Small Molecule therapeutics for tauopathy for neurodegenerative diseases
The Problem
The Solution
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.
