Drug Pipeline
19 drugs associated with Angelman Syndrome
| Drug | Phase | Sponsor | |
|---|---|---|---|
Circadin 10 mg | Phase 1 | Neurim Pharmaceuticals Ltd. | ↗ |
Circadin 2 mg | Phase 1 | Neurim Pharmaceuticals Ltd. | ↗ |
OV101 | Phase 1 | Healx AI | ↗ |
RO7248824 | Phase 1 | Hoffmann-La Roche | ↗ |
Alogabat | Phase 2 | Hoffmann-La Roche | ↗ |
EPI-743 | Phase 2 | Edison Pharmaceuticals Inc | ↗ |
GTX-102 Estrogen receptor alpha agonist | Phase 2 | Ultragenyx Pharmaceutical Inc | ↗ |
MVX-220 | Phase 2 | MavriX Bio, LLC | ↗ |
NNZ-2591 Insulin-like growth factor 1 (IGF-1) is a hormone secreted by glia and neurons of the CNS that plays an important role in normal brain development and response to pathologies. It is broken down into two separate molecules in the brain, both of which have synthetic analogues in development. [Glypromate], or GPE, comprises the last three peptides of IGF-1 and acts on glial cells, in contrast to its parent compound which tends to act on neurons - a synthetic analogue, [trofinetide], is currently un | Phase 2 | Neuren Pharmaceuticals Limited | ↗ |
Circadin 2/5/10 mg | Phase 3 | Neurim Pharmaceuticals Ltd. | ↗ |
Gaboxadol | Phase 3 | Healx AI | ↗ |
ION582 | Phase 3 | Ionis Pharmaceuticals, Inc. | ↗ |
Levodopa Levodopa by various routes crosses the blood brain barrier, is decarboxylated to form dopamine. This supplemental dopamine performs the role that endogenous dopamine cannot due to a decrease of natural concentrations and stimulates dopaminergic receptors. | Phase 3 | Wen-Hann Tan | ↗ |
Betaine Homocystinuria is a hereditary disorder characterized by high levels of the amino acid homocysteine. This condition can be caused by deficiencies or defects in cystathionine beta-synthase (CBS), 5,10-methylenetetrahydrofolate reductase (MTHFR), and cobalamin cofactor metabolism (cbl). CBS converts homocysteine to cystathionine, and a deficiency in this enzyme can lead to an accumulation of homocysteine. MTHFR is responsible for producing 5-methyltetrahydrofolate, a methyl donor that participates | unknown | University of California, San Diego | ↗ |
Creatine In the muscles, a fraction of the total creatine binds to phosphate - forming creatine phosphate. The reaction is catalysed by creatine kinase, and the result is phosphocreatine (PCr). Phosphocreatine binds with adenosine diphosphate to convert it back to ATP (adenosine triphosphate), an important cellular energy source for short term ATP needs prior to oxidative phosphorylation. | unknown | University of California, San Diego | ↗ |
Metafolin | unknown | University of California, San Diego | ↗ |
minocycline mechanism of action The tetracyclines are primarily bacteriostatic and are thought to exert their antimicrobial effect by the inhibition of protein synthesis. The tetracyclines, including minocycline, have a similar antimicrobial spectrum of activity against a wide range of gram-positive and gram-negative organisms. Cross-resistance of these organisms to tetracycline is common. | unknown | University of South Florida | ↗ |
Rugonersen | unknown | Oak Hill Bio Ltd | ↗ |
Vitamin B12 | unknown | University of California, San Diego | ↗ |