Can-Fite BioPharma (NYSE: CANF) reported that blinded overall survival in its ongoing pivotal Phase III study of Namodenoson for advanced hepatocellular carcinoma appears longer than originally anticipated.

The clinical-stage biotechnology company noted that the observed survival data reflects the pooled patient population receiving both Namodenoson and placebo. Consequently, no conclusions regarding treatment efficacy or differences between the study arms can be drawn from these preliminary observations.

Study Design And Methodology

The pivotal Phase III study is a randomized, double-blind, placebo-controlled trial evaluating Namodenoson in patients with advanced HCC and underlying Child-Pugh B7 cirrhosis. Participants are randomized 2:1 to receive either Namodenoson or a placebo. Overall survival serves as the primary efficacy endpoint for the investigation.

Next Steps For Analysis

In light of the longer-than-anticipated survival observed across the entire patient population, Can-Fite is evaluating an earlier timing for the study’s planned interim analysis. This interim analysis will enable an independent assessment of overall survival between the Namodenoson and placebo treatment groups. The assessment will proceed in accordance with the study’s statistical analysis plan and applicable regulatory requirements.

What The Numbers Show

The disclosure highlights a divergence between the actual blinded survival outcomes and the assumptions underlying the original study design. While this suggests potentially favorable kinetics in the pooled population, the lack of unblinded arm-specific data means the therapeutic signal remains unconfirmed until the formal interim analysis is conducted.

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.