Can-Fite BioPharma Ltd. announced a significant clinical finding from its compassionate use program, reporting the complete resolution of esophageal varices in a patient with decompensated liver cirrhosis treated with Namodenoson. The resolution was confirmed through endoscopic evaluation, representing an uncommon clinical outcome that may suggest a disease-modifying effect of the investigational drug.
The patient had previously been reported by Can-Fite to have experienced the disappearance of end-stage liver disease complications while receiving Namodenoson treatment. Esophageal varices represent a common and severe complication of advanced liver disease, carrying a high risk of life-threatening gastrointestinal bleeding.
Clinical Significance and Expert Commentary
"This case highlights the potential of Namodenoson to address life-threatening complications of advanced liver disease," stated Prof. Ohad Etzion, Chief of Gastroenterology and Liver Diseases at Soroka Medical Center in Israel. "We are encouraged by these findings, which may provide important insights for Namodenoson's ongoing clinical development."
Endoscopic images of the patient's esophagus before and after Namodenoson treatment demonstrate the disappearance of varices under compassionate use therapy, providing visual confirmation of the therapeutic response.
Current Clinical Development Program
Namodenoson is currently being evaluated in a Phase III clinical trial for the treatment of hepatocellular carcinoma (HCC) in patients with advanced liver disease (Child-Pugh B). The drug is also undergoing evaluation in a Phase IIb trial for the treatment of Metabolic Dysfunction-associated Steatohepatitis (MASH) and in a Phase IIa study in pancreatic cancer.
Data from compassionate use cases may provide valuable supplementary evidence regarding Namodenoson's broader therapeutic potential beyond its primary indications.
Drug Mechanism and Safety Profile
Namodenoson is a small orally bioavailable drug that binds with high affinity and selectivity to the A3 adenosine receptor (A3AR). The A3AR is highly expressed in diseased cells while showing low expression in normal cells. This differential expression may be one of the important factors that accounts for the excellent safety profile of the drug.
The company reports that these drugs have demonstrated an excellent safety profile with experience in over 1,600 patients in clinical studies to date.
Market Context and Unmet Medical Need
In 2017, an estimated 10.6 million people globally were affected by decompensated cirrhosis, with available treatment options remaining scarce, especially for patients who have reached advanced stages of the disease. The American Liver Foundation has highlighted the urgent need for new therapies, stating that there are more patients in need of a liver transplant than available organs, with some patients waiting over five years for a transplant.
The U.S. market for liver cirrhosis treatment is projected to grow to approximately $15 billion by 2030, underscoring the significant commercial opportunity for effective therapies.
Regulatory Status and Broader Pipeline
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. The drug has also shown proof of concept to potentially treat other cancers including colon, prostate, and melanoma.
Can-Fite BioPharma's broader pipeline includes Piclidenoson, which reported topline results in a Phase III trial for psoriasis and commenced a pivotal Phase III trial, and CF602, which has shown efficacy in the treatment of erectile dysfunction.
