STUDY 1053 DESIGN

LUMOXITI was studied in the largest trial to date (N=80) in patients with relapsed/refractory hairy cell leukemia (HCL) who failed at least 2 prior therapies

Study 1053 was a Phase 3 trial for LUMOXITI that enrolled 80 patients with hairy cell leukemia, including patients with variant hairy cell leukemia1

  • All patients received ≥2 prior therapies, including ≥1 purine nucleoside analog (PNA)1

Baseline Demographics (N=80)1

All patients received at least 1 prior PNA therapy, including 29% in combination with rituximab1

87.5% of patients in Study 1053 were treated with at least 2 prior lines of PNA therapy2

48.8% of patients were refractory to prior PNA therapy as monotherapy and/or in combination with rituximab2

37.5% of patients were deemed not eligible for treatment with PNAs due to risk of infection or active infection at screening2

The most common non-PNA prior treatment regimens included1:

LUMOXITI was evaluated in Study 1053, a single-arm Phase 3 trial (N=80)1

CR=complete response; HR=hematologic remission

  • Patients who achieved CR and then maintained hematologic remission for a minimum of 180 days achieved durable CR1
  • Safety follow-up ended at the 181-day evaluation16
  • All responses were assessed by blinded independent central review14

Study 1053: Key Inclusion and Exclusion Criteria4

INCLUSION CRITERIA
  • Age >18 years
  • Histologically confirmed hairy cell leukemia or variant hairy cell leukemia
  • At least 2 prior systemic therapies, including at least 1 course of PNA therapy
  • ECOG performance status ≤2
  • Adequate organ function
EXCLUSION CRITERIA
  • Chemotherapy, immunotherapy, or radiotherapy within previous 4 weeks
  • Pregnant or breastfeeding females
  • Active second malignancy requiring treatment
  • History of allogeneic bone marrow transplant
  • Decreased neutrophils or decreased platelets unless due to underlying disease

Study 1053 Efficacy Endpoints1

PRIMARY ENDPOINT

Durable Complete Response (CR) Following BICR-assessed CR, maintenance of hematologic remissionmore than 180 days.1

SECONDARY ENDPOINTS

Overall Response Rate (ORR) Best overall response of complete response or partial response.1
Complete Response (CR) Clearing of the bone marrow of hairy cells by routine Hematoxylin & Eosin stain, radiologic resolution of pre-existing lymphadenopathy and/or organomegaly, and hematologic remission.1
Partial Response (PR) ≥50% decrease or normalization (<500/mm3) in peripheral blood lymphocyte count, reduction of pre-existing lymphadenopathy and/or organomegaly, and hematologic remission.1
Duration of Response Median duration of response in months. 1

BICR=blinded independent central review.

aHematologic remission defined as hemoglobin ≥11 g/dL, neutrophils ≥1500/mm3, and platelets ≥100,000/mm3 without transfusions or growth factor for at least 4 weeks.