RAMAT GAN, Israel, Aug. 3, 2026
Can-Fite BioPharma Ltd. has launched its first clinical development program evaluating Piclidenoson for Lowe syndrome, a rare inherited genetic disorder with no approved disease-modifying therapies, by submitting a Phase 2 clinical study protocol to Bambino Gesù Children’s Hospital in Rome, Italy. The investigator-led study will be conducted under the leadership of Prof. Francesco Emma, a specialist in inherited kidney diseases, and is designed to evaluate the safety and efficacy of the company’s oral A3 adenosine receptor agonist in adults with genetically confirmed Lowe syndrome. The small open-label Phase 2 trial is intended to support future regulatory interactions and potentially establish a pathway toward registration if clinical outcomes are positive. The program follows a collaboration between Can-Fite BioPharma and Fondazione Telethon, supported by promising preclinical findings demonstrating restoration of OCRL-dependent cellular function, the underlying biological defect associated with the disease.
Phase 2 Study Targets Kidney Dysfunction in Rare Genetic Disorder
The Phase 2 clinical study will enroll five adult patients with genetically confirmed Lowe syndrome, a rare X-linked multisystem genetic disease caused by mutations in the OCRL gene. Participants will receive oral Piclidenoson twice daily for six months in a single-center, open-label trial conducted in Italy. The primary endpoint will assess improvement in 99mTc-DMSA renal uptake, an indicator of proximal tubular reabsorption capacity, while secondary endpoints will evaluate urinary biomarkers, Fanconi syndrome parameters, kidney function, and overall safety. Lowe syndrome is characterized by progressive kidney disease, neurological impairment, and eye abnormalities that often lead to chronic kidney disease and eventual kidney failure. Current treatment remains supportive, with no approved therapies addressing the underlying disease mechanism, highlighting the significant unmet medical need targeted by this investigational program.
Preclinical Evidence Supports Piclidenoson Development
The decision to advance Piclidenoson into clinical testing was supported by preclinical research conducted by Dr. Antonella De Matteis and collaborators at the Telethon Institute of Genetics and Medicine (TIGEM), which demonstrated restoration of OCRL-dependent cellular function associated with Lowe syndrome. These findings prompted the collaboration between Can-Fite BioPharma and Fondazione Telethon to evaluate the therapy in patients. Piclidenoson is a first-in-class, orally available A3 adenosine receptor agonist with an established safety profile from previous clinical studies. The investigational drug is currently undergoing pivotal Phase 3 evaluation for psoriasis under both U.S. Food and Drug Administration (FDA) and European Medicines Agency (EMA) oversight. Its mechanism of action involves modulation of inflammatory pathways through inhibition of IL-17 and IL-23, supporting its potential application across multiple inflammatory and rare disease indications.
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Rare Disease Program Expands Can-Fite’s Clinical Pipeline
The initiation of the Lowe syndrome program expands Can-Fite BioPharma’s clinical development portfolio beyond oncology, liver disease, and inflammatory disorders into the field of rare genetic diseases. The company believes the focused pilot study will provide critical data to guide future discussions with regulatory authorities regarding clinical development and a potential registration strategy for Piclidenoson in Lowe syndrome. Alongside this program, Can-Fite continues to advance Piclidenoson in Phase 3 psoriasis, Namodenoson in Phase 3 hepatocellular carcinoma (HCC) and mid-stage studies for MASH and pancreatic cancer, while also progressing additional pipeline candidates. If successful, the Lowe syndrome study could establish Piclidenoson as the first disease-modifying treatment for this ultra-rare inherited disorder, addressing a substantial unmet medical need in patients with progressive kidney disease and multisystem complications.
Source: Can-Fite BioPharma press release
