Encelto (revakinagene taroretcel-lwey) / Neurotech Pharma - LARVOL DELTA

LONG-TERM PROGRESSION OF ELLIPSOID ZONE LOSS AND ASSOCIATED FEATURES ON OPTICAL COHERENCE TOMOGRAPHY IN MACULAR TELANGIECTASIA TYPE 2. (PubMed, Ophthalmology) - "EZ loss in MacTel follows a characteristic sigmoid trajectory, with distinct morphologic types predicting progression velocity. While Pattern 1 lesions remain stable for longer periods, Pattern 2 and 3 indicate fast progression. These findings provide structural benchmarks that may help guide clinical management and treatment timing, particularly in light of emerging implant-based therapies such as Revakinagene taroretcel Encelto®."

Journal • Ophthalmology

Dual Intravitreal Implantation of NT-501 Encapsulated Cell Therapy for Glaucoma (clinicaltrials.gov) - P2 | N=30 | Active, not recruiting | Sponsor: Stanford University | Recruiting ➔ Active, not recruiting | Trial completion date: Dec 2025 ➔ Aug 2026

Enrollment closed • Trial completion date • Glaucoma • Ophthalmology

Study of NT-501 Encapsulated Cell Therapy for Glaucoma Neuroprotection and Vision Restoration (clinicaltrials.gov) - P2 | N=54 | Active, not recruiting | Sponsor: Stanford University | Trial completion date: Dec 2025 ➔ Aug 2026

Trial completion date • Glaucoma • Ophthalmology

Clinical histopathology and pathogenesis of macular telangiectasia type 2. (PubMed, Prog Retin Eye Res) - "We also review current neuroprotective strategies and provide targeted recommendations for future therapeutic development and histopathologic research. The conceptual framework developed in this work -grounded in rigorous analysis of the most consistent and methodologically validated histopathologic findings, and interpretation of their mechanistic context- may serve as a model for deciphering rare retinal diseases and for generating focused, hypothesis-driven questions to guide future investigation."

Journal • Review • CNS Disorders • Ophthalmology • Retinal Disorders

Conjunctival histopathological changes associated with netarsudil 0.02. (PubMed, Eye (Lond)) - "Conjunctiva treated with netarsudil may be more likely to demonstrate inflammation compared to those treated with other combinations of glaucoma medications. These findings may have surgical implications and additional studies correlating clinical outcomes are required."

Journal • Glaucoma • Inflammation • Ophthalmology

Updated Surgical Technique for Implantation of Revakinagene Taroretcel. (PubMed, Retina) - "The described surgical implantation technique was developed using learnings from the associated phase 2 and 3 trials and implemented to maximize the likelihood of long-term capsule stability and minimize the risk of adverse events including device extrusion."

Journal • Ophthalmology

Cell-Based Ciliary Neurotrophic Factor Therapy for Macular Telangiectasia Type 2. (PubMed, NEJM Evid) - P3 | "NT-501 for MacTel resulted in statistically significantly reduced EZA loss compared with sham procedures. (Funded by Neurotech Pharmaceuticals; ClinicalTrials.gov numbers, NCT03316300 and NCT03319849.)."

Clinical • Journal • Ophthalmology

Long-Term Durability of Ciliary Neurotrophic Factor-Releasing Revakinagene Taroretcel-lwey in Individuals With Retinal Degenerative Disorders. (PubMed, Invest Ophthalmol Vis Sci) - P1, P2, P3 | "There was no evidence of an immunological reaction to CNTF or the NTC-201-6A cells. This retrospective analysis suggests that a single, intraocular administration of revakinagene taroretcel-lwey provides sustained, bioactive CNTF for durations exceeding a decade, potentially providing a long-term treatment option for chronic retinal degenerative diseases."

Journal • Retrospective data

Surgical Explantation of Revakinagene Taroretcel Capsule Due to Non-Clearing Vitreous Hemorrhage Secondary to Fixation Loop Extrusion. (PubMed, Am J Ophthalmol) - No abstract available

Journal • Hematological Disorders • Ophthalmology • Retinal Disorders

Revakinagene Taroretcel: First Approval. (PubMed, Mol Diagn Ther) - "Revakinagene taroretcel has been granted Orphan Drug Designation for retinitis pigmentosa and Fast Track Designation for retinitis pigmentosa and dry age-related macular degeneration in the USA. This article summarises the milestones in the development of revakinagene taroretcel leading to this first approval for the treatment of adults with idiopathic MacTel type 2 in the USA."

Journal • Age-related Macular Degeneration • Dry Age-related Macular Degeneration • Gene Therapies • Genetic Disorders • Inherited Retinal Dystrophy • Macular Degeneration • Ocular Inflammation • Ophthalmology • Retinal Disorders • Retinitis Pigmentosa

Beyond the injection: delivery systems reshaping retinal disease management. (PubMed, Expert Opin Pharmacother) - "Specific innovations discussed include the ranibizumab port delivery system, EYP-1901 (Duravyu, vorolanib implant), KSI-301 (tarcocimab tedromer), KSI-501, OTX-TKI (Axpaxli, axitinib implant), 4D-150, revakinagene taroretcel-lwey (Encelto, NT-501, encapsulated cell therapy), Xipere (triamcinolone acetonide injectable suspension), AU-011 (belzupacap sarotalocan targeted delivery), ABBV-RGX-314, elamipretide, and OCS-01 (high concentration dexamethasone)...Challenges include overcoming the blood-retinal barrier, surgical complications with implantable devices, and ensuring patient adherence. Advances in smart delivery systems, drug formulations, and predictive models, alongside interdisciplinary collaboration, will be crucial in achieving personalized, effective, and sustainable retinal therapies."

Journal • Review • Age-related Macular Degeneration • Diabetic Macular Edema • Diabetic Retinopathy • Gene Therapies • Macular Degeneration • Ophthalmology • Retinal Disorders

NT-501 Capsule Stability and Encapsulated Cell Technology Platform Capabilities (ASGCT 2025) - "The simulated in vivo stability study supports that the NT-501 capsule remains integral and the capsule materials are stable for at least two years. Additionally, modifications can be made to the ECT capsule to alter the delivery levels or targets of therapeutics making it a versatile long term delivery platform for intraocular diseases. Disease Focus of Abstract:Ophthalmic Diseases"

Ophthalmology

Sustained Drug Delivery of Ciliary Neurotrophic Factor by Encapsulated Cell Technology: NT-501 Implants: (ASGCT 2025) - "Neurotech's ECT was developed to overcome the challenge of long-term therapeutic delivery to the retina. NT-501 consists of an encapsulated cell line that has been genetically engineered to produce a novel secreted form of human CNTF. Extensive characterization was conducted across product shelf life, stability, and on explanted devices which were implanted for up to 14.5 years."

Ophthalmology

Phase 4 Study: Long-term Safety and Efficacy of NT-501 in MacTel Type 2, Including Sham Procedure Participants (clinicaltrials.gov) - P4 | N=285 | Not yet recruiting | Sponsor: Neurotech Pharmaceuticals

New P4 trial • Ophthalmology

A Study to Determine the Safety and Efficacy of NT-501 With MHFM (clinicaltrials.gov) - P3 | N=12 | Active, not recruiting | Sponsor: Neurotech Pharmaceuticals | Recruiting ➔ Active, not recruiting

Enrollment closed • Ophthalmology

Revakinagene taroretcel (Encelto) - a gene therapy for idiopathic macular telangiectasia. (PubMed, Med Lett Drugs Ther) - No abstract available

Journal • Gene Therapies • Ophthalmology

Neuroprotective Characteristics of CNTF Delivered by Encapsulated Cells for the Treatment of Degenerative Diseases of the Retina (ARVO 2025) - "Purpose Neurotech's proprietary encapsulated cell therapy (ECT) utilizes a genetically engineered cell line, NTC-201.6A, to deliver ciliary neurotrophic factor (CNTF) to the retina...CNTF is a protein with demonstrated neuroprotective capabilities. We have analyzed CNTF that is delivered by encapsulated cells and shown that it retains expected activity and characteristics."

Ophthalmology

Determining the function of NT-501 produced CNTF at physiological levels in human retinas. (ARVO 2025) - "Layman Abstract (optional): Provide a 50-200 word description of your work that non-scientists can understand. Describe the big picture and the implications of your findings, not the study itself and the associated details."

Ophthalmology

Pharmacokinetics and Bioavailability of CNTF following Intraocular Implant of Encapsulated Cell Therapy (ECT) in NZW Rabbits (ARVO 2025) - "Each device contained an encapsulated cell line (NTC-201-6A) genetically engineered to secrete CNTF...Layman Abstract (optional): Provide a 50-200 word description of your work that non-scientists can understand. Describe the big picture and the implications of your findings, not the study itself and the associated details."

PK/PD data • Preclinical • Age-related Macular Degeneration • Dry Age-related Macular Degeneration • Glaucoma • Ocular Inflammation • Ophthalmology

A Study to Determine the Safety and Efficacy of NT-501 With MHFM (clinicaltrials.gov) - P3 | N=13 | Recruiting | Sponsor: Neurotech Pharmaceuticals | Not yet recruiting ➔ Recruiting

Enrollment open • Ophthalmology

Neurotech’s ENCELTO (revakinagene taroretcel-lwey) Approved by the FDA for the Treatment of Macular Telangiectasia Type 2 (MacTel) (Businesswire) - "Neurotech Pharmaceuticals...announced that the U.S. Food and Drug Administration (FDA), has approved ENCELTO (revakinagene taroretcel-lwey) for the treatment of Macular Telangiectasia type 2 (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."

FDA approval • Launch US • Ophthalmology

Ciliary Neurotrophic Factor Implant in Geographic Atrophy from Age-related Macular Degeneration: A Long-Term Case Study. (PubMed, Retin Cases Brief Rep) - "Notably, the implanted eye, exhibited less progression of macular GA and peripapillary atrophy. While acknowledging the limitations of a single case, we highlight the potential benefits of using the CNTF implant, a one-time surgical procedure, in reducing the treatment burden for atrophic AMD patients. We hope to see further reports from other sites that participated in this compassionate use protocol for NT-501 in atrophic AMD."

Journal • Age-related Macular Degeneration • Dry Age-related Macular Degeneration • Macular Degeneration • Ophthalmology • Retinal Disorders

Neurotech Provides Update on BLA for NT-501 as a Treatment for Macular Telangiectasia Type 2 (MacTel) (Businesswire) - "Neurotech Pharmaceuticals, Inc...announced that the U.S. Food and Drug Administration (FDA), has extended the Prescription Drug User Fee Act (PDUFA) goal date by three months to allow time required for the FDA to review additional data provided by the Company in response to recent requests from the FDA. The new PDUFA goal date for the biologics license application (BLA) of NT-501 (revakinagene taroretcel) as a treatment for Macular telangiectasia type 2 (MacTel) is March 18, 2025."

PDUFA • Ophthalmology

Study of NT-501 Encapsulated Cell Therapy for Glaucoma Neuroprotection and Vision Restoration (clinicaltrials.gov) - P2 | N=54 | Active, not recruiting | Sponsor: Stanford University | Trial completion date: Dec 2024 ➔ Dec 2025 | Trial primary completion date: Aug 2024 ➔ Aug 2025

Trial completion date • Trial primary completion date • Glaucoma • Ophthalmology

Pooled Visual Function Data of Neurotrophic Factor–Producing Revakinagene Taroretcel in People With Macular Telangiectasia Type 2 (AAO 2024) - No abstract available

Late-breaking abstract • Ophthalmology