Oligomerix, Inc., a privately held company pioneering the development of small molecule therapeutics targeting tau for Alzheimer's disease (AD) and related neurodegenerative disorders, announced that the company presented efficacy data for its lead program, a small molecule inhibitor of tau self-association, in two preclinical models of tauopathy. The findings were presented at the TAU2020 Global Conference on Wednesday, February 12, 2020.
Tau aggregation and the aggregated protein's evolution into neurofibrillary tangles in the brain of affected individuals is one of the hallmarks of AD. Abnormal tau pathology is also associated with several other neurodegenerative diseases.
To date, the majority of clinical studies for AD have focused on targeting amyloid beta and they have almost all failed in the clinic. Unlike the clinical failures seen to date with A? or other approaches, tau load and distribution have been shown to directly correlate with disease progression. Despite the promise of targeting tau to treat AD and other neurodegenerative diseases, there are surprisingly few programs in clinical development, and those that are in the clinic are focused on intervening after tau proteins have misfolded and oligomers have formed.
James Moe, Ph.D., MBA, President and CEO of Oligomerix, presented data showing that Oligomerix's lead compound reduced soluble tau self-association levels and caused a dose-dependent reduction of insoluble tau aggregates and phosphorylated insoluble tau aggregates in the brains of htau mice, which represents tauopathy in AD. The drug also showed dose-dependent reduction of self-associated tau, insoluble tau aggregates and phosphorylated tau in JNPL3 mice, which represents an inherited form of tauopathy.
A study by researchers from Oligomerix and the Feinstein Institutes for Medical Research demonstrating the compound's efficacy in the htau mouse model was recently published in the Journal of Alzheimer's Disease.
"These results in two separate models of tauopathy further validates Oligomerix's approach for inhibiting tau self-association, the initial step in tau oligomer formation, to block the downstream formation of larger aggregates," commented Dr. Moe. "As we continue conducting IND-enabling studies for this compound and develop an accompanying translational biomarker, we hope to bring the drug into human clinical trials in 2021," Dr. Moe added.
About Oligomerix's Lead Program
Oligomerix's lead candidate is a small molecule inhibitor of tau self-association and targets the beginning of the tau aggregation cascade. The activity of the drug translated from in vitro and cell assays to animal studies, validating the company's screening approach. In vivo proof-of-concept studies demonstrated compound efficacy in two transgenic mouse models with tau pathology representative of AD and frontotemporal dementia. In a study recently published in the Journal of Alzheimer's Disease, Oligomerix and the Feinstein Institutes for Medical Research reported that the compound inhibited hippocampal self-associated tau in the htau mouse model of tauopathy which expresses the six CNS isoforms of the human tau protein. Initial preclinical safety studies show the drug is well tolerated with no adverse events or behavioral abnormalities observed. The compound is being developed with an accompanying novel biomarker.
About Oligomerix, Inc.
Oligomerix is an emerging biotechnology company focused on developing disease-modifying therapeutics for Alzheimer's disease and related dementias by targeting tau self-association. The company's drug discovery platform has identified a pipeline of novel small molecule inhibitors, with preclinical IND-enabling studies in progress for the lead program. Oligomerix's small molecule approach blocks the formation of all tau aggregates by targeting tau self-association, known as the beginning of the aggregation cascade, whereas other companies have focused on targeting large tau aggregates formed downstream. The NYC-based company is located at the Ullmann Research Center for Health Sciences within the Albert Einstein College of Medicine.
Oligomerix is seeking strategic partners to support the acceleration and advancement of these important programs. For more information about Oligomerix, please visit http://www.oligomerix.com.
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