Can-Fite to Initiate Phase II Study in the Rare Genetic Disease Lowe Syndrome with Piclidenoson

Core Viewpoint - Can-Fite BioPharma Ltd. is advancing its clinical study for Piclidenoson, targeting the rare genetic disease Lowe Syndrome, with a Phase II trial set to begin soon [1][3]. Company Overview - Can-Fite BioPharma Ltd. is a biotechnology company focused on developing small molecule drugs for oncological and inflammatory diseases [1][11]. - The company’s lead drug candidate, Piclidenoson, is currently in pivotal Phase III trials for psoriasis and has shown efficacy in preclinical studies for Lowe Syndrome [6][11]. Clinical Study Details - The Phase II study for Lowe Syndrome will enroll 5 patients, who will receive 3 mg of Piclidenoson twice daily for 12 months, with the primary endpoint being the efficacy in increasing renal uptake [2][3]. - The study is led by Dr. Franchesca Emma from Bambino Gesù Children's Hospital in Rome, Italy [2]. Disease Background - Lowe Syndrome, an X-linked genetic condition, primarily affects males and is characterized by vision problems, kidney issues, and intellectual disabilities, with a prevalence of approximately 1 in 500,000 [4][5]. - There are currently no available drugs for Lowe Syndrome, highlighting the medical need for effective treatments [3][4]. Research Collaboration - Can-Fite has partnered with Fondazione Telethon for the clinical development of Piclidenoson, leveraging their expertise in rare genetic diseases [3][8]. Piclidenoson Overview - Piclidenoson is a first-in-class A3 adenosine receptor agonist with a strong safety profile and evidence of efficacy in both Phase II and Phase III studies [7][11]. - The drug works by inhibiting inflammatory cytokines IL-17 and IL-23 and inducing apoptosis in skin cell keratinocytes [7]. Fondazione Telethon Contributions - Fondazione Telethon has invested over 660 million euros in research, funding nearly 3,000 projects and studying 630 diseases, aiming to develop therapies for rare genetic conditions [9][10].