, receptor-blocked rats likely by binding to AT2 receptors. In addition, in cultured renal cells, this peptide stimulates the expression of many growth factors, proinflammatory mediators, and extracellular matrix proteins.

Saffron is a spice derived from the flower of Crocus sativus, commonly known as the "saffron crocus". Crocin is a water-soluble carotenoid pigment of saffron,that has been used as a spice for flavoring and coloring food preparations, and in Chinese traditional medicine as an anodyne or tranquilizer. Saffron is now used worldwide in folk medicine and is reputed to be useful in treating various human disorders such as heart and blood disorders. Stroke and heart attack are involved in reputed folkloric uses of saffron. Saffron is orally administrated as a decoction. Saffron extract exerts a protective effect on renal ischemia reperfusion induced oxidative damage in rats. Crocin suppresses tumor necrosis factor (TNF) alpha-induced apoptosis of pheochromocytoma (PC12) cells by modulating mRNA expressions of Bcl-2 family proteins, which trigger downstream signals culminating in caspase-3 activation followed by cell death. Depriving cultured PC12 cells of serum/glucose causes a rapid increase in cellular ceramide levels, followed by an increase in the risk of cell death. The accumulation of ceramide was found to depend on the activation of neutral sphingomyelinase (nSMase). Crocin prevented the activation of nSMase by enhancing the transcription of gamma-glutamylcysteinyl synthase, which contributes to a stable glutathione supply that blocks the activity of nSMase.

Arimoclomol citrate is an experimental drug developed by a biopharmaceutical company CytRx Corporation. In 2011 the worldwide rights to arimoclomol were bought by Danish biotech company Orphazyme ApS. The European Medicines Agency (EMA) and U.S. Food & Drug Administration (FDA) granted orphan drug designation to arimoclomol as a potential treatment for Niemann-Pick type C in 2014 and 2015 respectively. Arimoclomol is believed to function by stimulating a normal cellular protein repair pathway through the activation of molecular chaperones. Since damaged proteins, called aggregates, are thought to play a role in many diseases, CytRx believes that arimoclomol could treat a broad range of diseases.

Arimoclomol citrate is an experimental drug developed by a biopharmaceutical company CytRx Corporation. In 2011 the worldwide rights to arimoclomol were bought by Danish biotech company Orphazyme ApS. The European Medicines Agency (EMA) and U.S. Food & Drug Administration (FDA) granted orphan drug designation to arimoclomol as a potential treatment for Niemann-Pick type C in 2014 and 2015 respectively. Arimoclomol is believed to function by stimulating a normal cellular protein repair pathway through the activation of molecular chaperones. Since damaged proteins, called aggregates, are thought to play a role in many diseases, CytRx believes that arimoclomol could treat a broad range of diseases.

Zavegepant is a third generation, small-molecule, calcitonin gene-related peptide (CGRP) receptor antagonist being developed by Pfizer, under a license from Bristol-Myers Squibb, for the prevention and treatment of chronic and episodic migraine. In March 2023, zavegepant nasal spray (ZAVZPRET™) received its first approval in the USA for the acute treatment of migraine with or without aura in adults, based on two randomized, double-blind, placebo-controlled studies. Clinical development of an oral formulation of zavegepant is currently underway.