Trial Designed to Demonstrate Target Engagement, Safety, and Pharmacokinetics
CLEVELAND, April 7, 2026 /PRNewswire/ -- NeuroTherapia, Inc., a clinical-stage company focused on developing oral therapies for neurodegenerative diseases, has completed its Phase 2a clinical trial in Alzheimer's Disease (AD) patients. NTRX-07, the Company's lead molecule, was administered for 28 days in this double-masked, randomized clinical trial. In addition to monitoring safety, pharmacokinetics, and standard measures of clinical efficacy (ADAS-cog, MMSE, and Trail Making Test), the trial was designed to assess target engagement by analyzing biomarkers of neuroinflammation and neuronal function.
"We are pleased to see that NTRX-07 continues to be safe and well tolerated in patients with Alzheimer's Disease, and that drug exposure levels were in line with what we expected, demonstrating a positive outcome," commented Dr. Joseph Foss, the company's Chief Medical Officer. "Following the preliminary analysis of the exploratory endpoints, the study met its core Phase 2a objectives and produced a positive multi-domain signal package across safety, PK, biomarkers, MRI, EEG, and clinical outcomes. This supports confidence in the further development of this new approach to treating Alzheimer's Disease."
The primary objective of the study, safety and tolerability, was achieved, with overall Adverse Events (AEs) similar across the treatment and placebo groups. No treatment related Serious Adverse Events (SAEs) were reported in the active arm. Preliminary analysis of exploratory endpoints, including MRI, qEEG, and CSF biomarkers, indicates some encouraging results. Preliminary MRI findings, based on the hypothesis that neuroinflammation is important in AD progression, were directionally supportive, with changes trending toward stabilization on active treatment. Based in part on these encouraging results, NeuroTherapia is continuing to work with Oxford Brain Diagnostics Ltd on a more thorough analysis of the MRI data, including utilizing its CDM Explore platform to compare MRI results with changes in neuroinflammatory biomarkers, qEEG, and cognitive test results.
The trial was managed by CRU Global, a leading European CRO, and conducted at sites in Hungary, Poland, and the Czech Republic. The trial enrolled 48 subjects in a 1:1 randomized design across five sites.
"We were very pleased with the recruitment efforts at the sites, allowing the trial to complete on time. There was also considerable interest in participating in future trials expressed by site PIs, patients, and caregivers," commented Peter Kovacs, Ph.D., the President of CRU Global. "We are looking forward to working with NeuroTherapia on its next Phase 2 study, which we believe has an excellent opportunity to show clinical benefits to AD patients."
NTRX-07 is a small orally available molecule that targets receptors that increase in diseases, including Alzheimer's disease, amyotrophic lateral sclerosis (ALS), multiple sclerosis (MS), and chronic pain syndromes. In various animal models of AD, NTRX-07 restored normal function of the microglia (key immune cells in the brain), which in turn decreased microglial-induced inflammation, reduced levels of the Alzheimer's-associated Aß peptide in the brain, and substantially improved neuronal synaptic plasticity, learning, and memory. This study was being funded in part by a grant from the Alzheimer's Association Part the Cloud program.
About NeuroTherapia
At NeuroTherapia, our mission is to develop treatments that will improve the course of the most debilitating and deadly central nervous system (CNS) diseases patients and their families face today. NeuroTherapia, Inc. is a clinical-stage, privately held biotechnology company, spun out of the Cleveland Clinic, developing oral, small-molecule drugs to address neuroinflammatory conditions of the CNS, including Alzheimer's disease, Parkinson's disease, amyotrophic lateral sclerosis, and other CNS disorders. There is an emerging consensus that neuroinflammation plays a significant role in CNS disease. NeuroTherapia is a leader in developing novel therapeutics that modulate microglial activity, impacting the expression of proteins that drive inflammation in the CNS, thereby improving neuronal function and survival. Additional information about NeuroTherapia can be found at www.neurotherapia.com.
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SOURCE NeuroTherapia, Inc.
