Small Molecule Therapeutics for Tauopathy for Neurodegenerative Diseases
Our data-driven platform integrates high-throughput analytics and AI for next-generation drug discovery. We leverage novel insights into the foundational biologic pathways of neurodegenerative diseases to discover, evaluate, and develop disease-modifying therapeutics for Alzheimer’s disease, frontotemporal dementia (FTD) , amyotrophic lateral sclerosis (ALS), and other degenerative diseases of the nervous system.
The Problem
Neurodegenerative diseases, like Alzheimer’s disease, are a growing global health challenge as people live longer. These diseases are caused by harmful protein clumps, energy failures in brain cells, and chronic inflammation, leading to worsening memory, thinking, and movement problems. Unfortunately, current treatments only manage symptoms without slowing the disease. There is a critical need for new therapies that target these root causes, offering a real chance to slow or stop disease progression and improve patients’ lives.
The Solution
Tau and TDP-43 proteins are key proteins of the pathological neurodegenerative process manifesting through the presence of toxic clumps in brains of patients. Our novel orally available, brain-penetrant small molecules that prevent these proteins from forming harmful aggregates. Preclinical studies in animal models and human neurons demonstrate strong efficacy, showing that these compounds selectively eliminate neurotoxic aggregates of tau and TDP-43 while preserving their essential normal functions. Our insights in the fundamental functioning of these compounds allow us to position them as promising disease-modifying therapies for tau- and TDP-43-associated neurodegenerative diseases.
The Opportunity
The global neurodegenerative disease market was valued at approximately USD 59.06 billion in 2025 and is projected to reach USD 83.37 billion by 2030, reflecting a compound annual growth rate of 7.14% during this period. Currently, there are no FDA-approved treatments specifically targeting tau or TDP-43 pathologies, which are implicated in all major neurodegenerative diseases. The development of a novel small molecule targeting these proteins presents a significant opportunity to address this unmet medical need. Such a therapeutic could potentially qualify for orphan drug designation, expediting clinical development and regulatory approval, particularly for conditions like FTD and ALS. Moreover, the mechanism of action of this small molecule offers the potential for expansion into other neurodegenerative indications, thereby broadening its therapeutic application and market reach.
Status
We have performed comprehensive pharmacokinetic, safety pharmacology, and efficacy studies in animal models. In human neuronal models, our novel compounds have demonstrated strong safety and therapeutic efficacy combined with a favorable risk/benefit profiles. Preliminary non-human primate studies further validate the safety of our compounds, demonstrate their ability to reduce disease-related biomarkers, and confirm target engagement, strongly supporting their translational potential and suitability for oral administration in humans. With these promising results, our novel therapeutics are well-positioned to enter Phase I clinical development and we are getting ready to engage in discussions with the FDA.
Meet the Team
Xinglong Wang PhD
College of Pharmacy
Peter Nestler PhD
Tech Launch Arizona
Contact us today.
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