OCU300 as a Treatment for Ocular Graft Versus Host Disease
OCU300 is a re-purposed drug with an established safety record in ocular indications being developed under the U.S. Food and Drug Agency’s 505(b)(2) regulatory pathway. In an investigator-led exploratory observational study, OCU300 was found to improve both signs and symptoms in a majority of patients. We plan to confirm these findings in a controlled prospective study using objective end points.
About ocular Graft Versus Host Disease (oGVHD)
Ocular GVHD (oGVHD) is a common complication that occurs in 40-60% of patients who have undergone allogeneic hematological stem cell transplantation (allo-SCT), or bone marrow transplants. Driven by autoimmune inflammation, oGVHD can result in damage to the ocular surface and tear producing glands which over time significantly diminishes quality of life and restricts daily activities due to visual impairment. Common clinical manifestations include keratoconjunctivitis sicca and cicatricial conjunctivitis which combine to leave patients with dry, tearless eyes, vision issues, and pain. Patients may go on to develop eyelid inflammation and ocular scarring, and other forms of corneal and ocular surface disease.
Allogeneic hematological stem cell transplantation survivors continue to increase, and a recent multi-center study+ projected 234,000 allo-SCT survivors in the US by 2030. By 2020, oGVHD is estimated to impact approximately 63,000 patients in the US. Currently there are no approved pharmaceutical therapeutics for the prevention and/or treatment of oGVHD.
+Prevalence of Hematopoietic Cell Transplant Survivors in the United States. (Majhail, et al).