Ramat Gan, Israel — Can-Fite BioPharma Ltd. (NYSE American: CANF) (TASE:CANF), a biotechnology company advancing a pipeline of proprietary small molecule drugs that address oncological and inflammatory diseases, today announced the publication of research demonstrating that piclidenoson (IB-MECA), an orally administered A3 adenosine receptor agonist, corrects disease-associated cellular abnormalities and improves kidney protein reabsorption in preclinical models of Lowe syndrome. The findings were published in Signal Transduction and Targeted Therapy, a Nature Portfolio journal. https://doi.org/10.1038/s41392-026-03054-z
The study, entitled “An actionable GPCR restores phosphoinositide homeostasis and kidney function in Lowe syndrome,” was led by researchers including Dr. Leopoldo Staiano and Prof. Maria Antonietta De Matteis at the Telethon Institute of Genetics and Medicine (TIGEM), with collaborators at the University of Naples Federico II and other international institutions.
Lowe syndrome is a rare genetic disorder affecting the eyes, brain and kidneys, for which no curative therapy is currently available. Its kidney manifestations include impaired protein reabsorption, urinary protein loss and progressive kidney disease.
The researchers identified piclidenoson through screening of 1,280 compounds and demonstrated its activity across multiple experimental systems, including patient-derived kidney cells, human kidney organoids and a humanized mouse model. Piclidenoson restored cellular protein trafficking and protein reabsorption. In the mouse model, five months of treatment reduced urinary levels of a marker of low-molecular-weight proteinuria by 48–80% compared with untreated disease-model mice.
The study also identified the underlying mechanism: activation of the A3 adenosine receptor stimulates phospholipase C, correcting the abnormal accumulation of a membrane lipid that disrupts kidney cell function in Lowe syndrome.
“These findings demonstrate the potential of piclidenoson to address a fundamental cellular defect underlying kidney dysfunction in Lowe syndrome. The consistent activity across patient-derived cells, human kidney organoids and an animal model provides a compelling scientific foundation for advancing its clinical evaluation in this rare disease, said Prof. Pnina Fishman, Can-Fite CSO and Chairperson”.
A Phase II clinical study has been designed and is under review by Italian Health Authorities. The proposed Phase II study is an open-label, single-center clinical trial designed to evaluate the efficacy and safety of oral Piclidenoson administered twice daily for six months in 5 adult patients with genetically confirmed Lowe syndrome. The primary endpoint is improvement in renal uptake of 99mTc-DMSA as a measure of proximal tubular reabsorption capacity, with secondary endpoints.
About Piclidenoson
Piclidenoson is a robust anti-inflammatory agent, currently being evaluated in a pivotal Phase 3 psoriasis clinical study under approval of both the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA).
Piclidenoson is a novel, first-in-class, A3 adenosine receptor agonist (A3AR) small molecule, orally bioavailable drug with an excellent safety profile demonstrating evidence of efficacy in Phase II and Phase III clinical studies. The drug’s mechanism of action entails inhibition of the inflammatory cytokines interleukin 17 and 23 (IL-17 and IL-23) and the induction of apoptosis of patients’ skin cell keratinocytes involved with the disease pathogenicity.
About Fondazione Telethon
Fondazione Telethon ETS is one of the main Italian biomedical charities, founded in 1990 on the initiative of a group of patients suffering from muscular dystrophy. Its mission is to achieve the cure of rare genetic diseases through scientific research of excellence, selected according to the best practices shared internationally. Through a unique method in the Italian panorama, it follows the entire “research chain” dealing with fundraising, selection and funding of projects and the research activity itself carried out in the centers and laboratories of the Foundation. Telethon also develops collaborations with public health institutions and pharmaceutical industries to translate the results of research into therapies accessible to patients. Since its foundation, Telethon has invested more than 660 million euros in research, has funded 2,960 projects with 1,720 researchers involved and 630 diseases studied. To date, thanks to Fondazione Telethon, the first gene therapy with stem cells in the world has been made available, thanks to the collaboration with the pharmaceutical industry. This therapy is intended for the treatment of ADA-SCID, a severe immunodeficiency that compromises the body’s defenses from birth. In 2023, Fondazione Telethon became responsible for the production and distribution of the drug to eligible patients in the European Union
Another gene therapy resulting from Telethon research made available is the one for a serious neurodegenerative disease, metachromatic leukodystrophy. This therapeutic approach is in an advanced stage of development for another immunodeficiency, Wiskott-Aldrich syndrome. Other diseases on which the gene therapy developed by Telethon researchers has been evaluated in patients are beta thalassemia and two metabolic diseases of childhood, mucopolysaccharidosis type 6 and type 1. In addition, within the Telethon institutes a targeted therapeutic strategy is being studied or developed for other genetic diseases, such as hemophilia or various hereditary vision defects. In parallel, the study of basic mechanisms and potential therapeutic approaches for diseases still unanswered continues in all laboratories funded by Telethon.
About Can-Fite BioPharma Ltd.
Can-Fite BioPharma Ltd. (NYSE American: CANF) (TASE: CANF) is an advanced clinical stage drug development Company with a platform technology that is designed to address multi-billion dollar markets in the treatment of cancer, liver, and inflammatory disease. The Company’s lead drug candidate, Piclidenoson, is being evaluated in a pivotal Phase III study for the treatment of moderate-to-severe plaque psoriasis, which has completed enrollment of the first 247 patients for a pre-specified interim analysis, with interim efficacy and safety data expected in the first quarter of 2027. Can-Fite’s liver drug, Namodenoson, is being evaluated in a Phase III trial for hepatocellular carcinoma (HCC), a Phase 2b trial for the treatment of MASH, and in a Phase 2a study in pancreatic cancer. Namodenoson has been granted Orphan Drug Designation in the U.S. and Europe and Fast Track Designation as a second line treatment for HCC by the U.S. Food and Drug Administration. Namodenoson has also shown proof of concept to potentially treat other cancers including colon, prostate, and melanoma. CF602, the Company’s third drug candidate, has shown efficacy in the treatment of erectile dysfunction. These drugs have an excellent safety profile with experience in over 1,700 patients in clinical studies to date. For more information please visit: www.canfite.com.
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Can-Fite BioPharma
Motti Farbstein
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