This study is to determine the long-term levels of CNTF, and the clinical safety and efficacy of the NT-501 implants with the Medica Hollow Fiber Membrane (MHFM) in participants with macular telangiectasia type 2.

Start Date01 Apr 2025

Sponsor / Collaborator

Neurotech Pharmaceuticals, Inc.

NCT04729972

/ CompletedPhase 2

Phase II, Multicenter, Open-label Safety Study of Bilateral NT-501 in Participants With Macular Telangiectasia Type 2

This is a multi-center, open-label, 6-month study to evaluate the safety of bilateral CNTF implants in participants with Macular Telangiectasia Type 2.

Start Date13 Aug 2021

Sponsor / Collaborator

Neurotech Pharmaceuticals, Inc.

[+1]

NCT04577300

/ RecruitingPhase 2IIT

A Randomized, Sham Controlled, Masked Phase II Study to Evaluate the Safety and Efficacy of Dual Intravitreal Implantation of NT-501 Encapsulated Cell Therapy for the Treatment of Glaucoma

To determine the safety and efficacy over 24 months of dual NT-501 CNTF encapsulated cell therapy (ECT) on visual impairment related to glaucoma.

Start Date15 May 2021

Sponsor / Collaborator

Stanford University

100 Clinical Results associated with Revakinagene taroretcel

100 Translational Medicine associated with Revakinagene taroretcel

100 Patents (Medical) associated with Revakinagene taroretcel

Literatures (Medical) associated with Revakinagene taroretcel

01 Sep 2023·Ophthalmology science

Phase I NT-501 Ciliary Neurotrophic Factor Implant Trial for Primary Open-Angle Glaucoma

Article

Author: Satterfield, Kellie R ; Lam, Byron L ; Albini, Thomas A ; Nuñez, Mariana ; Beykin, Gala ; Goldberg, Jeffrey L

Purpose:

To assess the safety and efficacy of a ciliary neurotrophic factor (CNTF) intraocular implant on neuroprotection and neuroenhancement in glaucoma.

Design:

Open-label, prospective, phase I clinical trial.

Participants:

A total of 11 participants were diagnosed with primary open-angle glaucoma (POAG). One eye of each patient was assigned as the study (implant) eye.

Methods:

The study eye was implanted with a high-dose CNTF-secreting NT-501 implant, whereas the other eye served as a control. All patients were followed up for 18 months. Analysis was limited to descriptive statistics.

Main Outcome Measures:

Primary outcome was safety through 18 months after implantation assessed by serial eye examinations, structural and functional testing, and adverse events (AEs) recording. Parameters measured included visual acuity (VA), Humphrey visual field (HVF), pattern electroretinogram, scanning laser polarimetry with variable corneal compensation (GDx VCC), and OCT. These parameters were also used for secondary analysis of efficacy outcome.

Results:

All NT-501 implants were well tolerated with no serious AEs associated with the implant. The majority of AEs were related to the implant placement procedure and were resolved by 12 weeks after surgery. Foreign-body sensation was the most commonly reported AE and was self-limited to the postoperative period. The most common implant-related AE was pupil miosis; no patients underwent explant. Visual acuity and contrast sensitivity decreased more in fellow eyes than in study eyes (VA, -5.82 vs. -0.82 letters; and contrast sensitivity, -1.82 vs. -0.37 letters, for fellow vs. study eyes, respectively). The median HVF visual field index and mean deviation measurements worsened (decreased) in fellow eyes (-13.0%, -3.9 dB) and improved (increased) in study eyes (2.7%, 1.2 dB). Implanted eyes showed an increase in retinal nerve fiber layer thickness measured by OCT and by GDx VCC (OCT, 2.66 μm vs. 10.16 μm; and GDx VCC, 1.58 μm vs. 8.36 μm in fellow vs. study eyes, respectively).

Conclusions:

The NT-501 CNTF implant was safe and well tolerated in eyes with POAG. Eyes with the implant demonstrated both structural and functional improvements suggesting biological activity, supporting the premise for a randomized phase II clinical trial of single and dual NT-501 CNTF implants in patients with POAG, which is now underway.

Financial Disclosures:

Proprietary or commercial disclosure may be found after the references.

01 Mar 2021·Veterinary ophthalmologyQ4 · AGRICULTURAL & FORESTRY SCIENCE

Medical anti‐glaucoma therapy: Beyond the drop

Q4 · AGRICULTURAL & FORESTRY SCIENCE

Review

Author: Miller, Paul E. ; Eaton, Joshua Seth

Abstract:

Barriers to effective medical therapy are numerous and include difficulties with effective and sustained control of intraocular pressure (IOP) and adherence to prescribed anti‐glaucoma drop regimens. In an effort to circumvent these challenges, a number of new anti‐glaucoma therapies with sustained effects have emerged. Methods for sustained delivery of prostaglandin analogs are being intensely investigated and many are in human clinical trials. Intracameral devices include the following: Allergan's Durysta™ Bimatoprost SR, Envisia Therapeutics’ ENV515 travoprost implant, Glaukos’ iDose™, Ocular Therapeutix's OTX‐TIC travoprost implant, and Santen's polycaprolactone implant with PGE2‐derivative DE‐117. Other prostaglandin‐based technologies include Allergan's bimatoprost ring (placed in the conjunctival fornix), Ocular Therapeutics’ OTX‐TP intracanalicular travoprost implant, subconjunctival latanoprost in a liposomal formulation, and the PGE2 derivative PGN 9856‐isopropyl ester that is applied to the periorbital skin. Exciting breakthroughs in gene therapy include using viral vectors to correct defective genes such as MYOC or to modulate gonioimplant fibrosis, CRISPR technology to edit MYOC or to alter aquaporin to reduce aqueous humor production, and siRNA technology to silence specific genes. Stem cell technology can repopulate depleted tissues or, in the case of Neurotech's Renexus® NT‐501 intravitreal implant, serve as a living drug delivery device that continuously secretes neurotrophic factors. Other unique approaches involve nanotechnology, nasal sprays that deliver drug directly to the optic nerve and noninvasive alternating current stimulation of surviving cells in the optic nerve. Over time these modalities are likely to challenge the preeminent role that drops currently play in the medical treatment of glaucoma in animals.

01 Jan 2021·Ophthalmic researchQ4 · MEDICINE

The Use of Neuroprotective Agents in Treating Geographic Atrophy

Q4 · MEDICINE

Review

Author: Chong, Victor ; Giani, Andrea ; Scholl, Hendrik P N ; Boyer, David

Geographic atrophy (GA) secondary to age-related macular degeneration accounts for close to one-quarter of cases of legal blindness in the USA and the UK. Despite this notable disease burden, the pathophysiology of GA is complex and not fully understood, and there is currently no approved treatment to prevent or slow its progression. GA is heterogeneous in its appearance and extent, and underlying associated traits such as drusen and complement factor polymorphisms vary between patients and by ethnicity, posing a challenge for treatment development. The root cause of vision loss in GA is photoreceptor death; therefore, protecting photoreceptors from damage and delaying their degeneration are key to successful GA treatment. There are multiple neuroprotective pathways that may contribute to protecting photoreceptors from damage, and compounds that target these pathways include antioxidants, neurotrophic factors, and catalases. However, the efficacy of previously trialled neuroprotective therapies in GA, such as brimonidine, tandospirone, and NT-501, has been inconsistent; this may be due to their target of action, method of delivery, and/or suboptimal duration of action. Neurotrophic factors, or molecules involved in neuroprotective signalling cascades, may be ideal agents for further investigation for the treatment of GA. Future neuroprotective strategies in GA must focus on the development of agents with a long duration of action that can combat the progression of chronic damage in GA to provide clinically meaningful benefits for patients.

News (Medical) associated with Revakinagene taroretcel

11 Nov 2024

·www.pharmaceutical-technology.com

FDA delays priority review of Neurotech’s eye cell therapy implant

Neurotech’s NT-501 implant contains allogeneic retinal pigment epithelium (RPE) cells that produce therapeutic proteins and ciliary neurotrophic factor (CNTF) for targeted and continuous treatment. Image credit: GoodStudio / Shutterstock. Neurotech Pharmaceuticals’ eye cell therapy implant has faced a setback as the US Food and Drug Administration (FDA) has extended its Prescription Drug User Fee Act (PDUFA) date. The US regulatory agency has changed the PDUFA date for the therapy’s biologics license application (BLA) for NT-501 (revakinagene taroretcel) to 18 March 2025, three months after the original date of 17 December 2024 the FDA set back in June. The agency stated that it needed the extra time to “review additional data provided by the Neurotech in response to recent requests from the FDA”. The company is seeking approval for the NT-501 implant as a treatment for macular telangiectasia type 2 (MacTel), a rare ocular neurodegenerative disease. The implant received fast track designation for this indication in December 2018. The NT-501 cell therapy implant contains allogeneic retinal pigment epithelium (RPE) cells. The implant has a semipermeable membrane that allows the therapeutic proteins like the ciliary neurotrophic factor (CNTF), produced by the RPE cells, to exit whilst protecting these cells from the host’s immune system, providing a targeted and continuous treatment. The company has previously shared that two parallel Phase III trials (protocol A, NCT03316300; protocol B, NCT03319849) of NT-501 met its primary endpoint of the rate of change in ellipsoid zone area loss, which correlates to loss of photoreceptors and sight, from baseline through 24 months. The rate of reduction of progression of disease in the treatment group in protocol A and B studies was 56.4% and 29.2%, respectively. The company also noted that the implant was safe, durable, and well tolerated in both trials. Multiple companies are developing cell and gene therapies for eye disorders . SparingVision has an adeno-associated virus-based gene therapy, SPVN20 , for improving visual acuity in patients with advanced and late-stage retinitis pigmentosa. The therapy is being evaluated in patients with stage two and three retinitis pigmentosa patients in the Phase I/II PRODYGY study (NCT05748873). Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva . Editorial content is independently produced and follows the highest standards of journalistic integrity. Topic sponsors are not involved in the creation of editorial content. Free Whitepaper Cell and gene therapies: Pipe dream to pipeline The cell and gene industry is gaining momentum, with a new wave of therapies promising to transform the way doctors treat, and even cure, disease. In this report, Cytiva and GlobalData have collaborated to explore the rise of the cell and gene therapy industries, the current state of the market, present and future opportunities for advancement, and the challenges that lie ahead. Thank you. You will receive an email shortly. Please check your inbox to download the Whitepaper. By Cytiva Thematic By downloading this Whitepaper, you acknowledge that GlobalData may share your information with Cytiva Thematic and that your personal data will be used as described in their Privacy Policy

Phase 3Cell TherapyPriority ReviewGene TherapyFast Track

08 Nov 2024

·www.businesswire.com

Neurotech Provides Update on BLA for NT-501 as a Treatment for Macular Telangiectasia Type 2 (MacTel)

CUMBERLAND, R.I.--( BUSINESS WIRE )--Neurotech Pharmaceuticals, Inc., an innovator in sustained drug delivery for chronic retinal diseases, today 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. “We are committed to providing the Agency any information needed to complete the review of the NT-501 BLA,” said Richard Small, Chief Executive Officer. “Neurotech will continue in our effort to bring this important therapy to MacTel patients.” About Macular Telangiectasia (MacTel) Macular Telangiectasia Type 2 (MacTel), or idiopathic juxtafoveal macular telangiectasia type 2, is a rare neurodegenerative disease with characteristic localized retinal degeneration and secondary alterations of the retinal vasculature. 1 MacTel typically affects both eyes and causes a gradual deterioration in central vision. About Encapsulated Cell Therapy (ECT) The ECT platform is a cell-based delivery system designed to provide long-term, sustained delivery of therapeutic proteins for the treatment of chronic retinal 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 inserted into the patient’s vitreous and sutured to the sclera during an outpatient procedure. Once in place, the capsule's semi-permeable membrane allows essential nutrients to enter, while also permitting the therapeutic proteins to exit into the vitreous, providing targeted and continuous treatment. Simultaneously, the membrane protects the encapsulated RPE cells from the host's immune system, ensuring their long-term survival and functionality. About NT-501 Implant NT-501 leverages our ECT platform to deliver CNTF for the treatment of chronic retinal diseases, such as MacTel. CNTF is a potent neuroprotective protein that promotes the survival and maintenance of photoreceptors. This targeted therapy provides sustained delivery of CNTF, aiming to slow retinal degeneration and potentially improve long-term visual outcomes for patients. About Neurotech Pharmaceuticals, Inc. Neurotech Pharmaceuticals, Inc. is a private, clinical stage biotechnology company and an innovator in sustained drug delivery for chronic retinal diseases. The core platform technology, ECT, enables continuous production of therapeutic proteins to the back of the eye. 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.

Cell Therapy

20 Jun 2024

·www.biospace.com

Neurotech Pharmaceuticals, Inc. Receives Priority Review of Biologics License Application (BLA) for NT-501 (revakinagene taroretcel) as a Treatment for Macular Telangiectasia Type 2 (MacTel)

- Priority review granted with a PDUFA goal date set for December 17, 2024. CUMBERLAND, R.I.--(BUSINESS WIRE)-- Neurotech Pharmaceuticals, Inc., an innovator in sustained drug delivery for chronic retinal diseases, announces that the U.S. Food and Drug Administration (FDA), has determined that the Biologic License Application (BLA) for NT-501, an investigational encapsulated cell therapy for the treatment of MacTel, is sufficiently complete to permit a substantive review. The application has a prescription drug user fee act (PDUFA) goal date of December 17, 2024. MacTel is a progressive, neurodegenerative disease of the retina that results in the deterioration of central vision, significantly impacting patients' quality of life. NT-501 is an ocular implant designed to deliver sustained therapeutic doses of ciliary neurotrophic factor (CNTF) directly to the retina to slow the progression of the disease. “This is a significant achievement for Neurotech,” said Richard Small, Chief Executive Officer. “I would like to express my gratitude to our employees for reaching this important milestone.” About Macular Telangiectasia (MacTel) MacTel, or idiopathic juxtafoveal macular telangiectasia, is a rare neurodegenerative disease with characteristic localized retinal degeneration with secondary alterations of the retinal vasculature.1 MacTel typically affects both eyes and causes deterioration in central vision impacting patients’ quality of life. About Encapsulated Cell Therapy (ECT) The ECT platform is a cell-based delivery system that was designed to provide long-term, sustained delivery of therapeutic proteins for the treatment of chronic retinal diseases. This versatile platform consists of a small, semi-permeable capsule containing proprietary allogeneic RPE cells genetically engineered to produce specific therapeutic proteins for targeted disease treatment. The capsule is surgically inserted into the patient’s vitreous and sutured to the sclera during an outpatient procedure. Once in place, the capsule's semi-permeable membrane allows essential nutrients to enter, while also permitting the therapeutic proteins to exit into the vitreous, providing targeted and continuous treatment. Simultaneously, the membrane protects the encapsulated RPE cells from the host's immune system, ensuring their long-term survival and functionality. About NT-501 Implant NT-501 leverages our ECT platform to deliver CNTF for the treatment of chronic retinal diseases, such as MacTel. CNTF is a potent neuroprotective protein that promotes the survival and maintenance of photoreceptors. This targeted therapy provides sustained delivery of CNTF, aiming to slow retinal degeneration and potentially improve long-term visual outcomes for patients. About Neurotech Pharmaceuticals, Inc. Neurotech Pharmaceuticals, Inc. is a private, clinical stage biotechnology company and an innovator in sustained drug delivery for chronic retinal diseases. The core platform technology, ECT, enables continuous production of therapeutic proteins to the back of the eye. For more information about us, please visit . 1. Charbel Issa P, Gillies MC, Chew EY, Heeren TFC, Bird AC, Peto T, Holz FG, Scholl HPN (2013) Macular Telangiectasia Type 2.

Cell TherapyPriority Review

100 Deals associated with Revakinagene taroretcel

R&D Status

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Macular telangiectasia type 2	NDA/BLA	US	Neurotech Pharmaceuticals, Inc.	20 Jun 2024
Retinal Telangiectasis	Phase 3	AU	Neurotech USA, Inc.	19 Jan 2018
Retinitis Pigmentosa	Phase 3	US	Neurotech USA, Inc.	16 Apr 2007
Color Vision Defects	Phase 2	US	Neurotech USA, Inc.	07 Jul 2012
Achromatopsia 1	Phase 2	US	National Eye Institute	01 Jul 2012
Usher Syndromes	Phase 2	US	Neurotech USA, Inc.	20 Jan 2012
Geographic Atrophy	Phase 2	US	Neurotech USA, Inc.	-
Glaucoma	Phase 1	US	Neurotech USA, Inc.	-
Glaucoma	Phase 1	US	University of Miami	-
Optic Nerve Diseases	Phase 1	US	Neurotech USA, Inc.	-

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


EURETINA2024 Manual	Not Applicable	Macular telangiectasia type 2	-	NT-501	rfuwxtbdlp(irgxvglucu) = acriovadbk xpscjncogh (hfcdqjvyst ) View more	Positive	19 Sep 2024
				Sham	rfuwxtbdlp(irgxvglucu) = yofhncigxv xpscjncogh (hfcdqjvyst ) View more
EURETINA2024	Not Applicable	Macular telangiectasia type 2	-	NT-501	ucjpkubugw(yguiokiagv) = rwbcoeiftn ceqeehqsgv (ttiirqrdok, 0.05 - 0.10)	-	19 Sep 2024
				Sham treatment	ucjpkubugw(yguiokiagv) = pitvdwjbip ceqeehqsgv (ttiirqrdok, 0.14 - 0.19)
EURETINA2023	Not Applicable	Macular telangiectasia type 2	-	NT-501	lyxgwgtbzf(tjjviwrsih) = Non-ocular SAEs were comparable between treatment groups in NTMT-03-A (NT-501, 17.2% [10/58]; sham, 10.5% [6/57]) and NTMT-03-B (NT-501, 13.6% [8/59]; sham, 18.5% [10/54]), and none were related to treatment aebobtilnh (rhlyoekayq ) View more	-	05 Oct 2023
				Revakinagene Taroretcel (Sham treatment)
ARVO2023	Not Applicable	-	-	NT-501 Implant	vepuwyqbbb(svoqaacilw) = lwkeuptztr dftrpwqgld (meelaicckh )	-	23 Apr 2023
NCT03316300 (Protocol A, Biospace) Manual	Phase 3	Macular telangiectasia type 2	115	NT-501	umcutcauuw(hfqdwxsgyl) = hbnskxekmf tenbkgsank (wtyefjgxfv )	Positive	02 Nov 2022
				Sham Procedure	-
NCT03316300 (Protocol A, Pubmed) Manual	Phase 3	Macular telangiectasia type 2 \| Dystrophy, Macular	-	NT-501	rdaggjnxwh(dhuxyxmedn) = lvgvresiiq dphgchcuce (imizxfoflg, 0.19)	Positive	15 Mar 2021
				untreated	rdaggjnxwh(dhuxyxmedn) = lrebgtqvsk dphgchcuce (imizxfoflg, 0.11)

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