Praxis Bioresearch Gets FDA Nod for PRX-P4-003 IND in Alzheimer's Apathy

Praxis Bioresearch announced that the U.S. Food and Drug Administration (FDA) has approved the investigational new drug (IND) application for PRX-P4-003, a gut-activated stimulant designed to treat apathy in Alzheimer’s Disease. In previous exploratory microdose clinical trials with healthy volunteers, PRX-P4-003 showed successful activation via oral administration, which is the intended therapeutic route.

Jeffrey Cummings, MD, ScD, who is the director of the Chambers-Grundy Center for Transformative Neuroscience and a professor at the University of Nevada Las Vegas, highlighted that apathy is one of the most commonly observed symptoms in Alzheimer’s Disease, with no FDA-approved therapies currently available. He noted that PRX-P4-003 has promising chemical properties and preliminary clinical validation, suggesting its potential to build on pioneering studies conducted by the NIH-funded ADMET group.

PRX-P4-003 is a novel chemical entity discovered by Praxis Bioresearch. It acts as a selectively bioavailable dopamine and norepinephrine reuptake inhibitor, and it is specifically designed to be activated by pancreatic lipase, an enzyme present only in the digestive system. Sandeep Patil, MD, PhD, CEO and cofounder of Praxis Bioresearch, emphasized the significance of this milestone, expressing pride in the team's success in delivering a high-quality IND package. Patil stated that the next phase of development will focus on demonstrating the compound's effectiveness in alleviating apathy symptoms during clinical trials.

PRX-P4-003 is developed to deliver an active isomer of fencamfamine, a Schedule IV stimulant initially developed and marketed by E. Merck KG in Germany. Unlike current stimulants like methylphenidate and amphetamines, PRX-P4-003 is designed to be orally active, potentially reducing the risk of abuse. There is a pressing need for alternatives to current Schedule II stimulant medications in neuropsychiatric applications due to concerns about drug diversion.

Apathy is the most common neuropsychiatric symptom in Alzheimer’s dementia, affecting approximately 71% of patients throughout the progression of the disease. It is associated with increased patient suffering, caregiver distress, and additional disability. Currently, there is no approved treatment specifically for apathy in Alzheimer’s Disease.

Research mentioned in this publication has been supported partially by the National Institute of Aging of the National Institutes of Health under Award Number R44AG066378. The content is the sole responsibility of the authors and does not necessarily represent the official views of the National Institutes of Health.