Brain Related Drug Development
ONP-002: A Breakthrough Neurosteroid for Concussion Treatment
ONP-002 is a first-in-class neurosteroid designed to revolutionize the treatment of moderate to severe concussions. Engineered for rapid action and unparalleled brain penetration, ONP-002 aims to address the urgent unmet medical need in traumatic brain injuries.
Understanding Concussion: A Serious Brain Injury
A concussion is a type of traumatic brain injury (TBI) caused by a sudden impact to the head or body, resulting in rapid movement of the brain inside the skull. This can lead to:
- Chemical imbalances and cellular damage
- Disruptions in thinking, learning, behavior, and sleep
- Symptoms that range from mild confusion to severe cognitive impairment
Early intervention is crucial to preventing long-term complications. ONP-002 represents a transformative approach to concussion treatment, targeting the root causes of brain injury for faster recovery and better outcomes.
Download Our Whitepaper: Novel Drug Development to Treat Concussions, An Unmet Medical Need
Why ONP-002?
- Proven Efficacy: Preclinical studies in animal and cell models demonstrate rapid molecular and behavioral improvements within hours.
- Superior Safety Profile: Animal studies confirm excellent tolerability and no significant toxicity concerns.
- Optimized Delivery: Intranasal administration enables fast and targeted distribution to the brain.
- Cutting-Edge Formulation: A novel spray-dried nanoparticle formulation enhances bioavailability.
- Field-Ready Solution: Designed for one-time use via an innovative intranasal delivery device.
- Patented Innovation: Proprietary intellectual property covers its structure, synthesis, and therapeutic application.
How ONP-002 Works
ONP-002 penetrates cells to activate the ONP-002 receptor, found in neurons, glial cells, and the blood-brain barrier. This triggers a cascade of beneficial effects:
- Reduces inflammation, oxidative stress, and swelling
- Clears extracellular and intracellular debris to prevent long-term damage
- Maintains cognitive function without sedation, as it is non-GABAergic
Clinical Development
ONP-002 has successfully completed a Phase I Single and Multiple Ascending Dose (SAD/MAD) safety study, with ONP-002 demonstrating excellent tolerability and no severe adverse events. Oragenics is now advancing preparations for a Phase II clinical trial.
ONP-001: Pioneering Treatment for Rare Leukodystrophies
ONP-001 is an innovative neurosteroid designed to address rare leukodystrophies—a group of devastating genetic disorders that impair myelin production and disrupt brain function. By targeting the underlying cellular dysfunction, ONP-001 aims to restore neurological stability and slow disease progression.
Why ONP-001?
- Encouraging Preclinical Results: Animal models show significant improvements in cognition and motor function.
- Exceptional Safety Profile: No drug-related toxicity observed in extensive testing.
- Advanced Intranasal Formulation: Optimized for efficient brain targeting
- Proprietary Protection: Intellectual property covers synthesis and therapeutic use.
How ONP-001 Works
ONP-001 binds to an intracellular steroid receptor found in neurons, glial cells, and the blood-brain barrier, triggering essential protective mechanisms:
- Activates P-Glycoprotein (PGP): Clears toxic lipid and cholesterol buildup from brain cells.
- Reduces neuroinflammation and excess fluid accumulation.
- Enhances neuronal synaptogenesis for improved brain function.
- Non-GABAergic: No sedative or fatigue-inducing effects.
ONP-001 Toxicology and Development
Comprehensive acute and sub-acute dose-ranging studies in rats—both intranasal and intravenous—showed no adverse effects on hematology, blood chemistry, body weight, or organ function. IND-enabling studies are now in progress, paving the way for Phase I clinical trials.
ONP-001 and ONP-002 are currently in development and are not yet commercially available.