Signal12 Pharmaceuticals announced a significant regulatory milestone after aligning with the U.S. Food and Drug Administration (FDA) on the Phase 3 clinical trial strategy for Pro-ocular, a novel drop-free therapy developed to treat ocular Graft-versus-Host Disease (oGVHD). This agreement, reached during a Type C meeting with the FDA, paves the way for advanced clinical testing of what could become the first FDA-approved treatment for this debilitating 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."
Promising Phase 2 Results
The regulatory advancement follows compelling results from a Phase 2 trial conducted in collaboration with Harvard Medical School's Mass Eye and Ear Infirmary. The 10-week, double-masked, placebo-controlled study enrolled 33 patients who were randomized in a 2:1 ratio to receive either Pro-ocular 1% or placebo. At week 10, patients in the placebo group crossed over to the active treatment arm.
Results demonstrated statistically significant and clinically meaningful improvements across multiple efficacy endpoints. Notably, patients experienced improved corneal staining and reduced ocular symptom scores. These benefits were maintained during a two-year open-label extension phase, suggesting durable therapeutic effects.
The therapy also exhibited a favorable safety profile throughout the study period, with no drug-related serious adverse events reported. These key endpoints have been validated and accepted by the FDA, strengthening the foundation for the upcoming Phase 3 program.
Addressing a Critical Unmet Need
Ocular Graft-versus-Host Disease is a serious complication that can develop following allogeneic stem cell transplantation. It affects approximately 50,000 patients annually in the United States alone and currently has no FDA-approved treatments. The condition causes severe dry eye, ocular pain, and can significantly impair quality of life for affected individuals.
Marv Garrett, Senior Vice President of Regulatory Affairs at Signal12, emphasized the significance of the FDA's feedback: "The FDA's positive feedback on our Phase 3 trial design underscores the urgent need for new and effective interventions for oGVHD. 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."
Innovative Therapeutic Approach
Pro-ocular represents a departure from conventional eye drop therapies. The company describes it as a "drop-free therapy" that employs transappendageal delivery to precisely target the ophthalmic branch of the trigeminal nerve. This novel approach appears to not only boost tear production but also deliver significant relief from ocular pain.
The therapy's mechanism potentially offers advantages over traditional treatments for dry eye conditions, which often provide only temporary relief and require frequent administration. Pro-ocular's sustained efficacy observed in the Phase 2 extension study suggests it may offer a more convenient and effective treatment option.
Looking Toward Phase 3 and Beyond
Signal12 has indicated that initiation of the Phase 3 trial will begin promptly. The company is strategically positioned to advance a clinical program that could potentially transform the treatment landscape for oGVHD patients.
If successful in Phase 3 trials and subsequently approved, Pro-ocular would fill a significant therapeutic gap for patients suffering from this orphan condition. The therapy's potential to provide sustainable relief from both the signs and symptoms of oGVHD could represent a major advancement in post-transplant care.
The company has not disclosed a specific timeline for completion of the Phase 3 program or potential submission dates for regulatory approval. However, the alignment with FDA on trial design represents a critical step toward bringing this innovative therapy to patients with limited treatment options.
