T7117

Sapropterin dihydrochloride (6R-BH4 dihydrochloride) is phenylalanine hydroxylase agonist that is approved for the treatment of BH4 responsive PKU.

Dersimelagon (MT-7117) is an orally active, selective melanocortin 1 receptor (MC1R) agonist with EC50 values ​​of 8.16, 3.91, 1.14, and 0.251 nM for human (h), cynomolgus monkey (cm), mouse (m), and rat (r) MC1R, respectively. Dersimelagon exhibits good affinity for hMC1R and hMC4R, with Ki values ​​of 2.26 and 32.9 nM, respectively. Dersimelagon can be used in the study of skin pigmentation[1].

Dersimelagon phosphate is a selective melanocortin-1 receptor (MC1R) agonist that increases skin melanin without sun exposure and is being developed to increase light tolerance in EPP/XLP patients.

Pegcantratinib is a tropomyosin receptor kinase inhibitor for treatment for inherited CYLD defective skin tumours.

PBT434 is a novel, brain-penetrant, small molecule inhibitor of α-synuclein aggregation. In transgenic animal models of Parkinson disease (A53T) and MSA (PLP-α-Syn), PBT434 reduced α-synuclein aggregation, preserved neurons and improved motor function. Glial cell inclusions were also reduced in a murine MSA model. PBT434 is thought to act by redistributing reactive iron across membranes, thereby blocking intracellular protein aggregation and oxidative stress. The affinity of PBT434 for iron is greater than that of α-synuclein but lower than that of iron trafficking proteins, e.g., ferritin.