Can-Fite submits Phase 2 protocol testing Piclidenoson in Lowe syndrome
The company filed a study protocol with an Italian children's hospital for the first clinical test of its drug in this rare genetic disease.
Can-Fite BioPharma announced on August 3, 2026 that it had submitted a Phase 2 clinical study protocol to Bambino Gesù Children's Hospital in Rome, Italy, marking the first clinical evaluation of Piclidenoson in patients with Lowe syndrome, a rare inherited genetic disorder that currently has no approved disease-modifying therapies.1 Prof. Francesco Emma, described as an internationally recognized expert in inherited kidney diseases, will lead the study.1
Lowe syndrome is described in the release as a rare X-linked multisystem genetic disorder caused by mutations in the OCRL gene, resulting in severe renal, neurological, and ocular manifestations.1 The kidney component of the disease involves progressive damage to proximal tubule function that can lead to Fanconi syndrome, chronic kidney disease, and ultimately kidney failure.1 The release states that current treatment is only supportive, with no therapy addressing the disease's underlying mechanism.1
According to the company, Piclidenoson was chosen based on preclinical work showing it could restore OCRL-related cellular function, research led by Dr. Antonella De Matteis of the University of Naples Federico II and TIGEM in Italy.1 Can-Fite and Fondazione Telethon have entered a collaboration agreement to develop Piclidenoson for Lowe syndrome.1
The planned trial is an open-label, single-center study testing oral Piclidenoson taken twice daily for six months in five adult patients with genetically confirmed Lowe syndrome.1 The main measure of success will be improvement in kidney uptake of the imaging agent 99mTc-DMSA, used to gauge how well the proximal tubules reabsorb substances, while additional measures will track urinary markers of tubular function, signs of Fanconi syndrome, and safety.1
Can-Fite's CEO Motti Farbstein called the program's launch "an important milestone for Can-Fite."1 Separately, the release notes Piclidenoson is currently in a pivotal Phase 3 study for psoriasis conducted under agreements with both the FDA and the European Medicines Agency.1
Written by readthrough’s AI from the linked primary sources and fact-checked against them automatically before publishing. Not investment advice.