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ROCKstar STUDY DESIGN ROCKstar EFFICACY LONG-TERM ANALYSIS DESIGN LONG-TERM ANALYSIS DATA

ORRb FOLLOWING TREATMENT WITH REZUROCK 200 mg ONCE DAILY

  • Bar graph showing Best ORR through cycle 7 day 11 of 200 mg once daily (n=65c) with response rate % as x-axis. Text overlay states 75% ORR (95% CI, 63-85), PRIMARY END POINT.

    CR, n=4 (6%). PR, n=45 (69%).1

     

    aLong-term follow-up period is defined as 3 years.3

    bBest ORR was defined as the proportion of patients who achieved CR or PR according to the 2014 NIH cGVHD Consensus Criteria.4

    cBased on a final analysis by the FDA as seen through cycle 7 day 1 (n=65).1

    dBased on mITT population (n=77).2

  • Bar graph showing the Best ORR at any time2 of 200 mg once daily (n=77d) with response rate % as x-axis. Text overlay states 74% ORR (95% CI, 63-83), PRIMARY END POINT.

    CR, n=4 (5%). PR, n=53 (69%).3

     

    aLong-term follow-up period is defined as 3 years.3

    bBest ORR was defined as the proportion of patients who achieved CR or PR according to the 2014 NIH cGVHD Consensus Criteria.4

    cBased on a final analysis by the FDA as seen through cycle 7 day 1 (n=65).1

    dBased on mITT population (n=77).2

     

    Long-term analysisa study limitations

    This is an open-label follow-upa analysis of the mlTT population.2,3 Data presented are descriptive in nature. No conclusions of statistical significance or clinical significance can be drawn. Clinical data were analyzed based on all available data, including the potential continued involvement of responders and attrition of nonresponders. All patients received REZUROCK. Therefore, patients served as their own baseline control. This analysis includes data from an additional 20 patients who were enrolled in a subsequent biomarker cohort after the August 19, 2020, ROCKstar publication data cutoff, and therefore comprises 152 patients, including the addition of 11 patients in the once-daily arm and 9 patients in the twice-daily arm. The addition of these patients may impact the data due to their limited time on therapy.2-4 aLong-term follow-up period is defined as 3 years.3

RESPONSES BY ORGAN SYSTEM

  • Bar graph showing responses (%) of complete response (CR) and partial response (PR) by organ system with REZUROCK 200 mg once daily in the mITT population (n=66): Lower GI (n/N=4/6; 67%), 67% CR; Esophagus (n/N=9/19; 47%), 47% CR; Upper GI (n/N=8/13; 62%), 54% CR and 8% PR; Mouth (n/N=16/30; 53%), 50% CR and 3% PR; Joints/Fascia (n/N=38/51; 75%), 20% CR and 55% PR; Liver (n/N=3/9; 33%), 22% CR and 11% PR; Skin (n/N=18/55; 33%), 15% CR and 18% PR; Eyes (n/N=16/48; 33%), 17% CR and 16% PR; Lungs (n/N=7/24; 29%), 17% CR and 12% PR.

    aLong-term follow-up period is defined as 3 years.3

    ePercentages may not add up to the total due to rounding.

  • Bar graph showing responses (%) of complete response (CR) and partial response (PR) by organ system with REZUROCK 200 mg once daily in the mITT population (n=77): Lower GI (n/N=5/7; 71%), 71% CR; Esophagus (n/N=13/23; 57%), 57% CR; Upper GI (n/N=9/14; 64%), 57% CR and 7% PR; Mouth (n/N=23/41; 56%), 49% CR and 7% PR; Joints/Fascia (n/N=42/59; 71%), 19% CR and 53% PR; Liver (n/N=2/10; 20%), 10% CR and 10% PR; Skin (n/N=20/63; 32%), 14% CR and 18% PR; Eyes (n/N=24/56; 43%), 16% CR and 27% PR; Lungs (n/N=9/27; 33%), 19% CR and 15% PR.

    Data presented are descriptive in nature. No conclusions of statistical significance or clinical significance can be drawn.

     

    aLong-term follow-up period is defined as 3 years.3

    ePercentages may not add up to the total due to rounding.

MEDIAN TTNTf,g WITH REZUROCK 200 mg ONCE DAILY IN THE mITT POPULATION

  • Bar graph of 200 mg once daily (n=66) with 0 to 26 months on x-axis. Text overlay states 16.6 months (95% CI, 13-NA).

    aLong-term follow-up period is defined as 3 years.3

    fTTNT was defined as the time from the first dose of REZUROCK to the start of additional systemic cGVHD therapy.3

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

  • Bar graph showing the dose of REZUROCK 200 mg once daily (n=77) over 0 to 26 months with text overlay stating 24.2 months (95% CI, 13-NA).

    Data presented are descriptive in nature. No conclusions of statistical significance or clinical significance can be drawn.

     

    aLong-term follow-up period is defined as 3 years.3

    fTTNT was defined as the time from the first dose of REZUROCK to the start of additional systemic cGVHD therapy.3

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

FFSh WITH REZUROCK 200 mg ONCE DAILY IN THE mITT POPULATION

  • Kaplan-Meier graph showing the FFS rate was 73% at 6 months and 57% at 12 months. Number at risk starts with 66 at month 0 and decreases to 1 by month 24.


    Kaplan-Meier curve of estimated FFS.3

    aLong-term follow-up period is defined as 3 years.3

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

    hFFS was defined as the absence of relapse, nonrelapse mortality or a need for additional systemic therapy.4

  • Kaplan-Meier graph showing the FFS rate was 42% at 36 months. Number at risk starts with 77 at month 0 and decreases to 1 by month 42.

    Data presented are descriptive in nature. No conclusions of statistical significance or clinical significance can be drawn.


    Kaplan-Meier curve of estimated FFS.3

    aLong-term follow-up period is defined as 3 years.3

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

    hFFS was defined as the absence of relapse, nonrelapse mortality or a need for additional systemic therapy.4

OSi WITH REZUROCK 200 mg ONCE DAILY IN THE mITT POPULATION

  • Kaplan-Meier graph showing the 2-year OS rate was 87%. Number at risk starts with 66 at month 0 and decreases to 0 by month 24.

    Kaplan-Meier curve of estimated OS.3

    aLong-term follow-up period is defined as 3 years.3

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

    iOS was defined as the time from the first dose of REZUROCK to the date of death due to any cause.3

  • Kaplan-Meier graph showing the 2-year OS rate was 86%. Number at risk starts with 77 at month 0 and decreases to 0 by month 42.

    Data presented are descriptive in nature. No conclusions of statistical significance or clinical significance can be drawn.


    In the long-term follow-up analysis, 3-year OS data were not available. The 2-year OS includes 11 additional patients from a subsequent biomarker cohort after the August 19, 2020, ROCKstar publication data cutoff.3

     

    Kaplan-Meier curve of estimated OS.3

    aLong-term follow-up period is defined as 3 years.3

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

    iOS was defined as the time from the first dose of REZUROCK to the date of death due to any cause.3

     

     

     

CHANGE IN QOL SCORES

  • REZUROCK 200 mg ONCE DAILY IN AN
    EXPLORATORY ANALYSIS

    Blue icon that states 52% of patientsc reported a greater than or equal to 7-point reduction in LSSj summary score (n/N=34/65).

    aLong-term follow-up period is defined as 3 years.3

    cBased on a final analysis by the FDA as seen through cycle 7 day 1 (n=65).1

    dBased on the mITT population (n=77).2

    jThe LSS is a 30-item, 7-subscale symptom scale and QOL measurement tool that evaluates the AEs of cGVHD in the categories of skin, vitality, lung, nutritional status, psychological functioning, eye and mouth.5

  • REZUROCK 200 mg ONCE DAILY IN AN
    EXPLORATORY ANALYSIS

    Blue icon that states 58% of patientsd reported a greater than or equal to 7-point reduction in LSSj summary score (n/N=45/77).

    Data presented are descriptive in nature. No conclusions of statistical significance or clinical significance can be drawn.

     

    aLong-term follow-up period is defined as 3 years.3

    cBased on a final analysis by the FDA as seen through cycle 7 day 1 (n=65).1

    dBased on the mITT population (n=77).2

    jThe LSS is a 30-item, 7-subscale symptom scale and QOL measurement tool that evaluates the AEs of cGVHD in the categories of skin, vitality, lung, nutritional status, psychological functioning, eye and mouth.5

CHANGE IN CS AND CNI DOSE IN THE 200-mg ONCE-DAILY ARM

  • Patients who received CS therapy4

    Blue icon that states doses were reduced in 64% of patients (n/N=42/66).
    Blue icon that states treatment was discontinued in 20% of patients (n/N=13/66).

    Patients who received CNI therapy3

    Blue icon that states doses were reduced in 42% of patients (n/N=10/24).
    Blue icon that states treatment was discontinued in 17% of patients (n/N=4/24).

    aLong-term follow-up period is defined as 3 years.3

    fBased on mITT population (n=77).2

    mBased on mITT population (n=66).4

  • Patients who received CS therapy3

    Blue icon that states doses were reduced in 65% of patients (n/N=50/77).
    Blue icon that states treatment was discontinued in 27% of patients (n/N=21/77).

    Patients who received CNI therapy3

    Blue icon that states doses were reduced in 47% of patients (n/N=15/32).
    Blue icon that states treatment was discontinued in 22% of patients (n/N=7/32).

    Data presented are descriptive in nature. No conclusions of statistical significance or clinical significance can be drawn.

    aLong-term follow-up period is defined as 3 years.3

    dBased on mITT population (n=77).2
    kBased on mITT population (n=66).4

LEARN ABOUT THE SAFETY PROFILE FOR REZUROCK.

ROCKstar AND KD025-208 SAFETY
LONG-TERM ANALYSIS SAFETY

AE, adverse event; cGVHD, chronic graft-versus-host disease; CNI, calcineurin inhibitor; CR, complete response; CS, corticosteroid; FDA, US Food and Drug Administration; FFS, failure-free survival; GI, gastrointestinal; LSS, Lee Symptom Scale; mITT, modified intent-to-treat; MOA, mechanism of action; NA, not available; NIH, National Institutes of Health; ORR, overall response rate; OS, overall survival; PR, partial response; QOL, quality of life; TTNT, time to next treatment.

References: 1. REZUROCK. Package insert. Kadmon Pharmaceuticals, LLC. 2. Lee SJ, Cutler C, Pavletic S, Blazar BR, Yao Y, Ji R; on behalf of the ROCKstar Study Investigators. Belumosudil for chronic graft-versus-host disease after 2 or more lines of systemic therapy: 3-year follow-up to the ROCKstar study. Poster presented at: Tandem Meetings; February 21-24, 2024; San Antonio, TX. 3. Data on file. Sanofi. 4. 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 5. Lee SJ, Cook EF, Soiffer R, Antin JH. Development and validation of a scale to measure symptoms of chronic graft-versus-host disease. Biol Blood Marrow Transplant. 2002;8(8):444-452. doi:10.1053/bbmt.2002.v8.pm12234170

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