FDA Approves Neurotech's ENCELTO™ for MacTel Treatment

Neurotech Pharmaceuticals, Inc., a biotech company specializing in therapies for chronic eye conditions, has achieved a significant breakthrough with the approval of ENCELTO™ by the U.S. Food and Drug Administration (FDA). This development marks the first FDA-approved treatment for Macular Telangiectasia Type 2 (MacTel), a serious neurodegenerative retinal disease in adults that leads to progressive vision loss.

MacTel is characterized by the gradual degeneration of retinal cells, ultimately affecting a person's vision and quality of life. ENCELTO employs an innovative encapsulated cell therapy technology, which enables continuous delivery of ciliary neurotrophic factor (CNTF) to the retina. This approach aims to slow the progression of the disease by preserving the macular photoreceptors essential for vision. The approval of ENCELTO is based on successful outcomes from two phase 3 trials, which showed that the treatment notably slowed down the deterioration of vision in patients over a 24-month period.

Available in the United States starting June 2025, ENCELTO is designed to offer new hope to those affected by MacTel. Richard Small, CEO of Neurotech Pharmaceuticals, expressed his gratitude to all participants and teams involved in the clinical studies, highlighting this milestone as a significant achievement for the retina community and the company.

Dr. Charles C. Wykoff, a clinical investigator, emphasized the potential positive impact of ENCELTO on patients, noting that having an approved treatment can help preserve functional vision and thus improve patients' quality of life. Similarly, Dr. Thomas M. Aaberg Jr, Chief Medical Officer, underscored the historic nature of this approval for the MacTel community, as it provides the first therapeutic option for individuals grappling with this vision-threatening disease.

Neurotech's encapsulated cell therapy (ECT) platform is at the heart of this innovation. The ECT system involves a small, surgically-implanted capsule containing genetically modified retinal pigment epithelium (RPE) cells. These cells are engineered to produce and deliver therapeutic proteins directly to the retina. The semi-permeable membrane of the capsule allows necessary nutrients to enter while enabling the therapeutic proteins to exit and reach the retina. This design also protects the implanted cells from the body's immune response, ensuring their longevity and effectiveness.

MacTel itself is a bilateral, degenerative condition that wreaks havoc on retinal cells and disrupts the retinal vasculature. Over time, this leads to significant vision impairment as the retinal cells and vasculature deteriorate. The introduction of ENCELTO is poised to change the landscape of treatment for this condition by offering a means to slow its progression, thereby preserving vision for many patients.

Neurotech Pharmaceuticals is committed to advancing this pioneering technology to address chronic eye diseases and improve patient outcomes. This company's core ECT platform holds promise not only for treating MacTel but potentially for other chronic ocular diseases as well. As research and development continue, the focus remains on delivering innovative therapies that transform the standard of care for individuals suffering from debilitating eye conditions.