Phase 2 clinical study protocol submitted; Small Phase 2 study designed to support regulatory interactions and potential registration upon positive results

Ramat Gan, Israel, Aug. 03, 2026 (GLOBE NEWSWIRE) -- Can-Fite BioPharma Ltd. (NYSE:CANF) (TASE:CANF), a biotechnology company advancing a pipeline of proprietary small molecule drugs that address oncological and inflammatory diseases, today announced the submission of a Phase 2 clinical study protocol to Bambino Gesù Children's Hospital in Rome, Italy, for the first clinical evaluation of Piclidenoson in patients with Lowe syndrome, a rare inherited genetic disorder with no approved disease-modifying therapies. The study will be led by Prof. Francesco Emma, an internationally recognized expert in inherited kidney diseases.

Lowe syndrome is a rare X-linked multisystem genetic disorder caused by mutations in the OCRL gene, resulting in severe renal, neurological, and ocular manifestations. The renal disease is characterized by progressive proximal tubular dysfunction leading to Fanconi syndrome, chronic kidney disease, and eventual kidney failure. Current management is supportive, and no approved therapy addresses the underlying disease mechanism.

Piclidenoson was selected for clinical evaluation based on compelling preclinical studies demonstrating restoration of OCRL-dependent cellular function found by Dr. Antonella De Matteis, Professor of Biology, Department of Molecular Medicine and Medical Biotechnology at the University of Naples Federico II, and Program Coordinator of the Cell Biology and Disease Mechanisms at the Telethon Institute of Genetics and Medicine (TIGEM) in Italy. Can-Fite and Fondazione Telethon have signed a collaboration agreement for the clinical development of Piclidenoson for the treatment of Lowe Syndrome, a high medical need with no drug available.  

The Phase 2 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 evaluating urinary biomarkers of tubular function, Fanconi syndrome parameters, and safety.