Cumberland, R.I., March 6, 2025 – Neurotech Pharmaceuticals, Inc., a private biotech company focused on developing transformative therapies for chronic eye diseases, announced that the U.S. Food and Drug Administration (FDA), has approved ENCELTOTM (revakinagene taroretcel-lwey) for the treatment of Macular Telangiectasia type 2 (MacTel).
MacTel is a neurodegenerative disease of the retina in adults that causes progressive and irreversible vision loss, significantly impacting patients' quality of life. ENCELTO utilizes an encapsulated cell therapy technology designed to continually deliver therapeutic doses of ciliary neurotrophic factor (CNTF) to the retina to assist in slowing the progression of the disease. ENCELTO is the first and only FDA-approved treatment available for MacTel.
The approval was based on results from two phase 3 trials which demonstrated that after placement, of the implant, ENCELTO significantly slowed the loss of macular photoreceptors in MacTel patients over 24 months.
ENCELTO is expected to be available in the United States for patients starting in June of 2025.
“Today marks an extraordinary milestone for patients, the retina community, and Neurotech,” said Richard Small, Chief Executive Officer. “I would like to express my gratitude to clinical study participants, clinical investigators and their teams, and the entire Neurotech organization who have helped make this a reality.”
“I have seen the impact that MacTel can have on patients and their quality of life,” said Charles C.Wykoff, MD, PhD, Retinal Consultants of Texas, Houston, TX, a clinical investigator. “Now with an FDA-approved treatment, I am confident that ENCELTO will be able to meaningfully slow disease progression for many patients affected by MacTel, allowing them the opportunity to preserve more functional vision over time.”
"This is a historic moment for the MacTel community, as ENCELTO becomes the first-ever FDA-approved treatment for this vision-threatening disease,” said Thomas M. Aaberg Jr, MD, Chief Medical Officer. “For those who have been affected by MacTel and for all who have supported this journey, today we look forward to a future where vision loss from MacTel may be slowed."
About Macular Telangiectasia Type 2 (MacTel)
Macular Telangiectasia Type 2 (MacTel), or idiopathic macular telangiectasia type 2, is a bilateral, neurodegenerative disease in adults with characteristic localized retinal degeneration, a group of diseases that cause the gradual loss of cells in the retina, resulting in vision loss and secondary alterations of the retinal vasculature, the network of blood vessels that supplies oxygen and nutrients to
the retina.1
About Encapsulated Cell Therapy (ECT)
Neurotech’s ECT platform is a cell-based gene therapy delivery system designed to provide long-term,sustained delivery of therapeutic proteins for the treatment of chronic eye diseases. This versatile platform consists of a small, semi-permeable capsule containing proprietary allogeneic retinal pigment epithelium (RPE) cells genetically engineered to produce specific therapeutic proteins for targeted disease treatment. The capsule is surgically implanted. Once in place, the capsule's semi-permeable exterior membrane allows essential nutrients to enter, while also permitting the therapeutic proteins to exit into the eye where they can travel to the retina located at the back of the eye. The exterior membrane protects the encapsulated RPE cells from the host's immune system, contributing to their survival and functionality over time.
About Neurotech Pharmaceuticals, Inc
Neurotech Pharmaceuticals, Inc. is a private biotech company focused on developing transformative therapies for chronic eye diseases. The core platform technology, Encapsulated Cell Therapy (ECT) is a first in class, drug delivery platform designed to slow the progression of MacTel and other chronic eye diseases. For more information about us, please visit https://www.neurotechpharmaceuticals.com.
1.Charbel Issa P, Gillies MC, Chew EY, Heeren TFC, Bird AC, Peto T, Holz FG, Scholl HPN (2013) Macular Telangiectasia Type 2.