Announcement • Apr 14
Oragenics, Inc. Doses First Patient In Phase IIa Clinical Trial Of ONP-002 For Mild Traumatic Brain Injury
Oragenics, Inc. announced that the first patient has been dosed in its ongoing Phase IIa clinical trial evaluating ONP-002, the Company’s lead candidate for the treatment of concussion and mild traumatic brain injury (mTBI). The milestone was achieved at Mackay Hospital in Australia — the first site to be activated in the trial — within days of site activation on March 31, 2026. Concussion, aka, mild traumatic brain injury, represents the most prominent neurological conditions without an FDA-approved pharmacological treatment. According to the CDC, an estimated 1.7 to 3.8 million people in the U.S. experience traumatic brain injuries annually, with sports and recreational activities among the leading causes. Globally, an estimated 69 million individuals sustain traumatic brain injuries each year. Despite this scale, no pharmacological treatments have been approved — leaving patients, military personnel, athletes, and families with few effective options beyond rest and symptom management. If approved by the FDA, ONP-002 would be the first and only pharmacological standard of care for a global concussion market projected to reach over $9 billion by 2030. The commencement of patient dosing follows the receipt of Human Research Ethics Committee (HREC) approval in Australia and the activation of Mackay Hospital as the first clinical site. The rapid presentation of an eligible patient immediately upon site activation underscores the breadth and urgency of unmet clinical need in this population. Two additional Australian sites — Alfred Hospital (Melbourne) and Royal Adelaide Hospital (Adelaide) — are progressing through site governance approvals, with activation expected in the second quarter of 2026. ONP-002 is a first-in-class intranasal novel neurosteroid designed to address the underlying biology of brain injury — reducing neuroinflammation, oxidative stress, and cerebral edema — rather than simply managing symptoms. As an investigational neuroprotective intranasal drug, ONP-002 targets the biological cascade triggered by trauma, with the potential to represent a paradigm shift from symptom management to active neurological intervention. ONP-002 is delivered via Oragenics’ proprietary intranasal spray-dry powder device. The drug candidate serves a nasal drug delivery market expected to reach nearly $93 billion by 2030. Oragenics’ Phase IIa clinical trial is a randomized, placebo-controlled study designed to evaluate 40 patients who meet enrollment criteria based on CT scan findings, presenting symptoms, and emergency room or hospital admission. Patients will receive first dosing within 12 hours of concussion, followed by continued treatment for up to 30 days. The trial will assess safety and tolerability parameters through follow-up visits for nasal examinations, physical assessments, and neurocognitive testing. Feasibility will be determined according to tolerability and participant compliance. Oragenics expects that findings will support its planned investigational new drug (IND) application submission to the FDA, targeting Fourth Quarter 2026, for the next phase of clinical trials to be conducted in the U.S. The Phase 1 clinical trial of ONP-002 delivered a strong safety profile, with zero serious adverse events reported across all dose levels in 40 patients, supporting advancement to Phase 2. Preclinical data demonstrated reductions in swelling, inflammation, and oxidative stress in the brain, along with improvements in functional recovery. Southern Star Research, a full-service Australian clinical research organization (CRO), is managing Phase IIa trial operations. Sterling Pharma Solutions is providing cGMP drug manufacturing services from its facility in Cary, North Carolina. ONP-002 is an investigational neuroprotective, anti-inflammatory intranasal drug candidate targeting mild traumatic brain injury (mTBI) and concussion. Designed to interrupt biological pathways involved in inflammation, oxidative stress, and brain swelling following head trauma, ONP-002 has demonstrated safety and tolerability in Phase 1 clinical trials with zero serious adverse events across all dose levels. The drug candidate utilizes Oragenics’ proprietary intranasal delivery platform to enable rapid, targeted brain delivery — potentially representing a paradigm shift from symptom management to active neurological intervention. Oragenics is advancing ONP-002 through Phase IIa clinical trials in Australia, with U.S. Phase IIb trials planned to follow pending FDA investigational new drug application (IND) approval.
