LGBT Cancer Project

Graft-Versus-Host Disease (GVHD)

February 23, 2015

Mesoblast reported results of a phase III study of MSC-100-IV for severe steroid-resistant acute graft versus host disease (aGVHD). The pediatric trial enrolled 160 children. Overall response (defined as partial + complete response) at day +28 was 64%, and this response correlated with statistically significant improved survival compared to non-responders at day +100 after an MSC-100-IV infusion (81% v. 39%, p=0.0001). The day 28 overall responses by grade were 74% for grade B, 66% for grade C and 59% for grade D. Overall responses by organ involvement at day +28 were 62% for GI, 77% for skin and 53% for liver. Approximately 80% of grade B/C patients and over 50% of grade D patients survived to day 100. A single-arm, open-label, phase III registration trial to support product approval by the FDA, which will include approximately 60 pediatric patients with aGVHD, is in progress.

August 11, 2014

Quark Pharmaceuticals released results of a phase II trial of QPI-1002 for the prophylaxis of delayed graft function (DGF) in deceased donor kidney transplant patients. The multi-center, placebo-controlled, randomized, prospective and double-blinded study evaluated the clinical activity of QPI-1002 (administered as 10mg/kg single bolus IV dose at 30 minutes after circulatory reperfusion is achieved to the transplanted organ) in end-stage kidney disease dialysis-dependent patients undergoing deceased donor kidney transplantation. QPI-1002 treatment significantly increased the dialysis-free survival (time to first dialysis) in the first post-transplant month (Log rank p=0.04), reduced the mean duration of the first course of dialysis (13.4 v. 25.3 days) and reduced the number of dialysis sessions required in the first 30 days post-transplant (6.0 v. 11.2). By the end of the six-month study observation period, the total number of dialysis sessions in all efficacy evaluable patients treated with QPI-1002 was 1.5-fold lower compared to placebo group (375 v.. 561 p=0.059).

November 20, 2006

Osiris announced positive results from a phase II trial of Prochymal for the treatment of Graft vs. Host disease (GVHD). This trial enrolled 32 subjects who were divided into two groups. In addition to standard of care, subjects received two infusions of Prochymal, a low dose (2 million cells per kilogram) or a high dose (8 million cells per kilogram) three days apart, at the onset of moderate to severe (grades II-IV) GVHD. Treatment was well tolerated at both dose levels and through repeated dosing. No serious adverse events were reported. Complete response was seen in 74% of the subjects and the overall response rate was 94%. In the sub-population of subjects with gastrointestinal GVHD, 67% had a complete response and the overall response rate was 89%. In the sub-population of the subjects with the dermatological form of GVHD, 85% had a complete response and the overall response rate was 100%. Osiris is currently evaluating Prochymal in a phase III trial for this indication.

January 10, 2005

DOR BioPharma reported top-line results from a phase III trial of orBec (oral beclomethasone dipropionate), for the treatment of intestinal Graft-versus-Host Disease (iGVHD). The results indicate that orBec failed to meet its primary endpoint, significant improvement in time to treatment failure through day 50 vs. placebo (p=0.1177), nor was the treatment failure rate at 50 days statistically significant (p=0.0515). The drug did achieve significance in several secondary endpoints, including time to treatment failure at day 80 (p=0.0226), treatment failure rate at day 80 (0.0048), and mortality rate 200 days post transplant (p=0.006). The randomized, double-blind, placebo-controlled, multi-center clinical trial enrolled 129 post-bone marrow transplant patients, who received high-dose prednisone plus orBec or placebo for 10 days. If initial response was noted after 10 days, prednisone was rapidly tapered and subjects continued to receive orBec or placebo through day 50.

June 9, 2003

Isotechnika reported positive results from a phase IIa trial investigating ISA247, an immunosuppressive for the use in kidney transplants. Results showed that all of the primary and secondary endpoints of the study were achieved. Data demonstrated that stable kidney transplant subjects on ISA247 experienced no change in kidney function when compared to subjects taking cyclosporine. In addition, data also demonstrated that ISA247 achieved a level of immunosuppression comparable to cyclosporine at one-third the blood drug concentration. One of the secondary endpoints, the incidence of graft rejection, was designed to monitor safety parameters in kidney transplant patients receiving ISA247. The open label, randomized, multi-center study enrolled 132 subjects at twenty sites in Canada and the United States.

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