RARE Daily

Can-Fite Enters Collaboration with Fondazione Telethon to Co-Develop Treatment for Lowe Syndrome

August 24, 2023

Rare Daily Staff

Can-Fite BioPharma said it signed an agreement with Fondazione Telethon to co-develop its lead drug candidate, Piclidenoson, for the treatment of Lowe Syndrome, a rare, multisystemic disease.

Piclidenoson, Can-Fite’s lead experimental therapy, is in late-stage development to treat psoriasis. Recent findings, though, from researchers at The Telethon Institute of Genetics and Medicine in Italy, suggests it could be effective at treating Lowe syndrome.

Lowe Syndrome, also known as oculo-cerebro-renal syndrome (OCRL), is an X-linked genetic condition occurring almost exclusively in males. It is characterized by vision problems including cataracts that are present at birth, kidney problems that usually develop in the first year of life, and brain abnormalities associated with intellectual disabilities. Lowe Syndrome prevalence is estimated at approximately 1 in 500,000.

Piclidenoson is a novel, first-in-class, A3 adenosine receptor agonist (A3AR) small molecule and orally bioavailable drug with an excellent safety profile demonstrating evidence of efficacy in phase 2 and phase 3 clinical studies. The drug’s mechanism of action entails inhibition of the inflammatory cytokines interleukin 17 and 23 (IL-17 and IL-23) and the induction of apoptosis of patients’ skin cell keratinocytes involved with the disease pathogenicity.

“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. Importantly, we observed that Piclidenoson treatment in mouse models of Lowe syndrome leads to a significant decrease of the urinary loss of proteins in diseased animals,” said Antonella De Matteis, program coordinator of the Cell Biology and Disease Mechanisms at The Telethon Institute of Genetics and Medicine in Italy. “We chose to investigate Piclidenoson based on the availability of extensive scientific data showing its excellent safety, coupled with efficacy, in this disease which involves renal, cerebral, and ocular manifestations.”

Can-Fite estimates the total addressable market for the treatment of Lowe Syndrome at approximately $100 million in the United States, 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 and the European Medicines Agency (EMA). It may also qualify for the FDA’s Rare Pediatric Disease Priority Review Voucher, granting an accelerated review process for marketing approval, the company said.

“Based on Piclidenoson’s proven safety profile in clinical trials to date, and because Lowe is a rare disease in dire need of treatment, we plan to move into an advanced stage clinical study which may open a path to approval,” said Can-Fite Chairman Pnina Fishman.

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