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Study Design

STML-401-0114 clinical trial: The largest prospective study to date of patients with BPDCN to specifically target CD1231

  • The study was designed as a nonrandomized, multistage, open-label, multicenter evaluation of ELZONRIS as monotherapy in patients with BPDCN, regardless of whether they had received previous treatment1,2
  • FDA approval of ELZONRIS was based on the efficacy and safety observed in 13 treatment-naive patients in the pivotal cohort and 15 previously treated patients from other cohorts3,4
  • Following FDA approval, 40 additional patients (36 treatment-naive, 4 previously treated) were enrolled in a continued access cohort3,5

The primary outcome was CR/CRc3*†

Initial analysis

Initial analysis study design
  • Efficacy was confirmed in the pivotal cohort of 13 treatment-naive patients1,2
  • Median follow-up was ~2 years3

Long-term analysis

  • The long-term analysis included all 44 patients from the initial analysis, as well as 40 others enrolled in the continued access cohort1,3,5
    • 65 treatment-naive patients and 19 previously treated patients were efficacy evaluable1
  • Patients with CR/CRc received ELZONRIS until disease progression, unacceptable toxicity, or allogeneic (allo)/autologous (auto) SCT2,4
  • Median follow-up was ~3 years1

See the long-term study design

*Complete response (CR) criteria: normalization of blast percentage (≤5%) in the bone marrow; normalization of neutrophil count (≥1000/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.2

†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).2

‡Dosing period may be extended for dose delays up to day 10 of the cycle.4

Baseline patient characteristics (initial analysis)

Treatment-naive patients2,4

Characteristics Pivotal cohort
(n=13)		 Other cohorts
(n=16)		 Total§
(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
(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)
Visceral 15.4% (2) 12.5% (2) 13.8% (4)

Previously treated patients2-4

Characteristics Previously treated patients
(n=15)
Male, % (n) 86.7% (13)
Female, % (n) 13.3% (2)
Median age
(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)
Visceral 26.7% (4)
Prior treatments, % (n)  
Radiation therapy 33.3% (5)
Stem cell transplantation 26.7% (4)

Previous lines of therapy: Previously treated patients3

Number of lines Previously treated patients
1 60% (9)
2-3 27% (4)
≥4 13% (2)

§Total population (n=29) includes patients from the pivotal cohort (n=13).2,3

BPDCN, blastic plasmacytoid dendritic cell neoplasm; CT, computed tomography; ECOG PS, Eastern Cooperative Oncology Group performance status; SCT, stem cell transplantation.


Explore a treatment with durable efficacy in treatment-naive patients
  1. References:
  2. Pemmaraju N, et al. Long-term benefits of tagraxofusp for patients with blastic plasmacytoid dendritic cell neoplasm. J Clin Oncol. 2022;40(26):3032-3036.
  3. Data on file. Stemline Therapeutics, Inc.
  4. Pemmaraju N, et al. Tagraxofusp in blastic plasmacytoid dendritic-cell neoplasm. N Engl J Med. 2019;380(17):1628-1637.
  5. ELZONRIS [prescribing information]. New York, NY: Stemline Therapeutics, Inc.; November 2022.
  6. Pemmaraju N, et al. Long-term benefits of tagraxofusp for patients with blastic plasmacytoid dendritic cell neoplasm. J Clin Oncol. 2022;40(26):3032-3036 [supplementary appendix].

INDICATION

  • ELZONRIS is a CD123-directed cytotoxin indicated 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

  • Capillary leak syndrome (CLS), including life-threatening and fatal cases, has been reported among patients treated with ELZONRIS. In patients receiving ELZONRIS in clinical trials, the overall incidence of CLS was 53% (65/122), including Grade 1 or 2 in 43% (52/122) of patients, Grade 3 in 7% (8/122) of patients, Grade 4 in 1% (1/122) of patients, and four fatalities (3%). The median time to onset was 4 days (range - 1 to 46 days), and all but 5 patients experienced an event in Cycle 1.
  • Before initiating therapy with ELZONRIS, ensure that the patient has adequate cardiac function and serum albumin is greater than or equal to 3.2 g/dL. During treatment with ELZONRIS, monitor serum albumin levels prior to the initiation of each dose of ELZONRIS and as indicated clinically thereafter, and assess patients for other signs or symptoms of CLS, including weight gain, new onset or worsening edema, including pulmonary edema, hypotension or hemodynamic instability.

Hypersensitivity Reactions

  • ELZONRIS can cause severe hypersensitivity reactions. In patients receiving ELZONRIS in clinical trials, hypersensitivity reactions were reported in 43% (53/122) of patients treated with ELZONRIS and were Grade ≥ 3 in 7% (9/122). Manifestations of hypersensitivity reported in ≥ 5% of patients include rash, pruritus, and stomatitis. Monitor patients for hypersensitivity reactions during treatment with ELZONRIS. Interrupt ELZONRIS infusion and provide supportive care as needed if a hypersensitivity reaction should occur.

Hepatotoxicity

  • Treatment with ELZONRIS was associated with elevations in liver enzymes. In patients receiving ELZONRIS in clinical trials, elevations in ALT occurred in 79% (96/122) and elevations in AST occurred in 76% (93/122). Grade 3 ALT elevations were reported in 26% (32/122) of patients. Grade 3 AST elevations were reported in 30% (36/122) and Grade 4 AST elevations were reported in 3% (4/122) of patients. Elevated liver enzymes occurred in the majority of patients in Cycle 1 and were reversible following dose interruption.
  • Monitor alanine aminotransferase (ALT) and aspartate aminotransferase (AST) prior to each infusion with ELZONRIS. Withhold ELZONRIS temporarily if the transaminases rise to greater than 5 times the upper limit of normal and resume treatment upon normalization or when resolved.

ADVERSE REACTIONS:

Most common adverse reactions (incidence ≥ 30%) are capillary leak syndrome, nausea, fatigue, pyrexia, peripheral edema, and weight increase. Most common laboratory abnormalities (incidence ≥ 50%) are decreases in albumin, platelets, hemoglobin, calcium, and 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 indicated 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

  • Capillary leak syndrome (CLS), including life-threatening and fatal cases, has been reported among patients treated with ELZONRIS. In patients receiving ELZONRIS in clinical trials, the overall incidence of CLS was 53% (65/122), including Grade 1 or 2 in 43% (52/122) of patients, Grade 3 in 7% (8/122) of patients, Grade 4 in 1% (1/122) of patients, and four fatalities (3%). The median time to onset was 4 days (range - 1 to 46 days), and all but 5 patients experienced an event in Cycle 1.
  • Before initiating therapy with ELZONRIS, ensure that the patient has adequate cardiac function and serum albumin is greater than or equal to 3.2 g/dL. During treatment with ELZONRIS, monitor serum albumin levels prior to the initiation of each dose of ELZONRIS and as indicated clinically thereafter, and assess patients for other signs or symptoms of CLS, including weight gain, new onset or worsening edema, including pulmonary edema, hypotension or hemodynamic instability.

Hypersensitivity Reactions

  • ELZONRIS can cause severe hypersensitivity reactions. In patients receiving ELZONRIS in clinical trials, hypersensitivity reactions were reported in 43% (53/122) of patients treated with ELZONRIS and were Grade ≥ 3 in 7% (9/122). Manifestations of hypersensitivity reported in ≥ 5% of patients include rash, pruritus, and stomatitis. Monitor patients for hypersensitivity reactions during treatment with ELZONRIS. Interrupt ELZONRIS infusion and provide supportive care as needed if a hypersensitivity reaction should occur.

Hepatotoxicity

  • Treatment with ELZONRIS was associated with elevations in liver enzymes. In patients receiving ELZONRIS in clinical trials, elevations in ALT occurred in 79% (96/122) and elevations in AST occurred in 76% (93/122). Grade 3 ALT elevations were reported in 26% (32/122) of patients. Grade 3 AST elevations were reported in 30% (36/122) and Grade 4 AST elevations were reported in 3% (4/122) of patients. Elevated liver enzymes occurred in the majority of patients in Cycle 1 and were reversible following dose interruption.
  • Monitor alanine aminotransferase (ALT) and aspartate aminotransferase (AST) prior to each infusion with ELZONRIS. Withhold ELZONRIS temporarily if the transaminases rise to greater than 5 times the upper limit of normal and resume treatment upon normalization or when resolved.

ADVERSE REACTIONS:

Most common adverse reactions (incidence ≥ 30%) are capillary leak syndrome, nausea, fatigue, pyrexia, peripheral edema, and weight increase. Most common laboratory abnormalities (incidence ≥ 50%) are decreases in albumin, platelets, hemoglobin, calcium, and 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.