FDA Backs Phase 3 Trial of Pro-ocular for Ocular GVHD Treatment

US FDA

• Signal12 Pharmaceuticals has aligned with the FDA on its phase 3 clinical trial strategy for Pro-ocular, a novel drop-free therapy for ocular graft-vs-host disease (oGVHD).
• In a phase 2 trial, Pro-ocular demonstrated statistically significant and clinically meaningful improvements in corneal staining and ocular symptom scores.
• An upcoming phase 3 trial aims to confirm these findings in a larger patient population.

The FDA and Signal12 Pharmaceuticals have aligned their strategy for a phase 3 trial evaluating Pro-ocular, a novel drop-free therapy for the potential treatment of patients with oGVHD.¹ This alignment was achieved following a Type C meeting with the FDA.

"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, in a press release. "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 phase 2 trial, a 10-week, double-masked, placebo-controlled study, 33 patients were randomly assigned 2:1 to receive Pro-ocular or placebo.¹ At week 10 of treatment, patients receiving placebo crossed over to the active drug arm.

The study was conducted in collaboration with Harvard Medical School’s Mass Eye and Ear Infirmary.

Across multiple end points for efficacy, the findings were statistically significant and clinically meaningful. Significant improvements in corneal staining and ocular symptom scores were seen and maintained over a 2-year open-label extension. Pro-ocular also demonstrated a favorable safety profile, with no drug-related serious adverse events reported.

The key end points have been validated and accepted by the FDA.²

Now, a phase 3 study plans to further evaluate the therapy’s efficacy and safety, with the goal of providing a new treatment option for patients with oGVHD.¹ Initiation for the phase 3 trial will promptly begin, with results expected to pave the way for regulatory approval.

"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, in the press release. "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."

REFERENCES:

1. Signal12 achieves FDA alignment, accelerating Pro-ocular™ towards phase 3 clinical trials for ocular graft-versus-host disease. News release. Signal12, Inc. March 18, 2025. Accessed March 19, 2025. https://tinyurl.com/muyvdy8b

2. Clinical Trials. Signal12, Inc. Accessed March 19, 2025. https://signal12inc.com/clinical-trials/#pipeline