Sanofi Announces Summary Data from Clinical Trial of New Transplant Drug Rezurock

Sanofi today announced pooled data from a clinical trial of Rezurock (belumosudil) in chronic graft-versus-host disease (cGVHD)
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Results showed clinical remission in some organs with clinically meaningful changes (improvements) in patient-reported outcomes (PROs)
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Rezurock was developed by Kadmon Holdings and was approved by the US FDA in July 2021
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In September 2021, Sanofi acquired Kadmon for $1.
9 billion, and Rezurock acquired it
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Notably, Rezurock is the first and only ROCK2 small molecule inhibitor approved by the U.
S.
FDA for the treatment of pediatric patients (age ≥12 years) and adults with cGVHD who have failed at least 2 prior systemic therapies patients
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cGVHD is a possible complication after allogeneic stem cell transplantation, and the morbidity and mortality are not optimistic
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In cGVHD, transplanted immune cells (the graft) attack the patient's cells (the host), causing inflammation and fibrosis in multiple tissues including the skin, mouth, eyes, joints, liver, lungs, esophagus, and gastrointestinal tract
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It is estimated that there are approximately 14,000 patients with cGVHD in the United States
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Pooled analyses from the ROCKstar (KD025-213) and KD025-028 studies show a very strong, but not generalized, correlation between organ clinical response measures and clinically meaningful changes in PRO in patients treated with Rezurock
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Specifically, clinically meaningful changes in PRO were reported in skin, oral cavity, eye, upper gastrointestinal tract, lung (clinical spirometry score), and overall measures
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These data support the use of PRO for remission assessment in cGVHD clinical trials and patient care to help capture patient feelings
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A pooled analysis of clinical and PRO data included 170 (91.
3%) patients enrolled in 2 Rezurock studies (n=132, KD025-213; n=54, KD025-208) who had a baseline PRO assessment, at least One follow-up PRO, at least one response assessment
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Median age was 54.
5 years, 98 (58%) were male, median 3 prior treatments, 70% had severe cGVHD, median follow-up at data cutoff was 15 months (range 0.
6- 44 months)
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Organ remission was defined as complete remission (CR) and partial remission (PR)
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The pooled analysis showed organ remission rates: 35% (48/135) in the skin, 68% (84/124) in the joints, 57% (56/99) in the oral cavity, 41% (52/128) in the eye, and 73% in the upper GI tract (19/26), lower gastrointestinal 87% (13/15), pulmonary 23% (14/61); overall response rate was 77% (130/170)
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At least one PRO in all organs (except joints, esophagus, and lower GI tract) was statistically associated with National Institutes of Health (NIH) organ response, p<0.
05
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NIH clinical pulmonary remission, but not pulmonary remission based on FEV1% change, was associated with pulmonary PRO
.