Synonyms: OV935 | TAK-935
Compound class:
Synthetic organic
Comment: Soticlestat (TAK-935, previously OV935) is a first-in-class, clinical stage, orally administered cholesterol 24-hydroxylase (CYP46A1) inhibitor. It is being investigated as a novel anti-epileptic modality for pediatric forms of epilepsy [3], such as Dravet syndrome [1,4] and Lennox-Gastaut syndrome [5]. Cholesterol 24-hydroxylase is a brain-specific enzyme (in humans) that converts cholesterol to 24S-hydroxycholesterol. 24S-hydroxycholesterol acts as a positive allosteric modulator of N-methyl-D-aspartate (NMDA) receptors. Soticlestat blocks 24S-hydroxycholesterol production in the brain in an in vivo mouse model [2].
![]() Ligand Activity Visualisation ChartsThese are box plot that provide a unique visualisation, summarising all the activity data for a ligand taken from ChEMBL and GtoPdb across multiple targets and species. Click on a plot to see the median, interquartile range, low and high data points. A value of zero indicates that no data are available. A separate chart is created for each target, and where possible the algorithm tries to merge ChEMBL and GtoPdb targets by matching them on name and UniProt accession, for each available species. However, please note that inconsistency in naming of targets may lead to data for the same target being reported across multiple charts. ✖ |
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No information available. |
Summary of Clinical Use ![]() |
The first report of soticlestat's safety, tolerability, pharmacokinetics, pharmacodynamics and bioavailability (in healthy volunteers) was published in 2021 [6]. It has been progressed to larger clinical trials to determine efficacy in a range of developmental and/or epileptic encephalopathies. |
Clinical Trials | |||||
Clinical Trial ID | Title | Type | Source | Comment | References |
NCT02906813 | A Study to Determine the Bioavailability and Food Effect of a Single TAK-935 Dose in Healthy Participants | Phase 1 Interventional | Takeda | 6 | |
NCT02201056 | Safety, Tolerability, and Pharmacokinetics of Escalating Single Doses of TAK-935 | Phase 1 Interventional | Takeda | 6 | |
NCT03650452 | A Phase 2, Multicenter, Randomized, Double-blind, Placebo-controlled Study to Evaluate the Efficacy, Safety, and Tolerability of TAK-935 (OV935) as an Adjunctive Therapy in Pediatric Participants With Developmental and/or Epileptic Encephalopathies | Phase 2 Interventional | Takeda | ||
NCT03635073 | A Phase 2, Prospective, Interventional, Open-Label, Multi-Site, Extension Study to Assess the Long-Term Safety and Tolerability of TAK-935 (OV935) as Adjunctive Therapy in Patients With Rare Epilepsy | Phase 2 Interventional | Takeda | ||
NCT03166215 | Study of TAK-935 as an Adjunctive Therapy in Participants With Developmental and/or Epileptic Encephalopathies | Phase 1/Phase 2 Interventional | Takeda | ||
NCT03990649 | Study of TAK-935 as an Adjunctive Therapy in Adult Participants With Complex Regional Pain Syndrome (CRPS) | Phase 2 Interventional | Takeda |