Allopregnanolone.
In Alzheimer’s disease, the regenerative capacity of the brain is diminished. Allopregnanolone promotes the brain’s own system of regeneration, while simultaneously reducing the burden of Alzheimer’s Disease pathology, plaques and tangles. Exogenous administration of allopregnanolone promotes regeneration by activating neural stem cells to create new neurons.
It’s application in Alzheimer’s disease provides the first regenerative approach to treating a neurodegenerative disease.
Background.
Allopregnanolone is a neurosteroid that promotes the brain’s ability to regenerate itself. It is a naturally occurring molecule (a derivative of progesterone) that is produced endogenously from the earliest stages of development. It peaks in the 3rd stage of trimester when the brain of the developing fetus increases 4-fold in size in utero, harnessing the potential of Mother Nature. Allopregnanolone has been extensively researched since 1994 and during that time has undergone robust and rigorous safety and efficacy testing in both animal models and humans.
Phase 2b Clinical Trial
Allopregnanolone has been approved for a Phase 2b clinical trial, REGEN-BRAIN© which is currently enrolling participants at sites across the country.
What Makes Allopregnanolone Unique to Other Treatments for Alzheimer’s Disease.
Generates new neurons, new synapses and new neural circuits in the Alzheimer’s brain
Activates the energy system in the brain
Reduces Alzheimer’s Disease pathology – both beta amyloid plaques and tangles
First regenerative, safe therapeutic for Alzheimer’s disease
Mechanism of action – allopregnanolone is a positive allosteric modulator (PAM) of GABAA and is the only known molecule to uniquely* target and activate GABAA receptors (32) to generate new cells (neurogenesis).
Safe alternative to monoclonal antibodies that act by clearing amyloid plaques with no meaningful clinical impact on cognitive function.
Our Scientific Approach.
Firstly, and most importantly, our trials are designed to ensure early intervention.
At NeuTherapeutics, a patient-centric and compassionate approach is an integral part of our trial design because we have decades of peer-reviewed research in support of our therapeutic development and want all patients to benefit; shorter trials with open-label extension arms - the placebo arm crosses over to the active arm. This trial design facilitates faster patient recruitment as it’s more motivating for patients to enter the trial as they are guaranteed to receive the active molecule.
Preclinical work and translational science have established the optimal safe dosing regimen for allopregnanolone to promote regeneration in Alzheimer’s disease. The upcoming Phase II trial will further validate that dosing regimen.