– Lead Candidate NT-0796 Achieves Positive Interim Results from Phase 1 Study, Supporting Further Progression for Treatment of a Range of Inflammatory Diseases –
-Second Lead Candidate NT-0249 Advances to First-in-Human Phase 1 Study to Enable Clinical Development in Chronic Peripheral Inflammatory Disease-
-Third candidate NT-0527 announced as a novel brain-penetrating NLRP3 inflammasome inhibitor progressing in IND-enabled studies-
LEXINGTON, Massachusetts., May 10, 2022 /PRNewswire/ — Nod Thera, a clinical-stage biotechnology company developing a novel class of potent and selective small molecule oral inflammasome inhibitors NLRP3 to treat chronic inflammation-mediated diseases, today announced several key advances in the entire portfolio. NodThera’s lead candidate, NT-0796, has demonstrated positive interim results from its phase 1 single escalating dose (SAD) study. Additionally, the company has commenced first-in-man dosing in the Phase 1 study of its second lead candidate, NT-0249, and announced the selection of its third candidate, NT-0527 – an inhibitor of NLRP3 inflammasome entering the brain of a new chemotype.
Positive interim results from the SAD portion of the Phase 1 trial with NT-0796 represent early clinical evidence for the mechanism of NT-0796 as a potent NLRP3 inflammasome inhibitor. In all dosing cohorts, NT-0796 was safe and well tolerated and was shown to be orally bioavailable with a dose-proportional pharmacokinetic (PK) profile. This part of the study also showed a dose-dependent pharmacodynamic (PD) effect through the ability to reduce IL-1β and IL-18 levels in a ex-vivo NLRP3 stimulation test. These results confirm the criteria to advance the development of NT-0796 and continue the ongoing multiple ascending dose (MAD) portion of the Phase 1 study to assess brain exposure by cerebrospinal fluid (CSF) collection ).
“NT-0796 has demonstrated strong evidence of mechanism and translation from preclinical to human studies, further validating and de-risking the development of NT-0796 as an oral small molecule NLRP3 inflammasome inhibitor. potentially best-in-class,” said NodThera Chairman and CEO, Adam Keney. “We are encouraged by these first-in-man results as we strive to advance NT-0796 in inflammatory diseases affecting millions of patients, many of whom have few or no treatment options.
Building on the successful clinical progress of NT-0796, NodThera has also initiated dosing of the first healthy volunteers in the Phase 1 trial of the company’s second lead candidate, NT-0249. NT-0249 is a potent NLRP3 inflammasome inhibitor with favorable developmental characteristics supporting the advancement to treat chronic inflammatory diseases of the body. The primary objective of this study will be to assess the safety and tolerability of NT-0249, with secondary objectives to assess PK and PD (ability to lower levels of IL-1β and IL-18 ) after single and multiple ascending doses.
In addition to advancing two new candidates into clinical trials, NodThera is further expanding its diverse portfolio with the announcement of NT-0527 as the third oral small molecule NLRP3 inflammasome inhibitor of a new chemotype to be added to its pipeline. NT-0527 is specifically designed to inhibit the NLRP3 inflammasome in the brain, with the potential to treat a wide range of neuro-inflammatory diseases.
“Each of the three candidates in NodThera’s portfolio exhibit unique chemotypes that are distinct from each other, providing specific benefits that can be used collectively to cover a range of inflammatory diseases of the brain and body,” says NodThera’s Chief Scientific Officer. , Alan Watt. “With the recent addition of NT-0527 as a third candidate in our growing pipeline, NodThera is now advancing novel CNS penetrating and peripherally restricted NLRP3 inflammasome inhibitors with differentiated chemistry like no other. portfolio in the field.”
About Nod Thera
NodThera is a clinical-stage biotechnology company developing a new class of potent and selective NLRP3 inflammasome inhibitors for the treatment of diseases mediated by chronic inflammation. Led by an experienced management team, NodThera is leveraging new insights into inflammasome biology and chemistry to build a portfolio of highly differentiated small molecule NLRP3 inflammasome inhibitors. The company was founded in 2016 by Epidarex Capital and financed by 5am Ventures, Cowen Healthcare Investments, Epidarex Capital, F-Prime Capital, Novo Holdings, Sanofi Ventures and Sofinnova Partners. NodThera is headquartered in Lexington, MAwith additional locations in Cambridge, United Kingdom and Seattle, WA. Learn more about www.nodthera.com or follow us on LinkedIn.
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