CNTF

In macular telangiectasia type 2, small blood vessels at the center of the retina dilate and, in some cases, new blood vessels form under the retina, damaging patients' central vision and potentially leading to loss of detail vision. With a U.S. green light for its encapsulated cell therapy technology in the bag, privately held biotech Neurotech Pharmaceuticals is on the verge of making its commercial debut.The FDA last week approved (PDF) Neurotech’s cell therapy implant revakinagene taroretcel to treat the vision-robbing neurodegenerative disease macular telangiectasia (MacTel) type 2.The drug, which will adopt the brand name Encelto, is the first to win the FDA’s blessing in the rare vision disorder, Neurotech said in a release.In MacTel type 2, small blood vessels at the center of the retina dilate and, in some cases, new blood vessels form under the retina in a process known as neovascularization, according to the American Academy of Ophthalmology. Those blood vessels subsequently bleed or leak fluid, causing the light-sensitive portion of the retina, the macula, to swell or thicken, affecting patients’ central vision.MacTel type 2, which primarily occurs in middle-aged adults, can also lead to loss of detail vision. The type 2 version of the disease affects both eyes, though not always with the same severity. Encelto works by utilizing Neurotech’s proprietary encapsulated cell therapy (ECT) platform to continually deliver therapeutic doses of the protein ciliary neurotrophic factor straight to a patient’s retina, which is in turn expected to slow the progression of MacTel.Neurotech describes its ECT platform as a cell-based gene therapy delivery system designed to provide therapeutic proteins to patients with chronic eye diseases.The platform itself is made up of a small, semipermeable capsule loaded with allogeneic retinal pigment epithelium (RPE) cells that have been genetically engineered to produce therapeutic proteins.After the capsule is implanted via a surgical procedure, its semipermeable exterior allows essential nutrients to enter while therapeutic proteins can escape and travel to the retina at the back of the eye, Neurotech explained in its Thursday approval announcement.While Encelto currently stands alone against MacTel type 2, the drug boasts a clear convenience edge over other eye disorder drugs, which are often given via repeat injections or refillable delivery system implants. The FDA based its approval decision on the readouts of two late-stage trials that found Encelto significantly slowed the loss of macular photoreceptors in MacTel patients over a 24-month span, Neurotech said.In results from a pair of replicative phase 3 trials issued in late 2022, Neurotech reported that Encelto—then coded as NT-501—curbed the rate of MacTel progression by 56.4% and 29.2% compared to a simulated treatment through 24 months."This is a historic moment for the MacTel community, as Encelto becomes the first-ever FDA approved treatment for this vision-threatening disease,” Neurotech’s chief medical officer, Thomas Aaberg Jr., M.D., said in a statement. “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."Encelto is slated to become Neurotech’s first commercial product. The company plans to launch the product in the U.S. starting in June. 

Protein therapies for the eye must be dosed as frequent injections. Gene therapy offers one-time treatment, but must also be administered by injection. Neurotech Pharmaceuticals’ gene therapy comes in a surgical implant, and it’s now the first FDA-approved treatment for a rare vision-loss disorder. The approval handed out on March 5 covers the treatment of macular telangectasia type 2 (MacTel) in adults. The Neurotech therapy, known in development as revakinagene taroretcel, will be marketed under the brand name Encelto. There are two types of MacTel. In type 2, tiny blood vessels in the center of the retina become abnormal and may widen, according to the American Academy of Ophthalmology. The disease can also lead to the formation of new blood vessels under the retina that leak fluid. Over time, patients experience blurred vision and loss of central visions. MacTel type 2 affects both eyes, but not always with the same severity. The disease typically presents in middle-age adults. Its cause is unknown. presented by Artificial Intelligence What Keeps Healthcare CIOs Up at Night: Balancing Technology Investments with Consumer Expectations When it comes to managing inbound phone calls, underperformance has devastating cost implications. By Stephanie Baum Protein therapies for eye disorders require frequent injections because those engineered proteins have short half-lives. Neurotech, based in Cumberland, Rhode Island, has developed an alternative way to deliver therapeutic proteins to the retina. The privately held company’s platform technology is based on a line of cells that that can be engineered to continuously produce therapeutic proteins. The technology covers several classes of proteins, including antibodies, fusion proteins, and growth factors. For MacTel, the Neurotech technology delivers human ciliary neurotrophic factor (CNTF), a signaling protein produced in the body that promotes neuron survival. This protein has been studied for applications in neurodegenerative diseases and retinal degeneration. Neurotech’s approach employs a capsule surgically implanted in the eye during an outpatient procedure. Each capsule contains as many as 440,000 allogeneic retinal pigment epithelial cells, which express engineered versions of CNTF. The capsule component of Encelto is semi-permeable, which allows essential nutrients to enter and therapeutic proteins to leave, according to Neurotech. The exterior membrane protects the encapsulated cells from being attacked by the patient’s immune system. While Encelto’s mechanism of action is not completely understood, the proteins from this therapy are thought to trigger events that promote the survival of photoreceptors in the eye. Neurotech contends that its approach offers several advantages. The therapeutic protein is produced and released right in the eye, which means a relatively small amount of protein is needed compared to what would be required from a time-controlled drug delivery system. The Neurotech technology provides continuous and long-term secretion of therapeutic protein. Furthermore, treatment may be terminated by simply retrieving the implant. Healthcare Using Data to Help Healthcare Practices Succeed A new report from Relatient, A Data-Driven Guide to Patient Access Succes, highlights how focusing on data accuracy and relevance can enhance the performance of healthcare practices. By Relatient Regulatory review of Encelto was based on data from two Phase 3 clinical trials. In both studies, results showed statistically significant and clinically meaningful slowing of disease progression measured at 24 months. The implant was also shown to be safe and well tolerated in both clinical trials. “I have seen the impact that MacTel can have on patients and their quality of life,” Dr. Charles Wykoff, director of research at Retinal Consultants of Texas and an investigator in the Encelto clinical trials, said in Neurotech’s approval announcement. “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.” Neurotech has not disclosed a price for Encelto. The company expects the product, its first approved therapy, will become available in June.

The FDA on Thursday approved Neurotech Pharmaceuticals’ cell therapy for an eye disease known as macular telangiectasia type 2, which causes central vision to become distorted and get worse over time. The cell therapy will be marketed as Encelto, and is expected to be available in the US starting June 2025, according to Neurotech’s press release. The private biotech’s CEO, Richard Small, declined to disclose the list price for the treatment to Endpoints News , and said the company was still having internal discussions. Encelto is the first treatment approved by the FDA for macular telangiectasia type 2, known as MacTel for short, according to Neurotech. The therapy is delivered in a tiny, implanted membrane that encapsulates a type of eye cell called a retinal pigment epithelial cell, which has been genetically engineered to express a protein known as ciliary neurotrophic factor. That protein helps photoreceptors in the eye survive longer, slowing the disease. Small said the therapy is meant to be a long-term treatment for the disease, whereas other retinal diseases are treated by regular injections. The process for getting the therapy involves a surgical procedure that Small described as “straightforward.” MacTel is a neurodegenerative condition that leads to central vision loss. Data on its prevalence are limited: One estimate suggests that it impacts 0.1% of the population, and Small said there might be about 165,000 patients in the US. Neurotech’s cell therapy, also known as revakinagene taroretcel, reduced the rate of progression of the MacTel compared to a sham procedure in two Phase 3 studies by 56.4% and 29.2%. Neurotech said the therapy was generally well-tolerated in clinical trials. The adverse reactions listed on Encelto’s label include conjunctival hemorrhage, delayed dark adaptation, foreign body sensation and eye pain, among others.