To Evaluate the Safety and Preliminary Efficacy of Intravitreal EXG202 Injection in Patients with Wet (neovascular) Age-related Macular Degeneration (wAMD)

An Open-label, Dose-escalation Study to Evaluate the Safety and Efficacy of Intravitreal EXG202 Injection in Patients with Wet (neovascular) Age-related Macular Degeneration (wAMD)

The goal of this clinical trial is to evaluate the safety, tolerability and efficacy of intravitreal injection of EXG202 in subjects with neovascular age-related Macular Degeneration (nAMD).

Study Overview

• Status: Not yet recruiting
• Conditions: Wet Age-related Macular Degeneration; Wet Age-related Macular Degeneration (wAMD)
• Intervention / Treatment: Biological: EXG202

Detailed Description

EXG 202 is a gene therapy product developed for the treatment of neovascular age-related macular degeneration (nAMD). Neovascular AMD is the main cause of blindness among elderly individuals. The available therapies for treating nAMD require life-long intravitreal (IVT) injections every 4-12 weeks to maintain efficacy. Administration of EXG202 has the potential to treat nAMD by providing durable expression of therapeutic levels of intraocular protein and maintaining the vision of patients. EXG 202 is designed to reduce the current treatment burden which often results in undertreatment and vision loss in patients with nAMD receiving anti-VEGF therapy in clinical practice.

• Study Type: Interventional
• Enrollment (Estimated): 8
• Phase: Early Phase 1

Contacts and Locations

• Study Contact: Name: Sara Yang; Phone Number: 13957164092; Email: sarayang@exegenesisbio.com
• Study Contact Backup: Name: Lifei Wang, Ph.D

Study Locations

• China
  • Hebei
    • XingTai, Hebei, China, 054001
      • Hebei Eye Hospital
      • Contact: Lifei Wang, Ph.D; Phone Number: 13801057408; Email: wlfhb@126.com
  • Zhejiang
    • YiWu, Zhejiang, China, 322000
      • The Fourth Affiliated Hospital of Zhejiang University School of Medicine
      • Contact: Yongming Zhang, Ph.D; Email: zymcool@zju.edu.cn

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

1. Male or female, aged ≥50 years old;

2. The study eye must meet the following criteria:

   A. Diagnosed of wAMD and current active lesions; active, defined as the presence of any of the following lesions in the macular area upon OCT examination: a) intraretinal hemorrhage or fluid; b) subretinal fluid; c) subretinal hemorrhage; B. The BCVA score tested during screening using the ETDRS eye chart (see Appendix 3 for details) must be between 73-19 letters (including boundary values), (equivalent to Snellen visual acuity of 20/40 to 20/400);

3. Clinically meaningful response to standard anti-VEGF therapy within 6 months before screening or during the lead-in period of screening.
4. The subject has clear refractive media and sufficient pupil dilation at the time of screening to obtain high-quality retinal images for confirmation of diagnosis;
5. Subjects (including male subjects) have no pregnancy plans during the screening period and the entire trial period and voluntarily take effective contraceptive measures and have no sperm or egg donation plans;
6. Voluntarily participate in this clinical trial, understand the research procedures and sign the informed consent form before screening; have good compliance and are willing to abide by the research procedures.

Exclusion Criteria:

1. The study eye has any eye disease other than wAMD that may affect central vision and/or macular detection (such as macular hole, macular epiretinal membrane, etc.);
2. The study eye has a history of retinal detachment or retinal detachment during the screening period (such as rhegmatogenous retinal detachment, tractional retinal detachment, etc.);
3. The study eye has MNV caused by reasons other than wAMD (such as diabetic retinopathy, pathological myopia, retinal vein occlusion, angioid streak disease, ocular histoplasmosis, trauma, etc.), and a history of macular pathology unrelated to wAMD;
4. The study eye plans to undergo any intraocular surgery during the study period;

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Non-Randomized
• Interventional Model: Sequential Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Experimental: Dose escalation-Cohort A; Dose 1 :Administered via intravitreal injection	Biological: EXG202; EXG202 is a gene therapy product for the treatment of wet (neovascular) age-related macular degeneration（wAMD） with a single intravitreal injection and administration.
Experimental: Experimental: Dose escalation-Cohort B; Dose 2 :Administered via intravitreal injection	Biological: EXG202; EXG202 is a gene therapy product for the treatment of wet (neovascular) age-related macular degeneration（wAMD） with a single intravitreal injection and administration.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Safety and tolerability after EXG202 injection	Frequency, type, and intensity of ocular and non-ocular Adverse Events (AEs) and serious adverse events (SAE) at Week 52, to explore the maximum tolerated dose (MTD)	up to 52 weeks after treatment

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
To evaluate the efficacy after EXG202 injection	the Best Corrected Visual Acuity (BCVA) change from baseline	up to 52 weeks after treatment
To evaluate the efficacy after EXG202 injection	The optical coherence tomography (OCT ).	52 weeks following EXG202 treatment.

Collaborators and Investigators

• Sponsor: Hangzhou Jiayin Biotech Ltd

Study record dates

Study Major Dates

• Study Start (Estimated): March 28, 2025
• Primary Completion (Estimated): April 25, 2026
• Study Completion (Estimated): April 30, 2026

Study Registration Dates

• First Submitted: March 14, 2025
• First Submitted That Met QC Criteria: March 20, 2025
• First Posted (Actual): March 25, 2025

Study Record Updates

• Last Update Posted (Actual): March 25, 2025
• Last Update Submitted That Met QC Criteria: March 20, 2025
• Last Verified: March 1, 2025

More Information

Terms related to this study

• Keywords: AAV; wAMD
• Additional Relevant MeSH Terms: Eye Diseases; Retinal Diseases; Retinal Degeneration; Macular Degeneration
• Other Study ID Numbers: EXG202-011

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No