NeoTherapeutics is developing NeotrofinT (AIT-082, leteprinim potassium) for Alzheimer's disease. The drug is currently in Phase 2b/3 clinical trials and has shown clinical benefit to patients in the trials completed to date. Based on the neurotrophic actions of NeotrofinT, this drug may provide benefit to patients with other neurodegenerative diseases. Pre-clinical studies are ongoing in animal models of spinal cord injury, peripheral neuropathy, ALS and other diseases. The results from the current pre-clinical studies will determine whether or not NeotrofinT will enter clinical trials in one or more of these indications.
On October 8, 1998 NeoTherapeutics, Inc. announced that the company has been issued a new U.S. patent on gene regulation technology involved in treating neurodegenerative diseases.
U.S. Patent No. 5,801,184 describes how purine derivatives, a class of drug compounds which includes NeoTherapeutics' lead compound NEOTROFIN(tm) (AIT-082, leteprinim potassium), can be used to selectively control "turning on or off of genes" for the protein molecules involved in nerve regeneration. This patent represents new step treatment of neurodegenerative diseases utilizing oral medication to replace or augment the presence of neurotrophic factors in the brain and spinal cord.
NeoTherapeutics reports that the company does not know if all types of nerve cells will react in the same manner to NEOTROFIN(tm). There is no evidence at this time that NEOTROFIN(tm) is effective for ALS. However, the company is conducting an exploratory, pre-clinical experiment in animals to determine if NEOTROFIN(tm) demonstrates effectiveness for ALS.
NEOTROFIN, AIT-082, regulates the genetic expression of several neurotrophic factors in various areas of the brain and spinal cord. In each area, the factors it induces are those naturally needed by the nerve cells in that region. AIT-082 is the first drug in human clinical trials, which acts by regulating the production of the naturally occurring neurotrophic growth factors.
Neurotrophic factors are essential for the growth, maturation and survival of nerve cells. When present in appropriate concentrations, these factors also protect nerve cells against damage in stroke, trauma, and many neurodegenerative disorders including Alzheimer's and Parkinson's diseases. These factors are not produced by the nervous system in sufficient quantities to be fully effective in those conditions, and, because neurotrophic factors are proteins, they cannot be orally administered. These problems are circumvented by NeoTherapeutics' orally active purine derivative that controls the genetic expression of neurotrophic factors in the nervous system to treat neurodegenerative diseases.
NeoTherapeutics' research program is focused on designing and developing small molecules capable of promoting nerve regeneration and repair for a range of neurological diseases and conditions such as Alzheimer's and Parkinson's diseases, stroke and spinal cord injury.
