Sanofi Announces Pooled Clinical Trial Data of Rezurock (Belumosudil) Demonstrating Strong Correlation Between Organ-Specific Responses and Patient-Reported Outcomes in Chronic Graft-versus-Host Disease

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Recently, Sanofi announced the pooled analysis data from clinical trials of Rezurock (belumosudil) for the treatment of chronic graft-versus-host disease (cGVHD). The results showed that clinical responses in certain organs were associated with clinically meaningful changes (improvements) in patient-reported outcomes (PROs).

Rezurock, developed by Kadmon Holdings, was approved by the U.S. FDA in July 2021. In September 2021, Sanofi acquired Kadmon for $1.9 billion, bringing Rezurock under its ownership. Notably, Rezurock is the first and only FDA-approved ROCK2 small molecule inhibitor in the United States, indicated for the treatment of pediatric (aged ≥12 years) and adult patients with cGVHD who have failed at least two prior lines of systemic therapy.

cGVHD is a complication that may occur after allogeneic stem cell transplantation, with incidence and mortality rates being far from optimistic. In cGVHD, the transplanted immune cells (graft) attack the patient’s cells (host), leading to inflammation and fibrosis in multiple tissues including the skin, mouth, eyes, joints, liver, lungs, esophagus, and gastrointestinal tract. It is estimated that there are approximately 14,000 cGVHD patients in the United States.

Pooled analyses from the ROCKstar (KD025-213) and KD025-028 studies show a very strong but not universal correlation between organ clinical response measures and clinically meaningful changes in PROs in patients treated with Rezurock. Specifically, clinically meaningful changes in PROs were reported for skin, mouth, eyes, upper gastrointestinal tract, lungs (Clinical Pulmonary Scale score), and overall measurements. These data support the use of PROs for response assessment in cGVHD clinical trials and patient care to help capture patient experiences.

The pooled analysis of clinical and PRO data included 170 (91.3%) patients who participated in two Rezurock studies (n=132, KD025-213 study; n=54, KD025-208 study) and had baseline PRO assessments, at least one follow-up PRO, and at least one response assessment. The median age was 54.5 years, 98 (58%) were male, the median number of prior treatments was 3, 70% had severe cGVHD, and the median follow-up time at data cutoff was 15 months (range: 0.6–44 months).

Organ response is defined as complete response (CR) and partial response (PR). Pooled analysis showed organ response rates as follows: skin 35% (48/135), joints 68% (84/124), oral cavity 57% (56/99), eyes 41% (52/128), upper gastrointestinal tract 73% (19/26), lower gastrointestinal tract 87% (13/15), lungs 23% (14/61); the overall response rate was 77% (130/170). At least one patient-reported outcome (PRO) in all organs (except joints, esophagus, and lower gastrointestinal tract) was statistically correlated with the NIH organ response, p<0.05. NIH clinical lung response, but not lung response based on changes in FEV1%, was associated with lung PRO.

Reference Source: Media Update: Rezurock® (belumosudil) patient-reported outcomes correlated with clinical response in chronic graft-versus-host disease