Can-Fite launches a Phase 2 clinical trial of Piclidenoson for Lowe syndrome, evaluating its potential to improve kidney function in this rare genetic disorder.
Written By: Rishabh Sonawane, BPharm
Reviewed By: Pharmacally Editorial Team
Can-Fite BioPharma has submitted the protocol for a Phase 2 clinical study (NCT07410455) evaluating Piclidenoson in patients with Lowe syndrome. The pilot, open-label study will be conducted at Bambino Gesù Children’s Hospital in Rome under the leadership of Professor Francesco Emma.
The study is intended to generate initial clinical evidence to support future discussions with regulatory authorities and help define the subsequent clinical development pathway for Piclidenoson in Lowe syndrome, a rare genetic disorder for which no approved disease-modifying therapies currently exist. According to Can-Fite Chief Executive Officer Motti Farbstein, compelling preclinical findings provided the scientific rationale for advancing Piclidenoson into clinical evaluation, while the focused Phase 2 study is designed to facilitate regulatory discussions regarding future development.
Phase 2 Study Design
The Phase 2 study is an open-label, single-center pilot trial evaluating oral Piclidenoson administered twice daily for six months in five adults with genetically confirmed Lowe syndrome.
The primary endpoint is improvement in renal uptake of 99mTc-DMSA, a validated indicator of proximal tubular function that reflects renal tubular reabsorption, one of the key physiological processes impaired in Lowe syndrome.
Secondary endpoints include:
- Urinary biomarkers of tubular function
- Clinical parameters associated with Fanconi syndrome
- Safety and tolerability assessments
Although limited to five participants, the pilot study is intended to generate proof-of-concept clinical data that may guide future clinical development and regulatory discussions.
Preclinical Evidence Supporting Clinical Development
Can-Fite’s clinical program is supported by preclinical research conducted by Professor Antonella De Matteis and colleagues at the University of Naples Federico II and the Telethon Institute of Genetics and Medicine (TIGEM).
Their studies demonstrated restoration of OCRL-dependent cellular function following exposure to Piclidenoson. Since mutations in the OCRL gene disrupt essential cellular processes underlying Lowe syndrome, these findings provide the biological rationale for evaluating Piclidenoson as a potential therapeutic approach in affected patients.
To advance the program, Can-Fite has established a collaboration with Fondazione Telethon, combining the organization’s expertise in rare genetic diseases with the company’s clinical development capabilities.
Significant Unmet Medical Need
Lowe syndrome is a rare X-linked multisystem genetic disorder caused by mutations in the OCRL gene and is characterized by severe renal, neurological, and ocular manifestations.
Progressive proximal tubular dysfunction frequently results in Fanconi syndrome, chronic kidney disease, and eventual kidney failure. Current management remains supportive, as no approved therapies directly target the underlying molecular defect or modify disease progression.
Mechanism of Action
Piclidenoson is an orally administered, first-in-class A3 adenosine receptor (A3AR) agonist that selectively modulates inflammatory signaling pathways.
Activation of A3AR has been shown in previous clinical studies to suppress pro-inflammatory cytokines, including interleukin-17 (IL-17) and interleukin-23 (IL-23), while promoting apoptosis of pathogenic inflammatory cells. These anti-inflammatory effects form the therapeutic rationale for the drug’s development across inflammatory diseases.
In Lowe syndrome, however, the therapeutic hypothesis extends beyond its established anti-inflammatory activity. Preclinical studies indicate that Piclidenoson may restore OCRL-dependent cellular functions, providing the scientific basis for investigating its potential disease-modifying effects in this rare genetic disorder.
Clinical Development and Regulatory Perspective
The Lowe syndrome program represents Can-Fite’s expansion into rare genetic diseases and orphan drug development.
Should the Phase 2 pilot study demonstrate favorable safety findings together with preliminary evidence of biological activity, the results could support future discussions with regulatory authorities regarding subsequent clinical development and the design of larger confirmatory studies.
Piclidenoson is also being evaluated in a pivotal Phase 3 psoriasis study (NCT06643260) under U.S. Food and Drug Administration (FDA) and European Medicines Agency (EMA) oversight. According to the company, the drug has accumulated clinical safety experience through multiple Phase II and Phase III studies across inflammatory indications, supporting its continued investigation in additional therapeutic areas.
Regulatory Path
If the Phase 2 study demonstrates acceptable safety together with encouraging signals of efficacy, the findings are expected to help inform future regulatory interactions and define the next steps in the clinical development pathway for Piclidenoson in Lowe syndrome, a condition with no approved disease-modifying treatment options.
References
About the Writer
Rishabha Sonawane, B.Pharm (LinkedIn) is healthcare writer with a strong interest in medical writing, regulatory affairs, clinical research, and AI-driven drug discovery. He has completed specialized training from the NIH and ICMR in clinical pharmacology, clinical research, and scientific writing. Passionate about evidence-based healthcare communication, he focuses on translating complex scientific research into clear, accurate, and engaging medical content.