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FDA Approves Damona Pharmaceuticals' DPX-101 for Phase I Trial in Brain Disorders

• Damona Pharmaceuticals received FDA clearance for its IND application to begin a Phase I trial of DPX-101, a novel treatment for cognitive deficits. • DPX-101, a positive allosteric modulator targeting the α5-GABA-A receptor, aims to address cognitive disabilities in major depressive disorder and other brain disorders. • The Phase I trial will evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of DPX-101 in healthy volunteers through single and multiple ascending dose studies. • Preclinical data suggest DPX-101 selectively activates its target, improves electrophysiological effects, and enhances cognitive performance in relevant behavioral tests.

Damona Pharmaceuticals has received FDA approval for its Investigational New Drug (IND) application for DPX-101, paving the way for a Phase I clinical trial. This trial will assess the drug's potential in treating cognitive disabilities associated with major depressive disorder and other brain disorders.
DPX-101 is a positive allosteric modulator specifically designed to target the α5-GABA-A receptor in the brain. The Phase I trial is structured to evaluate the safety, tolerability, pharmacokinetic, and pharmacodynamic properties of DPX-101 in healthy individuals.

Trial Design and Endpoints

The Phase I trial will consist of two parts: a single-ascending dose (SAD) study and a multiple-ascending dose (MAD) study. The SAD study will involve five cohorts of participants, along with a comparator cohort receiving Zolpidem, an α1-GABA-A receptor agonist. This comparator arm aims to assess the selectivity of DPX-101 for the α5-GABA-A receptor.
The MAD study will randomize participants into three escalating dose cohorts, receiving daily doses of DPX-101 or a placebo for one week. The primary endpoints of the trial focus on the safety and tolerability of varying doses of DPX-101. Secondary endpoints will evaluate the pharmacokinetic profile of the drug.

Rationale and Preclinical Evidence

According to Damona Pharmaceuticals CEO John Reilly, the FDA clearance allows the company to initiate a Phase I clinical study of their lead asset, which "promises to establish a new standard of care for the treatment of cognitive deficits associated with brain disorders." Reilly added that preclinical studies and the compound's selectivity for α5-GABAAR suggest that DPX-101 could benefit a broad range of patients suffering from diseases that cause cognitive impairment.
Preclinical studies have demonstrated that DPX-101 exhibits highly selective activation of its target and the anticipated electrophysiological effects. Furthermore, the drug has shown improvements in cognitive performance in behavioral tests designed to assess functions of the frontal cortex and hippocampus.
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Reference News

[1]
Damona Pharmaceuticals given FDA approval for trial of DPX-101 for brain disorders
clinicaltrialsarena.com · Nov 5, 2024

The FDA approved Damona Pharmaceuticals' IND for DPX-101, a positive allosteric modulator targeting the α5-GABA-A recept...

[2]
Damona Pharmaceuticals given FDA approval for trial of DPX-101 for brain disorders
yahoo.com · Nov 5, 2024

FDA approves Damona Pharmaceuticals' IND for Phase I trial of DPX-101, a positive allosteric modulator targeting α5-GABA...

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