Develops small-molecule drug candidates designed to restore protein production for people with genetic diseases, focusing on rare kidney diseases and, with Almirall, rare skin diseases.
10-K for FY2025, filed Mar 17, 2026
Almirall licenses ZKN-013 directly and handles its development and commercialization for rare skin diseases; all license and service revenue to date has come from one customer.
10-K for FY2025, filed Mar 17, 2026
10-Q for Q2 FY2026, filed Aug 11, 2026
−$4.5MView ↗
Broader kidney pipeline
The older report centered its clinical kidney program on Alport syndrome; the latest adds exaluren development for nonsense-mutation ADPKD alongside Alport.
10-K for FY2022, filed Mar 31, 2023 10-K for FY2025, filed Mar 17, 2026We have multiple programs in our pipeline including a clinical program for the treatment of Alport syndrome with ELX-02 (subcutaneous…View ↗
Our pipeline also includes a preclinical program evaluating exaluren for the treatment of autosomal dominant polycystic kidney disease…View ↗
Partnered skin-drug development
The older report described the skin-disease program for RDEB, JEB and FAP; the latest assigns Almirall responsibility for developing and commercializing the skin-disease candidate.
10-K for FY2022, filed Mar 31, 2023 10-K for FY2025, filed Mar 17, 2026We are also advancing ZKN-013 as an oral agent for the treatment of RDEB/JEB and FAP patients with nonsense mutations. RDEB and JEB are…View ↗
We are also developing ZKN-013, an oral ribosome modulating agent (“RMA”) with structural similarity to azithromycin that induces PTC…View ↗
Competes with Travere Therapeutics, River Renal, Bayer, Boehringer Ingelheim, Otsuka, AstraZeneca, Vertex Pharmaceuticals, Novartis, PYC Therapeutics, AbbVie, GSK, XORTX Therapeutics, Maze Therapeutics, Krystal Biotech, Abeona Therapeutics, Chiesi, Castle Creek, RHEACELL, and Inmune Bio
10-K for FY2025, filed Mar 17, 2026There are currently no FDA-approved therapies approved specifically for the treatment of AS. Disease management typically consists of ACEi…View ↗
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