Amydis launches phase 1/2a trial to evaluate novel retinal tracer in patients with ALS, PD

Amydis Inc. announced that patient enrollment in underway for a phase 1/2a trial to evaluate AMDXP-2011P, a retinal imaging agent that targets protein biomarkers in patients with Parkinson’s disease and ALS.

According to a company press release, the “retinal tracer” is a fluorescent, nonradioactive imaging agent — designed to be used with currently available ocular imaging devices — that detects and quantifies deposits of alpha synuclein (ASYN) in patients with PD and TAR DNA-binding protein 43 (TDP-43) in patients with ALS. There are currently no approved diagnostics to detect these biomarkers, the company said.

“Beginning clinical evaluation of the first compound from our broad portfolio of ocular tracers marks a significant milestone in our efforts to serve the unmet needs of people with PD and ALS,” Amydis CEO and founder Stella Sarraf, PhD, said in the release. “AMDX-2011P is the first imaging agent to be investigated for direct visualization of ASYN and TDP-43 biomarkers in the retina, an accessible part of the CNS that can be imaged noninvasively at micron-level resolution.”

The PROBE trial is a prospective, randomized, blinded endpoint study designed to examine the safety, tolerability, pharmacokinetics and activity of a single IV bolus of AMDXP-2011P in patients with PD and ALS, the release stated. The trial will be conducted at two sites in Southern California.

“This is an important step forward in the field of ALS,” Merit Cudkowicz, MD, MSC, chief of neurology and director of the Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital and member of the safety and monitoring committee for PROBE. “To my knowledge, exploring a diagnosis of ALS through the eye is a novel and unique approach that may help us identify people at risk earlier in the disease and potentially help with drug development.”