On April 25, 2024 HOOKIPA Pharma Inc., a company developing a new class of immunotherapeutics based on its proprietary arenavirus platform, reported its final pivotal Phase 2/3 trial design for HB-200 in combination with pembrolizumab (Press release, Hookipa Biotech, APR 25, 2024, View Source [SID1234642344]). The Phase 2/3 trial design and protocol are based on alignment with the U.S. Food and Drug Administration (FDA) following the Company’s Type C meeting with the FDA.

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The seamless Phase 2/3 trial is for the investigational product HB-200 in combination with pembrolizumab for the treatment of patients with Human Papillomavirus 16-positive (HPV16+) recurrent/metastatic PD-L1 CPS ≥ 20 oropharyngeal squamous cell carcinoma (OPSCC) in the first line setting. The Company anticipates the first patient will be enrolled in the fourth quarter of 2024.

The Company also announced acceptance of its HB-200 study abstract as an oral presentation at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) 2024 Annual Meeting in the head and neck cancer session to be held on June 4, 2024. The presentation will include data for approximately 40 head and neck cancer patients treated with HB-200 in combination with pembrolizumab.

"It has been a dream of mine—and my HOOKIPA colleagues—to translate our science into a product that will make an impact for patients and help them fight cancer and infectious diseases. I am happy to say that we are taking a very big step forward to making this dream a reality," said Joern Aldag, Chief Executive Officer of HOOKIPA. "We believe our data is best-in-class and puts us in the lead position for OPSCC in the first line setting. We have alignment with the FDA on our pivotal trial design and protocol, as well as PRIME designation for HB-200 in combination with pembrolizumab for the treatment of patients with OPSCC in the first line setting from the European Medicines Agency. We believe our trial design and alignment with our primary regulators can help us reach a potential registration more quickly. Further, we have been accepted for an oral abstract presentation at ASCO (Free ASCO Whitepaper) in June 2024 where we will present an update from our Phase 1/2 trial with approximately 40 patients treated with the combination of HB-200 and pembrolizumab."

HB-200 in combination with pembrolizumab pivotal Phase 2/3 trial design summary

The trial will treat patients with HPV16+ recurrent/metastatic PD-L1 CPS ≥ 20 oropharyngeal squamous cell carcinoma in the first line setting.
The trial is expected to enroll approximately 250 patients across the seamless Phase 2/3 design.
Patients will be randomized one-to-one for HB-200 plus pembrolizumab or placebo plus pembrolizumab.
The primary endpoints are objective response rate for the Phase 2 portion and overall survival for the Phase 3 portion.
The Company may seek accelerated approval based on data from the Phase 2 portion of the trial, from approximately half of the Phase 2/3 study patients, if favorable.
Phase 2 primary analysis expected in 2026 with potential subsequent filing for accelerated approval.
The Company will host a conference call today where HOOKIPA’s Executive Team will discuss the full details of the trial design, and the Company’s clinical development strategy for oncology.

Call Details:
HOOKIPA HB-200 Phase 2/3 Clinical Trial Update
Thursday, April 25, 2024, 8:00 a.m. ET
Webcast Registration
Dial-in Registration

Abstract details: ASCO (Free ASCO Whitepaper) 2024 Annual Meeting

HB-200:
Title: HB-200 arenavirus-based immunotherapy plus pembrolizumab as first-line treatment of patients with recurrent/metastatic HPV16-positive head and neck cancer: Updated results
Presenter: Dr. Alan L. Ho, Head and Neck Oncologist at Memorial Sloan Kettering Cancer Center and a trial investigator
Abstract Type: Oral abstract
Session Name: Head and Neck Cancer
Session Date and Time: June 4, 2024; 9:45 AM-12:45 PM CDT
Abstract Number: 6005

Title: Neoadjuvant HPV16-specific arenavirus-based immunotherapy HB-200 plus chemotherapy followed by response-stratified de-intensification in HPV16+ oropharyngeal cancer: TARGET-HPV
Presenter: Dr. Ari Rosenberg, Principal Investigator, TARGET-HPV Trial, University of Chicago Medicine
Abstract Type: Rapid oral abstract
Session Name: Head and Neck Cancer
Session Date and Time: June 3, 2024; 8:00 AM-9:30 AM CDT
Abstract Number: 6017
Trial Sponsor: UChicago Medicine

HB-700
Title: Development of an arenavirus-based immunotherapy for treatment of KRAS mutant cancer
Abstract Type: Abstract only
Session Date: May 23, 2024
Abstract Number: e14672

About HB-200
HB-200 is HOOKIPA’s lead oncology candidate engineered with the company’s proprietary replicating arenaviral vector platform. It comprises two single-vector compounds with arenaviral backbones based on lymphocytic choriomeningitis virus (LCMV) and pichinde virus (PICV). Both express the same transgene encoding an E7E6 fusion protein derived from HPV16. HB-200 is an alternating 2-vector immunotherapy designed to further focus the immune response against the encoded antigen.

HB-200 in combination with pembrolizumab received Fast Track Designation from the U.S. Food and Drug Administration and PRIME designation from the European Medicines Agency for the treatment of first-line HPV16+ recurrent/metastatic oropharyngeal squamous cell carcinoma. These designations are supported by preliminary clinical evidence from the Phase 1/2, open-label, clinical trial (NCT04180215) evaluating safety, T cell response, and efficacy based on objective response rate (ORR) and disease control rate (DCR) as defined by RECIST 1.1. and iRECIST. As presented at the European Society for Medical Oncology Annual Congress 2023, HB-200 in combination with pembrolizumab showed a 42 percent confirmed ORR and disease control rate DCR of 74 percent across 19 evaluable patients, doubling the 19 percent ORR for pembrolizumab alone.

On April 25, 2024 Gilead Sciences, Inc. reported its first quarter 2024 results of operations (Press release, Gilead Sciences, APR 25, 2024, View Source [SID1234642343]).

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"Gilead delivered another strong quarter of revenue growth in the first quarter with 6% year-over-year growth in our base business driven by HIV, Oncology and Liver Disease," said Daniel O’Day, Gilead’s Chairman and Chief Executive Officer. "The acquisition of CymaBay brings us another potentially transformative therapy for people with liver disease, and a regulatory decision on seladelpar is expected in August. New HIV data demonstrates the continued progress in our long-acting HIV pipeline, and we look forward to providing updates on this and our broad Oncology portfolio throughout the rest of 2024."
First Quarter 2024 Financial Results
•Total first quarter 2024 revenue increased 5% to $6.7 billion, compared to the same period in 2023, primarily due to higher HIV, Oncology and Liver Disease sales.
•Diluted (loss) earnings per share ("EPS") was $(3.34) in the first quarter 2024, compared to $0.80 in the same period in 2023. The decrease was primarily driven by an acquired in-process research and development ("IPR&D") charge of $3.9 billion, or $3.14 per share, related to the acquisition of CymaBay Therapeutics, Inc. ("CymaBay"), as well as a pre-tax IPR&D impairment of $2.4 billion, or $1.46 per share, related to assets acquired by Gilead from Immunomedics, Inc. ("Immunomedics") in 2020.
•Non-GAAP diluted (loss) EPS was $(1.32) in the first quarter 2024, compared to $1.37 in the same period in 2023. The decrease was primarily driven by the charge related to the acquisition of CymaBay.
•As of March 31, 2024, Gilead had $4.7 billion of cash, cash equivalents and marketable debt securities, compared to $8.4 billion as of December 31, 2023.
•During the first quarter 2024, Gilead generated $2.2 billion in operating cash flow.
•During the first quarter 2024, Gilead paid dividends of $990 million and repurchased $400 million of common stock.
First Quarter 2024 Product Sales
Total first quarter 2024 product sales increased 5% to $6.6 billion, compared to the same period in 2023. Total product sales, excluding Veklury, increased 6% to $6.1 billion in the first quarter 2024, compared to the same period in 2023, primarily due to higher sales in HIV, Oncology and Liver Disease.
HIV product sales increased 4% to $4.3 billion in the first quarter 2024, compared to the same period in 2023, primarily driven by higher demand.
•Biktarvy (bictegravir 50mg/emtricitabine 200mg ("FTC")/tenofovir alafenamide 25mg ("TAF")) sales increased 10% to $2.9 billion in the first quarter 2024, compared to the same period in 2023, primarily driven by higher demand in the United States, Europe and other international markets.

April 25, 2024
2

•Descovy (FTC 200mg/TAF 25mg) sales decreased 5% to $426 million in the first quarter 2024, compared to the same period in 2023, primarily driven by lower average realized price due to channel mix, partially offset by higher demand.
The Liver Disease portfolio sales increased 9% to $737 million in the first quarter 2024, compared to the same period in 2023. This was primarily driven by favorable inventory dynamics, the timing of chronic hepatitis C virus ("HCV") purchases by the Department of Corrections in the United States, as well as higher demand across chronic hepatitis B virus ("HBV"), HCV and, in the European Union ("EU"), chronic hepatitis D virus ("HDV").
Veklury sales decreased 3% to $555 million in the first quarter 2024, compared to the same period in 2023, primarily driven by lower rates of COVID-19 related hospitalizations.
Cell Therapy product sales increased 7% to $480 million in the first quarter 2024, compared to the same period in 2023.
•Yescarta (axicabtagene ciloleucel) sales increased 6% to $380 million in the first quarter 2024, compared to the same period in 2023, primarily driven by strong demand in relapsed or refractory ("R/R") large B-cell lymphoma ("LBCL") outside the United States.
•Tecartus (brexucabtagene autoleucel) sales increased 13% to $100 million in the first quarter 2024, compared to the same period in 2023, with increased demand in R/R adult acute lymphoblastic leukemia and R/R mantle cell lymphoma.
Trodelvy (sacituzumab govitecan-hziy) sales increased 39% to $309 million in the first quarter 2024, compared to the same period in 2023, primarily driven by higher demand.
First Quarter 2024 Product Gross Margin, Operating Expenses and Effective Tax Rate
•Product gross margin was 76.6% in the first quarter 2024, compared to 77.8% in the same period in 2023, primarily driven by product mix and higher intangible asset amortization expenses. Non-GAAP product gross margin was 85.4% in the first quarter 2024, compared to 86.2% in the same period in 2023, primarily driven by product mix.
•Research & development ("R&D") expenses were $1.5 billion in the first quarter 2024, compared to $1.4 billion in the same period in 2023, primarily driven by costs related to the acquisition of CymaBay and restructuring expenses. Non-GAAP R&D expenses were $1.4 billion in the first quarter 2024, flat with the same period in 2023.
•Acquired IPR&D expenses were $4.1 billion in the first quarter 2024, primarily driven by the $3.9 billion charge related to the acquisition of CymaBay that closed on March 22, 2024.
•IPR&D impairment was $2.4 billion related to the assets acquired from Immunomedics in 2020 with no similar charges in 2023.
•Selling, general and administrative ("SG&A") expenses were $1.4 billion in the first quarter 2024, compared to $1.3 billion in the same period in 2023. This increase reflects costs related to the acquisition of CymaBay and restructuring expenses. Non-GAAP SG&A expenses were $1.3 billion in the first quarter 2024, flat with the same period in 2023.
•The effective tax rate ("ETR") was 7.0% in the first quarter 2024, compared to 24.3% in the same period in 2023, and non-GAAP ETR was (29.8)% in the first quarter 2024, compared to 18.9% in the same period in 2023. These changes primarily reflect the non-deductible acquired IPR&D charge for CymaBay.

April 25, 2024
3

Guidance and Outlook
For the full-year, Gilead expects:
(in millions, except per share amounts) 4/25/24 Guidance
Low End High End Comparison to Prior Guidance
Product sales $ 27,100 $ 27,500
Unchanged
Product sales, excluding Veklury $ 25,800 $ 26,200
Unchanged
Veklury $ 1,300 $ 1,300 Unchanged
Diluted EPS $ 0.10 $ 0.50
Previously $5.15 to $5.55
Non-GAAP diluted EPS $ 3.45 $ 3.85
Previously $6.85 to $7.25

Additional information and a reconciliation between GAAP and non-GAAP financial information for the 2024 guidance is provided in the accompanying tables. The financial guidance is subject to a number of risks and uncertainties. See the Forward-Looking Statements section below.
Key Updates Since Our Last Quarterly Release
Virology
•Presented data at the Conference on Retroviruses and Opportunistic Infections ("CROI") across Gilead’s HIV long-acting treatment pipeline. For once-weekly oral dosing, this included Phase 2 data evaluating lenacapavir in combination with Merck & Co., Inc.’s islatravir as well as initial Phase 1b data for GS-1720, Gilead’s novel, investigational integrase inhibitor. Additionally, updated results were presented from the twice-yearly injectable Phase 1b study of lenacapavir in combination with investigational broadly neutralizing antibodies, teropavimab and zinlirvimab.
•Announced data at CROI evaluating Biktarvy for treatment of people with HIV and coinfections of HBV or tuberculosis, as well as results from a Phase 2/3 study evaluating once-daily oral combination of bictegravir and lenacapavir.
•Presented multiple real-world analyses at CROI supporting the use of Veklury for people hospitalized with COVID-19, including in immunocompromised people. Additionally, presented a real-world analysis evaluating the impact of Veklury on the risk of developing long-COVID.
•Announced data at CROI evaluating the safety and efficacy of Hepcludex (bulevirtide) in people living with coinfections of HIV, HBV and HDV. In the United States and other areas outside of the EU and European Economic Area, bulevirtide is an investigational product and its safety and efficacy have not been established.
•Received approval by the U.S. Food and Drug Administration ("FDA") to expand Biktarvy’s label to include treatment of people with HIV who have suppressed viral loads with known or suspected M184V/I resistance.
•Received approval from FDA to expand the indication for Vemlidy (tenofovir alafenamide) to include treatment of chronic HBV in children six years and older who weigh at least 25 kg with compensated liver disease.
Oncology
•Announced a research collaboration, option and license agreement with Merus N.V. ("Merus") to discover novel antibody-based trispecific T-cell engagers in oncology.
•Entered into an exclusive license agreement with Xilio Therapeutics, Inc. ("Xilio") to develop and commercialize Xilio’s tumor-activated IL-12 program, including investigational candidate XTX301 in advanced solid tumors.

April 25, 2024
4

Inflammation
•Completed the acquisition of CymaBay for $4.3 billion in total equity value, or $3.9 billion net cash paid, adding investigational candidate seladelpar for the treatment of primary biliary cholangitis ("PBC") to Gilead’s Liver Disease portfolio. Seladelpar is an investigational, oral, selective peroxisome proliferator-activated receptor delta (PPARδ) agonist, with Orphan Drug Designation in the United States and Europe. PPARδ has been shown to regulate critical metabolic and liver disease pathways. FDA accepted the New Drug Application for seladelpar in February 2024 for priority review, with a Prescription Drug User Fee Act target action date of August 14, 2024.
Corporate
•Announced that Kevin Lofton is retiring from Gilead’s Board of Directors (the "Board"), effective at the conclusion of the Annual Meeting of Stockholders ("Annual Meeting") on May 8, 2024. Anthony Welters, if re-elected at the Annual Meeting, will succeed Mr. Lofton as Lead Independent Director.
•Recognized as one of America’s Most JUST Companies by Just Capital and CNBC, reflecting Gilead’s longstanding commitment to operate responsibly.
•The Board declared a quarterly dividend of $0.77 per share of common stock for the second quarter of 2024. The dividend is payable on June 27, 2024, to stockholders of record at the close of business on June 14, 2024. Future dividends will be subject to Board approval.
Certain amounts and percentages in this press release may not sum or recalculate due to rounding.
Conference Call
At 1:30 p.m. Pacific Time today, Gilead will host a conference call to discuss Gilead’s results. A live webcast will be available on View Source and will be archived on www.gilead.com for one year.

On April 25, 2024 Enterome, a clinical-stage company developing first-in-class immunomodulatory drugs for solid and liquid malignancies and inflammatory diseases based on its unique Mimicry platform, reported that clinical data from the ongoing Phase 1/2 trial of EO2463 in monotherapy and in combination with lenalidomide and/or rituximab in indolent non-Hodgkin lymphoma (NHL) will be presented at 2024 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting, to take place May 31 – June 4, in Chicago, Illinois (Press release, Enterome, APR 25, 2024, View Source [SID1234642342]).

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Poster presentation details – Abstract 7058

Title: Phase 1/2 of EO2463 immunotherapy as monotherapy and in combination with lenalidomide and/or rituximab in indolent NHL (EONHL1-20/SIDNEY)

Presenting Author: J.C. C. Villasboas Bisneto, M.D., Mayo Clinic

Session Name: Hematologic Malignancies—Lymphoma and Chronic Lymphocytic Leukemia

Session Date and Time: June 3rd, 9:00 AM-12:00 PM CDT

SIDNEY (EONHL1-20) is a Phase 1/2 multicenter, open-label, first-in-human study of EO2463 as monotherapy and in combination with lenalidomide and/or rituximab for the treatment of patients with indolent non-Hodgkin lymphoma (NHL). The study aims to assess the safety, tolerability, immunogenicity, and preliminary efficacy of EO2463 monotherapy and combination therapy in approximately 60 patients with follicular lymphoma (FL) and marginal zone lymphoma (MZL). For more information on the study, refer to Clinicaltrials.gov identifier: NCT04669171.

About EO2463:
EO2463 is an innovative, off-the-shelf immunotherapy candidate that combines four synthetic OncoMimic peptides. These non-self, microbial-derived peptides correspond to CD8 HLA-A2 epitopes that exhibit molecular mimicry with the B lymphocyte-specific lineage markers CD20, CD22, CD37, and CD268 (BAFF receptor). EO2463 also includes the helper peptide (CD4+ epitope) universal cancer peptide 2 (UCP2).

The unique ability of EO2463 immunotherapy to selectively target multiple B cell markers enables the destruction of malignant B lymphocytes that are abundant in iNHL. By ensuring broad target coverage across malignant B cells while avoiding a detrimental impact on normal peripheral B cells, this novel approach aims to simultaneously improve safety and maximize efficacy, reducing the tumor cells’ capacity to develop immune escape mechanisms.

On April 25, 2024 Candel Therapeutics, Inc. (Candel or the Company) (Nasdaq: CADL), a clinical stage biopharmaceutical company focused on developing multimodal biological immunotherapies to help patients fight cancer, reported that two abstracts were accepted for presentation at the 2024 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting taking place May 31 to June 4, 2024, in Chicago, IL (Press release, Candel Therapeutics, APR 25, 2024, View Source [SID1234642341]). The first presentation will feature data from the ongoing phase 1 clinical trial of CAN-3110 in patients with recurrent high-grade glioma. The presentation will focus on patients recruited in cohort C, treated with multiple injections of CAN-3110 (up to six), demonstrating that this approach is both feasible and well tolerated. The second presentation will show topline overall survival data from the phase 2 clinical trial of CAN-2409 plus valacyclovir, in combination with continued immune checkpoint inhibitor (ICI) treatment in patients with non-resectable, stage III/IV non-small cell lung cancer (NSCLC) who have an inadequate response to front line anti-PD(L)1 therapy.

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Details are as follows:

CAN-3110 – Recurrent High-Grade Glioma

•
Trials-in-Progress Poster Presentation Title: Longitudinal stereotactic injections of oncolytic immunoactivating rQNestin34.5v.2 (CAN-3110) with concomitant biopsies for "-omic" analyses in recurrent glioblastoma (GBM).

•
Presenter: David A. Reardon, MD, Professor of Medicine at Harvard Medical School; Clinical Director, Center for Neuro-Oncology at Dana Farber Cancer Institute

•
Session Title: Poster Session – Central Nervous System Tumors

•
Session Date/Time: Saturday, June 1, 2024; 9:00 AM – 12:00 PM CT

•
Location: Hall A, McCormick Place Convention Center, Chicago, IL

CAN-2409 – Non-Small Cell Lung Cancer

•
Poster Presentation Title: Overall survival after treatment with CAN- 2409, plus valacyclovir in combination with continued ICI in patients with stage III/IV NSCLC with inadequate response to ICI.

•
Presenter: Charu Aggarwal, MD, MPH, Associate Professor for Lung Cancer Excellence, Perelman School of Medicine, University of Pennsylvania

•
Session Title: Poster Session – Lung Cancer – Non-Small Cell Metastatic

•
Session Date/Time: Monday, June 3, 2024; 1:30 PM – 4:30 PM CT

•
Location: Hall A, McCormick Place Convention Center, Chicago, IL

Full abstracts will be released by ASCO (Free ASCO Whitepaper) on Thursday, May 23, 2024, at 5:00 PM ET. Details from the presentations will be available following the events on the Candel website at View Source

About CAN-2409

CAN-2409, Candel’s most advanced multimodal biological immunotherapy candidate, is an investigational, off-the-shelf, replication-defective adenovirus designed to deliver the herpes simplex virus thymidine kinase (HSV-tk) gene to a patient’s tumor and induce an individualized, systemic anti-tumor immune response. HSV-tk is an enzyme that locally converts orally administered valacyclovir into a toxic metabolite that kills nearby cancer cells, resulting in the release of a wide variety of cancer antigens. At the same time, the adenoviral serotype 5 capsid protein has the potential to elicit a pro-inflammatory response in the tumor microenvironment. Together, this regimen is designed to induce an individualized and specific CD8+ T cell mediated response against the injected tumor and uninjected distant metastases for broad anti-tumor activity, based on in situ vaccination against a variety of tumor antigens. As a result, CAN-2409 is an off-the-shelf drug candidate designed to result in an individualized anti-tumor immune response with the potential to treat a broad range of solid tumors. Encouraging monotherapy activity, as well as combination therapy activity with Standard of Care (SoC) radiotherapy, surgery, chemotherapy, and ICI, have previously been shown in several preclinical and clinical settings. Furthermore, to date, more than 1,000 patients have been dosed with CAN-2409 with a favorable tolerability profile supporting the potential for combination with other therapeutic strategies without inordinate concern of overlapping adverse events.

Currently, Candel is evaluating the effects of treatment with CAN-2409 in NSCLC, borderline resectable pancreatic ductal adenocarcinoma (PDAC), and localized, non-metastatic prostate cancer. CAN-2409 has been granted Fast Track Designation by the U.S. Food and Drug Administration (FDA) for treatment of PDAC, stage III/IV NSCLC in patients who are resistant to first line PD-(L)1 inhibitor therapy and who do not have activating molecular driver mutations or have progressed on directed molecular therapy, and treatment of localized, primary prostate cancer in combination with radiotherapy to

improve the local control rate, decrease recurrence and improve disease-free survival. Candel’s pivotal phase 3 clinical trial in prostate cancer is being conducted under a Special Protocol Assessment by FDA. The FDA has also granted Orphan Drug Designation to CAN-2409 for the treatment of PDAC.

About CAN-3110

CAN-3110 is a first-in-class, replication-competent herpes simplex virus-1 (HSV-1) oncolytic viral immunotherapy candidate designed with dual activity for oncolysis and immune activation in a single therapeutic. Its activity is designed to be conditional to the expression of Nestin in cancer cells. CAN-3110 is being evaluated in a phase 1b clinical trial in patients with recurrent HGG (rHGG). In October 2023, the Company announced that Nature published results from this ongoing clinical trial. CAN-3110 was well tolerated with no dose-limiting toxicity reported and CAN-3110 plus prodrug was associated with improved survival. Positive HSV-1 serology was a predictor of response and was associated with improved survival. Increased infiltrating immune cells in the tumor microenvironment and expansion of the T cell repertoire after treatment were also associated with improved survival. In the clinical trial, the investigators observed a nearly doubling of the expected median overall survival after a single CAN-3110 injection compared to historical reports of less than 6 to 9 months in this therapy-resistant condition. By comparison, survival in the anti-HSV1 positive patients treated with CAN-3110 was more than 14 months. The Company and academic collaborators are currently evaluating the effects of multiple CAN-3110 injections in rHGG, supported by the Break Through Cancer Foundation.

For more information about Candel, visit: www.candeltx.com.

On April 25, 2024 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, reported it published an article in the Experimental and Therapeutic Medicine Journal, titled "Long-term complete response to namodenoson in liver cancer with Child-Pugh B cirrhosis: A case report" (Link) (Press release, Can-Fite BioPharma, APR 25, 2024, View Source [SID1234642340]). The patient participated in the Phase II Liver Cancer Study and has been treated with namodenoson for >7 years under compassionate use program.

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The article describes a patient with advanced liver cancer that was enrolled in the former Can-Fite Phase II study, continues to receive treatment with namodenoson, and has now an overall survival of >7 years, with disappearance of ascites, normal liver function, good quality of life and defined as a long term complete response.

Liver Cancer designated as hepatocellular carcinoma (HCC), is a major global health problem due to its incidence, associated mortality, and lack of effective treatment modalities, particularly for patients with advanced hepatic dysfunction known as disease stage Child Pugh B.

Can-Fite has received agreement from both the U.S. Food and Drug Administration (FDA) and European Medicines Agency (EMA) on a pivotal Phase III clinical study which is now enrolling patients in Israel, Europe and the US in which = Namodenoson is being evaluated as a 2nd or 3rd line treatment for CPB7 patients in whom other approved therapies have not been effective. The study design includes a pre-planned interim analysis by an Independent Data Monitoring Committee (IDMC) upon enrollment of 50% patients.

Namodenoson has Orphan Drug status with both the FDA and EMA, as well as Fast Track Status with the FDA for the treatment of HCC. A compassionate use program has been ongoing in Israel and Romania.

"There is a market need for a safe and effective treatment of patients with advanced liver disease, defined as CPB7 where we believe Namodenoson has an advantage with its anti-cancer and liver protective effect. The complete response of this patient is a testimony for the potential efficacy of Namodenoson and we are optimistic that more patients in the current Phase III study will respond in a similar way," stated Can-Fite CEO Motti Farbstein.

According to the American Cancer Society, liver cancer accounts for more than 700,000 deaths globally each year. HCC is commonly aggressive with poor survival rates. As new drugs that effectively and safely treat HCC are developed and approved, the market for HCC treatments is estimated by Delveinsight to reach $3.8 billion by 2027 for the G8 countries.

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.