Squamous cell carcinoma, metastatic

The FDA has granted approval to BeiGene's Tevimbra (tislelizumab-jsgr), for treating adults with advanced or metastatic esophageal squamous cell carcinoma (ESCC) following prior systemic chemotherapy. The nod marks the first U.S. indication for Tevimbra, expected to be available in the latter half of 2024. Tevimbra’s approval was supported by the results of the global phase 3 RATIONALE 302 trial, which demonstrated a significant survival advantage over traditional chemotherapy. Patients treated with Tevimbra experienced a median overall survival of 8.6 months, compared to 6.3 months for those on chemotherapy. The drug has already received approval in Europe for the same condition and is under review for additional indications, including first-line ESCC treatment and as a therapy for non-small cell lung cancer. BeiGene is also conducting numerous trials to explore Tevimbra broader potential across various cancer types, with over 900,000 patients worldwide already treated. Initially, BeiGene and Novartis collaborated on developing Tevimbra under a 2021 agreement that allowed Novartis to lead regulatory efforts and commercialize the drug worldwide post-approval. They've now amended this arrangement, granting BeiGene exclusive global rights to develop, manufacture and sell Tevimbra.

VIENNA, Va.--( BUSINESS WIRE )--CEL-SCI Corporation (NYSE American: CVM) today issued a letter to its shareholders. This letter will be sent to the Company’s shareholders along with the proxy to the upcoming annual meeting. Dear CEL-SCI Shareholders: Last year we successfully moved our investigational Multikine ® (Leukocyte Interleukin, Injection)* cancer therapy forward on the clinical, manufacturing, and regulatory fronts by overcoming several challenges as the biotechnology sector went through very difficult times. The results from our Phase 3 trial presented us with a conundrum in that we had great survival data with Multikine as a pre-surgical treatment in head and neck cancer when followed by surgery and radiotherapy only, but not when followed by surgery, radiotherapy, and chemotherapy. The key issue was how to determine who should be treated with Multikine, which is given before surgery, while the determination of who gets chemotherapy added to their treatment is made after surgery. We solved this issue with great success and presented the selection criteria at a major oncology conference: CEL-SCI identified the target head and neck cancer patient population for Multikine that will be the basis for the Company’s regulatory filings for marketing clearance. In October 2023, the new data were presented at the 2023 European Society for Medical Oncology (ESMO) Congress. The target population, which saw its 5-year risk of death cut in half, can be identified prior to surgery upon diagnosis with tests that physicians routinely use in cancer screenings , a key finding for Multikine, which is a neoadjuvant therapy. A summary of Multikine’s results in the target population include the following: Risk of death cut in half at 5 years vs. control 73% survival for Multikine vs 45% in the control at 5 years 28.6% absolute 5-year overall survival benefit versus control; statistically significant p = 0.0015 and hazard ratio = 0.35 Tumor reduction rate >13% and tumor downstaging >35% Low PD-L1 tumor expression (as compared to high PD-L1 where Keytruda and Opdivo work best) No safety signals or toxicities vs standard of care Target population is an estimated 145,000 patients (global, annual) with newly diagnosed squamous cell carcinoma of the head and neck (SCCHN) who present with: No lymph node involvement (via PET scan) Low PD-L1 tumor expression (TPS<10) (via biopsy). Physicians routinely assess these features at baseline; no extra tests needed. These features make it easy to write a label for Multikine, which is essential for drug approval. CEL-SCI estimates that low PD-L1 patients represent about 70% of locally advanced primary SCCHN patients. In manufacturing, a major issue was the U.S. government’s rules prioritizing companies that make COVID products to receive medical supplies. This caused delays in our work commissioning our Multikine manufacturing facility. Fortunately, we were able to finish this critical work in January 2024: CEL-SCI’s cGMP state-of-the-art dedicated manufacturing facility commissioning was completed, a significant milestone toward a planned Biologics License Application (BLA) with several regulatory agencies for approval of Multikine. The Company’s manufacturing trade secrets, capabilities, and know-how are high-value key strategic assets that are very difficult for others to replicate. Usually companies pursue only one regulator, presumably because it is a great deal of work to pursue more. Since Multikine is a unique pre-surgical cancer drug, we decided to in parallel talk to the world’s leading regulators: FDA (U.S.), EMA (Europe), MHRA (UK) and Health Canada (Canada). In our discussions so far, we have been well received, and we have been told that clinically our results are very meaningful and promising. Regulators have been responsive and willing to help us. Here is an update of what we can share with you: CEL-SCI has submitted the target population data and a proposed study protocol for a confirmatory study of 212 patients to the U.S. Food and Drug Administration (FDA) in Q1 2024. Health Canada advised CEL-SCI to request advance consideration for approval under a Notice of Compliance with Conditions (NOCC) policy. The United Kingdom’s National Institute for Health and Care Excellence (NICE) selected Multikine to be evaluated as the potential new standard of care for SCCHN. NICE posted a detailed report from the UK’s National Institute for Health and Care Research (NIHR) regarding Multikine, its clinical data, and its potential to become a better standard of care in treating newly diagnosed head and neck cancer in the UK. The European Medicines Agency’s (EMA) Paediatric Committee granted CEL-SCI a product-specific waiver of strict requirements for commercialization of cancer drugs in the European Union (EU). The waiver is a big step forward for Multikine, as it removes a major hurdle on the path towards commercialization in Europe. Meetings with the UK regulators and the EMA are expected H1 2024. New PD-L1 biomarker findings from the Phase 3 study, which have been integrated into the new target population, demonstrated that Multikine significantly increases overall survival in patients with low levels of tumor cell PD-L1 expression. This is in stark contrast to approved checkpoint inhibitors, such as Keytruda and Opdivo, which most often show longer survival in a proportion of patients with a higher level of tumor cell PD-L1 expression, suggesting a combination therapy could boost patient outcomes. CEL-SCI filed a patent for the use of Multikine in tumors expressing low levels of PD-L1. We also presented new data at a number of leading scientific conferences: 10th European Congress on Head & Neck Oncology (ECHNO) 2023 “Leukocyte Interleukin Injection (LI) immunotherapy followed by radiotherapy extends overall survival (OS) in treatment naïve locally advanced primary squamous cell carcinoma of the head & neck: the IT-MATTERS Study” ( Link to data ) European Society for Radiotherapy and Oncology (ESTRO) 2023 “Histopathology population (HPP) confirms Multikine* [Leukocyte Interleukin Injection (LI)] treatment (Tx) outcome in naïve locally advanced primary head & neck squamous cell carcinoma SCCHN)” ( Link to data ) American Head and Neck Cancer Society’s (AHNS) 11th Annual International Conference on Head and Neck Cancer 2023 “Tumor cell PD-L1 biomarker confirms Leukocyte Interleukin Injection (LI) treatment (Tx) survival outcome advantage in naive locally advanced primary head & neck squamous cell carcinoma (SCCHN), the IT-MATTERS Study” ( Link to data ) European Society for Medical Oncology (ESMO) Annual Congress 2023 “Early response to Neoadjuvant Leukocyte Interleukin Injection (LI) immunotherapy extends overall survival (OS) in locally advanced primary squamous cell carcinoma (SCC) of the head & neck (HN): the IT-MATTERS Study” ( Link to data ) CEL-SCI Has All The Ingredients For Approval We can identify at diagnosis the patients most likely to have pre-surgical responses to Multikine. The survival statistics in this target population are so good that it is hard to imagine how they could be challenged or ignored. Multikine’s safety profile is very favorable compared to other oncology agents. And, we have a manufacturing plant that can make an estimated $2 billion worth of Multikine annually. CEL-SCI is ready — and we believe patients and doctors are, too. A new drug for our targeted disease (previously untreated locally advanced primary resectable squamous cell carcinoma of the head and neck) has not been approved by the FDA in decades. The current standard of care provides only a 50-50 chance of living past five years. Large companies have tried and failed to improve this figure. Patients desperately need better treatments. We now have the ingredients for delivering results to investors as well. While the biotech stock market always has its ups and downs, the clinical data will stand firm and drive the final success for CEL-SCI. CEL-SCI plans to seek approval for immediate patient access to Multikine without waiting on the results of a new trial wherever possible . There are regulatory pathways specifically designed for such approvals that CEL-SCI is pursuing worldwide. These pathways are called “conditional approvals” (or, in the U.S., accelerated approval) which means you can be approved first while a confirmatory study is ongoing and before that study is completed. Our situation—where we have selected a portion of the Phase 3 study for our target population—is precisely why these regulatory pathways were adopted by regulatory bodies, so that patients do not need to wait many years before gaining access to promising drugs that have already been shown to provide clinical benefit. Thank you for your continued support. Sincerely, Geert Kersten Chief Executive Officer Forward-Looking Statements This press release contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. When used in this press release, the words "intends," "believes," "anticipated," "plans" and "expects," and similar expressions, are intended to identify forward-looking statements. Such statements are subject to risks and uncertainties that could cause actual results to differ materially from those projected. Such statements include, but are not limited to, statements about the terms, expected proceeds, use of proceeds and closing of the offering. Factors that could cause or contribute to such differences include an inability to duplicate the clinical results demonstrated in clinical studies, timely development of any potential products that can be shown to be safe and effective, receiving necessary regulatory approvals, difficulties in manufacturing any of the Company's potential products, inability to raise the necessary capital and the risk factors set forth from time to time in CEL-SCI's filings with the Securities and Exchange Commission, including but not limited to its report on Form 10-K for the year ended September 30, 2023. The Company undertakes no obligation to publicly release the result of any revision to these forward-looking statements which may be made to reflect the events or circumstances after the date hereof or to reflect the occurrence of unanticipated events. * Multikine (Leukocyte Interleukin, Injection) is the trademark that CEL-SCI has registered for this investigational therapy. This proprietary name is subject to FDA review in connection with the Company's future anticipated regulatory submission for approval. Multikine has not been licensed or approved for sale, barter or exchange by the FDA or any other regulatory agency. Similarly, its safety or efficacy has not been established for any use.

Application based on results from global Phase 3 RATIONALE-305 trial demonstrating TEVIMBRA plus chemotherapy significantly improved overall survival in advanced gastric/GEJ cancer Prescription Drug User Fee Act target action date set for December 2024 BASEL, Switzerland & BEIJING & CAMBRIDGE, Mass.--(BUSINESS WIRE)-- BeiGene, Ltd. (NASDAQ: BGNE; HKEX: 06160; SSE: 688235), a global oncology company, today announced that the U.S. Food and Drug Administration (FDA) has accepted a Biologics License Application (BLA) for TEVIMBRA® (tislelizumab), in combination with fluoropyrimidine- and platinum-containing chemotherapy, for the treatment of patients with locally advanced unresectable or metastatic gastric or gastroesophageal junction (G/GEJ) adenocarcinoma. The FDA’s action date on the BLA is expected in December 2024. “There is an urgent need for new treatment options for gastric cancer, which is often diagnosed at the advanced or metastatic stage,” said Mark Lanasa, M.D., Ph.D., Chief Medical Officer, Solid Tumors at BeiGene. “In clinical trials, TEVIMBRA has demonstrated its potential to improve survival for patients with gastric and gastroesophageal junction cancer. This FDA acceptance brings us one step closer to delivering on a new treatment option for patients who often face poor prognoses.” The filing is based on results from the global RATIONALE-305 trial. The study met its primary endpoint of overall survival of 15.0 months for patients treated with TEVIMBRA in combination with investigator’s choice of chemotherapy compared to 12.9 months for patients treated with placebo plus chemotherapy (n=997; HR: 0.80 [95% CI: 0.70, 0.92]; P=0.0011), demonstrating a 20% reduction in the risk of death. Additionally, TEVIMBRA plus chemotherapy was associated with a higher objective response rate (47.3% vs. 40.5%) and median duration of response (8.6 months vs. 7.2 months) compared to placebo plus chemotherapy. Median progression-free survival for TEVIMBRA plus chemotherapy was 6.9 months vs. 6.2 months respectively; (HR: 0.78 [95% CI: 0.67, 0.90]). The safety pro TEVIMBRA in combination with chemotherapy was manageable and in line with the known safety pro anti-PD-1 antibodies. Grade ≥3 treatment-related adverse events (TRAEs) occurred in 53.8% of patients in the TEVIMBRA plus chemotherapy arm and 49.8% of patients in the placebo plus chemotherapy arm. The most common TRAEs of any grade with an incidence ≥30% were nausea, decreased appetite, platelet count decreased, neutrophil count decreased, vomiting, and anaemia. TEVIMBRA was recently approved by the European Commission for the treatment of patients with advanced or metastatic esophageal squamous cell carcinoma (ESCC) after prior chemotherapy. The FDA is also reviewing a BLA for TEVIMBRA as a first-line treatment for patients with unresectable, recurrent, locally advanced, or metastatic ESCC with a target action date in July 2024. A BLA for the treatment of patients with advanced or metastatic ESCC after prior chemotherapy is also under review by the FDA. BeiGene has launched more than 17 potentially registration-enabling trials with TEVIMBRA, of which 11 Phase 3 randomized trials and 4 Phase 2 trials have already had positive readouts. Through these trials, TEVIMBRA has demonstrated its ability to safely deliver clinically meaningful improvements in survival benefits and quality of life for hundreds of thousands of cancer patients across a range of tumor types – in many cases, regardless of PD-(L)1 status – both as monotherapy and in combination with other regimens. More than 900,000 patients have been prescribed TEVIMBRA globally to date. About RATIONALE-305 RATIONALE-305 (NCT03777657) is a randomized, double-blind, placebo-controlled, global Phase 3 that enrolled 997 patients with advanced unresectable or metastatic G/GEJ adenocarcinoma. The primary endpoint was OS, with prespecified hierarchy testing for the PD-L1 high population followed by the intent-to-treat (ITT) population. Results of the final analysis of the ITT population were presented as a late-breaking oral presentation during the European Society for Medical Oncology (ESMO) Congress 2023. About Gastric and Gastroesophageal Junction (G/GEJ) Adenocarcinoma Gastric (stomach) cancer is the fifth most common cancer worldwide and the fifth highest leading cause of cancer mortality.1 Nearly 1 million new patients were diagnosed with gastric cancer in 2022, and 660,000 deaths were reported globally. In the U.S., it is estimated there were approximately 27,000 patients diagnosed with gastric cancer and 11,000 deaths from the disease in 2024.2 The five-year survival rate for gastric cancer in the U.S. is 36%.3 Gastroesophageal junction adenocarcinoma occurs at the area where the esophagus joins the stomach, which is just beneath the diaphragm (the thin sheet of breathing muscle under the lungs).4 About TEVIMBRA® (tislelizumab) Tislelizumab is a uniquely designed humanized immunoglobulin G4 (IgG4) anti-programmed cell death protein 1 (PD-1) monoclonal antibody with high affinity and binding specificity against PD-1. It is designed to minimize binding to Fc-gamma (Fcγ) receptors on macrophages, helping to aid the body’s immune cells to detect and fight tumors. About BeiGene BeiGene is a global oncology company that is discovering and developing innovative treatments that are more affordable and accessible to cancer patients worldwide. With a broad portfolio, we are expediting development of our diverse pipeline of novel therapeutics through our internal capabilities and collaborations. We are committed to radically improving access to medicines for far more patients who need them. Our growing global team of more than 10,000 colleagues spans five continents, with administrative offices in Basel, Beijing, and Cambridge, U.S. To learn more about BeiGene, please visit and follow us on LinkedIn and X (formerly known as Twitter). Forward-Looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 and other federal securities laws, including statements regarding TEVIMBRA’s potential to improve survival for patients with gastric and gastroesophageal junction cancer; the ability of TEVIMBRA to be a treatment option for patients with poor prognoses; future regulatory approval of TEVIMBRA; TEVIMBRA’s ability to safely delivery clinically meaningful improvements in survival benefits and quality of life to cancer patients; BeiGene’s advancement, anticipated clinical development, regulatory submissions and commercialization of TEVIMBRA; and BeiGene’s plans, commitments, aspirations, and goals under the heading “About BeiGene.” Actual results may differ materially from those indicated in the forward-looking statements as a result of various important factors, including BeiGene's ability to demonstrate the efficacy and safety of its drug candidates; the clinical results for its drug candidates, which may not support further development or marketing approval; actions of regulatory agencies, which may affect the initiation, timing, and progress of clinical trials and marketing approval; BeiGene's ability to achieve commercial success for its marketed medicines and drug candidates, if approved; BeiGene's ability to obtain and maintain protection of intellectual property for its medicines and technology; BeiGene's reliance on third parties to conduct drug development, manufacturing, commercialization, and other services; BeiGene’s limited experience in obtaining regulatory approvals and commercializing pharmaceutical products and its ability to obtain additional funding for operations and to complete the development of its drug candidates and achieve and maintain profitability; and those risks more fully discussed in the section entitled “Risk Factors” in BeiGene’s most recent annual report on Form 10-K, as well as discussions of potential risks, uncertainties, and other important factors in BeiGene's subsequent filings with the U.S. Securities and Exchange Commission. All information in this press release is as of the date of this press release, and BeiGene undertakes no duty to update such information unless required by law. To access BeiGene media resources, please visit our News & Media site. 1 Ferlay J, Ervik M, Lam F, Laversanne M, Colombet M, Mery L, Piñeros M, Znaor A, Soerjomataram I, Bray F (2020). Global Cancer Observatory: Cancer Today. Lyon, France: International Agency for Research on Cancer. Available from: . Accessed February 9, 2024. 2 American Cancer Society. Cancer Facts & Figures 2024. Atlanta: American Cancer Society; 2024. 3 National Cancer Institute. Surveillance, Epidemiology, and End Results Program. Cancer stat facts: stomach cancer. Available at . Accessed January 22, 2024. 4 American Cancer Society. What Is Stomach Cancer? . View source version on businesswire.com: Contacts Investor: Liza Heapes +1 857-302-5663 ir@beigene.com Media: Kyle Blankenship +1 667-351-5176 media@beigene.com Source: BeiGene, Ltd. 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