Ocugen announced the decision to discontinue the Phase 3 trial of OCU300 for ocular Graft vs. Host Disease (oGVHD). The decision to stop the trial is based on results of a pre-planned interim sample size analysis conducted by an independent Data Monitoring Committee, which indicated the trial was unlikely to meet its co-primary endpoints upon completion. The study was not stopped based on safety concerns. Ocugen will analyze the full data when it is available.
The randomized, placebo-controlled, double-masked, multicenter phase 3 study planned to enroll 60 subjects to receive either Brimonidine Nanoemulsion Eye Drops 0.18% or ophthalmic buffered saline (placebo). The co-primary endpoints were 1) Validated Bulbar Redness (VBR), measured between baseline and 12 weeks of treatment, assessing ocular redness based on a 100-point scale measuring change in appearance, and 2) score on the 10-point Visual Analog Scale (VAS) between baseline and 12 weeks of treatment, showing measured change in ocular discomfort intensity.
OCU300 consists of an improved 0.18% ophthalmic nanoemulsion 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. Ocugen’s patented OcuNanoE™ technology is designed to enhance efficacy by prolonging retention of this potent anti-inflammatory drug on the eye surface. In addition, it allows OCU300 to be sterile filtered into single-use vials as preservative-free nanoemulsion, thereby eliminating potentially irritating effects of preservatives.
OCU300 was granted Orphan Drug Designation from the FDA for ocular GvHD.
About ocular Graft Versus Host Disease
Graft-versus-host disease (GVHD) is a common side effect of an allogeneic (donor source) bone marrow, stem cell or cord blood transplant (BMT) – approximately half of people who receive allogeneic transplants develop chronic GVHD. GVHD is an autoimmune disease that can affect many different parts of the body; the skin, eyes, mouth, stomach and intestines are affected most often. GVHD can range from mild to life-threatening. Ocular GVHD is a major complication that affects 60% – 80% of patients with chronic GVHD. Eye related symptoms include blurry vision, foreign body sensation, burning sensation, severe light sensitivity, chronic conjunctivitis (pink eye), dry eyes and eye pain.