Can-Fite to Initiate Phase II Study in the Rare Genetic Disease Lowe Syndrome with Piclidenoson
Can-Fite BioPharma (NYSE American: CANF) has announced the completion of Phase II study design for Piclidenoson in treating Lowe Syndrome, a rare genetic disease. The open study, led by Dr. Franchesca Emma from Bambino Gesù Children's Hospital, will enroll 5 patients treated with 3 mg Piclidenoson twice daily for 12 months.
The study aims to evaluate Piclidenoson's efficacy in increasing 99mTc-DMSA renal uptake. This initiative follows successful pre-clinical work by Dr. Antonella De Matteis at the University of Naples Federico II. Can-Fite has partnered with Fondazione Telethon for the clinical development.
Lowe Syndrome, occurring mainly in males, affects approximately 1 in 500,000 people and causes vision problems, kidney issues, and brain abnormalities. The condition currently has no available drug treatment, and patients rarely live beyond 40 years. Pre-clinical studies showed Piclidenoson significantly decreased urinary protein loss, making it the only effective compound found among thousands tested.
Can-Fite BioPharma (NYSE American: CANF) ha annunciato il completamento del design dello studio di Fase II per Piclidenoson nel trattamento della Sindrome di Lowe, una malattia genetica rara. Lo studio aperto, guidato dalla Dr.ssa Franchesca Emma dell'Ospedale Pediatrico Bambino Gesù, arruolerà 5 pazienti trattati con 3 mg di Piclidenoson due volte al giorno per 12 mesi.
Lo studio ha l'obiettivo di valutare l'efficacia del Piclidenoson nell'aumentare l'assorbimento renale di 99mTc-DMSA. Questa iniziativa segue i risultati preclinici positivi ottenuti dalla Dr.ssa Antonella De Matteis presso l'Università di Napoli Federico II. Can-Fite ha collaborato con la Fondazione Telethon per lo sviluppo clinico.
La Sindrome di Lowe, che si verifica principalmente nei maschi, colpisce circa 1 persona ogni 500.000 e causa problemi di vista, problemi renali e anomalie cerebrali. Attualmente non esiste un trattamento farmacologico disponibile per questa condizione, e i pazienti raramente superano i 40 anni. Gli studi preclinici hanno dimostrato che il Piclidenoson riduce significativamente la perdita di proteine nelle urine, rendendolo l'unico composto efficace trovato tra migliaia di testati.
Can-Fite BioPharma (NYSE American: CANF) ha anunciado la finalización del diseño del estudio de Fase II para Piclidenoson en el tratamiento de la Síndrome de Lowe, una enfermedad genética rara. El estudio abierto, dirigido por la Dra. Franchesca Emma del Hospital Infantil Bambino Gesù, inscribirá a 5 pacientes tratados con 3 mg de Piclidenoson dos veces al día durante 12 meses.
El estudio tiene como objetivo evaluar la eficacia del Piclidenoson en el aumento de la captación renal de 99mTc-DMSA. Esta iniciativa sigue al trabajo preclínico exitoso de la Dra. Antonella De Matteis en la Universidad de Nápoles Federico II. Can-Fite se ha asociado con la Fundación Telethon para el desarrollo clínico.
La Síndrome de Lowe, que ocurre principalmente en hombres, afecta aproximadamente a 1 de cada 500.000 personas y causa problemas de visión, problemas renales y anomalías cerebrales. Actualmente, no hay tratamiento farmacológico disponible para esta condición, y los pacientes rara vez viven más allá de los 40 años. Los estudios preclínicos mostraron que el Piclidenoson disminuyó significativamente la pérdida de proteínas en la orina, convirtiéndose en el único compuesto efectivo encontrado entre miles de probados.
Can-Fite BioPharma (NYSE American: CANF)는 Piclidenoson을 사용한 로우 증후군 치료를 위한 2상 연구 설계를 완료했다고 발표했습니다. 이 개방형 연구는 Bambino Gesù 아동 병원의 Franchesca Emma 박사가 주도하며, 12개월 동안 하루 두 번 3mg의 Piclidenoson으로 치료받는 5명의 환자를 모집할 예정입니다.
이 연구의 목적은 Piclidenoson이 99mTc-DMSA 신장 흡수를 증가시키는 효능을 평가하는 것입니다. 이 이니셔티브는 나폴리 페데리코 II 대학의 Antonella De Matteis 박사가 수행한 성공적인 전임상 연구에 이어 진행됩니다. Can-Fite는 임상 개발을 위해 Telethon 재단과 협력했습니다.
로우 증후군은 주로 남성에게 발생하며, 약 50만 명 중 1명에게 영향을 미치고 시각 문제, 신장 문제 및 뇌 이상을 초래합니다. 현재 이 질환에 대한 약물 치료는 없으며, 환자들은 40세를 넘기기 드뭅니다. 전임상 연구에서는 Piclidenoson이 소변의 단백질 손실을 유의미하게 감소시켰으며, 이는 수천 가지 테스트 중 유일하게 효과적인 화합물로 밝혀졌습니다.
Can-Fite BioPharma (NYSE American: CANF) a annoncé l'achèvement de la conception de l'étude de Phase II pour Piclidenoson dans le traitement du Syndrome de Lowe, une maladie génétique rare. L'étude ouverte, dirigée par Dr. Franchesca Emma de l'Hôpital Pédiatrique Bambino Gesù, recrutera 5 patients traités avec 3 mg de Piclidenoson deux fois par jour pendant 12 mois.
L'étude vise à évaluer l'efficacité du Piclidenoson à augmenter l'absorption rénale de 99mTc-DMSA. Cette initiative fait suite à des travaux précliniques réussis réalisés par Dr. Antonella De Matteis à l'Université de Naples Federico II. Can-Fite a établi un partenariat avec la Fondazione Telethon pour le développement clinique.
Le Syndrome de Lowe, qui survient principalement chez les hommes, touche environ 1 personne sur 500.000 et provoque des problèmes de vision, des problèmes rénaux et des anomalies cérébrales. Actuellement, il n'existe aucun traitement médicamenteux disponible pour cette condition, et les patients vivent rarement au-delà de 40 ans. Les études précliniques ont montré que le Piclidenoson réduisait significativement la perte de protéines urinaires, ce qui en fait le seul composé efficace trouvé parmi des milliers de tests.
Can-Fite BioPharma (NYSE American: CANF) hat den Abschluss des Studiendesigns der Phase II für Piclidenoson zur Behandlung des Lowe-Syndroms, einer seltenen genetischen Erkrankung, bekannt gegeben. Die offene Studie, geleitet von Dr. Franchesca Emma vom Bambino Gesù Kinderkrankenhaus, wird 5 Patienten einschreiben, die über einen Zeitraum von 12 Monaten zweimal täglich 3 mg Piclidenoson erhalten.
Das Ziel der Studie ist es, die Wirksamkeit von Piclidenoson bei der Erhöhung der Nierenaufnahme von 99mTc-DMSA zu bewerten. Diese Initiative folgt auf erfolgreiche präklinische Arbeiten von Dr. Antonella De Matteis an der Universität Neapel Federico II. Can-Fite hat sich mit der Fondazione Telethon für die klinische Entwicklung zusammengeschlossen.
Das Lowe-Syndrom tritt hauptsächlich bei Männern auf, betrifft etwa 1 von 500.000 Menschen und verursacht Sehprobleme, Nierenprobleme und Gehirnanomalien. Für diese Erkrankung gibt es derzeit keine verfügbare medikamentöse Behandlung, und die Patienten erreichen selten das 40. Lebensjahr. Präklinische Studien zeigten, dass Piclidenoson den Verlust von Urinproteinen signifikant verringerte und damit die einzige wirksame Verbindung unter Tausenden von getesteten darstellt.
- First potential drug treatment for Lowe Syndrome with no current therapeutic options
- Successful pre-clinical results showing significant decrease in urinary protein loss
- Fast-track potential due to rare disease status with FDA & EMA
- Partnership established with Fondazione Telethon for clinical development
- Small patient population (1 in 500,000) limiting market size
- Extended 12-month treatment period for Phase II study may delay results
- Early-stage development with no guaranteed clinical success
Insights
Can-Fite's advancement of Piclidenoson into Phase II trials for Lowe Syndrome represents a strategic pipeline expansion into the rare disease market, where treatments typically command premium pricing despite small patient populations. The company's market cap of just
The collaboration with Fondazione Telethon provides important external validation of Piclidenoson's potential in this indication. For rare genetic diseases like Lowe Syndrome (affecting approximately 1 in 500,000 individuals), regulatory agencies offer accelerated approval pathways, reduced clinical trial sizes, and extended market exclusivity periods – all factors that could benefit Can-Fite's development timeline and costs.
While the commercial opportunity is inherently by the extremely small patient population, the absence of any approved treatments for Lowe Syndrome creates an unmet medical need that could support favorable pricing if Piclidenoson proves effective. The 12-month treatment duration in the Phase II study means results won't materialize immediately, requiring patient capital.
This development leverages Can-Fite's existing Piclidenoson safety data from other indications, potentially streamlining the development process. For investors, this represents incremental pipeline value that diversifies the company's portfolio, though substantial revenue impact would still be years away given the early clinical stage.
The Phase II study of Piclidenoson in Lowe Syndrome addresses a significant void in treatment options for this rare X-linked genetic disorder. What makes this development scientifically noteworthy is Dr. De Matteis's statement that Piclidenoson was the only effective compound identified after screening thousands of candidates in preclinical models.
The study design is appropriately tailored for a rare disease investigation with just 5 patients – standard practice given the 1 in 500,000 prevalence. The primary endpoint focusing on 99mTc-DMSA renal uptake directly addresses the kidney manifestations of Lowe Syndrome, which include proteinuria and solute loss beginning in the first year of life.
Preclinical findings showing Piclidenoson's ability to significantly decrease urinary protein loss is particularly promising since renal complications are a major source of morbidity in these patients. The 12-month treatment duration is necessary to adequately assess impact on a chronic progressive condition affecting multiple body systems (ocular, cerebral, and renal).
The involvement of specialized research institutions (Bambino Gesù Children's Hospital and Telethon Institute) brings important expertise in rare genetic disorders. For a condition where life expectancy rarely exceeds 40 years, even modest improvements in disease manifestations could translate to meaningful clinical benefits, particularly if intervention can preserve kidney function over time.
FDA & EMA approvals for rare genetic diseases are fast and require clinical studies with small number of patients
Ramat Gan, Israel, March 19, 2025 (GLOBE NEWSWIRE) -- Can-Fite BioPharma Ltd. (NYSE American: CANF) (TASE:CANF), a biotechnology company advancing a pipeline of proprietary small molecule drugs that address oncological and inflammatory diseases, today announced that a Phase II design is completed and preparatory work is undergoing for the initiation of clinical study in the rare genetic disease Lowe Syndrome.
Dr. Franchesca Emma from the Division of Nephrology, Bambino Gesù Children's Hospital - IRCCS Rome Italy, will be the principal investigator of the study. The Phase II open study will enroll 5 patients that will be treated twice daily with 3 mg Piclidenoson for 12 months. The study’s primary end point will be the efficacy of Piclidenoson in increasing 99mTc-DMSA renal uptake.
The treatment of this rare genetic disease is based on successful pre-clinical work of 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 a collaboration agreement for the clinical development of Piclidenoson for the treatment of Lowe Syndrome, a high medical need with no drug available.
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 of 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 preclinical models of Lowe syndrome leads to a significant decrease of the urinary loss of proteins,” 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 in pre-clinical studies which involves renal, cerebral, and ocular manifestations.”
Can-Fite CSO&Chairperson Dr. Pnina Fishman commented, “We are very much enthusiastic by the breakthrough research of Dr. De Matteis showing that Piclidenoson is efficacious in treating pre-clinical models of Lowe Syndrome. Her impressive results are the basis for implementing Piclidenoson in the treatment of this rare genetic disease”. stated Dr. Pnina Fishman, Can-Fite CSO & Chairperson”.
About Piclidenoson
Piclidenoson is a robust anti-inflammatory agent, currently being evaluated in a pivotal Phase III psoriasis clinical study under approval of both the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA).
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 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 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 and is expected to commence a pivotal Phase III. Can-Fite's liver drug, Namodenoson, is being evaluated in a Phase III trial for hepatocellular carcinoma (HCC), a Phase IIb trial for the treatment of MASH, and in a Phase IIa study in pancreatic 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 1,600 patients in clinical studies to date. For more information please visit: https://www.canfite.com/.
Forward-Looking Statements
This press release may contain forward-looking statements, about Can-Fite’s expectations, beliefs or intentions regarding, among other things, its product development efforts, business, financial condition, results of operations, strategies or prospects. All statements in this communication, other than those relating to historical facts, are “forward looking statements”. Forward-looking statements can be identified by the use of forward-looking words such as “believe,” “expect,” “intend,” “plan,” “may,” “should” or “anticipate” or their negatives or other variations of these words or other comparable words or by the fact that these statements do not relate strictly to historical or current matters. Forward-looking statements relate to anticipated or expected events, activities, trends or results as of the date they are made. Because forward-looking statements relate to matters that have not yet occurred, these statements are inherently subject to known and unknown risks, uncertainties and other factors that may cause Can-Fite’s actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements. Important factors that could cause actual results, performance or achievements to differ materially from those anticipated in these forward-looking statements include, among other things, our history of losses and needs for additional capital to fund our operations and our inability to obtain additional capital on acceptable terms, or at all; uncertainties of cash flows and inability to meet working capital needs; the initiation, timing, progress and results of our preclinical studies, clinical trials and other product candidate development efforts; our ability to advance our product candidates into clinical trials or to successfully complete our preclinical studies or clinical trials; our receipt of regulatory approvals for our product candidates, and the timing of other regulatory filings and approvals; the clinical development, commercialization and market acceptance of our product candidates; our ability to establish and maintain strategic partnerships and other corporate collaborations; the implementation of our business model and strategic plans for our business and product candidates; the scope of protection we are able to establish and maintain for intellectual property rights covering our product candidates and our ability to operate our business without infringing the intellectual property rights of others; competitive companies, technologies and our industry; risks related to any resurgence of the COVID-19 pandemic and the war between Israel and Hamas; risks related to not satisfying the continued listing requirements of NYSE American; and statements as to the impact of the political and security situation in Israel on our business. More information on these risks, uncertainties and other factors is included from time to time in the “Risk Factors” section of Can-Fite’s Annual Report on Form 20-F filed with the SEC on March 28, 2024 and other public reports filed with the SEC and in its periodic filings with the TASE. Existing and prospective investors are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof. Can-Fite undertakes no obligation to publicly update or review any forward-looking statement, whether as a result of new information, future developments or otherwise, except as may be required by any applicable securities laws.
Contact
Can-Fite BioPharma
Motti Farbstein
info@canfite.com
+972-3-9241114
FAQ
What is the primary endpoint of Can-Fite's Phase II Lowe Syndrome trial for Piclidenoson (CANF)?
How many patients will be enrolled in Can-Fite's Phase II Lowe Syndrome study (CANF)?
What were the key findings from Piclidenoson's pre-clinical studies for Lowe Syndrome (CANF)?
Who is leading Can-Fite's Phase II Lowe Syndrome clinical study (CANF)?
