Clinical Study to Evaluate the Safety, Tolerability, and Preliminary Efficacy of Intravitreal (IVT) Injection of ABI-110 (AAV2.N54-VEGF Trap) in Subjects With Neovascular (Wet) Age-Related Macular Degeneration (wAMD), Including Symptomatic Macular Polypoidal Choroidal Vasculopathy (PCV)

Phase 1

Recruiting

• Conditions: Neovascular (Wet) AMD
• Interventions: Drug: ABI-110 High Dose; Drug: ABI-110 Low Dose; Drug: ABI-110 Medium Dose

• Registration Number: NCT06550011
• Lead Sponsor: Avirmax Biopharma Inc

Brief Summary

This will be a clinical study to assess initial safety and tolerability of IVT ABI-110 in patients diagnosed with wet macular degeneration (wAMD), including symptomatic macular PCV.

Detailed Description

This is a Phase 1/2a, open-label, multiple-cohort, dose-escalation study to evaluate the safety, tolerability, and preliminary efficacy of IVT ABI-110 (AAV2.N54-VEGF-Trap) injection in subjects with wAMD), including symptomatic macular PCV.

This trial will include a screening period, confirmation of response to EYLEA (aflibercept), single administration of ABI-110, and follow-up period of 104 weeks after IVT ABI-110 injection.

Study Timeline

• Study First Submitted: 2024-07-25
• Study Start: 2024-08-07
• Study First Submitted QC: 2024-08-08
• Study First Posted: 2024-08-12
• Status Verified: 2024-09
• Last Update Submitted: 2024-09-30
• Last Update Posted: 2024-10-02
• Primary Completion: 2026-01
• Study Completion: 2030-02

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 18

Inclusion Criteria

• General:

  1. Must be willing and able to provide written, signed informed consent.

  2. Age: Subjects ≥ 50 and ≤ 89 years of age at the time of signing the informed consent

     Study eye:

  3. Diagnosis of wAMD, including symptomatic macular PCV, confirmed by the Central Reading Center

  4. The macular neovascularization lesion must be confirmed as determined by the Central Reading Center based on SD-OCT imaging at screening.

  5. BCVA ≤ 20/32 and ≥ 20/250 (≤ 78L and ≥ 30L ETDRS letters)

  6. History of ≥ 2 anti-VEGF injections prior to trial entry with meaningful response to anti-VEGF and continued need for anti-VEGF treatment

  7. Response to anti-VEGF at trial entry

  8. Must be pseudophakic

Key

Exclusion Criteria

• Study or Fellow Eye:

  1. Prior gene therapy, either eye
  2. Any active ocular/intraocular infection or inflammation (e.g., blepharitis, infectious conjunctivitis, keratitis, scleritis, uveitis) or history of idiopathic or autoimmune-associated uveitis, either eye
  3. History of retinal disease other than wAMD or PCV, study eye
  4. Any condition preventing visual acuity improvement (e.g., fibrosis, atrophy, or retinal epithelial tear in the center of the fovea), study eye.
  5. History of (or active) retinal detachment, study eye
  6. Uncontrolled glaucoma (defined as IOP > 25 mmHg despite treatment), study eye, or history of steroid-response ocular hypertension/glaucoma in either eye
  7. History of intravitreal therapy, (other than anti-VEGF therapy) such as intravitreal steroid injection or investigational product, in the 6 months prior to screening, study eye
  8. Any prior treatment with photodynamic therapy or laser photocoagulation, study eye
  9. History of glaucoma filtration, vitrectomy, or other procedure that could affect drug distribution or clearance, study eye.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SEQUENTIAL

Arm && Interventions

Group	Intervention	Description
Dose level 3	ABI-110 High Dose	High Dose
Dose level 1	ABI-110 Low Dose	Low Dose
Dose level 2	ABI-110 Medium Dose	Medium Dose

Primary Outcome Measures

Name	Time	Method
Safety and Tolerability	52 weeks	Incidence of Treatment-Emergent Adverse Events \[Safety and Tolerability\]

Secondary Outcome Measures

Name	Time	Method
Preliminary Efficacy	52 weeks	Change from baseline to Week 52 after ABI-110 treatment in BCVA as assessed by ETDRS chart at a starting distance of 4M
Pharmacokinetics and Pharmacodynamics	52 weeks	Changes in PK and PD by ABI-110 dose level
Immunogenicity	52 weeks	Changes in Anti-AAV2 antibodies (ADA) and anti-VEGF-Trap antibodies in serum
Optimal Dose	52 weeks	To determine the optimal dose of IVT ABI-110 injection for later phases of study

Trial Locations

Locations (4)

California Retina Consultants; 🇺🇸 Bakersfield, California, United States

Bay Area Retina Associates; 🇺🇸 Walnut Creek, California, United States

Retina Consultants of Texas - San Antonio; 🇺🇸 San Antonio, Texas, United States

Retina Consultants of Texas; 🇺🇸 The Woodlands, Texas, United States