orBec® GVHD Prevention
Acute GI GVHD is a debilitating and painful disease and represents an unmet medical need. It is a common disorder among immunocompromised cancer patients after receiving hematopoietic cell transplantation. Unlike organ transplants where the patient's body may reject the organ, in GVHD it is the donor cells that begin to attack the patient's (host's) body - most frequently the gastrointestinal tract, liver and skin. Patients with mild-to-moderate GI GVHD typically develop symptoms of anorexia, nausea, vomiting and diarrhea. If left untreated, GI GVHD can progress to ulcerations in the lining of the GI tract, and in its most severe form, can be fatal.
orBec® was previously evaluated in a randomized, double-blind, placebo-controlled Phase 2 clinical trial for the prevention of acute GVHD after allogeneic hematopoietic cell transplantation (HCT) with myeloablative conditioning regimens. The trial was conducted by Paul Martin, MD, at the Fred Hutchinson Cancer Research Center in Seattle, WA and supported, in large part, by a National Institutes of Health (NIH) grant. Patients were started on study drug at the start of the conditioning regimen and continue through day 75 following HCT.
The results of this study indicate that orBec® is safe and well tolerated in this patient population, but did not achieve statistical significance in the primary endpoint, which was the proportion of subjects who developed acute GVHD with severity sufficient to require systemic immunosuppressive treatment on or before day 90 after transplantation. However, an encouraging result was that use of orBec® resulted in fewer cases of more severe acute GVHD grades IIb-IV (21% vs. 33% of patients receiving placebo), although this difference was not statistically significant. This result has the potential to be clinically relevant because GVHD grades IIb-IV are associated with more severe disease involving the skin and liver as well as being associated with poorer outcomes, including mortality rates that approach 100% in the grade IV patient population. Further analysis of the complete dataset continues and is aimed at identifying other potential effects seen with orBec® in preventing acute GVHD.
In addition to issued patents and pending worldwide patent applications held by or exclusively licensed to Soligenix, orBec® also benefits from orphan drug designations in the US for the prevention of GI GVHD, which provide for seven years of post-approval market exclusivity.
