Brain Related Drug Development

What is ONP-002?

ONP-002 is intended to be First-in-Class Neurosteroid being developed for the treatment of moderate to severe concussion.

• The IP includes structure, synthetic preparation, and methods of use
• Animal and cell culture models of neuronal injury show positive preclinical molecular and behavioral effects within hours
• Animal studies have shown the drug to have an excellent safety profile.
• Intranasal administration in animals showed quick and significant distribution to the brain
• The intranasal formulation was developed as a novel spray dried nanoparticle
• The formulation is loaded into a novel intranasal device for one-time field delivery

How does ONP-002 work?

ONP-002 diffuses intracellularly to induce the ONP-002 receptor which is an intracellular receptor found in neurons, glia, and the endothelium of the blood brain-barrier. The induction of the ONP-002 receptor activates the gene response elements leading to the production of proteins that reduce inflammation, oxidative stress, and swelling. In addition, transport channels are activated that reduce the build-up of extra- and intra-cellular debris that can cause chronic disease. Since ONP-002 is not a GABAergic compound, it causes no fatigue.

ONP-002 Phase I Clinical Trial

Recently, Odyssey Health completed a SAD/MAD Phase I human safety study. ONP-002 was well-tolerated with no severe adverse events. Oragenics is currently planning for a Phase II Clinical Trial.

What is ONP-001?

• ONP-001 is intended to be First-in-Class Neurosteroid and will be developed for the treatment of Niemann Pick Type-C Disease.
• The IP covers methods of preparation and methods of use
• Animal models of neuronal injury show positive preclinical effects on cognition and motor performance
• There has been no drug-related toxicity observed

How does ONP-001 work?

ONP-001 binds to an intracellular steroid receptor. The ONP-001 receptor is an intracellular receptor found in neurons, glia and the endothelium of the blood brain-barrier. The induction of the ONP-001 receptor activates gene response elements leading to the transcription of P-Glycoprotein (PGP), which pumps cholesterol and lipid debris, out of the brain cells and the brain circulation.

ONP-001 Toxicology

Acute and sub-acute dose ranging studies were executed in rats – intranasal and intravenous. No drug-related effects were noted in hematology, blood chemistry, or histopathology. IND-enabling studies are being scheduled. These studies will be followed by a Phase I safety trial.

? Whitepaper: Novel Drug Development to Treat Concussions, An Unmet Medical Need

Poster Presentation at Special Operations Medical Association (SOMA) - Scientific Assembly

What is Niemann Pick Type- C Disease?

Nieman Pick Type-C Disease is a rare brain disorder in children. It is considered an “orphan indication” by the FDA and therefore has special considerations in the FDA approval process.

• Niemann-Pick Type C disease (NPC) is an autosomal recessive, lysosomal storage disorder characterized by accumulation of cholesterol and gangliosides in the brain and liver.
• NPC is a rare (estimated U.S. prevalence of 1:150,000) neurodegenerative disorder. Prevalence is 5X higher with Arabian decent.
• Children with NPC demonstrate neurological dysfunction with cerebellar ataxia, dysarthria, seizures, visual palsy, motor impairment, dysphagia, psychotic episodes, and progressive dementia.
• There is no effective treatment for NPC, and it is a lethal disorder.