Oragenics, Inc. Receives Final HREC Approval In Australia To Begin Phase IIa Trial Of ONP-002 For Concussion Treatment

Oragenics, Inc., a clinical-stage biotechnology company developing brain-targeted therapeutics using proprietary intranasal delivery technology, announced that it has received final Human Research Ethics Committee (HREC) approval in Australia to initiate its Phase IIa clinical trial of its lead drug candidate, ONP-002, for the treatment of concussion, also known as mild traumatic brain injury (mTBI). With this approval, Oragenics has secured all necessary regulatory clearances and submitted all required filings to begin clinical site onboarding at three sites in Australia, with Bayside Health (Alfred Health) serving as the Lead Site.

Traumatic brain injury is among the most common neurological conditions without an FDA-approved pharmacological treatment. According to the CDC, between 1.7 and 3.8 million people in the United States experience traumatic brain injuries each year, with sports and recreational activities as leading causes. Globally, approximately 69 million individuals sustain traumatic brain injuries annually. Despite this high prevalence, no approved pharmacological therapies exist, leaving patients, athletes, military personnel, and families reliant on rest and symptom management. If approved by the FDA, ONP-002 would become the first pharmacological standard of care for concussions, a global market projected to exceed $9 billion by 2030.

ONP-002 is a first-in-class intranasal neurosteroid designed to target the underlying biology of mTBI rather than simply managing symptoms. The drug addresses neuroinflammation, oxidative stress, and cerebral edema triggered by brain trauma, potentially representing a paradigm shift from symptom-focused care to active neuroprotection. It would also participate in the broader nasal drug delivery market, projected to reach nearly $93 billion by 2030.

Janet Huffman, Chief Executive Officer of Oragenics, said that concussion and mTBI represent a major unmet medical need and that the company has worked responsibly and rigorously to reach this milestone. She noted that with HREC and governance approvals in place, Oragenics is ready to begin clinical site onboarding and patient enrollment in Australia, with the first patient expected to be dosed before the end of March. Huffman emphasized that the trial represents a meaningful step toward providing a potential breakthrough therapy for millions of patients who currently have no viable treatment options.

James Kelly, Chief Medical Officer, added that the HREC approval confirms the trial design, safety protocols, and investigator teams meet high standards. As a clinician experienced with concussion patients, he noted the significance of testing ONP-002, which targets the biological injury itself rather than just alleviating symptoms. The Phase 1 clinical trial demonstrated a strong safety profile, with no serious adverse events observed across all dose levels, providing confidence to advance to Phase IIa. Preclinical studies also showed reductions in brain swelling, inflammation, and oxidative stress, along with improvements in functional recovery.

The Phase IIa trial is a randomized, placebo-controlled study designed to enroll 40 patients who meet specific criteria based on CT scan findings, presenting symptoms, and emergency room or hospital admission. Patients will receive the first dose within 12 hours of injury and continue treatment for up to 30 days. Safety and tolerability will be monitored through follow-up visits, including nasal examinations, physical assessments, and neurocognitive testing. The study will also evaluate feasibility based on participant compliance and tolerability. 

Data from this trial are expected before the end of 2026 and will support Oragenics’ planned investigational new drug (IND) application to the FDA for further clinical development in the United States. Southern Star Research, a full-service Australian clinical research organization, will manage all aspects of the Phase IIa trial from initiation through completion, ensuring rigorous oversight and adherence to protocol standards.