Ganaxolone (CCD-1042; C-1042; Ztalmy) is a novel, CNS-active and potent GABAA modulator. As of March 2022, the US FDA has approved Ztalmy (ganaxolone) to treat seizures associated with cyclin-dependent kinase-like 5 (CDKL5) deficiency disorder (CDD) in patients 2 years of age and older. This is the first treatment for seizures associated with CDD and the […]

NCGC 84 (ML-154) is a novel, selective and brain penetrant NPS receptor antagonist. NCGC 84 blocks alcohol-induced ERK-phosphorylation in the central amygdala and decreases operant alcohol self-administration in rats.

Tinostamustine HCl (formerly known as EDO-S101 and Minomustine), the hydrochloride salt of Tinostamustine, is a dual alkylating agent and histone-deacetylase inhibitor (HDACi) and a fusion molecule. EDO-S101 is a functional pan-histone-deacetylase inhibitor and is assumed to potentiate the alkylating activity of the compound and/or may help to overcome resistance to other therapeutic agents.

Olodanrigan sodium (EMA401; PD126055; EMA-401), a novel, orally bioavailable, peripherally restricted and highly selective AT2R (angiotensin II type 2 receptor) antagonist, can be potentially used for treatment of postherpetic neuralgia (PHN). Inhibition of augmented AngII/AT2R induced p38 and p42/p44 MAPK activation, and hence inhibition of DRG neuron hyperexcitability and sprouting of DRG neurons. It is […]

Alloimperatorin is a novel and potent coumarin compound

LNT 1 is a potent flap endonuclease 1 (FEN1) inhibitor that induces DNA damage response.

TC-AQP1-1 is a novel and potent aquaporin 1 (AQP1) channel blocker.

Uridine triacetate (PN-401; RG 2133; Vistogard; Vistonuridine; Xuriden; Triacetyluridine; TAU), a prodrug of uridine, is an approved medication used for treatment of hereditary orotic aciduria or to treat patients following an overdose of chemotherapy drugs 5-fluorouracil or capecitabine. The acetyl groups of Vistonuridine increase the lipophilicity of uridine, thus enhancing its transport from the gastrointestinal […]

Warfarin sodium (trade names Coumadin among others) is an anticoagulant normally used in the prevention of thrombosis and thromboembolism, the formation of blood clots in the blood vessels and their migration elsewhere in the body, respectively. It is commonly used to treat blood clots such as deep vein thrombosis and pulmonary embolism and to prevent […]

Ciliobrevin D is a novel and potent AAA+ ATPase motor cytoplasmic dynein inhibitor, also inhibits Hedgehog (Hh) signaling and primary cilia formation and dynein-dependent microtubule gliding and ATPase activity in vitro.