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Kadmon (KDMN) Announces Pivotal Trial Data Published in the Journal Blood for REZUROCK (Belumosudil) in Chronic Graft-Versus-Host Disease

July 19, 2021 7:07 AM EDT

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Kadmon Holdings, Inc. (NASDAQ: KDMN) today announced that data from the pivotal ROCKstar clinical trial of REZUROCK™ (belumosudil) for the treatment of chronic graft-versus-host disease (cGVHD) were published in the journal Blood. The ROCKstar data served as the basis for the Company's New Drug Application (NDA) for REZUROCK for the treatment of cGVHD, which was granted full approval by the U.S. Food and Drug Administration (FDA) on July 16, 2021 for the treatment of adult and pediatric patients 12 years and older with cGVHD after failure of at least two prior lines of systemic therapy.

"Belumosudil is very effective in inducing responses across all organs affected by cGVHD and is safe and well tolerated. It is exciting to see the development of new interventions that can help patients," said Corey Cutler, MD, MPH, FRCPC, Associate Professor of Medicine, Harvard Medical School; Medical Director, Adult Stem Cell Transplantation Program, Dana-Farber Cancer Institute and ROCKstar study investigator.

"Leveraging ROCK inhibition to address both the immune and fibrotic components of cGVHD is a welcome step forward in the treatment of this debilitating disease."

"The results from ROCKstar showcase the benefit that belumosudil can provide to cGVHD patients, including reducing and eliminating corticosteroid and calcineurin inhibitor doses and achieving improvements in quality of life," said Harlan W. Waksal, M.D., President and CEO of Kadmon. "We are pleased by the recent FDA approval of REZUROCK and look forward to making the drug available to patients in the near term."

As previously reported, data from ROCKstar, the randomized, Phase 2 clinical trial of belumosudil, which enrolled 132 cGVHD patients who had received two to five prior lines of therapy, demonstrated an Overall Response Rates (ORR) of 74% (95% Confidence Interval (CI): 62, 84; p<0.0001) with 200 mg once daily (QD) and 77% (95% CI: 65, 87; p<0.0001) with 200 mg twice daily (BID). Responses were achieved across patient subgroups and complete responses were observed in all organ systems, including in lung. Responses were durable, with a median duration of 54 weeks, which represents the total combined response periods for each patient. Forty-four percent (44%) of responders maintained their response for ≥1 year. In addition, clinically meaningful improvement from baseline in the Lee Symptom Scale (LSS) score, a chronic GVHD symptom measurement, with belumosudil was observed in 61% of patients. During treatment, 65% of patients were able to reduce their corticosteroid dose, with 21% of patients completely discontinuing corticosteroid therapy. In addition, 22% of patients completely discontinued calcineurin inhibitor therapy. Belumosudil was well tolerated and adverse events have been consistent with those expected in patients with advanced cGVHD receiving corticosteroids and/or other immunosuppressants.

The Blood publication of the ROCKstar data is available online here.

About cGVHD

cGVHD is a complication that can occur following allogeneic stem cell transplantation, resulting in significant morbidity and mortality. In cGVHD, transplanted immune cells (graft) attack the patient's cells (host), leading to inflammation and fibrosis in multiple tissues, including skin, mouth, eye, joints, liver, lung, esophagus and gastrointestinal tract. Approximately 14,000 patients in the United States are living with cGVHD.

About REZUROCK™ (belumosudil)

REZUROCK™ (belumosudil) is the first and only approved therapy targeting Rho-associated coiled-coil kinase 2 (ROCK2), a signaling pathway that modulates inflammatory response and fibrotic processes. REZUROCK is approved in the United States for the treatment of adult and pediatric patients 12 years and older with cGVHD after failure of at least two prior lines of systemic therapy. For more information, visit www.REZUROCK.com.

Kadmon is also developing belumosudil for the treatment of systemic sclerosis. The FDA has granted Orphan Drug Designation to belumosudil for the treatment of systemic sclerosis.

INDICATIONS AND USAGE

REZUROCK is a kinase inhibitor indicated for the treatment of adult and pediatric patients 12 years and older with chronic graft-versus-host disease (chronic GVHD) after failure of at least two prior lines of systemic therapy.

SELECT IMPORTANT SAFETY INFORMATION

Warnings and Precautions

Embryo-Fetal Toxicity: Based on findings in animals and its mechanism of action, REZUROCK can cause fetal harm when administered to a pregnant woman. Advise females of reproductive potential and males with female partners of reproductive potential of the potential risk to a fetus and to use effective contraception.

Adverse Reactions

The most common (≥20%) adverse reactions, including laboratory abnormalities, in patients receiving REZUROCK were infections, asthenia, nausea, diarrhea, dyspnea, cough, edema, hemorrhage, abdominal pain, musculoskeletal pain, headache, phosphate decreased, gamma glutamyl transferase increased, lymphocytes decreased, and hypertension.

To report suspected adverse reactions, contact Kadmon at 1-877-377-7862 or the FDA at (800) FDA-1088 or www.fda.gov/medwatch.

Use in Specific Populations

Lactation: Advise not to breastfeed.

Please visit www.REZUROCK.com to see the full Prescribing Information for REZUROCK.



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