FDA Target Action Date is June 12, 2024
Amgen (NASDAQ:AMGN) today announced that the U.S. Food and Drug Administration (FDA) has accepted and granted Priority Review for the Company's Biologics License Application (BLA) for tarlatamab.
Tarlatamab is a potential first-in-class, investigational delta-like ligand 3 (DLL3) targeting Bispecific T-cell Engager (BiTE ® ) therapy for the treatment of adult patients with advanced small cell lung cancer (SCLC) with disease progression on or after platinum-based chemotherapy.
"The FDA's Priority Review designation for this application underscores the urgency to provide new treatment options for patients with advanced SCLC who have progressed following treatment with platinum-based chemotherapy," said David M. Reese , M.D., executive vice president of Research and Development at Amgen. "While first-line treatments often show strong responses, patients can experience aggressive recurrences and long-term survival remains a challenge. 2,3 Unfortunately, for patients who relapse, there are limited treatment options, emphasizing the importance of bringing new therapies to this patient population with advanced disease."
The FDA grants Priority Review to applications for medicines that offer, if approved, significant improvements over available options or may provide a treatment option where no adequate therapy currently exists. Based on the Priority Review designation, the Prescription Drug User Fee Action (PDUFA) date for tarlatamab is June 12, 2024 .
The BLA is based on the Phase 2 results from the DeLLphi-301 clinical trial that studied patients with advanced SCLC with disease progression on or after platinum-based chemotherapy. Results from the study were recently featured as part of a late-breaking presentation during the 2023 European Society for Medical Oncology (ESMO) Congress and simultaneously published in the New England Journal of Medicine . 4,5 The data presented demonstrated antitumor activity with a durable response and encouraging survival outcomes in previously treated SCLC. The safety profile was consistent with the Phase 1 trial. 6
Tarlatamab is being investigated in multiple studies including DeLLphi-302, a Phase 1b study evaluating tarlatamab in combination with an anti-PD-1 therapy in second-line or later SCLC; DeLLphi-303, a Phase 1b study investigating tarlatamab in combination with standard of care therapies in first-line SCLC; DeLLphi-304, a randomized Phase 3 trial comparing tarlatamab monotherapy with standard of care chemotherapy in second-line treatment of SCLC that is enrolling patients; DeLLphi-306, a recently-initiated, randomized Phase 3 trial of tarlatamab following chemoradiotherapy in earlier settings of SCLC; and DeLLpro-300, a Phase 1b study of tarlatamab in de novo or treatment-emergent neuroendocrine prostate cancer. 7 Amgen also plans to initiate an additional Phase 3 study of tarlatamab in first-line treatment of SCLC.
In October, tarlatamab was granted Breakthrough Therapy Designation by the FDA. The application is being reviewed by the FDA under the Project Orbis framework and Real Time Oncology Review (RTOR). Project Orbis is an initiative from the FDA Oncology Center of Excellence that provides a framework for concurrent submission of oncology products among certain countries.
About Small Cell Lung Cancer (SCLC)
SCLC is one of the most aggressive and devastating solid tumors with a median survival of approximately 12 months following initial therapy and a 7% five-year relative survival rate when all stages are combined. 8-10 Of the more than 2.2 million patients diagnosed with lung cancer worldwide each year, SCLC comprises 15% of cases. 11,1 Despite initial high response rates to platinum-based first-line chemotherapy, patients quickly relapse and require subsequent treatment options. 1
About Tarlatamab
Tarlatamab is an investigational, targeted therapy engineered by Amgen researchers that brings a patient's own T cells in close proximity to SCLC cells by binding both CD3 on T cells and DLL3 on SCLC cells. This results in the formation of a cytolytic synapse with lysis of the cancer cell. 12,13 DLL3 represents an exciting therapeutic target for patients with SCLC, as approximately 85% to 96% of patients have expression of DLL3 on the cell surface of SCLC cells, with minimal expression in normal cells. 6,14-16
About Tarlatamab Clinical Trials
Amgen's robust tarlatamab development program includes the DeLLphi clinical trials, which evaluate tarlatamab as a monotherapy and as part of combination regimens in earlier stages of SCLC, and DeLLpro clinical trials, which evaluate tarlatamab in neuroendocrine prostate cancer.
In the Phase 1 DeLLphi-300 study, tarlatamab showed responses in 23.4% of patients with encouraging durability in heavily pre-treated patients with SCLC. In the Phase 2 DeLLphi-301 study, tarlatamab administered as 10 mg dose every two weeks demonstrated an objective response rate of 40% in patients with advanced SCLC who had failed two or more prior lines of treatment. In both DeLLphi-300 and DeLLphi-301, the most frequent treatment-related adverse events were cytokine release syndrome (CRS; 52-55%), pyrexia (31-37%), and dysgeusia (22-26%), which were primarily grade 1-2. Treatment discontinuation for adverse events occurred in 3-4% of patients in the two trials. 5,6
For more information, please visit www.tarlatamabclinicaltrials.com .
About BiTE ® Technology
Bispecific T-cell Engager (BiTE ® ) technology is a targeted immuno-oncology platform that is designed to engage patient's own T cells to any tumor-specific antigen, activating the cytotoxic potential of T cells to eliminate detectable cancer. The BiTE ® immuno-oncology platform has the potential to treat different tumor types through tumor-specific antigens. The BiTE ® platform has a goal of leading to off-the-shelf solutions, which have the potential to make innovative T cell treatment available to all providers when their patients need it. Amgen is advancing multiple BiTE ® molecules across a broad range of hematologic malignancies and solid tumors, further investigating BiTE ® technology with the goal of enhancing patient experience and therapeutic potential. To learn more about BiTE ® technology, visit https://www.amgenoncology.com/bite-platform.html .
About Amgen
Amgen is committed to unlocking the potential of biology for patients suffering from serious illnesses by discovering, developing, manufacturing and delivering innovative human therapeutics. This approach begins by using tools like advanced human genetics to unravel the complexities of disease and understand the fundamentals of human biology.
Amgen focuses on areas of high unmet medical need and leverages its expertise to strive for solutions that improve health outcomes and dramatically improve people's lives. A biotechnology pioneer since 1980, Amgen has grown to be one of the world's leading independent biotechnology companies, has reached millions of patients around the world and is developing a pipeline of medicines with breakaway potential.
Amgen is one of the 30 companies that comprise the Dow Jones Industrial Average and is also part of the Nasdaq-100 index. In 2023, Amgen was named one of "America's Greatest Workplaces" by Newsweek, one of "America's Climate Leaders" by USA Today and one of the "World's Best Companies" by TIME.
For more information, visit Amgen.com and follow us on X (formerly known as Twitter), LinkedIn , Instagram , TikTok , YouTube and Threads .
Amgen Forward-Looking Statements
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CONTACT: Amgen, Thousand Oaks
Elissa Snook , 609-251-1407 (media)
Jessica Akopyan , 805-440-5721 (media)
Justin Claeys , 805-313-9775 (investors)
References
- Oronsky B, et al. J Cancer . 2022;13:2945-2953.
- Sabari JK, et al. Nat Rev Clin Oncol . 2017;14:549-561.
- Petty WJ, et al. Jama Oncol. 2023;9(3):419-429.
- Paz-Ares L, et al. Presented at ESMO 2023 Congress. Oct. 20-24, 2023 ; Madrid, Spain . Abstract #LBA92.
- Ahn MJ, et al. N Engl J Med . 2023;389:2063-2075.
- Paz-Ares L, et al. J Clin Oncol . 2023;41:2893-2903.
- ClinicalTrials.gov tarlatamab clinical trial listings. Available at www.clinicaltrials.gov . Accessed Dec. 4, 2023 .
- American Cancer Society. Lung Cancer Survival Rates. 2023. Available at: https://www.cancer.org/cancer/types/lung-cancer/detection-diagnosis-staging/survival-rates.html . Accessed on November 7, 2023 .
- Paz-Ares L, et al. ESMO Open . 2022;7:100408.
- Liu SV, et al. J Clin Oncol . 2021;39:619-630
- World Health Organization. Lung Cancer Factsheet. 2020. Available at: https://gco.iarc.fr/today/data/factsheets/cancers/15-Lung-fact-sheet.pdf . Accessed on November 7, 2023 .
- Baeuerle PA, et al. Curr Opin Mol Ther . 2009;11:22-30.
- Giffin MJ, et al. Clin Cancer Res . 2021;27:1526-1537.
- Rojo F, et al. Lung Cancer. 2020;147:237-243.
- Saunders LR, et al. Sci Transl Med. 2015;7:302ra136.
- Ahn MJ, et al. N Engl J Med . 2023;349:2063-2075.
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