NodThera Announces Clinical Progress for Lead NLRP3 Inflammasome Inhibitors and Candidate Selection of Novel Brain-Penetrant Compound

-Lead candidate NT-0796 achieves positive interim results from Phase 1 study, supporting further progression for the treatment of a range of inflammatory diseases-

-Second lead candidate NT-0249 advances into first-in-human Phase 1 study to enable clinical development in peripheral chronic inflammatory disease-

-Third candidate NT-0527 is announced as a novel brain-penetrant NLRP3 inflammasome inhibitor advancing through IND-enabling studies-

Excerpt from the Press Release:

LEXINGTON, Mass., May 10, 2022 /PRNewswire/ — NodThera, a clinical-stage biotechnology company developing a new class of potent and selective oral, small molecule NLRP3 inflammasome inhibitors to treat diseases driven by chronic inflammation, today announced several key advancements across the portfolio. NodThera’s lead candidate, NT-0796, demonstrated positive interim results from its Phase 1 single-ascending dose (SAD) study. Additionally, the company has commenced first-in-human dosing in the Phase 1 study of its second lead candidate, NT-0249, and announced the selection of its third pipeline candidate, NT-0527 – a brain-penetrant NLRP3 inflammasome inhibitor from a novel chemotype.

The positive interim results from the SAD portion of the Phase 1 trial with NT-0796 represent early clinical proof of mechanism for NT-0796 as a potent NLRP3 inflammasome inhibitor. Across all dosing cohorts, NT-0796 was safe and well tolerated and shown to be orally bioavailable with a dose-proportional pharmacokinetic (PK) profile. This portion of the study also showed a dose-dependent pharmacodynamic (PD) effect through the ability to lower IL-1β and IL-18 levels in an ex vivo NLRP3-stimulation assay. These results confirm the criteria to advance NT-0796 further in development and continue the ongoing multiple-ascending dose (MAD) portion of the Phase 1 study to assess brain exposure through cerebrospinal fluid (CSF) sampling.

“NT-0796 has demonstrated robust proof of mechanism and translation from preclinical studies to humans, both validating and further de-risking the development of NT-0796 as a potentially best-in-class, oral, small molecule NLRP3 inflammasome inhibitor,” said NodThera’s Chief Executive Officer, Adam Keeney. “We are encouraged by these first-in-human results as we work to progress NT-0796 in inflammatory diseases impacting millions of patients, many with limited to no treatment options.”

Click the button below to read the entire Press Release:

Continue Reading The Press Release