Drug Pipeline

14 drugs associated with Rett syndrome

DrugPhaseSponsor
Esketamine hydrochloride

Esketamine, the S-enantiomer of racemic ketamine, is a non-selective, non-competitive antagonist of the N -methyl- D -aspartate (NMDA) receptor, an ionotropic glutamate receptor. The mechanism by which esketamine exerts its antidepressant effect is unknown. The major circulating metabolite of esketamine (noresketamine) demonstrated activity at the same receptor with less affinity.

Phase 1Children's Hospital of Fudan University
ANAVEX2-73
Phase 2Anavex Life Sciences Corp.
Dextromethorphan

Dextromethorphan is a low-affinity uncompetitive NMDA antagonist and sigma-1 receptor agonist. It is also an antagonist of α3/β4 nicotinic receptors. However, the mechanism by which dextromethorphan's receptor agonism and antagonism translate to a clinical effect is not well understood.

Phase 2Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
EPI-743
Phase 2Edison Pharmaceuticals Inc
mirtazapine

**Summary** The mechanism of action of mirtazapine is not fully understood but may be explained by its effects on central adrenergic and serotonergic activity. This drug exhibits a fast onset of action, a high level of response, a manageable side-effect profile, and dual noradrenergic and serotonergic effects that are unique from the effects of other antidepressants. **Effects on various receptors** It has been shown that both noradrenergic and serotonergic activity increase following mirtaza

Phase 2University of Trieste
triheptanoin

Triheptanoin is a source of heptanoate fatty acids, which can be metabolized without the enzymes of long chain fatty acid oxidation. In clinical trials, patients with lc-FAODs treated with triheptanoin experienced improvements in hypoglycemia, cardiomyopathy, and rhabdomyolysis.

Phase 2Center for Rare Neurological Diseases, Norcross, GA
Bionetide
Phase 3Biomed Industries, Inc.
GWP42003-P

The exact mechanism of action of CBD and THC is not currently fully understood. However, it is known that CBD acts on cannabinoid (CB) receptors of the endocannabinoid system, which are found in numerous areas of the body, including the peripheral and central nervous systems, including the brain. The endocannabinoid system regulates many physiological responses of the body including pain, memory, appetite, and mood. More specifically, CB1 receptors can be found within the pain pathways of the br

Phase 3Jazz Pharmaceuticals
NTI164
Phase 3Fenix Innovation Group
risperidone

Though its precise mechanism of action is not fully understood, current focus is on the ability of risperidone to inhibit the D2 dopaminergic receptors and 5-HT2A serotonergic receptors in the brain. Schizophrenia is thought to result from an excess of dopaminergic D2 and serotonergic 5-HT2A activity, resulting in overactivity of central mesolimbic pathways and mesocortical pathways, respectively. D2 dopaminergic receptors are transiently inhibited by risperidone, reducing dopaminergic neurotra

Phase 3Janssen-Ortho Inc., Canada
Sarizotan high dose

Serotonin 1a (5-HT1a) receptor agonist; Dopamine D2 receptor partial agonist; Dopamine D3 receptor agonist; Dopamine D4 receptor agonist

Phase 3Newron Pharmaceuticals SPA
Sarizotan low dose

Serotonin 1a (5-HT1a) receptor agonist; Dopamine D2 receptor partial agonist; Dopamine D3 receptor agonist; Dopamine D4 receptor agonist

Phase 3Newron Pharmaceuticals SPA
Trofinetide

Most cases of Rett syndrome are associated with loss-of-function mutations in a gene encoding methyl CpG binding protein 2 (MECP2), a DNA binding protein with a role in epigenetic regulation of gene expression. These mutations are believed to lead to synaptic immaturation in the cortex, aberrant metabolism of brain cholesterol resulting in abnormal neuronal development, and abnormal neuronal signaling. Rett syndrome more commonly occurs in girls than boys. The exact mechanism of action of trofi

Phase 3ACADIA Pharmaceuticals Inc.
TSHA-102
Phase 3Taysha Gene Therapies, Inc.