OCU300 is in late-stage clinical development as a novel treatment for the debilitating immune condition called ocular graft versus host disease (oGVHD), which develops in many patients following an allogeneic bone marrow transplant. It consists of an improved ophthalmic nano-emulsion of brimonidine tartrate, an FDA-approved drug with established safety for ocular use, enabling Ocugen to develop OCU300 under the accelerated 505(b)(2) regulatory pathway. The proprietary nanoemulsion technology is designed to enhance efficacy by prolonging retention of this potent anti-inflammatory drug on the eye surface, and unlike other ophthalmic emulsion products, it also allows OCU300 to be sterile filtered, single-use vials, without the need for added preservatives.
Currently there are no FDA-approved products for the prevention or treatment of oGVHD, though dry eye treatments are sometimes used to manage certain symptoms. Given its properties to disrupt leukocyte extravasation to the ocular tissue, inhibit nociception, inhibit inflammatory response and mediate vasoconstriction, brimonidine can result in amelioration of clinical presentation of oGVHD, including reduction of ocular redness, reduction in ocular pain and improved patient satisfaction. In multiple investigator-led studies, brimonidine improved symptoms and signs in oGVHD patients. The scientific rationale for use and encouraging clinical experience led to OCU300 becoming the first and only program to receive Orphan Drug Designation from the FDA for this indication, which further emphasizes the unmet medical need for an effective therapeutic for oGVHD.
Ocugen is planning to confirm the efficacy of OCU300 in a controlled prospective Phase 3 study using objective end points, which the company expects to initiate in mid-2018.
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 bone marrow transplants. Driven by 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.
The addressable market for oGVHD treatments is expected to continue growing, as the survival rate for bone marrow transplant continues to increase. A recent multi-center study+ projected 105,000 allogeneic transplant survivors to be living in the U.S. by 2020, of whom up to 63,000 may develop oGVHD.
+Prevalence of Hematopoietic Cell Transplant Survivors in the United States. (Majhail, et al).