Signal12 Achieves FDA Alignment, Accelerating Pro-ocular™ Towards Phase 3 Clinical Trials for Ocular Graft-versus-Host Disease

BOSTON, March 18, 2025 /PRNewswire/ -- Signal12, Inc. (Signal12), a clinical-stage ophthalmic pharmaceutical company focused on first-of-a-kind patented technologies that enable existing drugs to act through unique neural pathways, today announced the alignment of its Phase 3 clinical trial strategy for Pro-ocular™ with the U.S. Food and Drug Administration (FDA). Pro-ocular™ is a novel drop-free therapy poised to revolutionize the treatment of ocular Graft-versus-Host Disease (oGvHD). This key regulatory milestone, achieved following a highly productive Type C meeting, sets the stage for Signal12's transformative Phase 3  clinical research in an area of high unmet medical need.

Ocular Graft-versus-Host Disease, an orphan condition impacting roughly 50,000 patients annually in the United States, currently has no FDA-approved treatment. Pro-ocular has demonstrated its potential to not only boost tear production but also deliver significant relief from ocular pain, offering a promising new lifeline for patients debilitated by this severe condition.

"This alignment on the Phase 3 requirements with the FDA represents a major step forward for patients suffering from oGvHD," said Tom Mitro, Chief Executive Officer at Signal12. "We believe Pro-ocular can significantly improve patient's quality of life by providing sustainable relief from the signs and symptoms of this highly debilitating disease. Our team looks forward to initiating our Phase 3 trial as soon as possible."

In a robust Phase 2 trial conducted over 10 weeks—a double-masked, placebo-controlled study involving 33 patients randomized 2:1 to Pro-ocular 1% or placebo—results revealed statistically significant and clinically meaningful improvements across multiple efficacy endpoints. These improvements, which included improved  corneal staining and ocular symptom scores , were sustained over a two-year open-label extension without any drug-related serious adverse events. The trial, executed in collaboration with Harvard Medical School's renowned Mass Eye and Ear Infirmary, reinforces Pro-ocular's potential as a breakthroughr therapy in oGvHD management. Detailed study outcomes are accessible at https://signal12inc.com/clinical-trials/.

"The FDA's positive feedback on our Phase 3 trial design underscores the urgent need for new and effective interventions for oGvHD," stated Marv Garrett, Senior Vice President of Regulatory Affairs at Signal12. "Our Phase 2 results, showing meaningful improvements in corneal staining and ocular dryness, highlight the disruptive potential of Pro-ocular as a potential treatment for this disease. We're highly enthusiastic about advancing to the next stage of clinical research."

Having achieved this important regulatory milestone, Signal12 is now strategically positioned to drive forward a Phase 3 clinical program that could redefine the treatment landscape for oGvHD. This development underscores the company's commitment to addressing critical therapeutic gaps.  

About Signal12 Inc.

Signal12 is a Phase 3-ready ophthalmic pharmaceutical company. Our proprietary topical drug, Pro-ocular™, has been shown to be safe and effective in Phase 2 clinical studies, demonstrating significant reductions in the signs and symptoms of ocular Graft-versus-Host Disease (oGvHD)—a chronic, debilitating condition following an allogeneic stem cell transplant. Pro-ocular™ is the only effective treatment for oGvHD, employing transappendageal delivery to precisely target the ophthalmic branch of the trigeminal nerve.

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SOURCE Signal12 Inc.