REZUROCK (belumosudil) tablets logo

For US Health Care
Professionals Only

THE ROCKstar STUDY

man wearing headphones while playing the guitar
man wearing headphones while playing the guitar

REZUROCK was evaluated in the pivotal ROCKstar (KD025-213) study in a real-world demographic of patients with cGVHD1

The FDA approval of REZUROCK was based on the pivotal ROCKstar study.1

The ROCKstar study included a broad range of patients who were representative of the general cGVHD population.1

These patients had

  • Early-stage cGVHD as well as late-stage disease1,2
  • Diverse ages (≥12 years)1
  • NIH-defined moderate (27%) or severe (70%) cGVHD2
  • A wide range of organ involvement, including fibrotic manifestations1,2
  • A diverse treatment history2
  • Median of 3 prior lines of systemic therapy (30% had received ruxolitinib and 33% had received ibrutinib)

Study design for ROCKstar2

ROCKstar study criteria with REZUROCK treatment arms and end points, including overall response rate (ORR), safety, duration of response (DOR), Lee Symptom Scale (LSS) score, steroid doses, failure-free survival (FFS) and overall survival (OS) ROCKstar study criteria with REZUROCK treatment arms and end points, including overall response rate (ORR), safety, duration of response (DOR), Lee Symptom Scale (LSS) score, steroid doses, failure-free survival (FFS) and overall survival (OS)

ROCKstar was a pivotal phase 2, open-label, randomized, multicenter study that evaluated the efficacy and safety of REZUROCK in patients with cGVHD after receiving 2 to 5 prior lines of systemic therapy.2

Exclusion criteria: Patients were excluded from the study if they had a relapse of their underlying malignancy, had developed posttransplant lymphoproliferative disease, were currently receiving ibrutinib2 or had any of the following laboratory results: platelets were < 50 × 109/L; absolute neutrophil count < 1.5 × 109/L; AST or ALT > 3 × ULN; total bilirubin > 1.5 × ULN; QTc(F) >480 ms; eGFR <30 mL/min/1.73 m2; or FEV1 ≤39%.1

Screening for eligibility was conducted within 14 days of C1D1.2

Certain concurrent immunosuppressive medications were allowed, as drug-drug interactions were not anticipated.2

aThe Karnofsky/Lansky Performance Status Scale is used in outcome-based analyses to determine the functional status of a patient. The Karnofsky Scale is designed for patients aged ≥16 years, whereas the Lansky Scale is designed for patients aged ≤16 years. The scale range for both is 10 to 100.3

bThe final FDA interpretation of the ROCKstar study omitted 1 patient from the REZUROCK 200-mg once-daily arm. As a result, there are minor differences between the ROCKstar publication, where n=66, and the Prescribing Information, where n=65.

cORR was defined as the proportion of patients who achieved CR or PR. All responses were assessed by the study site investigators.1

dPrespecified secondary end points; not powered to show statistical significance.

eDOR was measured from the time of initial PR or CR until documented progression from best response of cGVHD, time from initial response to start of additional systemic cGVHD therapy or death.2

fThe 7-day LSS summary score was calculated based on the developer recommendations and was compared with the score from baseline in an exploratory analysis. An improvement of ≥7 points was considered clinically meaningful.2

gFFS was defined as the absence of relapse, nonrelapse mortality or a need for additional systemic therapy.2

hOS was defined as the time from the first dose of REZUROCK to the date of death due to any cause.4

Select baseline characteristics2,4

The baseline demographics demonstrate the diversity of the patient population in the ROCKstar study. Many patients presented with challenging characteristics, including advanced or complex disease and having received multiple prior lines of systemic therapy.

CharacteristicsREZUROCK 200 mg once daily (n=66)b
Median age, y (range) 53 (21-77)
Male, n (%) 42 (64)
Median prior lines of systemic therapy, n 3
Median time from cGVHD diagnosis to enrollment, mo (range) 25 (2-162)
Median prednisone-equivalent dose at enrollment, mg/kg/d (range) 0.20 (0.03-0.95)
Concomitant PPI use, n (%) 33 (50)
≥4 organs involved, n (%) 33 (50)
Previous aGVHD, n (%) 42 (64)
Refractory to prior line of systemic therapy, n (%i) 44 (79)
NIH-defined cGVHD severity, n (%)
Severe 46 (70)
Moderate 18 (27)
Mild 2 (3)
Prior systemic cGVHD therapy type, n (%)
CS (prednisone) 65 (99)
Tacrolimus 40 (61)
ECP 31 (47)
Sirolimus 29 (44)
Ibrutinib 22 (33)
Ruxolitinib 20 (30)
Cyclosporine 4 (6)
Imatinib 3 (5)

bThe final FDA interpretation of the ROCKstar study omitted 1 patient from the REZUROCK 200-mg once-daily arm. As a result, there are minor differences between the ROCKstar publication, where n=66, and the Prescribing Information, where n=65.

iDenominator excludes patients with unknown status.1

Most patients in the ROCKstar study had moderate or severe cGVHD based on the 2014 NIH cGVHD Consensus Criteria2

horizontal bar graph of cGVHD severity in patients treated with REZUROCK 200 mg once daily
hhorizontal bar graph of cGVHD severity in patients treated with REZUROCK 200 mg once daily

bThe final FDA interpretation of the ROCKstar study omitted 1 patient from the REZUROCK 200-mg once-daily arm. As a result, there are minor differences between the ROCKstar publication, where n=66, and the Prescribing Information, where n=65.

A broad range of organ involvement4

Organ involvementPatients (n=66),b
n (%)
Eyes 48 (73)
Skin 55 (83)
Mouth 30 (46)
Joints/Fascia 51 (77)
Lungs 24 (36)
Upper GI 13 (20)
Esophagus 19 (29)
Lower GI 6 (9)
Liver 9 (14)

bThe final FDA interpretation of the ROCKstar study omitted 1 patient from the REZUROCK 200-mg once-daily arm. As a result, there are minor differences between the ROCKstar publication, where n=66, and the Prescribing Information, where n=65.

Efficacy was achieved

in a real-world demographic of patients.1

See the results

aGVHD, acute graft-versus-host disease; ALT, alanine aminotransferase; AST, aspartate aminotransferase; cGVHD, chronic graft-versus-host disease; C1D1, cycle 1 day 1; CNI, calcineurin inhibitor; CR, complete response; CS, corticosteroid(s); DOR, duration of response; ECP, extracorporeal photopheresis; eGFR, estimated glomerular filtration rate; FDA, US Food and Drug Administration; FEV1, forced expiratory volume in 1 second; FFS, failure-free survival; GI, gastrointestinal; HCT, hematopoietic cell transplant; LFT, liver function test; LSS, Lee Symptom Scale; mITT, modified intent-to-treat; MOA, mechanism of action; NIH, National Institutes of Health; NRM, nonrelapse mortality; ORR, overall response rate; OS, overall survival; PPI, proton pump inhibitor; PR, partial response; QOL, quality of life; TTR, time to response; ULN, upper limit of normal.

References: 1. REZUROCK. Package insert. Kadmon Pharmaceuticals, LLC; 2021. 2. Cutler C, Lee SJ, Arai S, et al; on behalf of the ROCKstar Study Investigators. Belumosudil for chronic graft-versus-host disease after 2 or more prior lines of therapy: the ROCKstar Study. Blood. 2021;138(22):2278-2289. doi:10.1182/blood.2021012021 3. Center for International Blood and Marrow Transplant Research. Karnofsky/Lansky performance status. Accessed April 20, 2022. https://www.cibmtr.org/DataManagement/TrainingReference/Manuals/DataManagement/Documents/appendix-l.pdf 4. Data on file. Kadmon Pharmaceuticals, LLC; 2021.

Please verify that you are a US health care professional

This information is intended for US health care professionals.

I am a US health care
professional.