Suzhou China Mar 4, 2019 – CStone partner Agios Pharmaceuticals, Inc. (NASDAQ:AGIO) recently announced that the U.S. Food and Drug Administration (FDA) has accepted the company's supplemental New Drug Application (sNDA) for TIBSOVO® (ivosidenib) for the treatment of patients with newly diagnosed acute myeloid leukemia (AML) with an isocitrate dehydrogenase 1 (IDH1) mutation who are not eligible for standard therapy. The sNDA was granted Priority Review and has been given a Prescription Drug User Fee Act (PDUFA) action date of June 21, 2019. CStone is responsible for ivosidenib's development and commercialization in Greater China Region under an exclusive collaboration and license agreement with Agios.
AML, a cancer of blood and bone marrow characterized by rapid disease progression, is the most common acute leukemia affecting adults. The vast majority of patients do not respond to chemotherapy and progress to relapsed/refractory AML. The five-year survival rate for AML is approximately 27 percent. IDH1 mutations are present in about 6 to 10 percent of AML cases.
TIBSOVO® is a first-in-class, oral, targeted inhibitor of mutant IDH1 and was approved by the U.S.FDA in 2018 for the treatment of patients with relapsed or refractory AML (R/R AML) and a susceptible IDH1 mutation detected by a U.S. FDA approved companion diagnostic test.
The sNDA submission is based on results from the untreated AML patients from the Phase 1 dose-escalation and expansion study of ivosidenib in patients with newly diagnosed AML ineligible for standard treatment. Data from the untreated AML portion of this study were presented at the 2018 American Society of Hematology (ASH) Annual Meeting.
In addition, the FDA accepted the TIBSOVO® sNDA under its Real-Time Oncology Review pilot program, which aims to make the review of oncology drugs more efficient by allowing the FDA access to clinical trial data before the information is formally submitted to the agency.
“In less than seven months since TIBSOVO's approval in relapsed or refractory AML, we are pleased to be working with the FDA to expand its labeled indication into the frontline setting,” said Chris Bowden, M.D., chief medical officer of Agios. “Patients with newly diagnosed AML who are not eligible for standard treatments, such as intensive and non-intensive chemotherapy, are currently offered only palliative care. There is tremendous need for new treatment options, and we believe AML patients with IDH1 mutations have the potential to benefit from this targeted therapy.”
About CStone
CStone Pharmaceuticals is a biopharmaceutical company focused on developing and commercializing innovative immuno-oncology and molecularly targeted drugs to address significant unmet medical needs for cancer patients in China and worldwide. Since the Company's inception in 2015, CStone has assembled a world-class management team that has a full spectrum of complementary skillsets from preclinical research to clinical development and commercialization. Through dual sources of innovation, comprised of internal research and external partnership, the Company has built a rich oncology pipeline of 14 oncology drug candidates with significant mono- and combination-therapy potential and synergies, including 4 assets exclusively in-licensed in Greater China from Agios and Blueprint Medicines. Among CStone's portfolio, 4 late-stage drug candidates are at or near pivotal trials. CStone's business model has a clear focus on clinical development, while at the same time, the Company is rapidly developing its commercial and manufacturing capabilities. The Company is backed by prestigious VC and PE funds with record-breaking amounts of equity investment, raising a combined total of approximately USD 412 million in two pre-IPO financing rounds. With an experienced team, a rich pipeline, a robust clinical development-driven business model, and substantial funding, CStone's vision is to become globally recognized as a leading Chinese biopharmaceutical company by bringing innovative and differentiated oncology therapies to cancer patients worldwide.
For more information about CStone Pharmaceuticals, please visit: www.cstonepharma.com.
About Agios
Agios is focused on discovering and developing novel investigational medicines to treat cancer and rare genetic diseases through scientific leadership in the field of cellular metabolism. In addition to an active research and discovery pipeline across both therapeutic areas, Agios has two approved oncology precision medicines and multiple first-in-class investigational therapies in clinical and/or preclinical development. All Agios programs focus on genetically identified patient populations, leveraging our knowledge of metabolism, biology and genomics. For more information, please visit the company's website at www.agios.com.
