Amydis Inc., a privately held clinical-stage company pioneering a platform of ocular tracers that enable imaging of disease-related molecular biomarkers in the eye, announced a Phase 2 grant award of $2.5 million from the National Institute on Ageing at the National Institutes of Health (NIH). The award supports Amydis' novel approach to address a critical gap in medicine: the lack of a diagnostic test for TAR DNA-binding protein 43 (TDP-43) associated with neurodegenerative diseases such as amyotrophic lateral sclerosis (ALS), frontotemporal dementia (FTD), and Limbic-predominant Age-related TDP-43 Encephalopathy (LATE).
Approximately 97 per cent of people diagnosed with ALS have TDP-43 pathology, making it a central biological hallmark of the disease. Yet today, there is no blood test or brain scan that can directly detect TDP-43 in living patients. ALS remains a diagnosis of exclusion, often taking months or even years due to overlapping symptoms with other conditions—time that patients and families cannot afford to lose. Earlier diagnosis could accelerate access to therapeutic intervention and clinical trials, enabling more opportunities to impact the disease
To address this unmet need, Amydis is developing a non-invasive, fluorescent ocular tracer designed to function as a simple eye test capable of detecting TDP-43 using routine ophthalmic imaging technologies and workflows. Because patients routinely visit eye care providers when they notice vision changes, this approach has the potential to integrate molecular biomarker detection of neurodegenerative disease into everyday clinical practice.
"For patients and families facing ALS and related dementias, time is everything," said Dr. Stella Sarraf, Founder and CEO of Amydis. "We believe the eye represents a new frontier in neurodegenerative disease detection. Earlier detection has the potential to change how ALS and related diseases are diagnosed, studied, and ultimately treated."
The Phase 2 grant award was based on the successful completion of a Phase 1 NIH grant where Amydis demonstrated its proprietary fluorescent tracer can detect TDP-43 deposits in the retinal cadaver tissues of people with ALS, FTD and LATE. This foundational work was made possible with support from Target ALS and through collaborations with Georgetown University, Washington University in St. Louis, Mass General Brigham, Cedars-Sinai and Banner Health. The results were recently presented at the NEALS conference in November and the International Symposium on MND/ALS in December. A manuscript is being prepared for submission to a peer-reviewed journal.
This new Phase 2 grant supports continued analysis of retinal cadaver tissues to further characterise and map TDP-43 deposits, applying artificial intelligence to define disease-specific patterns across ALS, FTD, and LATE, to distinguish these conditions at a molecular level for use in precision medicine.
"Prodromal detection of TDP-43 in people with ALS would be a step forward for our field," said Dr. Merit Cudkowicz, Executive Director of the Mass General Brigham Neuroscience Institute and Director of the Sean M. Healey & AMG Centre for ALS. "A molecular biomarker test to detect TDP43 in the eye has the potential to facilitate clinical trial design and drug targeting, as well as accelerate patient enrollment in clinical trials, providing earlier access to promising investigational therapies."
