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Important Safety Information

Prescribing Information

MDS Study Design

ASCERTAIN: A Phase 3 MDS and CMML clinical trial1

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
Graphic showing the randomized 1:1 crossover study design of the ASCERTAIN trial, which compared INQOVI and intravenous decitabine treatment sequences across multiple therapy cycles in patients receiving HMA therapy for MDS. Graphic showing the randomized 1:1 crossover study design of the ASCERTAIN trial, which compared INQOVI and intravenous decitabine treatment sequences across multiple therapy cycles in patients receiving HMA therapy for MDS.

aFive days.

Endpoints evaluated in the ASCERTAIN Phase 3 trial

ENDPOINTS PHASE 3 (N=133)1,2 PHASE 3 LONG-TERM
FOLLOW-UP (N=133)1
PRIMARY ENDPOINTS 5-day area under the curve (AUC) between INQOVI and IV decitabine for Cycles 1 and 2 5-day AUC between INQOVI and IV decitabine for Cycles 1 and 2
KEY SECONDARY ENDPOINTS
  • Complete response (CR)
  • Rate of conversion from transfusion dependence to transfusion independence
  • Clinical response
  • Transfusion independence
  • Median overall survival (OS) and leukemia-free survival (LFS)
  • Safety
  • Pharmacodynamics
OTHER ENDPOINTS
  • Median duration of CR
  • Median time to CR
  • Median duration of CR and best response
  • Median time to first response
ENDPOINT PHASE 3 (N=133)13,14
PRIMARY ENDPOINTS 5-day under the curve (AUC) between oral decitabine-cedazuridine and IV decitabine for Cycles 1 and 2
KEY SECONDARY ENDPOINTS
  • Complete response (CR)
  • Rate of conversion from transfusion dependence to transfusion independence
OTHER ENDPOINTS
  • Median duration of CR
  • Median time to CR
ENDPOINT PHASE 3 LONG-TERM FOLLOW-UP (N=133)1
PRIMARY ENDPOINTS 5-day AUC between oral decitabine-cedazuridine and IV decitabine for Cycles 1 and 2
KEY SECONDARY ENDPOINTS
  • Clinical response
  • Transfusion independence
  • Median overall survival (OS) and leukemia-free survival (LFS)
  • Safety
  • Pharmacodynamics
OTHER ENDPOINTS
  • Median duration of CR and best response
  • Median time to first response

Patient demographics and disease characteristics at baseline1,2

BASELINE PATIENT CHARACTERISTIC PHASE 3 (N=133)
AGE (YEARS)
Median (range) 71 (44-88)
SEX (%)
Male 65
Female 35
RACE (%)
White 91
Black or
African American 		3
Asian 2
Other or not reported 4
ECOG PERFORMANCE
SCORE (PS) (%)
0 41
1 59
2 0
DISEASE CATEGORY/IPSS (%)
MDS intermediate-1 risk 44
MDS intermediate-2 risk 20
MDS high-risk 16
MDS low-risk 8
CMML 12
PRIOR HMA THERAPY (%)a
Prior azacitidine 5
Prior decitabine 3
TRANSFUSION DEPENDENCE (%)b
RBC transfusion dependence 39
Platelet transfusion dependence 8

aOne cycle only, per the exclusion criteria.

bDefined as documentation of ≥2 units of transfusion within 56 days of the first day of study treatment.

MDS efficacy

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

Explore MDS efficacy

MDS safety

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

View MDS safety

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.