Prothena presents TDP-43 data at neuroscience conference

Prothena Corporation plc (NASDAQ: PRTA) announced it will present preclinical data on its TDP-43 CYTOPE program at the Neuroscience 2025 conference in San Diego on November 19.

The poster presentation will detail results from studies using the company's CYTOPE drug delivery technology, which aims to deliver large molecules into cells to target intracellular disease pathways. The technology uses an endosomal escape mechanism designed to preserve membrane integrity following systemic administration.

In a transgenic mouse model of ALS expressing human mutant TDP-43, systemically administered TDP-43 CYTOPE distributed to the brain and internalized into the cytosol. The treatment reduced intracellular pTDP-43 pathology in the motor cortex and neuromuscular junction of the rNLS8 mice, according to the company's statement.

Laboratory studies using rat and human-derived neuronal cell lines and human iPSC motor neurons showed the TDP-43 CYTOPE internalized into the cytosol and colocalized with cytosolic pTDP-43 aggregates. The treatment promoted clearance of these aggregates and reduced RNA dysregulation caused by cryptic exon inclusions, which are pathogenic features associated with ALS and other TDP-43 proteinopathies.

The presentation is scheduled for 1 p.m. PT during the session titled "ALS and Motor Neuron Disease: Human Studies and Preclinical Therapy Development."

Prothena develops investigational therapeutics for neurodegenerative and rare peripheral amyloid diseases. The company's pipeline includes programs for ATTR amyloidosis with cardiomyopathy, Alzheimer's disease, and Parkinson's disease.

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