Can-Fite BioPharma Targets Lowe Syndrome with New Clinical Study
The approval process for therapies targeting rare genetic diseases is notably swift, often necessitating clinical trials involving a limited cohort of patients.
Can-Fite BioPharma Ltd. (NYSE American: CANF) (TASE: CANF), a dynamic biotechnology firm focused on developing proprietary drugs for oncological and inflammatory disorders, has announced the completion of its Phase II study design and is gearing up to commence its clinical trials for the rare genetic condition known as Lowe Syndrome.
Study Overview and Key Collaborators
The principal investigator for this initiative is Dr. Franchesca Emma from the renowned Bambino Gesù Children's Hospital in Rome, Italy. The Phase II study, which is set to be open-label, will involve five patients who will be administered 3 mg of Piclidenoson twice daily over a 12-month period. A vital objective of this investigation will be to assess the efficacy of Piclidenoson in enhancing renal uptake, measured using 99mTc-DMSA.
This project draws upon previous successful pre-clinical research led by Dr. Antonella De Matteis, an esteemed Professor in the Department of Molecular Medicine and Medical Biotechnology at the University of Naples Federico II. Furthermore, Can-Fite has entered into a collaboration agreement with Fondazione Telethon to facilitate the clinical development of Piclidenoson specifically for Lowe Syndrome, a condition that currently has no available treatment options.
Lowe Syndrome: A Brief Overview
Lowe Syndrome, also referred to as oculo-cerebro-renal syndrome (OCRL), is an X-linked genetic disorder predominantly affecting males. This multisystem condition manifests through significant visual impairments like congenital cataracts, kidney issues that typically arise in infancy, and neurological complications leading to intellectual disabilities. The life expectancy for individuals suffering from this syndrome often does not exceed 40 years. The prevalence of Lowe Syndrome is estimated at approximately 1 in 500,000 births.
Research Findings and Expert Insights
Dr. De Matteis remarked, “After testing thousands of compounds searching for a viable treatment for Lowe Syndrome, Piclidenoson is the sole candidate that has demonstrated effectiveness in pre-clinical environments. Notably, we observed a significant reduction in the urinary protein loss in our models treated with Piclidenoson.” She emphasized the drug’s extensive scientific backing regarding its safety and efficacy.
Dr. Pnina Fishman, the Chief Scientific Officer and Chairperson of Can-Fite, expressed her enthusiasm regarding these promising findings from Dr. De Matteis’s research, stating, “Piclidenoson’s efficacy in pre-clinical models of Lowe Syndrome lays the groundwork for its potential application in treating this challenging genetic condition.”
Understanding Piclidenoson
Piclidenoson stands out as a notable anti-inflammatory agent that is currently under evaluation in a pivotal Phase III clinical study for psoriasis, receiving endorsements from both the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA). It is classified as a first-in-class A3 adenosine receptor agonist, which signifies its innovative approach to treatment.
The drug’s mechanism of action involves the inhibition of inflammatory cytokines, specifically interleukin 17 and 23 (IL-17 and IL-23), alongside promoting the programmed cell death (apoptosis) of skin cell keratinocytes implicated in disease progression. This multifaceted benefit underscores its potential beyond just psoriasis treatment.
Partnership with Fondazione Telethon
Founded in 1990, Fondazione Telethon ETS is a pivotal Italian biomedical charity dedicated to overcoming rare genetic diseases through superior scientific research. They strive to translate groundbreaking research findings into effective therapies accessible to patients. The foundation’s continuous investment in research has led to the successful development of treatments for conditions like ADA-SCID and metachromatic leukodystrophy, showcasing its commitment to tackling rare diseases comprehensively.
About Can-Fite BioPharma
Can-Fite BioPharma Ltd. (NYSE American: CANF) (TASE: CFBI) is at the forefront of drug development aimed at addressing significant markets in oncological, liver, and inflammatory disease treatments. Their lead candidate, Piclidenoson, has shown promising topline results in a Phase III trial for psoriasis, with ongoing evaluations in various stages for other conditions, including hepatocellular carcinoma (HCC) and pancreatic cancer. Moreover, with over 1,600 patients participating in clinical studies to date, Can-Fite maintains an excellent safety profile.
Frequently Asked Questions
What is Piclidenoson used for?
Piclidenoson is primarily evaluated for treating psoriasis and is being studied for its potential application in Lowe Syndrome.
Who is leading the Phase II study for Lowe Syndrome?
The principal investigator for the clinical study is Dr. Franchesca Emma from Bambino Gesù Children's Hospital in Rome, Italy.
What challenges does Lowe Syndrome present?
Lowe Syndrome is a complex genetic disorder with severe visual, renal, and neurological manifestations and currently lacks effective treatment options.
What regulatory bodies are involved in the approval of Piclidenoson?
Both the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) are involved in the approval processes for Piclidenoson.
What is the significance of Can-Fite's collaboration with Fondazione Telethon?
This collaboration aims to expedite clinical development efforts for treating Lowe Syndrome and leverage Telethon's expertise in rare genetic disorders.
