Neurotech Updates on NT-501 BLA for MacTel Treatment

Neurotech Pharmaceuticals, Inc., a pioneering company in the field of sustained drug delivery for chronic retinal diseases, has announced a significant update regarding its NT-501 (revakinagene taroretcel) treatment. The U.S. Food and Drug Administration (FDA) has extended the Prescription Drug User Fee Act (PDUFA) goal date by three months, now setting it to March 18, 2025. This extension is to allow the FDA additional time to review further data provided by Neurotech in response to their recent inquiries.

Richard Small, the CEO of Neurotech Pharmaceuticals, expressed the company's dedication to working with the FDA to ensure the completion of the NT-501's Biologics License Application (BLA) review process. The therapy, designed for the treatment of Macular Telangiectasia Type 2 (MacTel), is a crucial development in addressing this rare neurodegenerative disease that leads to gradual loss of central vision.

MacTel, specifically Macular Telangiectasia Type 2 or idiopathic juxtafoveal macular telangiectasia type 2, is characterized by localized retinal degeneration and subsequent changes in the retinal blood vessels. This condition typically impacts both eyes, resulting in a progressive decline in central vision, which is essential for tasks such as reading and recognizing faces.

Central to Neurotech's approach is their Encapsulated Cell Therapy (ECT) platform. This innovative system is designed to offer long-term, sustained delivery of therapeutic proteins to treat chronic retinal conditions. The ECT platform involves a small, semi-permeable capsule that houses genetically modified allogeneic retinal pigment epithelium (RPE) cells. These cells are engineered to produce specific therapeutic proteins aimed at treating targeted diseases. The capsule is surgically placed into the vitreous of the patient's eye and secured to the sclera during a minimally invasive outpatient procedure.

Once implanted, the capsule's semi-permeable membrane allows necessary nutrients to pass through while also enabling the therapeutic proteins to be released into the vitreous humor of the eye. This design provides continuous and targeted treatment to the retina. Additionally, the membrane shields the encapsulated RPE cells from the patient's immune system, ensuring their long-term survival and functionality.

The NT-501 implant utilizes the ECT platform to deliver Ciliary Neurotrophic Factor (CNTF), a potent neuroprotective protein, for treating chronic retinal diseases such as MacTel. CNTF plays a crucial role in promoting the survival and maintenance of photoreceptors, which are essential for vision. By providing sustained delivery of CNTF, NT-501 aims to slow the progression of retinal degeneration and potentially enhance long-term visual outcomes for patients suffering from MacTel.

Neurotech Pharmaceuticals, Inc. is a private, clinical-stage biotechnology company that focuses on innovative, sustained drug delivery solutions for chronic retinal diseases. Their core technology, ECT, is pivotal in enabling the continuous production of therapeutic proteins directly to the back of the eye, offering a promising approach to treating debilitating retinal conditions.