Can-Fite BioPharma Ltd. announced it plans to develop its lead drug candidate, Piclidenoson, for the treatment of Lowe Syndrome based on recent efficacy findings by Dr. Antonella De Matteis, Professor of Biology, Department of Molecular Medicine and Medical Biotechnology at the University of Naples Federico II, and Program Coordinator of the Cell Biology and Disease Mechanisms at The Telethon Institute of Genetics and Medicine (TIGEM) in Italy. Can-Fite and Fondazione Telethon have signed an agreement outlining their collaboration for the development of Piclidenoson for the treatment of Lowe Syndrome, a high medical need with no drug available. Fondazione Telethon is an Italian nonprofit organization whose mission is to advance biomedical research toward the diagnosis, cure, and prevention of genetic diseases, and it established TIGEM in 1994.

Lowe Syndrome, also known as oculo-cerebro-renal syndrome (OCRL), an X-linked genetic condition occurring almost exclusively in males, is a multisystem disorder characterized by vision problems including clouding of the lenses of the eyes (cataracts) that are present at birth, kidney problems (consisting in urinary loss of proteins and solutes) that usually develop in the first year of life, and brain abnormalities associated with intellectual disabilities, and a life span that rarely exceeds 40 years. Lowe Syndrome prevalence is estimated at approximately 1 in 500,000. Can-Fite estimates the total addressable market for the treatment of Lowe Syndrome at approximately $100 million in the U.S. alone, based on incidence and cost of treatment for other rare genetic pediatric syndromes.

The Company is not aware of any drug candidates currently in development for the systemic treatment of Lowe Syndrome. As a rare genetic pediatric disorder, Lowe Syndrome may qualify for Orphan Drug Designation by the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA), granting special development status to accelerate development at a reduced cost. The FDA?s Orphan Drug designation grants 7 years of market exclusivity, tax credits, waiver of the Prescription Drug User Fee Amendments fee (currently $3.1 million in 2022), and access to additional grants and support from the FDA.

Piclidenoson for the treatment of Lowe Syndrome may also qualify for the FDA?s Rare Pediatric Disease Priority Review Voucher, granting an accelerated review process for marketing approval.