Company targets Phase 2 trial of oral Alzheimer’s drug that inhibits the activity of microglia to combat neuroinflammation.
US biotech NeuroTherapia has secured $12.3 million in the first close of a Series B financing round to progress its mission to tackle central nervous system diseases, with an initial focus on Alzheimer’s. While many companies are targeting the removal of toxic proteins like beta amyloid, tau and alpha synuclein, the Cleveland Clinic spinout is addressing neuroinflammation, a factor increasingly recognized as crucial in the progression of CNS diseases.
NeuroTherapia’s focus is on developing orally available small-molecule drugs that inhibit the activity of microglia, immune cells in the CNS that contribute to neuroinflammation. By modulating these cells, the company aims to reduce inflammation without disrupting the clearance of toxic proteins, thereby improving neuronal function and survival.
Microglia, the central immune cells in the brain, can exist in two states: the neuroprotective M2 phenotype, which helps clear amyloid β, and the neurotoxic M1 phenotype, which contributes to inflammation and synaptic damage. In Alzheimer’s disease and other neurodegenerative conditions, there is an increased prevalence of the M1 state, exacerbating the disease.
NeuroTherapia’s lead candidate, NTRX-07, is designed to activate the cannabinoid type 2 (CB2) receptor, promoting the transition of microglia from the harmful M1 state to the beneficial M2 state. This mechanism is expected to reduce neuronal damage, enhance the clearance of amyloid β, and inhibit neuroinflammation, which could ultimately slow or reverse the progression of Alzheimer’s.
The new funding will primarily support the continued clinical development of NTRX-07, including advancing it through a Phase 2a trial to further investigate the drug’s potential to inhibit neuroinflammation and improve cognitive biomarkers in patients with Alzheimer’s. Preliminary results from a Phase 1b clinical trial have already shown promising outcomes, including a favorable safety profile, pharmacokinetics consistent with preclinical expectations, and a trend toward cognitive improvement in treated patients.
The funding round was led by Cleveland Clinic Innovations, with participation from the Brain Trust Accelerator Fund II, Dolby Family Ventures, and the Alzheimer’s Drug Discovery Foundation (ADDF), Foundation for a Better World and CRUINT.
“We were pleased with the Phase 1b trial results that demonstrated a trend toward cognitive benefits in Alzheimer’s disease and wanted to continue its development as rapidly as possible,” said Joseph Rich, senior director at Cleveland Clinic Innovations. “We believe that the company’s planned Phase 2a trial has the potential to not only demonstrate the ability of NTRX-07 to inhibit neuroinflammation, but it could also lead to an improvement in biomarkers of cognitive function in patients with Alzheimer’s disease.”
In addition to progressing NTRX-07, NeuroTherapia is also exploring the drug’s potential in a preclinical model of amyloid-related imaging abnormalities (ARIA), a significant side effect associated with therapeutic monoclonal antibodies used in Alzheimer’s treatment. The company hypothesizes that NTRX-07 could mitigate or prevent ARIA, potentially enhancing the efficacy and safety of monoclonal antibody therapies.
NeuroTherapia is also planning for the development of second-generation molecules targeting other conditions linked to neuroinflammation, such as Parkinson’s disease, amyotrophic lateral sclerosis (ALS), and pain.