Can-Fite BioPharma Ltd. (NYSE American: CANF) (TASE: CANF) has finalized the design of a Phase II clinical trial for its drug candidate Piclidenoson in the treatment of Lowe Syndrome, a rare genetic disease with no currently approved therapies. The company is now undertaking preparatory work to initiate the study.
The Phase II open-label trial will enroll five patients who will receive 3 mg of Piclidenoson twice daily for a 12-month period. Dr. Franchesca Emma from the Division of Nephrology at Bambino Gesù Children's Hospital in Rome, Italy, will serve as the principal investigator. The study's primary endpoint will measure the efficacy of Piclidenoson in increasing 99mTc-DMSA renal uptake.
A Breakthrough for an Underserved Rare Disease
Lowe Syndrome, also known as oculo-cerebro-renal syndrome (OCRL), is an X-linked genetic condition that occurs almost exclusively in males. This multisystem disorder affects approximately 1 in 500,000 individuals and is characterized by:
- Vision problems, including congenital cataracts
- Kidney dysfunction, particularly urinary loss of proteins and solutes, typically developing in the first year of life
- Neurological abnormalities associated with intellectual disabilities
- Reduced life expectancy, rarely exceeding 40 years
The clinical development program stems from successful preclinical research conducted by Dr. Antonella De Matteis, Professor of Biology at the University of Naples Federico II and Program Coordinator at the Telethon Institute of Genetics and Medicine (TIGEM) in Italy.
"Having tested thousands of compounds in search of a treatment for Lowe Syndrome, Piclidenoson is the only compound we've found to date that has shown to be effective in pre-clinical studies," Dr. De Matteis explained. "Importantly, we observed that Piclidenoson treatment in preclinical models of Lowe syndrome leads to a significant decrease of the urinary loss of proteins."
Strategic Collaboration for Rare Disease Development
Can-Fite has established a collaboration agreement with Fondazione Telethon for the clinical development of Piclidenoson in Lowe Syndrome. This partnership leverages Telethon's expertise in rare genetic diseases and Can-Fite's drug development capabilities.
Dr. Pnina Fishman, Can-Fite's CSO and Chairperson, expressed enthusiasm about the program: "We are very much enthusiastic by the breakthrough research of Dr. De Matteis showing that Piclidenoson is efficacious in treating pre-clinical models of Lowe Syndrome. Her impressive results are the basis for implementing Piclidenoson in the treatment of this rare genetic disease."
The development pathway for Lowe Syndrome may benefit from accelerated regulatory processes, as both the FDA and EMA offer expedited approval pathways for rare genetic diseases, typically requiring smaller clinical studies.
Piclidenoson: Mechanism and Development Status
Piclidenoson is a novel, first-in-class, A3 adenosine receptor agonist (A3AR) small molecule with an established safety profile. The drug's mechanism of action involves:
- Inhibition of inflammatory cytokines interleukin 17 and 23 (IL-17 and IL-23)
- Induction of apoptosis in pathogenic cells
The compound is currently being evaluated in a pivotal Phase III clinical study for psoriasis under the approval of both the U.S. Food and Drug Administration and the European Medicines Agency. Its demonstrated safety profile across more than 1,600 patients in clinical studies to date provides a solid foundation for its investigation in Lowe Syndrome.
Addressing an Unmet Medical Need
The development of Piclidenoson for Lowe Syndrome represents a significant step toward addressing a high unmet medical need. Currently, no approved pharmacological treatments exist for this rare genetic condition, with management limited to supportive care for specific symptoms.
The collaboration between Can-Fite and Fondazione Telethon exemplifies the growing trend of partnerships between biopharmaceutical companies and disease-focused foundations to accelerate therapeutic development for rare conditions.
If successful, this Phase II study could provide the first evidence of a potential treatment option for patients with Lowe Syndrome, potentially changing the treatment landscape for this devastating rare disease.
