ixoberogene soroparvovec (ADVM-022) / Adverum Biotech - LARVOL DELTA

Efficacy and Safety Study of Ixoberogene Soroparvovec (Ixo-vec) in Participants With Neovascular Age-related Macular Degeneration (AQUARIUS) (clinicaltrials.gov) - P3 | N=534 | Recruiting | Sponsor: Adverum Biotechnologies, Inc. | N=284 ➔ 534

Enrollment change • Age-related Macular Degeneration • Macular Degeneration • Ophthalmology • Retinal Disorders • Wet Age-related Macular Degeneration

Adverum Reports 5-year OPTIC Extension Data for Investigational Gene Therapy for Wet AMD (Eyewire News) - "According to the company, patients treated with Ixo-vec experienced an 87% cumulative reduction in mean annualized anti-VEGF injections through 5 years compared with their pretreatment injection frequency. Before enrollment, patients had required a mean of 9.9 annualized anti-VEGF injections, suggesting a sustained reduction in treatment burden over the follow-up period....The long-term extension data also demonstrated durable anatomic outcomes, with a mean central subfield thickness (CST) improvement of 131.6 µm below baseline through 5 years. Visual outcomes were maintained in the previously treated, high-treatment-need population, with a mean best-corrected visual acuity (BCVA) change of -6.1 ETDRS letters over the same period."

P1 data • Wet Age-related Macular Degeneration

Ixo-vec is being evaluated in two phase 3 trials— ARTEMIS and AQUARIUS. (Eyewire News) - "In ARTEMIS, enrollment has been completed and topline data are expected in the first quarter of 2027. In AQUARIUS, enrollment is ongoing."

Enrollment status • P3 data: top line • Wet Age-related Macular Degeneration

Ixoberogene Soroparvovec (Ixo-vec) Contralateral Dosing Study in Participants With Neovascular Age-related Macular Degeneration (clinicaltrials.gov) - P2 | N=15 | Recruiting | Sponsor: Adverum Biotechnologies, Inc.

New P2 trial • Age-related Macular Degeneration • Gene Therapies • Macular Degeneration • Ophthalmology • Retinal Disorders • Wet Age-related Macular Degeneration

ARTEMIS: Efficacy and Safety Study of Ixoberogene Soroparvovec (Ixo-vec) in Participants With Neovascular Age-Related Macular Degeneration (clinicaltrials.gov) - P3 | N=311 | Active, not recruiting | Sponsor: Adverum Biotechnologies, Inc. | Recruiting ➔ Active, not recruiting

Enrollment closed • Age-related Macular Degeneration • Macular Degeneration • Ophthalmology • Retinal Disorders • Wet Age-related Macular Degeneration

Ixoberogene soroparvovec (Ixo-vec) intravitreal gene therapy for neovascular age-related macular degeneration (nAMD): Phase 2 LUNA Trial 2-year results (ARVO 2026) - P2, P3 | "Eligible participants were randomized to Ixo-vec 6x1010 vg/eye (6E10) or 2x1011 vg/eye (2E11) and one of four prophylactic regimens: topical difluprednate ± IVT dexamethasone ± oral prednisone. Conclusions Administration of a single IVT injection of Ixo-vec to individuals with nAMD and a high injection burden was well tolerated and resulted in stable BCVA and CST and a marked reduction in injection burden through 2 years. A Phase 3 trial evaluating Ixo-vec in individuals with nAMD is underway (ARTEMIS, NCT06856577)."

Gene therapy • P2 data • Age-related Macular Degeneration • Macular Degeneration • Ophthalmology • Retinal Disorders • Wet Age-related Macular Degeneration

Ixoberogene soroparvovec (Ixo-vec) intravitreal gene therapy for neovascular age-related macular degeneration (nAMD): Long-term results from the OPTIC trial (ARVO 2026) - P | "Conclusions Long-term outcomes in the OPTIC trial demonstrated that a single IVT injection of Ixo-vec was well tolerated and resulted in sustained intraocular aflibercept concentrations, preserved visual acuity, and a marked reduction in injection burden in a high-need nAMD population. Final 5-year results from OPTIC are anticipated in early 2026."

Gene therapy • Age-related Macular Degeneration • Cataract • Macular Degeneration • Ophthalmology • Retinal Disorders • Wet Age-related Macular Degeneration

Efficacy and Safety Study of Ixoberogene Soroparvovec (Ixo-vec) in Participants With Neovascular Age-related Macular Degeneration (AQUARIUS) (clinicaltrials.gov) - P3 | N=284 | Recruiting | Sponsor: Adverum Biotechnologies, Inc.

New P3 trial • Age-related Macular Degeneration • Macular Degeneration • Ophthalmology • Retinal Disorders • Wet Age-related Macular Degeneration

Emerging drugs for diabetic retinopathy: an overview of therapeutic innovation. (PubMed, Ther Adv Ophthalmol) - No abstract available

Journal • Diabetic Retinopathy • Retinal Disorders

AAV-based gene therapies for neovascular AMD. (PubMed, Gene Ther) - "This review summarizes the rationale for AAV use in ocular gene therapy, compares major delivery routes, and highlights leading clinical candidates, including RGX-314, ADVM-022, 4D-150, and NG101. Future directions include next-generation capsids, combination regimens, and precision patient selection. Collectively, these developments position AAV-based gene therapy as a promising strategy to redefine the therapeutic landscape of nAMD."

Journal • Review • Age-related Macular Degeneration • Gene Therapies • Immune Modulation • Immunology • Inflammation • Macular Degeneration • Ophthalmology • Retinal Disorders • Wet Age-related Macular Degeneration

Long-term Study of ADVM-022 in Neovascular (Wet) AMD [OPTIC-EXT] (clinicaltrials.gov) - P=N/A | N=23 | Active, not recruiting | Sponsor: Adverum Biotechnologies, Inc. | Trial completion date: Jun 2025 ➔ Jun 2030 | Trial primary completion date: Jun 2025 ➔ Jun 2030

Trial completion date • Trial primary completion date • Age-related Macular Degeneration • Macular Degeneration • Ophthalmology • Retinal Disorders • Wet Age-related Macular Degeneration

Adverum Biotechnologies Announces Completion of Screening for Pivotal Phase 3 ARTEMIS Trial of Ixo-vec for Wet Age-Related Macular Degeneration (GlobeNewswire) - "Adverum Biotechnologies...announced it has notified sites that it plans to complete screening in ARTEMIS...by September 30, seven months after initiation, because the company now expects full enrollment of at least 284 treatment-naïve and treatment experienced patients in 4Q 2025 and data readout in 1Q 2027."

P3 data • Trial status • Wet Age-related Macular Degeneration

Emerging solutions for neovascular age-related macular degeneration. (PubMed, Curr Opin Ophthalmol) - "Therapeutic innovation in nAMD is transitioning from incremental refinements in intravitreal anti-VEGF delivery to strategies aimed at extending durability or targeting alternative contributory pathways. Long-term safety, efficacy, and durability will determine which of these candidates redefine standard care."

Journal • Age-related Macular Degeneration • Gene Therapies • Macular Degeneration • Ophthalmology • Retinal Disorders • Wet Age-related Macular Degeneration • FGF2

ARTEMIS: Efficacy and Safety Study of Ixoberogene Soroparvovec (Ixo-vec) in Participants With Neovascular Age-Related Macular Degeneration (clinicaltrials.gov) - P3 | N=284 | Recruiting | Sponsor: Adverum Biotechnologies, Inc. | Trial completion date: Mar 2030 ➔ Nov 2030 | Trial primary completion date: Apr 2027 ➔ Dec 2026

Trial completion date • Trial primary completion date • Age-related Macular Degeneration • Macular Degeneration • Ophthalmology • Retinal Disorders • Wet Age-related Macular Degeneration

Lilly to Acquire Adverum Biotechnologies (PRNewswire) - "Under the terms of the merger agreement, Lilly will commence a tender offer to acquire all of the outstanding shares of Adverum common stock for a per share price of (1) $3.56 per share in cash payable at closing plus (2) one non-transferrable contingent value right (CVR) that entitles the holder to receive up to an additional $8.91 per CVR in cash upon the achievement of two milestones described below, for total potential per share consideration of up to $12.47."

M&A • Wet Age-related Macular Degeneration

ARTEMIS: Efficacy and Safety Study of Ixoberogene Soroparvovec (Ixo-vec) in Participants With Neovascular Age-Related Macular Degeneration (clinicaltrials.gov) - P3 | N=284 | Recruiting | Sponsor: Adverum Biotechnologies, Inc. | Trial primary completion date: Mar 2030 ➔ Apr 2027

Trial primary completion date • Age-related Macular Degeneration • Macular Degeneration • Ophthalmology • Retinal Disorders • Wet Age-related Macular Degeneration

A Semimechanistic Ocular Pharmacokinetic Model for ADVM-022 Gene Therapy Describing the Dose-Exposure Relationship in Monkeys and the Scaling to Human. (PubMed, Mol Pharm) - "Increasing doses within the lower range of preclinical studies (3 × 1010-2 × 1013 vg/eye) lead to increased transduction and expression, plateauing at upper limits of approximately 12.7 μg/day·cm3 for the retina, and 0.785 μg/day for extra-retinal tissues at higher doses. Assuming similar transduction efficiency between humans and monkeys, with adjustments for anatomical differences, the model provided predictions of ocular aflibercept concentrations that aligned with observations from the two dose groups in the phase 1 OPTIC clinical trial, supporting the utility of this approach."

Journal • PK/PD data • Age-related Macular Degeneration • Gene Therapies • Macular Degeneration • Ophthalmology • Retinal Disorders • Wet Age-related Macular Degeneration

Gene Therapy in Age-related Macular Degeneration. (PubMed, Int Ophthalmol Clin) - "Current trials in wet AMD include ADVM-022 and RGX-314. Current trials in dry AMD include GT-005 and JNJ-1887."

Journal • Review • Age-related Macular Degeneration • Dry Age-related Macular Degeneration • Gene Therapies • Macular Degeneration • Ophthalmology • Retinal Disorders • Wet Age-related Macular Degeneration

Biodistribution and Expression of Ixoberogene Soroparvovec (Ixo-vec) in Non-Human Primates: A Comprehensive Study on Intraocular and Systemic Distribution (ASGCT 2025) - P1, P2 | "Additionally, these data confirm the potential to safely dose the second eye for patients with bilateral nAMD. Disease Focus of Abstract:Ophthalmic Diseases"

Age-related Macular Degeneration • Gene Therapies • Macular Degeneration • Ophthalmology • Retinal Disorders • Wet Age-related Macular Degeneration

Gene Therapy in Diabetic Retinopathy and Diabetic Macular Edema: An Update. (PubMed, J Clin Med) - "Preliminary results from the SPECTRA (4D-150) and ALTITUDE (ABBV-RGX-314) studies are promising, demonstrating an improvement in the diabetic retinopathy severity score and a reduction in the treatment burden. In contrast, the INFINITY (ADVM-022) trial was complicated by several cases of severe inflammation and hypotony that led the sponsor to discontinue further development of this product for DME."

Journal • Review • Diabetic Macular Edema • Diabetic Retinopathy • Gene Therapies • Glaucoma • Inflammation • Ocular Inflammation • Ophthalmology • Retinal Disorders • Uveitis

Gene therapy in neovascular age related macular degeneration: an update. (PubMed, Graefes Arch Clin Exp Ophthalmol) - "Ixoberogene soroparvovec, RGX-314 and 4D-150 are the leading NV-AMD genetic treatment programs. Pre-clinical models suggest that genome surgery with clustered regularly interspaced short palindromic repeats (CRISPR) may be another option in the future."

Journal • Review • Age-related Macular Degeneration • Gene Therapies • Macular Degeneration • Oncology • Ophthalmology • Retinal Disorders • Wet Age-related Macular Degeneration

Long-Term Follow-up Study of ADVM-022 in DME (INFINITY-EXT) (clinicaltrials.gov) - P=N/A | N=22 | Active, not recruiting | Sponsor: Adverum Biotechnologies, Inc. | Enrolling by invitation ➔ Active, not recruiting

Enrollment closed • Diabetic Macular Edema • Diabetic Retinopathy • Ophthalmology • Retinal Disorders

Adverum Biotechnologies Reports Fourth Quarter and Full Year 2024 Financial Results and Provides Pipeline Highlights and Updates to Anticipated Milestones (GlobeNewswire) - "Upcoming Anticipated Milestones: 2H 2025: Initiate the global AQUARIUS Phase 3 trial; 4Q 2025: Present Phase 2 LUNA two-year long-term follow-up data."

New P3 trial • P2 data • Wet Age-related Macular Degeneration

Ixoberogene Soroparvovec (Ixo-vec) Intravitreal Gene Therapy for Neovascular Age-Related Macular Degeneration: Phase 2 LUNA 52-Week Data (ARVO 2025) - P2 | "Methods Participants were randomized across two Ixo-vec doses, 6x1010 and 2x1011 vg/eye (6E10 and 2E11) across multiple corticosteroid prophylactic regimens including topical difluprednate ± IVT dexamethasone ± oral prednisone (NCT05536973). 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."

Gene therapy • P2 data • Age-related Macular Degeneration • Macular Degeneration • Ophthalmology • Retinal Disorders • Wet Age-related Macular Degeneration

ARTEMIS: Efficacy and Safety Study of Ixoberogene Soroparvovec (Ixo-vec) in Participants With Neovascular Age-Related Macular Degeneration (clinicaltrials.gov) - P3 | N=284 | Recruiting | Sponsor: Adverum Biotechnologies, Inc. | Not yet recruiting ➔ Recruiting

Enrollment open • Age-related Macular Degeneration • Macular Degeneration • Ophthalmology • Retinal Disorders • Wet Age-related Macular Degeneration