Can-Fite's Piclidenoson Demonstrates Up to 80% Improvement in Kidney Disease Marker in Lowe Syndrome Preclinical Study: Published in Nature Portfolio Journal
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Rhea-AI Summary
Can-Fite BioPharma (CANF) announced published preclinical findings showing piclidenoson improved kidney protein reabsorption in experimental models of Lowe syndrome. The research appeared in Signal Transduction and Targeted Therapy, a Nature Portfolio journal. Activity was demonstrated in patient-derived kidney cells, human kidney organoids and a humanized mouse model. After five months, treated mice had 48–80% lower urinary levels of a protein-loss marker than untreated disease-model mice.
A proposed Phase II study is under review by Italian Health Authorities. The open-label, single-center trial would evaluate twice-daily oral treatment for six months in 5 adults with genetically confirmed Lowe syndrome. Its primary endpoint measures improvement in kidney uptake of 99mTc-DMSA to assess protein reabsorption capacity.
Positive
- Minor pointPiclidenoson reduced a urinary protein-loss marker by 48–80% versus untreated disease-model mice after five months.
- Minor pointPiclidenoson restored cellular protein trafficking and protein reabsorption across preclinical experimental systems.
- Minor point. Forward-looking: it has not happened yet and may not happen.A proposed Phase II trial would evaluate twice-daily oral piclidenoson for six months in Lowe syndrome.
Negative
- Minor pointThe proposed Phase II study remains under review by Italian Health Authorities.
- Minor point. Forward-looking: it has not happened yet and may not happen.The proposed trial is open-label and single-center, with 5 adult patients.
Key Figures
- Urinary proteinuria marker reduction
- 48–80%
- After five months of treatment in a mouse model, compared with untreated disease-model mice
- Compounds screened
- 1,280 compounds
- Screening identified piclidenoson for Lowe syndrome research
- Proposed Phase II study enrollment
- 5 adults
- Genetically confirmed Lowe syndrome; study under review by Italian Health Authorities
- Proposed treatment regimen
- Twice daily for six months
- Oral piclidenoson in the proposed Phase II study
Historical Context
-
Eurasian patent readiness notice covered Piclidenoson use in Lowe syndrome and described the planned five-adult Phase 2 study.
24h Move is the share-price change in the day after each event; other market factors may also have contributed.
Key Terms
a3 adenosine receptor agonist medical
human kidney organoids medical
phospholipase c medical
99mtc-dmsa medical
open-label medical
AI-generated analysis. How Rhea-AI works. Not financial advice.
The research describes compelling preclinical findings providing the foundation for a planned Phase II clinical study, currently under review by health authorities in Italy
Ramat Gan, Israel, Nov. 12, 2026 (GLOBE NEWSWIRE) -- 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–
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.
Forward-Looking Statements
This press release may contain forward-looking statements, about Can-Fite’s expectations, beliefs or intentions regarding, among other things, its product development efforts of Piclidenoson for the treatment of Lowe syndrome including the timing and outcome of regulatory review of the planned Phase II study in Lowe syndrome, Can-Fite’s ability to initiate and enroll patients in such study, and the results of any such study. All statements in this communication, other than those relating to historical facts, are “forward looking statements”. Forward-looking statements can be identified by the use of forward-looking words such as “believe,” “expect,” “intend,” “plan,” “may,” “should” or “anticipate” or their negatives or other variations of these words or other comparable words or by the fact that these statements do not relate strictly to historical or current matters. Forward-looking statements relate to anticipated or expected events, activities, trends or results as of the date they are made. Because forward-looking statements relate to matters that have not yet occurred, these statements are inherently subject to known and unknown risks, uncertainties and other factors that may cause Can-Fite’s actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements. Important factors that could cause actual results, performance or achievements to differ materially from those anticipated in these forward-looking statements include, among other things, our market and other conditions, history of losses and needs for additional capital to fund our operations and our inability to obtain additional capital on acceptable terms, or at all; uncertainties of cash flows and inability to meet working capital needs; the initiation, timing, progress and results of our preclinical studies, clinical trials and other product candidate development efforts; our ability to advance our product candidates into clinical trials or to successfully complete our preclinical studies or clinical trials; our receipt of regulatory approvals for our product candidates, and the timing of other regulatory filings and approvals; the clinical development, commercialization and market acceptance of our product candidates; our ability to establish and maintain strategic partnerships and other corporate collaborations; the implementation of our business model and strategic plans for our business and product candidates; the scope of protection we are able to establish and maintain for intellectual property rights covering our product candidates and our ability to operate our business without infringing the intellectual property rights of others; competitive companies, technologies and our industry; risks related to not satisfying the continued listing requirements of NYSE American; and statements as to the impact of the political and security situation in Israel on our business. More information on these risks, uncertainties and other factors is included from time to time in the “Risk Factors” section of Can-Fite’s Annual Report on Form 20-F filed with the SEC on March 26, 2026 and other public reports filed with the SEC and in its periodic filings with the TASE. Existing and prospective investors are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof. Can-Fite undertakes no obligation to publicly update or review any forward-looking statement, whether as a result of new information, future developments or otherwise, except as may be required by any applicable securities laws.
Contact
Can-Fite BioPharma
Motti Farbstein
+972-3-9241114
FAQ
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What did Can-Fite's piclidenoson study show in Lowe syndrome?
In a humanized mouse model, five months of piclidenoson treatment reduced urinary levels of a low-molecular-weight proteinuria marker by 48–80% compared with untreated disease-model mice. The research also demonstrated activity in patient-derived kidney cells and human kidney organoids. These are preclinical findings.
How does piclidenoson act on kidney cell abnormalities in Lowe syndrome?
Piclidenoson activates the A3 adenosine receptor, which stimulates the enzyme phospholipase C. In the preclinical study, this corrected abnormal accumulation of a membrane lipid that disrupts kidney cell function in Lowe syndrome.