PERSIST-2 is a randomized, active-controlled, phase 3 study (N=311) in patients with MF and platelet counts ≤100 x 109/L.1

Study Design1,2

Infographic of PERSIST-2 study design, including randomization and coprimary endpoints.
Infographic of PERSIST-2 study design, including randomization and coprimary endpoints.

PERSIST-2 enrolled patients with intermediate or high-risk primary or secondary (PPV or PET) MF with splenomegaly and platelet count ≤100 × 109/L.1

Patient demographics
  • Median age was 68 years (range 32 to 91)
  • 55% were male; 45% were female
  • 86% Caucasian; 14% non-Caucasian
Hematologic parameters
  • Platelet counts: Median baseline was 55 × 109/L
    • 45% of patients had Grade ≥3 thrombocytopenia (plt <50 × 109/L)
  • Hemoglobin level: Median baseline was 9.5 g/dL
    • 59% of patients were anemic (Hgb <10 g/dL) in the VONJO arm (vs 57% in the BAT arm)3
    • 23% of patients were RBC transfusion dependent at study entry
Pertinent medical history
  • Ruxolitinib history: Patients in both the VONJO arm (46%) and BAT arm (51%) had prior ruxolitinib therapy
  • In the VONJO arm, 43% of patients had ≥2 prior therapies (vs 48% in the BAT arm)3
  • Disease history: 68% of patients had primary MF, 20% had PPV MF, and 12% had PET MF
  • Baseline median spleen length was 14 cm
  • *The 400 mg once daily dose could not be established to be safe, so further information on this arm is not provided.
  • The efficacy population included patients who received VONJO 200 mg BID (n=31) or BAT (n=32), and had baseline platelet counts <50 × 109/L.
  • Limitation: No conclusions regarding the benefits or risks of VONJO can be established based on the TSS data from PERSIST-2. These data are not included in the VONJO Prescribing Information.

Best available therapy arm in the efficacy population1

BAT included any physician-selected treatment (including JAK2 inhibitors, such as ruxolitinib) and could have included watch-and-wait or symptom-directed treatment without MF-specific treatment.§

  • In the BAT arm, most patients on ruxolitinib were started on 5 mg BID2
  • Patients could cross over to VONJO after 6 months on BAT (or sooner if needed due to disease progression)2
  • At the time of crossover, those patients discontinued all BAT therapies, including erythropoietic agents4
  • VONJO was initiated within 1 week after discontinuing previous MF therapy, with no washout period4

Most Common BAT Agents Used in the BAT Treatment Arm (n=32) in Patients With Platelet Counts ≤50 × 109/L

Best available therapy in PERSIST-2 study included ruxolitinib, watch and wait, and hydroxyurea
  • §BAT agents could be used alone, in combinations, sequentially, and intermittently, as clinically indicated by standards of care. BAT may have included ruxolitinib, hydroxyurea, glucocorticoids, erythropoietic agents, immunomodulatory agents, mercaptopurine, danazol, interferons, cytarabine, and melphalan.
  • BAT=best available therapy; BID=twice daily; JAK2=janus kinase 2; QD=once daily; RBC=red blood cell.
  • References: 1. VONJO. Prescribing information. CTI BioPharma Corp.; 2023. 2. Mascarenhas J, et al. JAMA Oncol. 2018;4(5):652-659. 3. Mascarenhas J, et al. JAMA Oncol. 2018. Accessed September 14, 2023. PMC5885169/bin/jamaoncol-e175818-s001.pdf 4. CTI BioPharma Corp. PERSIST-2 Protocol. 2013. Accessed September 14, 2023.