Piclidenoson

August 3, 2026: Can-Fite BioPharma Ltd. (NYSE: CANF; TASE: CANF) announced submission of a Phase 2 protocol to Bambino Gesù Children’s Hospital in Rome for the first clinical evaluation of Piclidenoson in Lowe syndrome, a rare inherited OCRL-related disorder with no approved disease-modifying therapies. The study, led by Prof. Francesco Emma, is intended to support regulatory interactions and a potential registration path if results are positive.

The open-label, single-center Phase 2 trial will enroll 5 adult patients with genetically confirmed Lowe syndrome and test oral Piclidenoson twice daily for 6 months. The primary endpoint is improvement in renal uptake of 99mTc-DMSA as a measure of proximal tubular reabsorption capacity; secondary endpoints include urinary biomarkers of tubular function, Fanconi syndrome parameters, and safety.

Can-Fite said the program is supported by preclinical findings from Dr. Antonella De Matteis showing restoration of OCRL-dependent cellular function, and it has signed a collaboration agreement with Fondazione Telethon for clinical development in Lowe syndrome.

Separately, the company said Piclidenoson is also being evaluated in a pivotal Phase 3 psoriasis study under FDA and EMA approval.