Chemistry:Soticlestat
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| Other names | TAK-935; OV-935 |
| Routes of administration | By mouth |
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| Formula | C23H23N3O2 |
| Molar mass | 373.456 g·mol−1 |
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Soticlestat (TAK-935, OV-935) is an experimental anticonvulsant and cholesterol 24-hydroxylase inhibitor being investigated as a treatment for Dravet syndrome, Lennox–Gastaut syndrome, tuberous sclerosis complex, dup15q syndrome, and CDKL5 deficiency disorder.[1][2] The development rights to the drug were purchased by Takeda Pharmaceuticals from Ovid Therapeutics in 2021.[3]
Soticlestat was designated as an orphan drug by the FDA in 2017 for the treatment for both Dravet syndrome and Lennox–Gastaut syndrome.[4] In a phase 2 study called ELEKTRA, soticlestat was well tolerated and reduced seizure frequency in patients with Dravet syndrome and Lennox–Gastaut syndrome.[5]
Mechanism of action
Soticlestat functions by blocking cholesterol 24-hydroxylase (CH24H, also known as CYP46A1), an enzyme in the brain that converts cholesterol to the oxysterol 24S-hydroxycholesterol (24S-HC). Reduction of 24S-HC has been shown to reduce glutamatergic signaling, which reduces seizures.[1][2] Soticlestat may also have neuroprotective and anti-inflammatory properties via glial cell modulation.[2]
References
- ↑ 1.0 1.1 "CDKL5 Deficiency Disorder-Related Epilepsy: A Review of Current and Emerging Treatment". CNS Drugs 36 (6): 591–604. June 2022. doi:10.1007/s40263-022-00921-5. PMID 35633486.
- ↑ 2.0 2.1 2.2 "Epilepsy: expert opinion on emerging drugs in phase 2/3 clinical trials". Expert Opinion on Emerging Drugs 27 (1): 75–90. March 2022. doi:10.1080/14728214.2022.2059464. PMID 35341431.
- ↑ "Takeda buys epilepsy treatment rights from Ovid for up to $856 million". 3 March 2021. https://www.reuters.com/article/us-takeda-pharma-ovid/takeda-buys-epilepsy-treatment-rights-from-ovid-for-up-to-856-million-idUSKBN2AV1C9.
- ↑ Pena, Ana (22 October 2019). "Soticlestat Can Reduce Seizure Frequency in Adults With Dravet, Other Rare Epilepsies, Initial Data Shows". https://dravetsyndromenews.com/news/soticlestat-can-reduce-seizure-frequency-dravet-syndrome-other-rare-epilepsies/.
- ↑ "A phase 2, randomized, double-blind, placebo-controlled study to evaluate the efficacy and safety of soticlestat as adjunctive therapy in pediatric patients with Dravet syndrome or Lennox–Gastaut syndrome (ELEKTRA)". Epilepsia 63 (10): 2671–2683. October 2022. doi:10.1111/epi.17367. PMID 35841234.
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