Novartis receives FDA fast track designation for sabatolimab (MBG453) in myelodysplastic syndromes

May 25, 2021

Novartis today announced that the US Food and Drug Administration (FDA) has granted fast track designation for sabatolimab (MBG453) for the treatment of adult patients with myelodysplastic syndromes (MDS) defined with an IPSS-R risk category of high or very high risk in combination with hypomethylating agents. Fast track designation facilitates the development and expedites the review of drugs to treat serious conditions and fill unmet medical needs1.

  • MDS, a group of rare and often underdiagnosed blood cancers, is characterized by a dysfunctional immune system and leukemic stem cell proliferation2-4.
  • Despite treatment with HMAs – the last treatment innovation in higher-risk (HR) MDS over the past 15 years – patients face poor outcomes, including a limited duration of response, and have a median overall survival rate of less than a year5,6.
  • Sabatolimab is a first-in-class investigational immuno-myeloid therapy that binds to TIM-3, a novel target expressed on multiple immune cell types and leukemic cells and blasts, but not on the normal stem cells that induce blood formation; it is in development for HR-MDS and acute myeloid leukemia (AML)7,8.

The STIMULUS clinical trial program includes multiple studies evaluating sabatolimab as part of different combination therapies in patients with MDS and AML, including the Phase II STIMULUS-MDS1, Phase III STIMULUS-MDS2, Phase II STIMULUS-MDS3 and Phase II STIMULUS-AML1 studies9-12.

  1. U.S. Food and Drug Administration (FDA). Fast Track. Available from: https://www.fda.gov/patients/fast-track-breakthrough-therapy-accelerated-approval-priority-review/fast-track.
  2. Brunner AM, et al. Recent advances in the cellular and molecular understanding of myelodysplastic syndromes: implications for new therapeutic approaches. Clin Adv Hematol Oncol. 2018 Jan;16(1):56-66.
  3. Steensma, DP, et al. Myelodysplastic Syndromes: Diagnosis and Treatment. Mayo Clin Proc. July 2015;90(7):969-983.
  4. Glenthøj A, et al. Immune Mechanisms in Myelodysplastic Syndrome. Int J Mol Sci. 2016 Jun; 17(6): 944.
  5. Stein EM, et al. Treatment patterns and outcomes in patients with myelodysplastic syndromes treated with hypomethylating agents: a SEER-Medicare analysis. Leuk Lymphoma. 2021 Jan 11:1-16.
  6. Faber MG, et al. Current state of myelodysplastic syndromes: standard treatment practices and therapeutic advances. J Clin Pathways. 2019;5(6):43-47.
  7. Borate, U., et al. Phase Ib Study of the Anti-TIM-3 Antibody MBG453 in Combination with Decitabine in Patients with High-Risk Myelodysplastic Syndrome (MDS) and Acute Myeloid Leukemia (AML). Blood (2019) 134(1): 570.
  8. Wolf Y., et al. TIM3 comes of age as an inhibitory receptor. Nat Rev Immunol. 2020 Mar 20(3):173-185.
  9. “A Study of MBG453 in Combination With Hypomethylating Agents in Subjects With IPSS-R Intermediate, High or Very High Risk Myelodysplastic Syndrome (MDS). (STIMULUS-MDS1).” ClinicalTrials.gov, U.S. National Institutes of Health, 2019, https://clinicaltrials.gov/ct2/show/NCT03946670.
  10. “Study of Efficacy and Safety of MBG453 in Combination With Azacitidine in Subjects With Intermediate, High or Very High Risk Myelodysplastic Syndrome (MDS) as Per IPSS-R, or Chronic Myelomonocytic Leukemia-2 (CMML-2) (STIMULUS-MDS2).” ClinicalTrials.gov, U.S. National Institutes of Health, 2020, https://clinicaltrials.gov/ct2/show/NCT04266301.
  11. “A Study of Sabatolimab in Combination With Azacitidine and Venetoclax in High or Very High Risk MDS Participants (STIMULUS-MDS3).” ClinicalTrials.gov, U.S. National Institutes of Health, 2021, https://clinicaltrials.gov/ct2/show/NCT04812548.
  12. “A Study of MBG453 in Combination With Azacitidine and Venetoclax in AML Patients Unfit for Chemotherapy (STIMULUS-AML1).” ClinicalTrials.gov, U.S. National Institutes of Health, 2019, https://clinicaltrials.gov/ct2/show/NCT04150029.

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