Drug Trials Snapshots: KOMZIFTI
HOW TO USE THIS SNAPSHOT
The information provided in Snapshots highlights who participated in the key clinical trials that supported the original FDA approval of this drug, and whether there were differences among sex, race, age, and ethnic groups. The “MORE INFO” bar shows more detailed, technical content for each section. The Snapshot is intended as one tool for consumers to use when discussing the risks and benefits of the drugs.
LIMITATIONS OF THIS SNAPSHOT
Do not rely on Snapshots to make decisions regarding medical care. Always speak to your healthcare provider about the benefits and risks of a drug.
Some of the information in this Snapshot is for presentation purposes and does not represent the approved conditions of use of this drug. Refer to the KOMZIFTI Prescribing Information for all the approved conditions of use of this drug (e.g., indication(s), population(s), dosing regimen(s), safety information).
Snapshots are limited to the information available at the time of the original approval of the drug and do not provide information on who participated in clinical trials that supported later approvals for additional uses of the drug (if applicable).
KOMZIFTI (ziftomenib)
kom-ZIF-tee
Kura Oncology, Inc.
Approval date: November 13, 2025
DRUG TRIALS SNAPSHOT SUMMARY:
What is the drug for?
KOMZIFTI is a cancer drug that is used to treat adult patients with acute myeloid leukemia (AML) with a susceptible nucleophosmin 1 (NPM1) mutation that has come back or has not improved after previous treatment(s) and who have no satisfactory alternative treatment options.
How is this drug used?
KOMZIFTI is a capsule that is taken orally once a day. The recommended dose is 600 mg once daily.
Who participated in the clinical trials?
The FDA approved KOMZIFTI based on evidence from one clinical trial (KO-MEN-001) of 112 patients with AML with an NPM1 mutation whose disease had come back or had not improved after previous treatment(s). Patients were enrolled at 42 sites in seven countries including the United States, Canada, Belgium, France, Germany, Italy, and Spain. The study included 52 patients in the United States.
How were the trials designed?
The benefits and side effects of KOMZIFTI were evaluated in one open-label clinical trial in 112 adult patients with AML whose disease had come back or had not improved after previous treatment(s). All patients had a mutation in a certain gene called NPM1.
Patients received KOMZIFTI 600 mg orally once daily. Treatment continued until disease worsened or patients experienced unacceptable side effects.
The benefit of KOMZIFTI was evaluated by measuring the number of patients who had no evidence of disease (complete remission) with full or partial recovery of blood counts after treatment and the percentage of patients who no longer required transfusions after treatment.
How were the trials designed?
The efficacy and safety of KOMZIFTI were evaluated in 112 adult patients with relapsed or refractory AML with an NPM1 mutation. Patients were pooled from two cohorts of the open-label, single-arm, multicenter clinical trial (Study KO-MEN-001, NCT04067336; KOMET-001), 20 patients in the Phase 1b cohort and 92 in the Phase 2 cohort. All 112 patients received the recommended KOMZIFTI dose of 600 mg once daily; patients continued receiving KOMZIFTI until disease progression or unacceptable toxicity. Patients were permitted to resume treatment with KOMZIFTI following hematopoietic stem cell transplant (HSCT).
The primary endpoint was rate of complete remission (CR) and complete remission with partial hematologic recovery (CRh). Efficacy was established based on the rate of CR+CRh, duration of CR+CRh, and the rate of conversion to transfusion independence.
DEMOGRAPHICS SNAPSHOT
Figure 1 summarizes how many male and female patients were enrolled in the clinical trial used to evaluate the efficacy of KOMZIFTI.
Figure 1. Baseline Demographics by Sex
Source: Adapted from FDA Review
Figure 2 summarizes how patients by race were enrolled in the clinical trial used to evaluate the efficacy of KOMZIFTI.
Figure 2. Baseline Demographics by Race
Source: Adapted from FDA Review
Figure 3 summarizes how patients by age were enrolled in the clinical trial used to evaluate the efficacy of KOMZIFTI.
Figure 3. Baseline Demographics by Age
Source: Adapted from FDA Review.
Figure 4 summarizes how patients by ethnicity were enrolled in the clinical trial used to evaluate the efficacy of KOMZIFTI.
Figure 4. Baseline Demographics by Ethnicity
Source: Adapted from FDA Review.
Who participated in the trials?
Table 1. Baseline Demographics and Disease Characteristics, Efficacy Population
Demographics and Disease Characteristics | KOMZIFTI N=112 |
|---|---|
Age, years | |
Median (min, max) | 69 (22, 86) |
Age group, years, n (%) | |
<65 | 42 (38) |
≥65 | 70 (63) |
Sex, n (%) | |
Male | 49 (44) |
Female | 63 (56) |
Race, n (%) | |
White | 88 (79) |
Black or African American | 2 (2) |
Asian | 4 (4) |
Other | 2 (2) |
Unknown | 16 (14) |
Ethnicity, n (%) | |
Hispanic or Latino | 3 (3) |
Not Hispanic or Latino | 87 (78) |
Unknown | 22 (20) |
Type of AML, n (%) | |
De novo AML | 95 (85) |
Secondary AML | 17 (15) |
Disease status, n (%) | |
Primary refractory | 7 (6) |
Refractory relapse | 37 (33) |
Untreated relapse | 68 (61) |
Median number of prior lines of therapy (range) | 2 (1, 7) |
Prior stem cell transplantation, n (%) | 26 (23) |
Source: Adapted from FDA Review
*REDEMPLO 50 mg is not an approved dosing regimen
Abbreviations: AML, acute myeloid leukemia
What are the benefits of this drug?
The trial measured the number of patients with no evidence of disease with full (CR) or partial (CRh) recovery of blood counts. In the trial, 24 of the 112 patients (21.4%) experienced CR or CRh after treatment, with a median duration of five months. The median time to response was 2.7 months.
Of the 66 patients who needed blood and/or platelet transfusions before treatment with KOMZIFTI, 14 (21.2%) no longer needed transfusions for at least 56 days during treatment. Of the 46 patients who did not need transfusions of both blood and platelets before treatment with KOMZIFTI, 12 (26.1%) went at least 56 days without needing a transfusion during treatment.
What are the benefits of this drug (results of trials used to assess efficacy)?
Table 2. Efficacy Results, Efficacy Population
Endpoint | KOMZIFTI N=112 |
|---|---|
CRa+CRhb, n (%) | 24 (21.4) |
95% CI | 14.2, 30.2 |
Median DOCR+CRhc (months) | 5.0 |
95% CI | 1.9, 8.1 |
CRa, n (%) | 19 (17.0) |
95% CI | 10.5, 25.2 |
Median DOCRd (months) | 5.0 |
95% CI | 2.8, 8.1 |
CRhb, n (%) | 5 (4.5) |
95% CI | 1.5, 10.1 |
Observed DOCRhe (months) | 0.0+, 1.5+, 1.5, 1.6, 11.4 |
Source: KOMZIFTI Prescribing Information
a CR is defined as bone marrow blasts <5%, absolute neutrophil count (ANC) >1.0x109/L, and platelet count >100x109/L.
b CRh is defined as bone marrow blasts <5%, ANC >:0.5x10⁹/L; platelet count >50x109/L.
c Duration of CR+CRh (DOCR+CRh) is defined as the time from first CR/CRh to the first documented relapse or death, whichever occurs first.
d Duration of CR (DOCR) is defined as the time from first CR to the first documented relapse or death, whichever occurs first.
e Duration of CRh (DOCRh) is defined as the time from first CRh to the first documented relapse or death, whichever occurs first.
The 95% CI rate was calculated using the exact method based on binomial distribution.
Abbreviations: CI, confidence interval; CR, complete remission; CRh, complete remission with partial hematologic recovery; +, censor
Were there any differences in how well the drug worked in clinical trials among sex, race, and age?
- Sex: KOMZIFTI worked similarly in males and females.
- Race: The number of patients of races other than White was small; therefore, differences in how KOMZIFTI worked among races could not be determined.
- Age: KOMZIFTI worked similarly in patients younger and older than 65 years of age.
Were there any differences in how well the drug worked in clinical trials among sex, race, and age?
Table 3. Efficacy Results (CR + CRh Rate) by Subgroup
Subgroup | Pooled Efficacy Cohort, N=112 | |
|---|---|---|
n/Ns (%) | 95% CI | |
Sex | ||
Female | 15/63 (24) | 14, 36 |
Male | 9/49 (18) | 9, 32 |
Race | ||
White | 18/88 (20) | 13, 30 |
Asian/Black or African American/Other | 2/8 (25) | 3, 65 |
Unknown | 4/16 (25) | 7, 52 |
Age group, years | ||
<65 | 8/42 (19) | 9, 34 |
≥65 | 16/70 (23) | 14, 34 |
Source: Adapted from FDA Review
Abbreviations: CI, confidence interval; CR, complete remission; CRh, complete remission with partial hematologic recovery; N, number of patients in treatment arm; n, number of patients meeting criteria for CR or CRh; Ns, total number of patients for each specific subgroup
What are the possible side effects?
The most common side effects of KOMZIFTI are infections (including bacterial and viral infections), bleeding (hemorrhage), diarrhea, nausea, feeling tired (fatigue), swelling in the arms and legs (edema), joint pain (musculoskeletal pain), itching (pruritus), fever with decreased white blood cell counts (febrile neutropenia), and changes in liver function tests (transaminases increased).
KOMZIFTI may cause a serious side effect called differentiation syndrome, which may be life-threatening or lead to death if not treated. KOMZIFTI may cause changes in the electrical activity of the heart (QT prolongation) and may cause harm to an unborn baby.
What are the possible side effects (results of trials used to assess safety)?
Table 4. Safety Results, Safety Population
Adverse Reaction | KOMZIFTI, N=112 | |
|---|---|---|
All Grades† % | Grade 3 or 4 % | |
Infections and infestations | ||
Infection without identified pathogena | 52 | 38 |
Bacterial infectionb | 28 | 17 |
Viral infectionc | 16 | 5 |
Vascular disorders | ||
Hemorrhaged | 38 | 8 |
Hypertension | 11 | 5 |
Gastrointestinal disorders | ||
Diarrheae* | 36 | 5 |
Nauseaf* | 35 | 2 |
General disorders and administration site conditions | ||
Fatigueg | 34 | 8 |
Edemah* | 30 | 3 |
Musculoskeletal and connective tissue disorders | ||
Musculoskeletal paini | 28 | 4 |
Neoplasms benign, malignant, and unspecified (incl cysts and polyps) | ||
Differentiation syndrome | 26 | 13 |
Skin and subcutaneous | ||
Pruritusj* | 23 | 0 |
Blood and lymphatic system disorder | ||
Febrile neutropenia | 22 | 22 |
Leukocytosisk* | 16 | 5 |
Investigations | ||
Transaminases increasedl | 21 | 8 |
Electrocardiogram QT prolonged* | 12 | 8 |
Renal impairmentm | 19 | 6 |
Respiratory, thoracic, and mediastinal disorders | ||
Hypoxia | 9 | 5 |
Source: KOMZIFTI Prescribing Information
† Includes the following fatal adverse reactions: infection (n=1) and differentiation syndrome (n=2).
* No Grade 4 events were reported for this adverse reaction.
a Includes abdominal abscess, abscess limb, anal abscess, anorectal infection, bronchitis, conjunctivitis, device related infection, device related sepsis, diverticulitis, emphysematous cystitis, enterocolitis infectious, gingivitis, hordeolum, infection, large intestine infection, nail infection, necrotizing fasciitis, penile infection, periodontitis, perirectal abscess, peritonitis, pneumonia, postoperative abscess, respiratory tract infection, rhinitis, sepsis, septic shock, sinusitis, skin infection, soft tissue infection, tooth infection, upper respiratory tract infection, urosepsis, vaginal infection, vascular device infection, and wound infection.
b Includes bacteremia, bacterial infection, bacterial pyelonephritis, breast cellulitis, cellulitis, clostridial infection, clostridium difficile colitis, clostridium difficile infection, enterobacter sepsis, enterococcal bacteremia, erysipelas, escherichia bacteremia, escherichia infection, escherichia sepsis, escherichia urinary tract infection, klebsiella bacteremia, klebsiella infection, klebsiella urinary tract infection, paronychia, pneumonia bacterial, pneumonia klebsiella, pseudomonal bacteremia, and staphylococcal bacteremia.
c Includes adenovirus infection, COVID-19, genital herpes, herpes simplex reactivation, herpes virus infection, herpes zoster, nasal herpes, oral herpes, pneumonia influenzas, pneumonia parainfluenza viral, respiratory syncytial virus infection, and rhinovirus infection.
d Includes angina bullosa hemorrhagic, bladder tamponade, catheter site hematoma, conjunctival hemorrhage, contusion, disseminated intravascular coagulation, ecchymosis, epistaxis, gastric hemorrhage, gastrointestinal hemorrhage, gingival bleeding, hematemesis, hematochezia, hematoma, hematuria, hemoptysis, hemorrhage, hemorrhoidal hemorrhage, hyperfibrinolysis, injection site hematoma, injection site hemorrhage, lower gastrointestinal hemorrhage, melaena, mouth hemorrhage, oral blood blister, petechiae, purpura, rectal hemorrhage, retinal hemorrhage, shock hemorrhagic, skin hemorrhage, subdural hematoma, tongue hemorrhage, traumatic hematoma, upper gastrointestinal hemorrhage, vaginal hemorrhage, and vitreous hemorrhage.
e Includes diarrhea, colitis, and colitis erosive.
f Includes nausea and vomiting.
g Includes fatigue, asthenia, and malaise.
h Includes edema, edema peripheral, generalized edema, localized edema, and peripheral swelling.
i Includes arthralgia, back pain, bone pain, flank pain, musculoskeletal chest pain, musculoskeletal pain, myalgia, neck pain, and pain in extremity.
j Includes pruritus and nasal pruritus.
k Includes white blood cell count increased, leukocytosis, and hyperleukocytosis.
l Includes transaminases increased, alanine aminotransferase increased, aspartate aminotransferase increased, gamma-glutamyl transferase increased, and hypertransaminasemia.
m Includes renal impairment: acute kidney injury, azotemia, blood creatinine increased, blood urea increased, chronic kidney disease, postrenal failure, and renal failure.
Abbreviations: incl, including
Were there any differences in side effects among sex, race, and age?
- Sex:
- Race:
- Age:
Were there any differences in side effects of the clinical trials among sex, race, and age groups?
Table 5. Safety Results by Subgroup
Source: Adapted from FDA Review
Abbreviations: N, number of patients in treatment arm; n, number of patients meeting criteria; Ns, total number of patients for each specific subgroup
GLOSSARY
CLINICAL TRIAL: Voluntary research studies conducted in people and designed to answer specific questions about the safety or effectiveness of drugs, vaccines, other therapies, or new ways of using existing treatments.
EFFICACY: How well the drug achieves the desired response when it is taken as described in a controlled clinical setting, such as during a clinical trial.
SUBGROUP: A subset of the population studied in a clinical trial. Demographic subsets include sex, race, and age groups.