Drugs / Sonlicromanol
Trials 7
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 11 trial | ||||||
| Phase 1 | NCT02544217 | May → Sep 2015 | Leber hereditary optic neuropathy, Leigh syndrome, MELAS syndrome | Khondrion BV | Completed | No outcome recorded |
| Phase 25 trials | ||||||
| Phase 2 | NCT07298005 | Apr 2026 → Feb 2027 expected | post-COVID-19 disorder | Michele van Vugt | Recruiting | No outcome recorded |
| Phase 2 | NCT04604548 | Aug 2021 → Jun 2023 | MELAS syndrome, maternally-inherited diabetes and deafness, progressive external ophthalmoplegia | Khondrion BV | Completed | No outcome recorded |
| Phase 2 | NCT04846036 | Feb 2021 → Dec 2025 | MELAS syndrome, necrotizing encephalomyelopathy, subacute, of Leigh, adult | Khondrion BV | Suspended | No outcome recorded Stop: Business |
| Phase 2 | NCT04165239 | Oct 2019 → May 2022 | MELAS syndrome, pure mitochondrial myopathy | Khondrion BV | Completed | No outcome recorded |
| Phase 2 | NCT02909400 | Sep 2016 → Jul 2017 | MELAS syndrome, pure mitochondrial myopathy | Khondrion BV | Completed | No outcome recorded |
| Phase 31 trial | ||||||
| Phase 3 | NCT06451757 | Apr 2026 → Sep 2028 expected | MELAS syndrome, maternally-inherited diabetes and deafness | Khondrion BV | Recruiting | No outcome recorded |
News releases announcing trial results or a regulatory action · 14
| Date | Issuer | Release |
|---|---|---|
| 2025-08-04 | Khondrion BV | Regulatory Khondrion Secures EU Orphan Drug Designation for Lead Compound Sonlicromanol in Treatment of Inherited Mitochondrial Oxidative Phosphorylation Defects khondrion.com ↗
Khondrion, a Phase 3 clinical-stage Dutch biopharmaceutical company pioneering therapies for primary mitochondrial diseases, caused per definition by defects in mitochondrial Oxidative Phosphorylation (OXPHOS), today announced that the European Commission has granted Orphan Drug Designation (ODD) to its lead investigational therapy, sonlicromanol, for the treatment of all inherited mitochondrial oxidative phosphorylation (OXPHOS) defects. |
| 2024-11-14 | Khondrion BV | Regulatory Khondrion receives FDA clearance of IND application for pivotal Phase 3 clinical trial of sonlicromanol for the treatment of primary mitochondrial disease khondrion.com ↗
Khondrion, a clinical stage biopharmaceutical company discovering and developing therapies targeting primary mitochondrial disease (PMD), today announced that it has received clearance from the U.S. Food and Drug Administration (FDA) for its Investigational New Drug (IND) application for sonlicromanol. |
| 2024-11-07 | Khondrion BV | Regulatory Khondrion receives FDA clearance of IND application for pivotal Phase 3 clinical trial of sonlicromanol for the treatment of primary mitochondrial disease khondrion.com ↗
Khondrion, a clinical stage biopharmaceutical company discovering and developing therapies targeting primary mitochondrial disease (PMD), today announced that it has received clearance from the U.S. Food and Drug Administration (FDA) for its Investigational New Drug (IND) application for sonlicromanol. |
| 2022-11-22 | Khondrion BV | Results Khondrion announces sonlicromanol Phase IIb progress supporting Phase III development in MELAS spectrum disorders khondrion.com ↗
Both randomised placebo-controlled Phase IIb study and ongoing Phase IIb open label extension study show statistically significant and/or clinically meaningful results in multiple outcome measures with acceptable safety profile |
| 2021-07-12 | Khondrion BV | Results Khondrion announces publication in PLOS ONE of new research showing normalisation of prostate cancer stem cell mPGES-1 overexpression and inhibition of cancer spheroid growth by sonlicromanol’s active metabolite khondrion.com ↗
The research team found that inhibition of mPGES-1 by sonlicromanol’s metabolite was able to considerably reduce spheroid growth in the human prostate cancer cell line DU145, potentially offering a novel approach in treating prostate cancer and other malignancies with high expression of mPGES-1. |
| 2021-01-18 | Khondrion BV | Regulatory Khondrion receives EMA agreement on its Paediatric Investigation Plan for sonlicromanol khondrion.com ↗
Khondrion, a clinical-stage biopharmaceutical company discovering and developing therapies targeting mitochondrial disease, today announces that the European Medicines Agency (EMA) has accepted its Paediatric Investigation Plan (PIP) for the development of its wholly-owned lead asset, sonlicromanol, for children. |
| 2021-01-13 | Khondrion BV | Results Khondrion announces publication in Scientific Reports outlining an additional anti-inflammatory mode of action for its lead phase IIb-stage drug candidate sonlicromanol khondrion.com ↗
KH176m was found to selectively decrease the level of the inflammatory lipid modulator prostaglandin E2 (PGE2) in these fibroblast cells as well as in a mouse macrophage-like cell line RAW264.7. |
| 2020-09-28 | Khondrion BV | Regulatory Khondrion Receives Rare Pediatric Disease Designation for Sonlicromanol from US FDA khondrion.com ↗
Khondrion, a clinical-stage biopharmaceutical company discovering and developing therapies targeting mitochondrial disease, today announces that it received a rare pediatric disease (RPD) designation from the United States (US) Food and Drug Administration (FDA) for sonlicromonal for the treatment of patients with MELAS syndrome. |
| 2019-07-02 | Khondrion BV | Regulatory Khondrion granted Orphan Drug Designation for KH176 for the treatment of MIDD from European Commission khondrion.com ↗
Khondrion, a clinical-stage pharmaceutical company discovering and developing therapies targeting mitochondrial disease, today announces that the European Commission has granted Orphan Drug Designation (ODD) to its lead asset, KH176, for the treatment of patients with maternally inherited diabetes and deafness (MIDD). |
| 2018-09-25 | Khondrion BV | Results Publication of the KHENERGY study results khondrion.com ↗
The study, in 18 patients with an m.3243A>G mutation in their mitochondrial genome, showed that KH176 was well tolerated and appeared safe at the 100mg twice-daily oral dosing regimen. |
| 2015-12-03 | Khondrion BV | Results KH176 Phase 1 Clinical trials khondrion.com ↗
The study deemed that KH176 is well tolerated and has an excellent pharmacokinetic profile. |
| 2015-11-24 | Khondrion BV | Regulatory EMA Orphan Drug Designation khondrion.com ↗
Khondrion has received an Orphan Drug Designation from the European Commission of its frontrunner compound KH176, in the treatment of Leigh syndrome (EU/3/14/1336) |
| 2015-08-11 | Khondrion BV | Regulatory EMA Orphan Drug Designation for MELAS syndrome khondrion.com ↗ |
| 2014-11-24 | Khondrion BV | Regulatory FDA Orphan Drug Designation khondrion.com ↗
Khondrion has received an Orphan Drug Designation from the FDA of its frontrunner compound KH176, in the treatment of inherited mitochondrial respiratory chain diseases. |
All press releases naming this drug 25 releases
Evidence & citations 4 cited values
Every value below carries the sentence it was read from. 8 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | Sonlicromanol | ClinicalTrials.gov intervention name — accepted as the source's own label NCT07298005 ↗ |
| Known as | KH176 | “Sonlicromanol (KH176)” NCT06451757 ↗ |
| Modality | Small molecule | “KH176 is an orally bio-available small molecule under development for the treatment of mitochondrial(-related) diseases.” PMID 29037240 ↗ Oct 2017 |
| Route | Oral | “KH176 is an orally bio-available small molecule under development for the treatment of mitochondrial(-related) diseases.” PMID 29037240 ↗ Oct 2017 |