Ocugen Inc., an early stage biopharmaceutical company based in Pennsylvania, has announced the licensing of a clinical stage technology for the treatment of ocular graft versus host disease (oGVHD). The company describe the compound, OCU300, as a repurposed drug with a safety track record in ocular applications. In previous exploratory studies, OCU300 was shown to improve symptoms in almost 90% of patients with ocular graft versus host disease, for which there is currently no treatment indicated.
The company is already in the process of developing two other experimental treatments for ocular indications – OCU100 and OCU200. OCU100 is a recombinant protein based on lens epithelial derived growth factor, shown to rescue photoreceptors in models of retinal degeneration and with potential application to both retinitis pigmentosa (RP) and geographic atrophy (GA). OCU200 is a fusion protein based on tumstatin and transferrin which has demonstrated superior performance in head-to-head studies against anti-VEGF treatment of neovascular disease. The most recent licensing acquisition of OCU300 now develops a further ocular indication for the company. Ocular graft versus host disease (oGVHD) is a complication in some allogenic bone marrow transplant pricedures where the donor’s leukocytes attack the recipient’s immuno-compromised sysytem, most commonly affecting the ocular surface. According to Ocugen, ocular GVHD develops in approximately 40-60% of allogenic transplant patients and manifests through a number of common clinical manifestations, including keratoconjunctivitis sicca and cicatricial conjunctivitis. The company is aiming to establish a dose-finding study for OCU300 with further plans for a Phase III clinical trial in 2017. In addition, it is understood that OCU300 is being developed through the FDA’s 505(b)(2) pathway. Ocugen announced the execution of the exclusive worldwide license agreement to OCU3000 with the University of Illinois at Chicago (UIC).
Commenting on the commercial milestone Dr. Shankar Musunuri, Chairman and CEO of Ocugen stated that, “Patients who develop oGVHD after receiving a bone marrow transplant suffer from significantly irritating and discomforting symptoms of severe dry eye, which can cause long-term vision problems. With OCU300, we have a clear and rapid development path forward, and we hope to bring these patients a new treatment option that has the potential to provide significant relief.”