NeuroTherapia Announces EMA Approval of its Phase 2 Trial for the Treatment of Alzheimer's Disease

Trial Designed to Demonstrate Target Engagement, in addition to Standard Safety and Pharmacokinetics

CLEVELAND, Feb. 4, 2025 /PRNewswire/ -- NeuroTherapia, Inc., a clinical-stage company focused on developing oral therapies for neurodegenerative diseases, announced it has received approval for its Phase 2 clinical trial from the European Medicines Agency (EMA). NTRX-07, the Company's lead molecule, will be administered to Alzheimer's disease (AD) participants for 28 days in this double-masked, randomized clinical trial. In addition to monitoring safety, pharmacokinetics and standard measures of clinical efficacy (ADAS-cog, MMSA and Trails Making Test), the trial is also designed to give an indication of target engagement by analyzing various biomarkers of neuroinflammation and neuronal function.

 "After seeing some encouraging trends in improving neuronal function in our Phase 1b trial, we are excited to begin this study to determine if these trends remain following a longer treatment duration and with more patients," commented Dr. Joseph Foss, the company's Chief Medical Officer. "Identification of biomarkers in the CSF that correspond to changes in neuroinflammation assessed with MRI or in improvements in neuronal function with quantitative EEG (qEEG) would demonstrate that NTRX-07 is providing AD patients with benefits of reducing neuroinflammation and improving neuronal function similar to what we have seen in preclinical studies."

The trial will be managed by CRU Global, a leading European CRO, and conducted at sites in Hungary, Poland, and the Czech Republic. An Investigators Meeting was in early January with representatives and Key Opinion Leaders from the experienced sites in EMA region. Sites are currently completing training for administrating qEEG and P300 ERP recordings, with patient enrollment expected to begin in February. The investigators expressed enthusiasm for participating in the trial and exploring the effects of NTRX-07 for their patients due to its novel mechanism of action and the data from the Phase 1b study.

"AD remains a devastating disease with few good treatment options," said Tony Giordano, Ph.D., NeuroTherapia's President and CEO. "We hope that this trial not only demonstrates the potential of our novel neuroinflammatory inhibitor to treat AD effectively, but by analyzing biomarkers, we might be able to identify specific biomarkers that show treatment response. These biomarkers could then be applied in subsequent trials to select patients who might best benefit from treatment with NTRX-07."

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 Ab peptide in the brain, and substantially improved neuronal synaptic plasticity, learning, and memory. Phase 1a and 1b trials have recently been completed, demonstrating that administration of the drug product is well tolerated at doses expected to be efficacious based on modeling of the preclinical efficacy data. The study is being funded in part by a grant from the Alzheimer's Association Part the Cloud program.

About NeuroTherapia

NeuroTherapia, Inc. is a clinical-stage, privately held biotechnology company, spun out of Cleveland Clinic, developing oral, small-molecule drugs to address neuroinflammatory conditions of the central nervous system (CNS), including Alzheimer's disease, Parkinson's disease, amyotrophic lateral sclerosis, and other CNS conditions. There is an emerging consensus that neuroinflammation plays a significant role in CNS disease. NeuroTherapia is developing therapeutics that modulate microglia cell activity, which is known to express proteins that drive inflammation in the CNS without affecting the clearance of toxic beta-amyloid. We believe this approach will result in improved neuronal function and survival. At NeuroTherapia, our mission is to improve  the course of the most debilitating and deadly CNS diseases that patients and their families face today. Additional information about NeuroTherapia can be found at www.neurotherapia.com.

SOURCE NeuroTherapia, Inc.