Amydis announced the successful completion of a pre-IND (investigational new drug) meeting with the FDA regarding the development plans for a small-molecule tracer to detect TDP43 in the retina of amyotrophic lateral sclerosis (ALS) patients.
The FDA agreed with the proposed phase 1/2a first-in-human clinical research in ALS patients. Amydis intends to begin the clinical trial in the first half of 2022.
The proposed study's design may allow for an early read-out on the retinal tracer's potential ability to aid in the diagnosis and management of ALS patients.
ALS is a neurological illness that causes paralysis and death within three to five years of diagnosis. There are currently no objective diagnostics for detecting, quantifying, and monitoring TDP43, the most distinguishing biomarker protein linked with ALS.
TDP43 deposits are assumed to be a primary cause of disease pathogenesis in 97% of ALS patients and 50% of Frontal Temporal Dementia (FTD) cases, which has genetic roots with ALS.
“We are thrilled to have FDA’s clear guidance on the development path to introduce our novel retinal tracer to address the unmet need in ALS,” Stella Sarraf, PhD, Founder and Chief Executive Officer of Amydis, said in a company news release.
“The potential benefits of a retinal test for ALS to visualize TDP43 with commercially available and routinely used ophthalmic imaging devices represents a potentially game-changing breakthrough in clinical care of ALS patients. This may also aid in the clinical development of next generation therapeutics for this devastating disease through enhanced patient recruitment and monitoring.”
In collaboration with Target ALS, a non-profit medical organization committed to the search for new ALS treatments, Amydis was the first to investigate whether TDP43 appears in the retina, which is often regarded as a "window to the brain" due to its accessibility.
Dr. Merit Cudkowicz, Director of the Sean M. Healey and AMG Center for ALS at Mass General Hospital, commented, “To my knowledge, exploring a diagnosis of ALS through the eye is a novel and unique approach and we are excited to collaborate with Amydis in their future ALS clinical trial.”