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IDHIFA Clinical Trial Design

Approval of IDHIFA was based on results from a
CLINICAL TRIAL OF 199 ADULT PATIENTS WITH R/R AML AND AN IDH2 MUTATION

IDHIFA was evaluated in an open-label, single-arm, multicenter, 2 cohort clinical trial of 199 adult patients with R/R AML and an IDH2 mutation. Patients' IDH2 mutations were identified by a local diagnostic test and retrospectively confirmed by or prospectively identified by the Abbott RealTime™ IDH2 assay. IDHIFA was given orally at a starting dose of 100 mg daily until disease progression or unacceptable toxicity. Dose reductions were allowed to manage side effects. Patients had a median age of 68 years (range, 19 to 100) and received a median of 2 prior anticancer regimens (range, 1 to 6). More than half (52%) were refractory to previous therapy.

  • Efficacy was established on the basis of the rate of complete response (CR)/complete response with partial hematologic recovery (CRh), the duration of CR/CRh, and the rate of conversion from transfusion dependence to transfusion independence
  • The median follow-up was 6.6 months (range, 0.4 to 27.7 months)

Patient Demographics

Demographic and disease characteristics

BASELINE DEMOGRAPHIC AND DISEASE CHARACTERISTICS IN PATIENTS WITH R/R AML

Demographic and disease characteristics
R/R AML, N=199

100-mg daily dose

Median age (years)
  • 68 (range, 19-100)
Age categories, n (%)
<65 years76 (38)
≥65 years to <75 years74 (37)
≥75 years49 (25)
Sex, n (%)
Male103 (52)
Female96 (48)
Race, n (%)
White153 (77)
Black10 (5)
Asian1 (1)
Native Hawaiian/other Pacific Islander1 (1)
Other/not provided34 (17)
ECOG PSa, n (%)
046 (23)
1124 (62)
228 (14)
Relapsed AML, n (%)95 (48)
Refractory AML, n (%)104 (52)
IDH2 mutationb, n (%)
R140155 (78)
R17244 (22)
Median time from initial AML diagnosis
(months) (172 patients)
11.3 (range, 1.2-129.1)
Cytogenetic risk status, n (%)
Intermediate98 (49)
Poor54 (27)
Missing/failure47 (24)
Prior stem cell transplants for AML, n (%)25 (13)
  • Transfusion dependent at baselinec, n (%)
  • 157 (79)
Number of prior anticancer regimensd, n (%)
189 (45)
264 (32)
≥346 (23)
Median number of prior therapies2 (range, 1-6)

a1 patient had missing baseline ECOG PS.

b For 3 patients with different mutations detected in bone marrow compared to blood, the result of blood is reported.

c Patients were defined as transfusion dependent at baseline if they received any RBC or platelet transfusions within the 8-week baseline period.

d Includes intensive and/or nonintensive therapies.

ECOG PS, Eastern Cooperative Oncology Group performance status; RBC, red blood cell.

IDHIFA Clinical Trial Results

IDHIFA efficacy outcomes

EFFICACY RESULTS IN PATIENTS WITH R/R AML (N=199)

 CRaCRhbCR/CRh
n (%)37 (19%)
(95% CI, 13%-25%)
9 (4%)
(95% CI, 2%-8%)
46 (23%)
(95% CI, 18%-30%)
Median DORc (months)8.2
(95% CI, 4.7-19.4)
9.6
(95% CI, 0.7-NA)
8.2
(95% CI, 4.3-19.4)
 n (%)Median DORc (months)
CRa37 (19%)
(95% CI, 13%-25%)
8.2
(95% CI, 4.7-19.4)
CRhb9 (4%)
(95% CI, 2%-8%)
9.6
(95% CI, 0.7-NA)
CR/CRh46 (23%)
(95% CI, 18%-30%)
8.2
(95% CI, 4.3-19.4)

aCR was defined as <5% of blasts in the bone marrow, no evidence of disease, and full recovery of peripheral blood counts (platelets >100,000/μL and ANC >1,000/μL).

b CRh was defined as <5% of blasts in the bone marrow, no evidence of disease, and partial recovery of peripheral blood counts (platelets >50,000/μL and ANC >500/ μL).

c DOR was defined as time since first response in CR or CRh to relapse or death, whichever is earlier.

ANC, absolute neutrophil counts; CI, confidence interval; DOR, duration of response; NA, not available.

MEDIAN TIME TO FIRST AND BEST RESPONSE

 Time to first response
(months)
Time to best response of CR/CRh
(months)
Patients achieving CR/CRh (n=46/199)1.9
(range, 0.5-7.5)
3.7
(range, 0.6-11.2)

Of the 46 patients who achieved a best response of CR/CRh, 39 (85%) did so within 6 months of initiating IDHIFA

rbc- and platelet-transfusion independence

Among the 157 patients who were dependent on RBC and/or platelet transfusions at baseline, 53 (34%) became independent of RBC and platelet transfusions during any 56-day post-baseline period.

Of the 42 patients who were independent of both RBC and platelet transfusions at baseline, 32 (76%) remained transfusion independent during any 56-day post-baseline period.

Indication

IDHIFA® (enasidenib) is indicated for the treatment of adult patients with relapsed or refractory acute myeloid leukemia (AML) with an isocitrate dehydrogenase-2 (IDH2) mutation as detected by an FDA-approved test.

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Important Safety Information

WARNING: DIFFERENTIATION SYNDROME

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

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WARNINGS AND PRECAUTIONS

Differentiation Syndrome: See Boxed WARNING. In the clinical trial, 14% of patients treated with IDHIFA experienced differentiation syndrome. Differentiation syndrome has been observed with and without concomitant hyperleukocytosis, as early as 10 days and at up to 5 months after IDHIFA initiation. If differentiation syndrome is suspected, initiate systemic corticosteroids and hemodynamic monitoring until improvement. Taper corticosteroids only after resolution of symptoms. Differentiation syndrome symptoms may recur with premature discontinuation of corticosteroids. If severe pulmonary symptoms requiring intubation or ventilator support and/or renal dysfunction persist for more than 48 hours after initiation of corticosteroids, interrupt IDHIFA until signs and symptoms are no longer severe. Hospitalization for close observation and monitoring of patients with pulmonary and/or renal manifestation is recommended.

Embryo-Fetal Toxicity: Based on animal embryo-fetal toxicity studies, IDHIFA can cause embryo-fetal harm when administered to a pregnant woman. Advise females of reproductive potential and males with female partners of reproductive potential to use effective contraception during treatment with IDHIFA and for at least 1 month after the last dose. Pregnant women, patients becoming pregnant while receiving IDHIFA, or male patients with pregnant female partners should be apprised of the potential risk to the fetus.

ADVERSE REACTIONS

  • The most common adverse reactions (≥20%) included total bilirubin increased (81%), calcium decreased (74%), nausea (50%), diarrhea (43%), potassium decreased (41%), vomiting (34%), decreased appetite (34%), and phosphorus decreased (27%)
  • The most frequently reported ≥Grade 3 adverse reactions (≥5%) included total bilirubin increased (15%), potassium decreased (15%), phosphorus decreased (8%), calcium decreased (8%), diarrhea (8%), differentiation syndrome (7%), non-infectious leukocytosis (6%), tumor lysis syndrome (6%), and nausea (5%)
  • Serious adverse reactions were reported in 77.1% of patients. The most frequent serious adverse reactions (≥2%) were leukocytosis (10%), diarrhea (6%), nausea (5%), vomiting (3%), decreased appetite (3%), tumor lysis syndrome (5%), and differentiation syndrome (8%). Differentiation syndrome events characterized as serious included pyrexia, renal failure acute, hypoxia, respiratory failure, and multi-organ failure

LACTATION

Many drugs are excreted in human milk and because of the potential for adverse reactions in breastfed infants, advise women not to breastfeed during treatment with IDHIFA and for at least 1 month after the last dose.

Please see full Prescribing Information, including Boxed WARNING.

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Celgene Commercial Co-pay Program Terms and Conditions

Eligibility

Patients must meet the following criteria to enroll:

  • Covered by commercial or private insurance
  • Reside in the United States or US territory
  • Not participating in a federal or state-funded healthcare program, including, but not limited to, Medicare (Parts B, C, and D) or Medicaid, Medigap, CHAMPUS, VA, DOD, or Tricare
  • Gross annual household income must not exceed $100,000
    • Gross household income is the total income before income tax deductions from all people living in your household. Gross income refers not only to the salaries and benefits received, but also to receipts from any personal business, investments, dividends, and other income

Program Benefits

  • For Celgene oral hematology products, Celgene provides assistance to reduce the co-pay of eligible patients to $25 per prescription with a maximum benefit of $10,000 per enrollment period
  • For Celgene IV products, the Program will cover the co-pay for each prescription of a Celgene product up to a maximum of $10,000 per enrollment period
    • In order to receive the Program benefits for a Celgene IV product, patients or their providers must submit an Explanation of Benefits (EOB) form
  • Patients are responsible for any costs that exceed the Program’s $10,000 maximum
  • The Program will not cover, and shall not be applied toward, the cost of any dosing procedure, any other healthcare provider service or supply charges or other treatment costs, or any costs associated with a hospital stay

Program Timing

  • If eligible, patients will be enrolled from the date of enrollment through the end of the then current calendar year

Additional Terms and Conditions of the Celgene Commercial Co-pay Programs

  • Patients, pharmacists, and healthcare providers must not seek reimbursement from health insurance or any third party for any part of the benefit received by the patient through this Program. Patients must not seek reimbursement from any health savings, flexible spending, or other healthcare reimbursement accounts for the amount of assistance received from the Program
  • Acceptance of this offer confirms that this offer is consistent with your insurance and that you will report the value of the co-pay assistance you receive as may be required by your insurance provider
  • Only valid in the United States and US territories; this offer is void where prohibited by law, taxed or restricted. Absent a change in Massachusetts law, effective July 1, 2019, Massachusetts residents will no longer be eligible to participate in this Program
  • The Program benefits are nontransferable
  • Acceptance in this Program is not conditioned on any past, present, or future purchase, including additional doses
  • The Program cannot be combined with any other coupon, rebate, voucher, free trial, or similar offer
  • The Program is not insurance
  • Celgene reserves the right to rescind, revoke, or amend this Program at any time without notice

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