Namodenoson Earns FDA Orphan Drug Designation in Pancreatic Cancer

Pancreatic cancer, 3D rendering: © matthieu - stock.adobe.com

• The FDA has granted orphan drug designation to namodenoson (CF102) in pancreatic cancer.
• Orphan drug designation provides Can-Fite BioPharma Ltd, the sponsor, with regulatory advantages.
• A phase 2 study (NCT06387342) will evaluate the agent in the intent-to-treat population.

Namodenoson has been granted FDA orphan drug designation for the potential treatment of patients with pancreatic cancer.¹

With this designation, Can-Fite, the sponsor, is eligible for tax credits for clinical trials, exemption from user fees, and a potential 7 years of market exclusivity upon approval.²

”We are advancing our plans to start our phase 2 study in pancreatic cancer and aim to commence the study by the end of year;we are thrilled that the FDA has granted orphan drug status,” said Can-Fite chief executive officer Motti Farbstein in a press release.¹

The phase 2 study evaluating namodenoson will be a multicenter, open-label trial in patients with advanced pancreatic adenocarcinoma whose disease progressed on at least first-line therapy. Patients will receive namodenoson 25 mg orally twice daily for consecutive 28-day cycles. The study plans to enroll 20 evaluable patients.

The study’s primary end point will be safety, and secondary end points will include objective response rate, progression-free survival, disease control rate, duration of response, and overall survival.

Patients with an ECOG performance status of 2 or lower, adequate laboratory values, and a life expectancy of at least 8 weeks will be eligible to enroll. Those with uncontrolled thyroid disease, active infection, uncontrolled hypertension, or another active malignancy will not be able to participate in the study.³

About Namodenoson

Namodenoson is an orally available with high affinity and selectivity to the A3 adenosine receptor (A3AR) on the surface of liver and pancreatic cancer cells, inducing apoptosis. A3AR is minimally expressed in healthy cells, giving the potential for a strong safety profile with minimal off-target toxicity.¹

The agent was previously investigated in a phase 1/2 study (NCT00790218) for the treatment of advanced hepatocellular carcinoma (HCC)⁴ and a phase 2 study (NCT02128958) for the treatment of HCC with Child-Pugh B cirrhosis.⁵ A phase 3 study (NCT05201404) of namodenoson in advanced HCC with Child-Pugh B7 cirrhosis is currently recruiting.⁶

REFERENCES:

1. FDA grants orphan drug designation to Can-Fite’s namodenoson for pancreatic cancer. News release. Can-Fite BioPharma. October 9, 2024. Accessed October 9, 2024. https://tinyurl.com/59umvkb9

2. Designating an orphan product: drugs and biological products. FDA. Updated August 12, 2024. Accessed October 9, 2024. https://tinyurl.com/24muw8am

3. Namodenoson treatment of advanced pancreatic cancer. ClinicalTrials.gov. Updated August 21, 2024. Accessed October 9, 2024. https://clinicaltrials.gov/study/NCT06387342

4. A phase 1-2 study of CF102 in patients with advanced hepatocellular carcinoma. ClinicalTrials.gov. Updated May 17, 2022. Accessed October 9, 2024. https://clinicaltrials.gov/study/NCT00790218

5. Phase 2, randomized, double-blind, placebo-controlled of the efficacy and safety of CF102 in hepatocellular carcinoma (HCC). ClinicalTrials.gov. Updated October 4, 2022. Accessed October 9, 2024. https://clinicaltrials.gov/study/NCT02128958

6. Namodenoson in the treatment of advanced hepatocellular carcinoma in patients with Child-Pugh class B7 cirrhosis (LIVERATION). ClinicalTrials.gov. Updated July 31, 2024. Accessed October 9, 2024. https://clinicaltrials.gov/study/NCT05201404