NodThera announces positive inflammatory disease data


NodThera has announced positive data from the first human studies of its lead therapy candidates, NT-0249 and NT-0796.

In the study, both candidates were shown to unequivocally inhibit the NLRP3 inflammasome, a highly validated drug target that plays an important role in controlling inflammatory disease. The distinct design characteristics of each candidate allow them to penetrate different brain areas for optimal drug distribution in various NLRP3-driven diseases.

Brain penetration and anti-inflammatory effects in both clinical programs

Data from the recently completed multiple-ascending dose (MAD) cohort of the first human study NT-0249 confirmed a potentially best-in-class pharmacokinetic/pharmacodynamic (PK/PD) profile suitable for once-daily dosing. NT-0249 exhibited significant anti-inflammatory effects in healthy volunteers, with reductions in the main inflammatory biomarkers, C-reactive protein (CRP) and fibrinogen, which were maintained throughout treatment. The level of NT-0249 measured in cerebrospinal fluid (CSF) also indicates a high level of brain penetration.

Findings from the first completed human study of NT-0796, originally disclosed in 2022, also confirm its excellent PK/PD profile, brain penetration and anti-inflammatory effects in healthy volunteers.

Both candidates were well tolerated, most of the treatment effects were mild and there were no serious side effects (SAE).

The Parkinson’s disease program is run by NodThera

Development of NT-0796 is now progressing in a pilot phase Ib/IIa study that is rich in biomarkers in Parkinson’s disease. This study explored the candidate’s effects on inflammation and disease-specific biomarkers in blood and CSF using a panel of clinical biomarkers, designed using NT-0796’s preclinical profile on cytokines, chemokines, and microgliosis and astrogliosis markers relevant to NLRP3 inhibition.

Early-stage studies, in healthy elderly volunteers, are ongoing, investigating modified formulations of drug candidates designed for use in future patient research arms.

Alan Watt, chief executive officer of NodThera, said: “As the burden of non-communicable diseases continues to increase globally, targeting chronic low-grade inflammation, through selective modulation of NLRP3 inflammation, has great potential for the treatment of this disease. Our strategy to design highly differentiated brain penetrant molecules, which combines deep understanding of NLRP3 inhibition, pharmaceutical neuroscience expertise, and precision molecular design, holds promise that modulating NLRP3 inflammation can change the treatment paradigm for chronic peripheral and neurodegenerative diseases.

“The outstanding clinical data from these two clinical candidates reinforces our belief that NodThera has the clinical tools to address this challenge.”


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