Phanes Biotech’s novel molecule, P021, has been proven in preliminary studies to shift the balance from neurodegeneration to neuroregeneration, thereby altering the way we think about and treat AD.

P021 promotes and rescues neural formation (neurogenesis) and the brain’s ability to rewire (neuronal plasticity) by competitively enhancing the activity of brain-derived neurotrophic factor (BDNF), leading to both prevention of the destruction of some nerve cells and regeneration and repair of destroyed nerve cells.

By its significant interactions, P021 alters, prevents, and repairs tau and AβPP pathologies, resulting in effective disease-modifying activity in AD. In a recent study in mice, P021 was proven to prevent not only cognitive-type deficits in AD but also AD-type pathological changes, including tau and AβPP pathologies, post-synaptic deficits, and neural inflammation, which are considered hallmarks of AD pathology.

AD is a progressive neurodegenerative disease characterized clinically by cognitive impairment associated with extracellular senile plaques and intracellular neurofibrillary tangles. Shifting the balance from neural degeneration to neural regeneration and neuronal and synaptic plasticity is a novel strategy in the development of disease-modifying treatment and prevention of AD and other neurodegenerative disorders. One strategy for shifting this balance would be to enhance the activity of neurotrophic factors such as BDNF while inhibiting leukemia inhibiting factor (LIF). Data obtained from years of transgenic and animal studies indicate that P021 enhances BDNF, which activates GSK3β, thereby decreasing tau’s hyperphosphorylation and the amyloidogenic processing of AβPP. LIF signaling leads to prevention of formation of neural progenitor cells during the process of neurodegeneration. By its potent activity on BDNF and LIF, P021 results in the rescue of neurogenesis and neuroplasticity deficits, reduction of both tau and Aβ pathologies, and improvement of cognitive impairment. The mechanism of action and characteristics of P021 are unique, targeting neuronal health via neurogenesis and neuronal plasticity and offering the great potential of being a disease-modifying drug for AD and other neurodegenerative diseases.