THOUSAND OAKS, Calif., Dec. 8, 2020 /PRNewswire/ -- Amgen (NASDAQ: AMGN) today announced that the U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy designation for its investigational KRASG12C inhibitor, sotorasib, for the treatment of patients with locally advanced or metastatic non-small cell lung cancer (NSCLC) with KRAS G12C mutation, as determined by an FDA-approved test, following at least one prior systemic therapy.
"For more than 40 years, scientists have been trying to target KRAS. Today's news is a welcome update for the many non-small cell lung cancer patients with the KRAS G12C mutation, who currently have no targeted therapies," said Bonnie J. Addario, cofounder and board chair of the GO2 Foundation for Lung Cancer. "We are pleased that the FDA and Amgen recognize the unmet need for these patients and are working to make new treatment options available as quickly as possible."
KRAS G12C is the most common KRAS mutation in NSCLC.1,2 In the U.S., about 13% of patients with NSCLC adenocarcinoma harbor the KRAS G12C mutation3 and each year approximately 25,000 new patients in the U.S. are diagnosed with KRAS G12C-mutated NSCLC.4 Unmet need remains high and options are limited for NSCLC patients with the KRAS G12C mutation that have failed first-line treatment. The outcomes with current therapies are suboptimal with response rates of approximately 9-18% and a median progression-free survival of approximately 4 months for second-line NSCLC.5,6,7
Amgen has taken on one of the toughest challenges of the last 40 years in cancer research8 by developing sotorasib. Sotorasib was the first KRASG12C inhibitor to enter the clinic and is being studied in the broadest clinical program exploring 10 combinations with global sites spanning across 4 continents. In just over two years, the sotorasib clinical program has also established the deepest clinical data set with more than 600 patients studied across 13 tumor types.
"Breakthrough Therapy designation and Real-Time Oncology Review bring Amgen closer to potentially providing a targeted therapy to patients with a KRAS G12C mutation and establishing sotorasib as the foundational therapy in KRAS G12C-driven cancers," said David M. Reese, M.D., executive vice president of Research and Development at Amgen. "We are pleased to receive these regulatory designations and plan to submit a new drug application by end of year as we rapidly work to get sotorasib to the patients who need it."
A Breakthrough Therapy designation is designed to expedite the development and regulatory review of medicines that may demonstrate substantial improvement on a clinically significant endpoint over available medicines.9 The Real-Time Oncology Review (RTOR) pilot program aims to explore a more efficient review process that ensures safe and effective treatments are made available to patients as early as possible.10
The designation and RTOR are supported by positive Phase 2 results in patients with advanced NSCLC from the CodeBreaK 100 clinical study, whose cancer had progressed despite prior treatment with chemotherapy and/or immunotherapy. In the study, treatment with sotorasib provided durable anticancer activity with a positive benefit-risk profile.11
The CodeBreaK clinical development program for Amgen's investigational drug sotorasib is designed to treat patients with an advanced solid tumor with the KRAS G12C mutation and address the longstanding unmet medical need for these cancers. As the most advanced KRAS G12C clinical development program, CodeBreaK has enrolled more than 600 patients across 13 tumor types since its inception.
CodeBreaK 100, the Phase 1 and 2, first-in-human, open-label multicenter study, enrolled patients with KRAS G12C-mutant solid tumors. Eligible patients must have received a prior line of systemic anticancer therapy, consistent with their tumor type and stage of disease. The primary endpoint for the Phase 2 study was centrally assessed objective response rate. The Phase 2 trial in NSCLC enrolled 126 patients, 123 of whom had centrally evaluable lesions by RECIST at baseline. The Phase 2 trial in colorectal cancer (CRC) is fully enrolled and topline results are expected in 2021.
A global Phase 3 randomized active-controlled study comparing sotorasib to docetaxel in patients with KRAS G12C-mutated NSCLC (CodeBreaK 200) is currently recruiting. Amgen also has several Phase 1b combination studies across various advanced solid tumors (CodeBreaK 101) open for enrollment.
For information, please visit www.codebreaktrials.com.
About Amgen Oncology
Amgen Oncology is searching for and finding answers to incredibly complex questions that will advance care and improve lives for cancer patients and their families. Our research drives us to understand the disease in the context of the patient's life – not just their cancer journey – so they can take control of their lives.
For the last four decades, we have been dedicated to discovering the firsts that matter in oncology and to finding ways to reduce the burden of cancer. Building on our heritage, Amgen continues to advance the largest pipeline in the Company's history, moving with great speed to advance those innovations for the patients who need them.
At Amgen, we are driven by our commitment to transform the lives of cancer patients and keep them at the center of everything we do.
To learn more about Amgen's innovative pipeline with diverse modalities and genetically validated targets, please visit AmgenOncology.com. For more information, follow us on www.twitter.com/amgenoncology.
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.
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CONTACT: Amgen, Thousand Oaks
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1 Pakkala S, et al. JCI Insight. 2018;3:e120858.
2 Arbour KC, et al. Clin Cancer Res. 2018;24:334-340.
3 Amgen, Data on File. 2020.
4 American Cancer Society, Cancer Facts and Figures. 2020. https://www.cancer.org/content/dam/cancer-org/research/cancer-facts-and-statistics/annual-cancer-facts-and-figures/2020/cancer-facts-and-figures-2020.pdf. Accessed November 23, 2020.
5 Herbst RS, et al. Lancet. 2016;387-1540-1550.
6 Aggarwal S, et al. Poster presentation at ESMO Virtual Congress 2020, Sep. 19-21, 2020. Poster 1339P.
7 Hayashi H, et al. Clin Lung Cancer. 2013;14:261-266.
8 Kim D, et al. Cell. 2020. doi:10.1016/j.cell.2020.09.044.
9 U.S. Food and Drug Administration. Breakthrough Therapy. https://www.fda.gov/patients/fast-track-breakthrough-therapy-accelerated-approval-priority-review/breakthrough-therapy. Accessed November 10, 2020.
10 U.S. Food and Drug Administration. Real Time Oncology Review https://www.fda.gov/about-fda/oncology-center-excellence/real-time-oncology-review-pilot-program . Accessed November 20, 2020.
11 Hong DS, et al. N Engl J Med. 2020; 383:1207-1217.