U.S. Department of Health & Human Services Divider Arrow National Institutes of Health Divider Arrow NCATS

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Showing 11 - 20 of 47384 results

Nirogacestat (PF-3084014) is a tetralin imidazole gamma-secretase inhibitor. Gamma-secretase, a proteolytic enzyme complex, mediates processing of several integral membrane proteins including amyloid precursor protein and Notch. This compound can inhibit both Notch-related pathway in neoplasia and reduces amyloid-β production. Nirogacestat (PF-3084014) is under development by Pfizer for the treatment of cancer.

Class (Stereo):
CHEMICAL (ACHIRAL)


Conditions:

Mitapivat, also known as PKM2 activator 1020, is an activator of a pyruvate kinase PKM2, an enzyme involved in glycolysis. It was disclosed in a patent publication WO 2011002817 A1 as compound 78.

Class (Stereo):
CHEMICAL (ABSOLUTE)

Trofinetide (NNZ 2566), a proprietary small molecule analogue of glycine-proline-glutamate [Glypromate®], is being developed by Neuren Pharmaceuticals for the treatment of brain injuries, fragile X syndrome, Rett syndrome. Trofinetide is a synthetic analogue of a naturally occurring neurotrophic peptide derived from IGF-1, a growth factor produced by brain cells. In animal models, trofinetide exhibits a wide range of important effects including inhibiting neuroinflammation, normalizing the role of microglia and correcting deficits in synaptic function. Trofinetide is being developed both in intravenous and oral formulations for a range of acute and chronic conditions. The intravenous form of trofinetide is presently in a Phase 2 clinical trial in patients with moderate to severe traumatic brain injury. The oral form of trofinetide is in Phase 2 development in Rett syndrome, Fragile X syndrome and mild traumatic brain injury (concussion). Trofinetide has been granted Fast Track Status and Orphan Drug Designation in the U.S. and Orphan Drug Designation in Europe for both Rett syndrome and Fragile X syndrome.

Showing 11 - 20 of 47384 results