ASCERTAIN: A Phase 3 MDS and CMML clinical trial1

MDS Study Design

Designed to evaluate the only oral HMA therapy for patients with MDS and CMML1-3

INQOVI is the only oral hypomethylating agent (HMA) therapy approved for adults with intermediate- to high-risk myelodysplastic syndromes (MDS), including chronic myelomonocytic leukemia (CMML).2,3

ASCERTAIN was an open-label, randomized, Phase 3 crossover trial (N=133) that evaluated systemic decitabine exposure, demethylation activity, and safety between INQOVI and intravenous (IV) decitabine in a broad range of adult patients with MDS or CMML.1,2

The trial allowed for intrapatient comparison in the first 2 randomized treatment cycles, then assessment of long-term efficacy and safety of INQOVI as a single arm. Median follow-up was approximately 2.6 years.1,2

Eligibility criteria2

  • Diagnosis of MDS or CMML (International Prognostic Scoring System [IPSS] intermediate-1, intermediate-2, or high-risk)
  • Treatment naïve or ≤1 prior cycle of decitabine or azacitidine
  • No restrictions on body weight or surface area

MDS efficacy

INQOVI delivered equivalent systemic exposure to IV decitabine.1,2

MDS safety

INQOVI showed a safety profile similar to IV decitabine in the Phase 3 clinical trial.1,2

References: 1. Garcia-Manero G, McCloskey J, Griffiths EA, et al. Oral decitabine-cedazuridine versus intravenous decitabine for myelodysplastic syndromes and chronic myelomonocytic leukaemia (ASCERTAIN): a registrational, randomised, crossover, pharmacokinetics, phase 3 study. Lancet Haematol. 2024;11(1):e15-e26. 2. INQOVI [package insert]. Princeton, NJ: Taiho Oncology, Inc.; 2026. 3. Zeidan AM, Perepezko K, Salimi T, Washington T, Epstein RS. Patients’ perspectives on oral decitabine/cedazuridine for the treatment of myelodysplastic syndromes/neoplasms. Ther Adv Hematol. 2024;15:20406207241257313.