Can-Fite Applies for FDA Orphan Drug Designation for Namodenoson in the Treatment of Pancreatic Cancer
Can-Fite BioPharma, a biotechnology firm focused on oncological and inflammatory diseases, has submitted an application to the FDA for Orphan Drug Designation for Namodenoson, aimed at treating pancreatic carcinoma. An orphan drug designation provides benefits including seven-year marketing exclusivity, tax credits, fee waivers, and grant eligibility. Can-Fite plans to initiate a Phase IIa multicenter open-label trial to assess Namodenoson's safety, clinical activity, and pharmacokinetics in patients with advanced pancreatic adenocarcinoma. The trial will involve approximately 20 patients receiving oral Namodenoson 25 mg twice daily over 28-day cycles. Key metrics will include safety profile, Objective Response Rate (ORR), Progression-Free Survival (PFS), Disease Control Rate (DCR), Duration of Response (DoR), and Overall Survival (OS). Namodenoson has already been granted Orphan Drug Status for advanced liver cancer by the FDA and EMA.
- Submission of FDA Orphan Drug Designation application for Namodenoson in pancreatic carcinoma.
- Potential market exclusivity for seven years upon approval.
- Eligibility for 50% tax credit on clinical investigation expenses.
- Waiver of Prescription Drug User Fee Act (PDUFA) application fees.
- Assistance in the drug development process and eligibility for Orphan Products Grant funding.
- Namodenoson already has Orphan Drug Status for advanced liver cancer by both FDA and EMA.
- None.
Insights
The submission of an application for Orphan Drug Designation for Namodenoson in the treatment of pancreatic cancer is a significant milestone. Pancreatic cancer is a particularly aggressive form of cancer with limited treatment options and a generally poor prognosis. The potential benefits of Namodenoson, if granted this designation, include longer-term market exclusivity and financial incentives that could expedite its development and availability to patients.
Namodenoson has previously shown promising results in other indications, including advanced liver cancer. The planned Phase IIa clinical trial will be important to assess its efficacy and safety in patients who have failed first-line therapies for pancreatic adenocarcinoma. This trial's focus on safety and various clinical activity metrics like Objective Response Rate (ORR) and Overall Survival (OS) is essential to determine whether Namodenoson can provide a tangible benefit in this hard-to-treat cancer.
However, it is essential to approach these developments with cautious optimism. While Orphan Drug Designation can accelerate the development process, the actual clinical efficacy and safety outcomes will be the ultimate determinants of success. Investors should keep a close eye on the trial results, especially given the high-risk nature of oncology drug development.
The application for Orphan Drug Designation for Namodenoson by Can-Fite BioPharma is potentially significant from a financial perspective. If approved, the benefits include seven years of market exclusivity, substantial tax credits and reduced regulatory fees, all of which can considerably lower the drug's development cost and enhance profitability. These incentives can provide a competitive edge that might attract further investments or partnerships.
Historically, the biotech sector reacts positively to such regulatory milestones, often leading to a short-term boost in stock price. However, investors should also consider the high-risk nature of clinical trials in oncology. While Namodenoson has shown promise in earlier studies, the upcoming Phase IIa trial's results will be critical. Any negative outcomes could adversely impact stock performance and market confidence.
In the broader context, Can-Fite's strategy seems to hinge on leveraging regulatory designations to advance their pipeline. This approach is prudent, but the company's long-term financial health will heavily depend on successful trial outcomes and subsequent market adoption of its therapies.
The FDA Orphan Drug Designation application for Namodenoson highlights Can-Fite's commitment to addressing rare and hard-to-treat diseases. Orphan designation not only provides financial and regulatory incentives but also underscores the significance of the unmet medical need in pancreatic cancer. This form of cancer has a high mortality rate, with limited effective treatments available.
The planned Phase IIa trial focusing on multiple endpoints like Progression-Free Survival (PFS) and Disease Control Rate (DCR) will provide comprehensive data on Namodenoson's clinical benefits. The open-label nature of the trial allows for real-time assessment of the drug's efficacy and safety, which is particularly important for conditions with poor prognosis. However, it's worth noting that the small sample size (approximately 20 patients) may limit the generalizability of the findings.
The previous Orphan Drug Status for Namodenoson in advanced liver cancer adds a layer of credibility to this new application. Still, investors should remain vigilant about the trial's outcomes and any subsequent regulatory feedback. While the potential for success is significant, the high attrition rates in oncology drug development necessitate a balanced perspective on this news.
PETACH TIKVA, Israel, July 11, 2024 (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 it has submitted an application to the U.S. Food and Drug Administration (FDA) for Orphan Drug Designation for its drug candidate Namodenoson in the treatment of pancreatic carcinoma.
An orphan drug is defined in the 1984 amendments of the U.S. Orphan Drug Act (ODA) as a drug intended to treat a condition affecting fewer than 200,000 persons in the United States. Orphan designation qualifies the sponsor of the product for seven-year marketing exclusivity to the first sponsor obtaining FDA approval of a designated drug, a tax credit equal to
Can-Fite plans to start shortly a Phase IIa clinical study that will be a multicenter open-label trial in patients with advanced pancreatic adenocarcinoma whose disease has progressed on at least first line therapy. The trial will evaluate the safety, clinical activity, and pharmacokinetics (PK) of Namodenoson in this population. All patients will receive oral Namodenoson 25 mg administered twice daily for consecutive 28-day cycles. Patients will be evaluated regularly for safety. Approximately 20 evaluable patients will be enrolled. The primary objective of this trial is to characterize the safety profile of Namodenoson and the secondary objective is to evaluate the clinical activity as determined by the Objective Response Rate (ORR) using Response Evaluation Criteria in Solid Tumors (RECIST 1.1), Progression-Free Survival (PFS), Disease Control Rate (DCR), Duration of Response (DoR), and Overall Survival (OS). Can-Fite has already been granted Orphan Drug Status for Namodenoson for the indication of advanced liver cancer by the FDA and also by the EMA.
“The Orphan Drug application for Namodenoson underscores the high unmet medical need for a safe and efficacious drug for this devastating disease," said Motti Farbstein, CEO of the Company. "This application further validates our belief that Namodenoson may potentially offer efficacy on top of the drug safety that has been already proved in other clinical indications. Upon marketing approval, receiving market exclusivity for Namodenoson would be significantly beneficial to Can-Fite.”
1 https://www.fda.gov/media/83372/download
About Namodenoson
Namodenoson is a small orally bioavailable drug that binds with high affinity and selectivity to the A3 adenosine receptor (A3AR). Namodenoson was evaluated in Phase II trials for two indications, as a second line treatment for hepatocellular carcinoma, and as a treatment for non-alcoholic fatty liver disease (NAFLD) and non-alcoholic steatohepatitis (NASH). A3AR is highly expressed in diseased cells whereas low expression is found in normal cells. This differential effect accounts for the excellent safety profile of the drug.
About Can-Fite BioPharma Ltd.
Can-Fite BioPharma Ltd. (NYSE American: CANF) (TASE: CANF) 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. Can-Fite's liver drug, Namodenoson, is being evaluated in a Phase IIb trial for the treatment of NASH a Phase III trial for hepatocellular carcinoma (HCC), and the Company is planning 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: 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
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