NodThera has announced positive results from its Phase Ib/IIa clinical trial of NT-0796, an oral, brain-penetrant nucleotide-binding domain, leucine-rich–containing family, pyrin domain–containing-3 (NLRP3) inflammasome inhibitor, in patients with Parkinson’s disease.
The study focused on assessing the effects of NT-0796 on inflammatory and disease-specific biomarkers in blood and cerebrospinal fluid (CSF).
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It demonstrated a mean reduction in key pro-inflammatory biomarkers in the CSF over 28 days versus baseline, with levels nearing those of healthy elderly controls. This data suggested a reversal of NLRP3-mediated neuroinflammation.
Additionally, decline in neurodegenerative markers, such as neurofilament light chain (NfL) and soluble triggering receptor expressed on myeloid cells 2 (sTREM2), were observed after oral dosing of NT-0796.
In subjects with higher acute phase biomarkers c-reactive protein (CRP) and fibrinogen, significant reductions were noted, aligning with the peripheral anti-inflammatory effects of NT-0796 seen in elderly healthy subjects in an earlier phase of the study.
Furthermore, NT-0796 was found to be safe and well-tolerated, with most adverse events being mild and transient in nature.
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By GlobalDataNo serious adverse events were reported in the trial.
Pharmacokinetic data suggested the potential for once-daily dosing of NT-0796, with drug levels in the brain, measured by CSF concentration, maintained over 24 hours.
These findings indicate that NT-0796 can deliver anti-neuroinflammatory and anti-inflammatory changes within seven days, which lasted for 28 days, showing great potential for long-term oral treatment in Parkinson’s disease patients.
NodThera’s ongoing Phase Ib/IIa clinical study of NT-0796 in obese subjects with cardiovascular risk is expected to yield results by the end of the second quarter this year.
This trial is assessing the change from baseline to day 28 in CRP levels, a peripheral inflammatory marker and known cardiovascular disease risk predictor, as well as the potential impact on body weight over the same period.
NodThera CEO Alan Watt said: “This is the inaugural demonstration of an NLRP3 inhibitor’s potential to not only address Parkinson’s disease but also offer a broader impact on neurodegenerative diseases. Given that existing Parkinson’s treatments primarily manage symptoms, our innovative, disease-modifying strategy presents a significant shift, aiming to stop the disease progression.
“NT-0796’s demonstrated efficacy in reducing neuroinflammation in patients heralds a substantial advancement towards halting this devastating disease.”
