Drugs / ELX-02
ELX-02
Trials 7
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 12 trials | ||||||
| Phase 1 | NCT03776539 | Jan → Jul 2019 | impaired renal function disease | Eloxx Pharmaceuticals, Inc. | Completed | No outcome recorded |
| Phase 1 | NCT03309605 | Oct 2017 → Jun 2019 | disease of genetic or genomic mechanism | Eloxx Pharmaceuticals, Inc. | Completed | No outcome recorded |
| Phase 25 trials | ||||||
| Phase 2 | NCT07523581 | Jun 2026 → Jun 2027 expected | X-linked Alport syndrome, autosomal recessive Alport syndrome | Eloxx Pharmaceuticals, Inc. | Recruiting | No outcome recorded |
| Phase 2 | NCT05448755 | Nov 2022 → Sep 2023 | Alport syndrome | Eloxx Pharmaceuticals, Inc. | Completed | No outcome recorded |
| Phase 2 | NCT04135495 | Nov 2019 → Oct 2022 | cystic fibrosis | Eloxx Pharmaceuticals, Inc. | Completed | No outcome recorded |
| Phase 2 | NCT04126473 | Nov 2019 → Apr 2022 | cystic fibrosis | Eloxx Pharmaceuticals, Inc. | Completed | No outcome recorded |
| Phase 2 | NCT04069260 | Aug → Dec 2019 | cystinosis | Eloxx Pharmaceuticals, Inc. | Terminated | No outcome recorded |
| Comparator or background therapy2 trials | ||||||
| Phase 1 | NCT03292302 Comparator | Sep → Dec 2017 | disease of genetic or genomic mechanism | Eloxx Pharmaceuticals, Inc. | Completed | No outcome recorded |
| Phase 1 | NCT02807961 Comparator | Jul 2016 → Feb 2017 | disease of genetic or genomic mechanism | Eloxx Pharmaceuticals, Inc. | Terminated | No outcome recorded |
News releases announcing trial results or a regulatory action · 2
| Date | Issuer | Release |
|---|---|---|
| 2023-09-07 | Eloxx Pharmaceuticals, Inc. | Results Eloxx Pharmaceuticals Provides Program Updates on ELX-02 and ZKN-013 eloxxpharma.com ↗
All 3 Alport patients (100% response rate) treated with ELX-02 had biopsy-confirmed disease regression. |
| 2022-06-01 | Eloxx Pharmaceuticals, Inc. | Results Exaluren Induces COL4A5 Readthrough Across 49 Nonsense Variants in Alport Syndrome eloxxpharma.com ↗
Omachi et al. published in iScience a systematic screen of exaluren across 49 COL4A5 nonsense variants, demonstrating readthrough activity across all variants tested, with some mutations showing greater than 40-fold enhancement. |
All press releases naming this drug 3 releases
| Date | Issuer | Release |
|---|---|---|
| 2026-09-11 | Eloxx Pharmaceuticals, Inc. | Eloxx Pharmaceuticals Announces First Patients Dosed in Phase 2b EXACT Study of Exaluren in Nonsense Mutation Alport Syndrome eloxxpharma.com ↗ |
| 2023-09-07 | Eloxx Pharmaceuticals, Inc. | Eloxx Pharmaceuticals Provides Program Updates on ELX-02 and ZKN-013 eloxxpharma.com ↗ |
| 2022-06-01 | Eloxx Pharmaceuticals, Inc. | Exaluren Induces COL4A5 Readthrough Across 49 Nonsense Variants in Alport Syndrome eloxxpharma.com ↗ |
Evidence & citations 8 cited values
Every value below carries the sentence it was read from. 10 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | ELX-02 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT04069260 ↗ |
| Known as | Exaluren | ClinicalTrials.gov intervention name — accepted as the source's own label NCT07523581 ↗ |
| Known as | Exalurene | ChEMBL registry synonym — accepted as the source's own label CHEMBL4297744 ↗ |
| Known as | Exalureno | ChEMBL registry synonym — accepted as the source's own label CHEMBL4297744 ↗ |
| Known as | NB-124 | ChEMBL registry synonym — accepted as the source's own label CHEMBL4297744 ↗ |
| Action | Restore | “optimized as a translational read-through drug (TRID) for the treatment of genetic conditions caused by nonsense mutations” NCT03292302 ↗ |
| Modality | Small molecule | “advanced synthetic aminoglycoside” NCT02807961 ↗ |
| Route | Subcutaneous | “Subcutaneously Administered ELX-02” NCT03309605 ↗ |