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
  • 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)

Study design1,2

ROCKstar was a pivotal phase 2, open-label, randomized, multicenter study that evaluated the efficacy and safety of REZUROCK in patients with cGVHD.

aThe 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.

bProportion of patients who achieved CR or PR according to the 2014 NIH cGVHD Consensus Criteria.1

cPrespecified secondary end point; not powered to show statistical significance.

Select baseline characteristics2

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.

baseline characteristics of patients, including median prior lines of systemic therapy, organ involvement, previous acute GVHD (aGVHD) and other characteristics
baseline characteristics of patients, including median prior lines of systemic therapy, organ involvement, previous acute GVHD (aGVHD) and other characteristics

aThe 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.

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

horizontal bar graph of REZUROCK 200-mg once-daily treatment arm, REZUROCK 200-mg twice-a-day (BID) treatment arm and total study participants
horizontal bar graph of REZUROCK 200-mg once-daily treatment arm, REZUROCK 200-mg twice-a-day (BID) treatment arm and total study participants

aThe 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.

chart of organ involvement, including the eyes, skin, mouth, joints/fascia, lungs, upper GI tract, esophagus, lower GI tract and liver
chart of organ involvement, including the eyes, skin, mouth, joints/fascia, lungs, upper GI tract, esophagus, lower GI tract and liver

aThe 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

across a diverse range of patients.1

See the results

aGVHD, acute graft-versus-host disease; BID, twice a day; cGVHD, chronic graft-versus-host disease; CNI, calcineurin inhibitor; CS, corticosteroids; DOR, duration of response; ECP, extracorporeal photopheresis; FFS, failure-free survival; GI, gastrointestinal; HCT, hematopoietic cell transplant; LSS, Lee Symptom Scale; MOA, mechanism of action; NIH, National Institutes of Health; ORR, overall response rate; OS, overall survival; PPI, proton pump inhibitor; QOL, quality of life; TTR, time to response.

References: 1. REZUROCK. Package insert. Kadmon Pharmaceuticals, LLC; 2021. 2. Cutler CS, Lee SJ, Arai S, et al. Belumosudil for chronic graft-versus-host disease (cGVHD) after 2 or more prior lines of therapy: the ROCKstar study. Blood. 2021;blood.2021012021. doi:10.1182/blood.2021012021. 3. Data on file. Kadmon Pharmaceuticals, LLC; 2021.