ASRS 2024: Neurotech Pharmaceuticals update on the NT-501 device

Thomas Aaberg, MD, gives an update on Neurotech Pharmaceuticals Encapsulated Cell Therapy platform for the potential treatment of Macular Telangiectasia Type 2, with a projected PDUFA goal date from the FDA at the annual ASRS meeting in Stockholm, Sweden.

Video Transcript:

Editor's note: The below transcript has been lightly edited for clarity.

Thomas Aaberg, MD:

Hello, I am Tom Aaberg. I am a practicing retina specialist in Grand Rapids, Michigan, and I'm the chief medical officer for Neurotech Pharmaceuticals. I'm at ASRS Stockholm 2024.

I'm excited to be here. On the first day of the program, Dr. Paul Bernstein and Dr. Jack Wells presented updates to our clinical program in Mactel 2. Mactel 2 is what is considered to be an orphan disease. It's actually a neurodegeneration of the retina, rather than its name implies, which would be a vascular cause. This is a progressive disease that affects the macula. It begins with paracentral scotomas. Patients notice metamorphopsia, blind spots, difficulty reading. Near tasks are particularly challenging for these patients. Unfortunately, it can be very subtle in its initial presentation, and is often either undiagnosed or misdiagnosed for years, which can lead to some obvious frustrations for the patient. OCT has been critical for these patients. It's been key in diagnosing Mactel. The advancements of OCT-A has even been more valuable for these patients

For those unfamiliar with Neurotech, we have a first-in-class encapsulated cell therapy. This innovative approach harnesses the power of allogenic retinal pigment epithelial cells that we can genetically engineer to produce a variety of therapeutic factors. Our NT-501 device, in particular, is designed to produce ciliary neurotrophic factor for the treatment of Mactel 2. As demonstrated by Dr. Wells and Dr. Bernstein, our NT-501 has been shown to be efficacious in 2 parallel Phase 3 trials and maintains a favorable safety profile. We've submitted our biologic license application to the FDA. They have granted us a PDUFA goal date of December 17, 2024. We at Neurotech are super excited... look forward to hopefully a positive outcome. We're also very excited for the potential benefit this could have for Mactel 2 patients and look forward to any future advances.