We are advancing a pipeline of small-molecule therapeutics with the goal of bringing new precision neuromedicines to patients living with cognitive deficits associated with CNS disorders, including major neurodegenerative and psychiatric disorders such as Alzheimer’s disease, depression, and schizophrenia.
DPX-101 is a Precision-Targeted α5-GABA-A PAM
DPX-101 is a receptor-positive allosteric modulator (PAM) that targets the α5 subunit-containing GABA-A receptor to augment its function to restore contacts between neurons.
DPX-101 is unique in its subtype specificity, and we expect its highly targeted mechanism to sustain efficacy while minimizing many of the risks and side effects associated with α1-selective modulators of GABA-A, positioning DPX-101 as a best-in-class molecule targeting the α5-GABA-A receptor.
In the 5xFAD mouse model of Alzheimer’s disease-related β-amyloid pathology, DPX-101 improved working memory deficits and prevented neuronal shrinkage and spine loss in early and late stages of β-amyloid deposition. In gold-standard mouse models for major depressive disorder, DPX-101 augmented the function of the α5-GABA-A receptor to produce robust pro-cognitive, antidepressant, and brain-protective effects. These findings provide the basis for our plans to initiate a Phase 1 clinical study to evaluate the safety, tolerability, and pharmacokinetics of DPX-101 in humans.
Our Initial Focus
Alzheimer’s disease (AD) is the most common form of dementia, and nearly 100 million people worldwide are affected by Alzheimer’s or related dementia and its prodromal stage. It is a progressive neurological condition that leads to memory loss, cognitive decline, and changes in behaviors, significantly impacting the lives of patients and their families. Up to 50% of people with Mild Cognitive Impairment due to AD progress to Alzheimer’s dementia within five to 10 years, which impacts a person’s ability to remain independent. Currently, there are no treatment options available.
An estimated 11.2 million Major Depressive Disorder (MDD) patients in the U.S. suffer from cognitive impairment. Cognitive impairment is a core symptom of MDD that affects many areas of cognition, including learning and memory, executive functioning, processing speed, and attention and concentration. Cognitive impairment occurs during and between episodes of MDD, contributing significantly to occupational and functional disability and impacting quality of life.