NEW YORK--(BUSINESS WIRE)--Kadmon Holdings, Inc. (NYSE: KDMN) today announced updated efficacy and safety results from an ongoing Phase 2 clinical trial evaluating KD025 in patients with steroid-dependent or refractory chronic graft-versus-host disease (cGVHD). Results from the study will be presented in an oral session on Saturday, June 16th, 2018 at the 23rd Congress of the European Hematology Association (EHA) in Stockholm, Sweden. The presentation is now available on the Kadmon website.
The updated study results include four additional months of patient follow-up, through May 2, 2018. The Overall Response Rate (ORR) continues to be 65% (11/17) in Cohort 1 (KD025 200 mg QD; n=17) and 69% (11/16) in Cohort 2 (KD025 200 mg BID; n=16). Responses have been durable, with 47% (8/17) of patients in Cohort 1 having sustained a response for at least 20 weeks, up from 41% (7/17) in the last reported analysis. Initial durability data from Cohort 2 demonstrated that 38% (6/16) of patients have sustained a response for at least 20 weeks, which is comparable to durability data observed in Cohort 1 at the same median treatment duration. Durability data continue to mature across Cohorts 1 and 2. Sixty-six percent (66%; 22/33) of all patients were able to reduce doses of corticosteroids and 15% (5/33) have completely discontinued steroid use. In addition, 55% (18/33) of patients across Cohorts 1 and 2 saw a clinically meaningful improvement in cGVHD symptoms, as measured by at least a 7-point decrease in the Lee Symptom Scale score. KD025 continued to be well tolerated, with no treatment-related serious adverse events and no apparent increased risk of infection.
“KD025 continues to achieve robust and durable responses across multiple organs affected by cGVHD, while simultaneously allowing the majority of patients to taper doses of steroids,” said Amandeep Salhotra, M.D., Assistant Professor, Hematology and Hematopoietic Cell Transplantation, City of Hope, Duarte, CA, and study investigator. “In addition, the favorable tolerability profile of KD025 has allowed patients to continue on treatment and maintain responses, highlighting its potential for long-term use as a backbone of cGVHD therapy.”
Kadmon plans to initiate an open-label, pivotal study of KD025 in cGVHD in Q3 2018, based on FDA guidance received in a Type C meeting. The planned pivotal study (KD025-213) will enroll adults who have received at least two prior lines of systemic therapy for cGVHD. Patients will be randomized to receive KD025 200 mg QD or 200 mg BID (63 patients per cohort), and either dose may be considered by the FDA for the registrational dose. The primary endpoint is ORR, supported by a key secondary endpoint of Duration of Response.
“These study results continue to show accumulation of clinical benefit and underscore the significant potential of KD025 in cGVHD, particularly in patients with multiple organs involved,” said Harlan W. Waksal, M.D., President and CEO at Kadmon. “We have a clear regulatory path forward for KD025 and we look forward to initiating our pivotal study in the third quarter of this year.”
KD025-208 is an ongoing Phase 2 clinical trial of KD025, Kadmon’s Rho-associated coiled-coil kinase 2 (ROCK2) inhibitor, in adults with steroid-dependent or steroid-refractory cGVHD and active disease. The dose-finding trial includes 48 patients divided into three cohorts at different dose levels (KD025 200 mg QD, 200 mg BID and 400 mg QD), enrolled sequentially following a safety assessment of each cohort. In October 2017, KD025 received orphan drug designation from the U.S. Food and Drug Administration for cGVHD.
cGVHD is a potentially life-threatening condition following hematopoietic stem cell transplantation. With 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 products for significant unmet medical needs. Our product pipeline is focused on inflammatory and fibrotic diseases.
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