Brain Related Drug Development

What is PRV-002?

PRV-002 is a new chemical entity (NCE) with proven in-vivo efficacy in animal concussion models through gene amplification of anti-inflammatories, antioxidants, and anti-edematous channels.

  • Safety margin > 200 fold in animal toxicology studies

  • The IP covers composition of matter, synthetic routes, and methods of use

  • Mouth to Nose (breath propulsion) delivery system

  • Nano-particle spray-dried formulation drives drug into the brain in minutes

How does PRV-002 work?

PRV-002 induces an intracellular steroid receptor found in brain cells. The stimulation of PRV-002 receptoractivates multiple gene response elements promoting transcription of anti-inflammatories, anti-oxidants, and efflux channels that remove excess fluid and debris. Since PRV-002 is not a GABAergic compound, it avoids drug-induced fatigue and any cognitive decline associated with prolonged use. PRV-002 weakly activates the progesterone receptor, avoiding side effects such as clotting or infertility.

 
 

In development, not commercially available.

 

What is PRV-001?

PRV-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 PRV-001 work?

PRV-001 binds to an intracellular steroid receptor. The PRV-001 receptor is an intracellular receptor found in neurons, glia and the endothelium of the blood brain-barrier. The induction of the PRV-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 PRV-001 is not a GABAergic compound, it causes no fatigue. Furthermore, PRV-001 promotes neuronal synatopgenesis in neuronal cell culture models.

PRV-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.

 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.