KL1333
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|---|---|
| Legal status | |
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| E number | {{#property:P628}} |
| ECHA InfoCard | {{#property:P2566}} |
KL1333 is an investigational NAD+ modulator developed by the South Korean pharmaceutical company Yungjin Pharm. In 2017, Abliva (previously NeuroVive Pharmaceutical) was granted exclusive rights to develop and commercialize KL1333 globally, except in Korea and Japan where Yungjin Pharm retains its exclusive rights.[1]
Research[edit]
KL1333 appears to upregulate the mitochondrial regulator PGC-1α, possibly mediated by SIRT1 and AMPK.
In some cell lines, including MELAS fibroblasts, KL1333 has been shown to induce the activation of the SIRT1/AMPK/PGC-1α signaling network through NQO1-mediated oxidation of NADH to NAD+, significantly increasing the NAD+/NADH ratio. It exhibits higher activity toward NQO1 than idebenone. In cell-free enzyme assays, KL1333 completely converted NADH to NAD+.[2]
Regulatory status[edit]
On April 18th 2018 it was designated an orphan drug for treatment of inherited mitochondrial respiratory chain diseases by the FDA. In the EU, Orphan Drug Designation has been obtained for MELAS syndrome.[1]
References[edit]
- ↑ 1.0 1.1 "NeuroVive's KL1333 Receives FDA Orphan Drug Designation for Treatment of Mitochondrial Diseases" (Press release). Lund, Sweden: NeuroVive Pharmaceutical. April 18, 2018. Retrieved September 5, 2018.
- ↑ Seo, Kang-Sik; et al. (2018). "KL1333, a Novel NAD+ Modulator, Improves Energy Metabolism and Mitochondrial Dysfunction in MELAS Fibroblasts". Frontiers in Neurology. 9: 552. doi:10.3389/fneur.2018.00552. PMC 6041391. PMID 30026729.
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