A precision approach to rare kidney disease
Eloxx is advancing exaluren for genetically defined patient subsets with Alport syndrome and ADPKD caused by nonsense mutations.
Have Alport syndrome or ADPKD with a known nonsense mutation?
Disease information, clinical trial access, and patient advocacy resources.
Prescribers & Investigators
Mechanism of action, preclinical and clinical evidence, referral pathways, and publications.
Investor Relations
SEC filings, press releases, investor presentations, and contact information.
Exaluren is a novel eukaryotic ribosome-selective glycoside product candidate designed to induce premature termination codon readthrough in the presence of nonsense mutations and promote the restoration of full-length proteins.
How it works →Nonsense mutation readthrough
Selectively modulates the human ribosome at premature stop codons, enabling translation to produce a functional full-length protein.
Kidney-concentrated delivery
Administered subcutaneously; transported and concentrated into kidney cells via megalin.
Gene-agnostic mechanism
Targets the premature stop codon regardless of which gene is affected — enabling investigation across COL4A3, COL4A4, and COL4A5 (Alport) and PKD1/PKD2 (ADPKD).
One molecule. Two genetically defined kidney diseases.
Exaluren is being developed across two indications united by a shared genetic mechanism — nonsense mutation readthrough.
| Compound | Target | Indication | Status |
|---|---|---|---|
exaluren |
COL4A3 / COL4A4 / COL4A5 nonsense mutations | Nonsense mutation Alport syndrome (NMAS) |
EXACT Phase 2b NCT07523581 → EXACT Study information → Alport program → |
exaluren |
PKD1 / PKD2 nonsense mutations | Nonsense mutation ADPKD (nmADPKD) |
In Development Program details → |
ZKN-013* Licensed to Almirall |
COL7A1 nonsense mutations | RDEB & JEB (rare skin diseases) | Phase 1 |
* Eloxx has exclusively licensed ZKN-013 to Almirall, S.A., who is developing it for the treatment of rare skin diseases caused by nonsense mutations (RDEB and JEB). Almirall is responsible for all development and commercialization of ZKN-013.
Advancing exaluren for people who have waited long enough
Eloxx is investigating exaluren as a potential therapy targeting full-length functional protein restoration in Alport syndrome and ADPKD in patients with nonsense mutations.