- Can-Fite Signed agreement with Fondazione Telethon for
co-development of Piclidenoson for the treatment of Lowe syndrome
an estimated $100 M market in the U.S. only with no drug
available
- FDA & EMA approvals for rare genetic diseases are fast and
require clinical studies with small number of patients
- 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 BioPharma Ltd. (NYSE American: CANF) (TASE: CANF), a
biotechnology company advancing a pipeline of proprietary small
molecule drugs that address inflammatory, cancer and liver
diseases, announced today 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.
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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
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. (Graphic: Business
Wire)
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.
“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,” Dr. De Matteis
stated. “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 Chairman Dr. Pnina Fishman commented, “We are hopeful
that Piclidenoson can offer a much needed treatment for infants,
children, and young people living with Lowe Syndrome. 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. Dr. De Matteis and her team have made an
impactful discovery with Piclidenoson and we look forward to
working with her and Fondazione Telethon.”
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.
About Fondazione Telethon
Fondazione Telethon ETS is one of the main Italian biomedical
charities, founded in 1990 on the initiative of a group of patients
suffering from muscular dystrophy. Its mission is to achieve the
cure of rare genetic diseases through scientific research of
excellence, selected according to the best practices shared
internationally. Through a unique method in the Italian panorama,
it follows the entire "research chain" dealing with fundraising,
selection and funding of projects and the research activity itself
carried out in the centers and laboratories of the Foundation.
Telethon also develops collaborations with public health
institutions and pharmaceutical industries to translate the results
of research into therapies accessible to patients. Since its
foundation, Telethon has invested more than 660 million euros in
research, has funded 2,960 projects with 1,720 researchers involved
and 630 diseases studied. To date, thanks to Fondazione Telethon,
the first gene therapy with stem cells in the world has been made
available, thanks to the collaboration with the pharmaceutical
industry. This therapy is intended for the treatment of ADA-SCID, a
severe immunodeficiency that compromises the body's defenses from
birth. In 2023, Fondazione Telethon became responsible for the
production and distribution of the drug to eligible patients in the
European Union
Another gene therapy resulting from Telethon research made
available is the one for a serious neurodegenerative disease,
metachromatic leukodystrophy. This therapeutic approach is in an
advanced stage of development for another immunodeficiency,
Wiskott-Aldrich syndrome. Other diseases on which the gene therapy
developed by Telethon researchers has been evaluated in patients
are beta thalassemia and two metabolic diseases of childhood,
mucopolysaccharidosis type 6 and type 1. In addition, within the
Telethon institutes a targeted therapeutic strategy is being
studied or developed for other genetic diseases, such as hemophilia
or various hereditary vision defects. In parallel, the study of
basic mechanisms and potential therapeutic approaches for diseases
still unanswered continues in all laboratories funded by
Telethon.
About Piclidenoson
Piclidenoson is a novel, first-in-class, A3 adenosine receptor
agonist (A3AR) small molecule, orally bioavailable drug with an
excellent safety profile demonstrating evidence of efficacy in
Phase II and Phase III 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.
About Can-Fite BioPharma Ltd.
Can-Fite BioPharma Ltd. (NYSE American: CANF) (TASE: CFBI) is an
advanced clinical stage drug development Company with a platform
technology that is designed to address multi-billion dollar markets
in the treatment of cancer, liver, and inflammatory disease. The
Company's lead drug candidate, Piclidenoson recently reported
topline results in a Phase III trial for psoriasis. Can-Fite's
liver drug, Namodenoson, is being evaluated in a Phase IIb trial
for the treatment of non-alcoholic steatohepatitis (NASH), and
enrollment is expected to commence in a Phase III trial for
hepatocellular carcinoma (HCC), the most common form of liver
cancer. Namodenoson has been granted Orphan Drug Designation in the
U.S. and Europe and Fast Track Designation as a second line
treatment for HCC by the U.S. Food and Drug Administration.
Namodenoson has also shown proof of concept to potentially treat
other cancers including colon, prostate, and melanoma. CF602, the
Company's third drug candidate, has shown efficacy in the treatment
of erectile dysfunction. These drugs have an excellent safety
profile with experience in over 1,500 patients in clinical studies
to date. For more information please visit: www.can-fite.com.
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Can-Fite BioPharma Motti Farbstein info@canfite.com
+972-3-9241114
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