What is ONP-002?
ONP-002 is a First-in-Class Neurosteroid being developed for the treatment of moderate to severe concussion.
· The IP includes structure, synthetic preparation, and methods of use
· There are very few competing products currently in human trials
· Animal and cell culture models of neuronal injury show positive preclinical molecular and behavioral effects within hours
· There has been no drug-related toxicity observed
· 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. Odyssey is actively engaged with clinical sites for a Phase II Clinical Trial.
What is ONP-001?
· ONP-001 is a First-in-Class Neurosteroid being developed for the treatment of Niemann Pick Type-C Disease.
· The IP covers methods of preparation and methods of use
· There are very few competing products currently in human trials
· Animal models of neuronal injury show positive preclinical effects on cognition and motor performance
· There has been no drug-related toxicity observed
· The intranasal formulation is in preparation
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.
In development, not commercially available.
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.
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Brain Related Drug Development
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What is ONP-001?
ONP-001 is a First-in-Class Neurosteroid for the treatment of Niemann Pick Type-C Disease.
The IP covers methods of preparation and methods of use
There are very few competing products currently in human trials
Animal and cell culture models of neuronal injury show positive preclinical effects
There has been no drug-related toxicity observed
The intranasal formulation is complete
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 the gene response element leading to the transcription of P-Glycoprotein (PGP), which pumps toxins, cholesterol, and lipid debris, as well as excess fluid, out of the brain cells and brain circulation. Since ONP-001 is not a GABAergic compound, it causes no fatigue. Furthermore, ONP-001 promotes neuronal synatopgenesis in neuronal cell culture models.
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, BW, FC, or histopathology.