NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines®) recommend ivosidenib (TIBSOVO®) for newly diagnoseda and R/R AML with an IDH1 mutation1
aAt least 60 years and not eligible for or declines intensive remission induction therapy. See NCCN Guidelines® for full recommendation.

TIBSOVO® was studied in a patient population reflective of that seen in clinical practice1

Selected baseline demographic and disease characteristics (N=174)1
Median age (years) (min, max) 67 (18, 87)
ECOG PS
0 21%
1 56%
2 22%
3 1%
IDH1 mutation
R132C 59%
R132H 25%
R132G 7%
R132S 6%
R132L 4%
Cytogenetic risk status
Intermediate 60%
Poor 27%
Missing/unknown 13%
Relapse type
Primary refractory 37%
Refractory relapse 26%
Untreated relapse 37%
Prior stem cell transplantation for AML 23%
Transfusion dependent at baselinea 63%
Type of AML
De novo AML 67%
Secondary AML 33%
aPatients were defined as transfusion dependent at baseline if they received any RBC or platelet transfusion occurring within 56 days prior to the first dose of TIBSOVO.1
ECOG PS, Eastern Cooperative Oncology Group Performance Status.

Many patients in the study had challenging disease characteristics1,2

  • 26%

    of patients who were refractory in relapse1

  • 37%

    of patients who were primary refractory1

58% of patients had ≥2 prior anticancer therapies2

  • Median number of prior therapies (min, max): 2 (1, 6)1

23% of patients had prior stem cell transplantation for AML1
33% of patients had secondary AML1

TIBSOVO delivered strong and durable responses as an oral, single agent in difficult-to-treat disease1

  • 24.7% CR rate (43/174) (95% CI, 18.5-31.8);
    8.0% CRh rate (14/174) (95% CI, 4.5-13.1)1
  • CR+CRh rate appeared to be consistent across all baseline demographic and baseline disease characteristics with the exception of number of prior regimens1
  • For patients who achieved CR or CRh, the median time to CR or CRh was 2 months (range, 0.9-5.6 months)1
47% of patients who had received 1 prior regimen (35/74) achieved CR or CRh (95% CI, 35.6-59.3)2
Medium Duration of Response, Graph
  • Median treatment duration: 4.1 months (range, 0.1-39.5 months)1
  • Median follow-up: 8.3 months (range, 0.2-39.5 months)1
bDOCR (duration of CR) and DOCR+CRh (duration of CR+CRh) were defined as time since first response of CR or CR/CRh, respectively, to relapse or death, whichever is earlier.1

Transfusion independence was seen in 37% of transfusion-dependent patients who received TIBSOVO1

  • 20.0% (22/110)

    Patients who achieved CR or CRh2

  • 17.3% (19/110)

    Patients who did not achieve CR or CRh2

  • 59% of patients who were transfusion independent at baseline (38/64) remained so1
  • Patients were defined as transfusion dependent at baseline if they received any RBC or platelet transfusions within 56 days prior to the first dose of TIBSOVO. Patients were defined as transfusion independent if they became independent of transfusions during any 56-consecutive day postbaseline period1
12% of patients (21/174) went on to receive a stem cell transplant following treatment with TIBSOVO1
RBC, red blood cell.

References: 1. TIBSOVO [package insert]. Cambridge, MA: Agios Pharmaceuticals, Inc.; 2019. 2. Data on file. Agios Pharmaceuticals, Inc.
 

TIBSOVO® was studied as a single agent in both the newly diagnosed and R/R AML settings1

TIBSOVO is a first-in-class agent that inhibits the mutant IDH1 enzyme to induce myeloid differentiation1

  • The pivotal trial for TIBSOVO was an open-label, single-arm, multicenter trial1
  • IDH1 mutations were identified by a local or central diagnostic test and confirmed retrospectively using the Abbott RealTimeTM IDH1 assay, which is the FDA-approved test for selection of patients with AML for treatment with TIBSOVO1
Patients were assigned a starting dose of TIBSOVO 500 mg daily and received treatment until disease progression, development of unacceptable toxicity, or undergoing hematopoietic stem cell transplantation1
  • 28 IC-ineligible patients with newly diagnosed AML were evaluated for safety and efficacy
  • 179 patients were evaluated for safety and 174 for efficacy in the R/R AML population

Efficacy was established based on the rate of CR and/or CRh, duration of CR+CRh, as well as on the rate of conversion from transfusion dependence to transfusion independence1

CR, complete remission, defined as <5% blasts in the bone marrow, no evidence of disease, and full recovery of peripheral blood counts (platelets >100,000/microliter and absolute neutrophil counts >1000/microliter); CRh, complete remission with partial hematological recovery, defined as <5% blasts in the bone marrow, no evidence of disease, and partial recovery of peripheral blood counts (platelets >50,000/microliter and absolute neutrophil counts >500/microliter); IC, intensive chemotherapy; R/R, relapsed or refractory.1

Reference: 1. TIBSOVO [package insert]. Cambridge, MA: Agios Pharmaceuticals, Inc.; 2019.
 

INDICATIONS

TIBSOVO is indicated for the treatment of acute myeloid leukemia (AML) with a susceptible isocitrate dehydrogenase-1 (IDH1) mutation as detected by an FDA-approved test in:

  • Adult patients with newly diagnosed AML who are ≥75 years old or who have comorbidities that preclude use of intensive induction chemotherapy.
Indications & Important Safety Information

IMPORTANT SAFETY INFORMATION

WARNING: DIFFERENTIATION SYNDROME

Patients treated with TIBSOVO have experienced symptoms of differentiation syndrome, which can be fatal if not treated. Symptoms may include fever, dyspnea, hypoxia, pulmonary infiltrates, pleural or pericardial effusions, rapid weight gain or peripheral edema, hypotension, and hepatic, renal, or multi-organ dysfunction. If differentiation syndrome is suspected, initiate corticosteroid therapy and hemodynamic monitoring until symptom resolution.