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Efficacy
Study Design
STML-401-0114 clinical trial: The largest prospective study of patients with BPDCN to specifically target CD1231
In the largest prospective clinical trial of treatment-naïve and previously-treated patients with BPDCN, ELZONRIS was evaluated in an open-label, multicenter clinical study (N=44). The pivotal cohort consisted of 13 treatment-naïve patients.1,2
*Complete response (CR) criteria: normalization of blast percentage (≤ 5%) in the bone marrow; normalization of neutrophil count (≥ 1,000/µL) and platelet count (≥ 100,000/µL) in the peripheral blood; absence of leukemic blasts in the peripheral blood; 100% clearance of all skin lesions from baseline; no new lesions in patients without lesions at baseline; regression of nodal masses to normal size on CT; no palpable nodules on the spleen or liver.3
†Clinical complete response with minimal residual skin abnormality (CRc) criteria: marked clearance of all skin lesions from baseline; residual hyperpigmentation or abnormality with BPDCN identified on biopsy (or no biopsy performed).3
‡Dosing period may be extended for dose delays up to day 10 of the cycle.2
Baseline patient characteristics
Key baseline demographics in the study reflect the patient population and variable presentation of BPDCN.1-3
Baseline Characteristics: Treatment-Naïve Patients2,3§
| Characteristics | Pivotal Cohort (N=13) |
Other Cohorts (N=16) |
All Cohorts (N=29) |
|---|---|---|---|
| Male, % (n) | 84.6% (11) | 75.0% (12) | 79.3% (23) |
| Female, % (n) | 15.4% (2) | 25.0% (4) | 20.7% (6) |
| Median age, years (min, max) | 65.0 (22, 84) | 67.5 (28, 84) | 67.0 (22, 84) |
| ECOG PS, % (n) | |||
| 0 | 61.5% (8) | 43.8% (7) | 51.7% (15) |
| 1 | 38.5% (5) | 56.3% (9) | 48.3% (14) |
| BPDCN, % (n) | |||
| Skin | 100.0% (13) | 93.8% (15) | 96.6% (28) |
| Bone marrow | 53.8% (7) | 43.8% (7) | 48.3% (14) |
| Peripheral blood | 23.1% (3) | 25.0% (4) | 24.1% (7) |
| Lymph nodes | 46.2% (6) | 43.8% (7) | 44.8% (13) |
| Viscera | 15.4% (2) | 12.5% (2) | 13.8% (4) |
Baseline Characteristics: Previously-Treated Patients1-3
| Characteristics | Previously-Treated (N=15) |
|---|---|
| Male, % (n) | 86.7% (13) |
| Female, % (n) | 13.3% (2) |
| Median age, years (min, max) | 72 (44, 80) |
| ECOG PS, % (n) | |
| 0 | 33.3% (5) |
| 1 | 66.7% (10) |
| BPDCN, % (n) | |
| Skin | 86.7% (13) |
| Bone marrow | 60.0% (9) |
| Peripheral blood | 6.7% (1) |
| Lymph nodes | 53.3% (8) |
| Viscera | 26.7% (4) |
| Prior treatments, % (n) | |
| Radiation therapy | 33.3% (5) |
| Stem cell transplantation | 26.7% (4) |
Previous lines of therapy: previously-treated patients1
| Number of Lines | Patients, % (n) |
|---|---|
| 1 | 60% (9) |
| 2-3 | 27% (4) |
| ≥ 4 | 13% (2) |
§All-cohorts population (N=29) includes patients from the pivotal cohort (N=13).
CT = computed tomography; ECOG PS = Eastern Cooperative Oncology Group performance status.
References: 1. Pemmaraju N, Lane AA, Sweet KL, et al. Tagraxofusp in blastic plasmacytoid dendritic-cell neoplasm. N Engl J Med. 2019;380(17):1628-1637. 2. ELZONRIS [prescribing information]. New York, NY, US: Stemline Therapeutics, Inc.; December 2018. 3. Data on file. Stemline Therapeutics, Inc.
INDICATION
- ELZONRIS is a CD123-directed cytotoxin for the treatment of blastic plasmacytoid dendritic cell neoplasm (BPDCN) in adults and in pediatric patients 2 years and older
IMPORTANT SAFETY INFORMATION
Boxed WARNING: CAPILLARY LEAK SYNDROME
- Capillary Leak Syndrome (CLS), which may be life-threatening or fatal, can occur in patients receiving ELZONRIS. Monitor for signs and symptoms of CLS and take actions as recommended
WARNINGS AND PRECAUTIONS
Capillary Leak Syndrome
- ELZONRIS can cause capillary leak syndrome (CLS), which may be life-threatening or fatal if not properly managed. The overall incidence of CLS in clinical trials was 55% in patients receiving ELZONRIS, including 46% in Grades 1 or 2, 6% in Grade 3, 1% in Grade 4, and 2 fatal events. Common signs and symptoms (incidence ≥ 20%) associated with CLS that were reported during treatment with ELZONRIS include hypoalbuminemia, edema, weight gain, and hypotension
- Before initiating therapy with ELZONRIS, ensure that the patient has adequate cardiac function and serum albumin is ≥ 3.2 g/dL
- During treatment with ELZONRIS, ensure that serum albumin levels are ≥ 3.5 g/dL and have not been reduced by ≥ 0.5 g/dL from the albumin value measured prior to dosing initiation of the current cycle. Monitor serum albumin levels prior to the initiation of each dose or more often as indicated clinically thereafter. Additionally, assess patients for other signs or symptoms of CLS, including weight gain, new onset or worsening edema including pulmonary edema, hypotension, or hemodynamic instability
- Counsel patients to seek immediate medical attention should signs or symptoms of CLS occur at any time
Hypersensitivity Reactions
- ELZONRIS can cause severe hypersensitivity reactions. Grade 3 or higher events were reported in 10% of patients in clinical trials. Monitor patients for hypersensitivity reactions during treatment with ELZONRIS. Interrupt ELZONRIS infusion and provide supportive care as needed if a hypersensitivity reaction should occur. If the reaction is severe, discontinue ELZONRIS permanently
Hepatotoxicity
- Elevations in liver enzymes can occur with ELZONRIS. Grade 3 or higher elevations in liver enzymes occurred in approximately 40% of patients in clinical trials
- Monitor alanine aminotransferase (ALT) and aspartate aminotransferase (AST) prior to each infusion with ELZONRIS. Temporarily withhold ELZONRIS if the transaminases rise to greater than 5 times the upper limit of normal (ULN) and resume treatment upon normalization or when resolved
ADVERSE REACTIONS:
The most common adverse reactions in the clinical trials (incidence ≥ 30%) are capillary leak syndrome, nausea, fatigue, peripheral edema, pyrexia, and weight increase. The most common laboratory abnormalities (incidence ≥ 50%) are decreases in albumin, platelets, hemoglobin, calcium, sodium, and increases in glucose, ALT, and AST.
Please see full Prescribing Information, including Boxed WARNING.
To report SUSPECTED ADVERSE REACTIONS, contact Stemline Therapeutics, Inc. at 1-877-332-7961 or contact the FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.
IMPORTANT SAFETY INFORMATION
INDICATION
- ELZONRIS is a CD123-directed cytotoxin for the treatment of blastic plasmacytoid dendritic cell neoplasm (BPDCN) in adults and in pediatric patients 2 years and older
IMPORTANT SAFETY INFORMATION
Boxed WARNING: CAPILLARY LEAK SYNDROME
- Capillary Leak Syndrome (CLS), which may be life-threatening or fatal, can occur in patients receiving ELZONRIS. Monitor for signs and symptoms of CLS and take actions as recommended
WARNINGS AND PRECAUTIONS
Capillary Leak Syndrome
- ELZONRIS can cause capillary leak syndrome (CLS), which may be life-threatening or fatal if not properly managed. The overall incidence of CLS in clinical trials was 55% in patients receiving ELZONRIS, including 46% in Grades 1 or 2, 6% in Grade 3, 1% in Grade 4, and 2 fatal events. Common signs and symptoms (incidence ≥ 20%) associated with CLS that were reported during treatment with ELZONRIS include hypoalbuminemia, edema, weight gain, and hypotension
- Before initiating therapy with ELZONRIS, ensure that the patient has adequate cardiac function and serum albumin is ≥ 3.2 g/dL
- During treatment with ELZONRIS, ensure that serum albumin levels are ≥ 3.5 g/dL and have not been reduced by ≥ 0.5 g/dL from the albumin value measured prior to dosing initiation of the current cycle. Monitor serum albumin levels prior to the initiation of each dose or more often as indicated clinically thereafter. Additionally, assess patients for other signs or symptoms of CLS, including weight gain, new onset or worsening edema including pulmonary edema, hypotension, or hemodynamic instability
- Counsel patients to seek immediate medical attention should signs or symptoms of CLS occur at any time
Hypersensitivity Reactions
- ELZONRIS can cause severe hypersensitivity reactions. Grade 3 or higher events were reported in 10% of patients in clinical trials. Monitor patients for hypersensitivity reactions during treatment with ELZONRIS. Interrupt ELZONRIS infusion and provide supportive care as needed if a hypersensitivity reaction should occur. If the reaction is severe, discontinue ELZONRIS permanently
Hepatotoxicity
- Elevations in liver enzymes can occur with ELZONRIS. Grade 3 or higher elevations in liver enzymes occurred in approximately 40% of patients in clinical trials
- Monitor alanine aminotransferase (ALT) and aspartate aminotransferase (AST) prior to each infusion with ELZONRIS. Temporarily withhold ELZONRIS if the transaminases rise to greater than 5 times the upper limit of normal (ULN) and resume treatment upon normalization or when resolved
ADVERSE REACTIONS:
The most common adverse reactions in the clinical trials (incidence ≥ 30%) are capillary leak syndrome, nausea, fatigue, peripheral edema, pyrexia, and weight increase. The most common laboratory abnormalities (incidence ≥ 50%) are decreases in albumin, platelets, hemoglobin, calcium, sodium, and increases in glucose, ALT, and AST.
Please see full Prescribing Information, including Boxed WARNING.
To report SUSPECTED ADVERSE REACTIONS, contact Stemline Therapeutics, Inc. at 1-877-332-7961 or contact the FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.