NEW YORK, Oct. 17, 2018 (GLOBE NEWSWIRE) — Kadmon Holdings, Inc. (NYSE: KDMN) today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to KD025, the company’s ROCK2 inhibitor, for the treatment of patients with chronic graft-versus-host disease (cGVHD) after failure of two or more lines of systemic therapy. Kadmon is currently enrolling patients in a pivotal Phase 2 clinical trial of KD025 in patients with cGVHD.
The FDA selectively grants Breakthrough Therapy Designation to expedite the development and review of a drug that is intended to treat a serious condition and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over existing therapies.
“We are pleased that the FDA has recognized the therapeutic potential of KD025 for cGVHD, a serious condition for which new treatments are urgently needed,” said Harlan W. Waksal, M.D., President and CEO at Kadmon. “This designation is a key regulatory milestone and we look forward to working closely with the FDA to expedite the development of KD025 for cGVHD.”
The Breakthrough Therapy Designation of KD025 in cGVHD is supported by data from an ongoing Phase 2 clinical trial (KD025-208). In the study, KD025 was well tolerated and demonstrated clinical activity in approximately two-thirds of patients across Cohort 1 (KD025 200 mg QD) and Cohort 2 (KD025 200 mg BID). Preliminary results from the trial were previously presented at the 23rd Congress of the European Hematology Association (EHA) in June 2018 and at the Blood and Marrow Transplantation (BMT) Tandem Meetings in February 2018.
Enrollment is ongoing in KD025-213, the pivotal Phase 2 clinical trial of KD025 in adults with cGVHD who have received at least two prior lines of systemic therapy. KD025-213 is an open-label trial in which patients are randomized to receive either KD025 200 mg QD or 200 mg BID, enrolling 63 patients per arm. Either KD025 dose may be considered by the FDA for the registrational dose.
KD025 is a selective oral inhibitor of Rho-associated coiled-coil kinase 2 (ROCK2), a signaling pathway that modulates inflammatory response. In October 2017, the FDA granted orphan drug designation to KD025 for the treatment of cGVHD.
cGVHD is a common and often fatal complication following hematopoietic stem cell transplantation. 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 GI tract.
About Kadmon Holdings, Inc.
Kadmon Holdings, Inc. is a fully integrated biopharmaceutical company developing innovative product candidates for significant unmet medical needs. Our product pipeline is focused on inflammatory and fibrotic diseases.
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