Can-Fite BioPharma Ltd. (NYSE: CANF) (TASE: CANF) submitted a Phase 2 clinical study protocol to Bambino Gesù Children's Hospital in Rome, Italy, on Aug. 03, 2026, marking the first clinical evaluation of Piclidenoson in patients with Lowe syndrome. This submission represents a significant milestone for the biotechnology company, which is advancing proprietary small molecule drugs for oncological and inflammatory diseases. The move addresses a critical unmet medical need, as Lowe syndrome is a rare inherited genetic disorder with no approved disease-modifying therapies currently available.

The study will be led by Prof. Francesco Emma, an internationally recognized expert in inherited kidney diseases. Can-Fite BioPharma entered into a collaboration agreement with Fondazione Telethon to support the clinical development of Piclidenoson for this high-need indication. The selection of Piclidenoson for clinical evaluation was based on compelling preclinical studies demonstrating the restoration of OCRL-dependent cellular function.

These preclinical findings were identified by Dr. Antonella De Matteis, Professor of Biology at the Department of Molecular Medicine and Medical Biotechnology at the University of Naples Federico II. Dr. De Matteis also serves as Program Coordinator of Cell Biology and Disease Mechanisms at the Telethon Institute of Genetics and Medicine (TIGEM) in Italy. Her research provided the foundational evidence for pursuing clinical trials in humans.

Lowe syndrome is a rare X-linked multisystem genetic disorder caused by mutations in the OCRL gene. The condition results in severe renal, neurological, and ocular manifestations. The renal disease is characterized by progressive proximal tubular dysfunction, leading to Fanconi syndrome, chronic kidney disease, and eventual kidney failure. Current management strategies are supportive, with no approved therapy addressing the underlying disease mechanism.

The Phase 2 study is designed as an open-label, single-center clinical trial. It aims to evaluate the efficacy and safety of oral Piclidenoson administered twice daily for six months in five adult patients with genetically confirmed Lowe syndrome. The small study size is intended to support regulatory interactions and potential registration upon positive results.

Study Design and Endpoints

The trial focuses on specific renal and safety metrics to determine the drug's impact on proximal tubular function. The primary and secondary endpoints are structured to provide comprehensive data on therapeutic potential.

What the Numbers Show

The decision to proceed with a single-center, five-patient Phase 2 trial reflects the rarity of Lowe syndrome and the lack of existing treatment options. By focusing on renal uptake of 99mTc-DMSA as the primary endpoint, Can-Fite BioPharma is prioritizing a direct measure of the drug's ability to restore proximal tubular reabsorption capacity. This approach allows for early assessment of efficacy in a highly controlled environment before expanding to larger, multi-center studies. The collaboration with Fondazione Telethon and the involvement of leading experts like Prof. Francesco Emma and Dr. Antonella De Matteis underscore the scientific rigor behind the development strategy.

Can-Fite BioPharma Ltd. announced that an abstract highlighting positive results from its Phase 2a study of Namodenoson in patients with advanced pancreatic ductal adenocarcinoma (PDAC) has been accepted for poster presentation at the European Society for Medical Oncology (ESMO) Congress 2026. The presentation will take place at one of the world's premier scientific meetings in oncology, providing a platform to showcase clinical findings to the global community. Predominantly third-line pancreatic cancer patients demonstrated durable survival despite advanced disease, and a patient who received Namodenoson as second-line therapy remains alive more than 18 months.

The Phase 2a study evaluated oral Namodenoson in patients with advanced pancreatic cancer who had progressed following prior standard therapies. The study successfully achieved its primary safety endpoint and demonstrated encouraging survival outcomes together with durable disease stabilization in this difficult-to-treat patient population.

Namodenoson is a highly selective A3 adenosine receptor agonist with a unique mechanism of action that induces apoptosis of cancer cells while exhibiting an excellent safety profile. The drug has demonstrated anti-tumor activity across multiple preclinical models, including pancreatic cancer, and is also being developed for hepatocellular carcinoma and MASH.

Clinical Development Plans

Can-Fite is currently planning the next stage of clinical development for Namodenoson in pancreatic cancer. A Phase 2b study is designed to evaluate Namodenoson in combination with chemotherapy, based on encouraging clinical findings and supportive preclinical evidence demonstrating synergistic anti-tumor activity.

Key Study Details

"We are pleased that our abstract has been selected for presentation at ESMO, one of the most prestigious international oncology conferences," said Pnina Fishman, Ph.D., Chairperson and Chief Scientific Officer of Can-Fite BioPharma. "Acceptance by ESMO provides important scientific recognition of our pancreatic cancer program and offers an opportunity to present our clinical findings to the global oncology community. We believe these data further support the continued development of Namodenoson for patients with advanced pancreatic cancer."

Additional details regarding the poster presentation, including presentation date, session information, and poster number, will be announced when they become available.