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

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

Biodistribution and Expression of Ixoberogene soroparvovec (Ixo-vec) in Non-Human Primate Eye (ARVO 2025) - P1, P2 | "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

Ixoberogene Soroparvovec (Ixo-vec) Intravitreal Gene Therapy for Neovascular AMD: 4-Year Safety and Efficacy Results from the OPTIC Trial (ARVO 2025) - P | "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."

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

In Situ Hybridization Demonstrates Ocular Distribution of Ixo-vec IVT Gene Therapy in Human Cadaver Eyes with wAMD and Supports Durable Clinical Efficacy (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."

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

Nonclinical study of ixo-vec gene therapy for nAMD supports efficacy for a human dose of 6E10 vg/eye and staggered dosing of fellow eyes. (PubMed, Mol Ther Methods Clin Dev) - P1, P2 | "Staggered dosing, administered 2 months apart, did not exacerbate IOI, and both eyes maintained therapeutic aflibercept levels. These findings support the tolerability and efficacy of staggered dosing, indicating the potential for bilaterally relevant aflibercept levels with ixo-vec, due to immune response confinement to the dosed eye."

Journal • Age-related Macular Degeneration • Gene Therapies • Inflammation • Macular Degeneration • Ocular Inflammation • 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 | Not yet recruiting | Sponsor: Adverum Biotechnologies, Inc.

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

Adverum Biotechnologies Initiates ARTEMIS Phase 3 Study Evaluating Ixo-vec for Wet AMD (GlobeNewswire) - "Adverum Biotechnologies, Inc...today announced the initiation of the ARTEMIS Phase 3 study. This pivotal trial is designed to evaluate the efficacy and safety of Ixo-vec (ixoberogene soroparvovec) as a one-time intravitreal (IVT) injection for patients with neovascular (wet) age-related macular degeneration (AMD)....ARTEMIS is a US-based study evaluating a single administration of Ixo-vec (6E10 vg/eye) compared to aflibercept (2mg) every 8 weeks in approximately 284 patients with wet AMD, including both treatment-naïve and previously treated patients."

New P3 trial • Wet Age-related Macular Degeneration

Safety and Efficacy of ADVM-022 in Treatment-Experienced Patients With Neovascular Age-related Macular Degeneration [LUNA] (clinicaltrials.gov) - P2 | N=69 | Active, not recruiting | Sponsor: Adverum Biotechnologies, Inc. | Trial completion date: Nov 2028 ➔ Aug 2028 | Trial primary completion date: Aug 2028 ➔ Aug 2024

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

Adverum Biotechnologies Announces Webcast to Report 52-Week LUNA Phase 2 Clinical Data and 4-Year OPTIC Clinical Data, and to Provide Key Pivotal Program Design Elements (GlobeNewswire) - "Adverum Biotechnologies, Inc...plans to host a webcast to report 52-week LUNA phase 2 clinical data and 4-year OPTIC clinical data, and to provide key pivotal program design elements Monday, November 18th at 7:30 a.m. EST."

P1 data • P2 data • Age-related Macular Degeneration • Ophthalmology • Wet Age-related Macular Degeneration

Adverum Biotechnologies Reports Third Quarter 2024 Financial Results, Provides Corporate Highlights and Updates Anticipated Milestones (GlobeNewswire) - "Upcoming Anticipated Milestones: 4Q 2024: LUNA 52-week data update, including all-available safety follow-up; 4Q 2024: OPTIC 4-year clinical data update; 4Q 2024: Phase 3 pivotal trial design update; H1 2025: Planned initiation of Phase 3 trial...Research and development expenses were $20.4 million for the three months ended September 30, 2024 compared to $20.7 million for the same period in 2023. Research and development expenses decreased due to lower facilities related expenses partially offset by higher spending on Ixo-vec clinical development and higher compensation expenses. Stock-based compensation expense included in research and development expenses was $1.1 million for the third quarter of 2024."

Commercial • New P3 trial • P1 data • P2 data • Age-related Macular Degeneration • Diabetic Macular Edema • Ophthalmology • Wet Age-related Macular Degeneration

Adverum Biotechnologies Appoints Jason L. Mitchell as Chief Commercial Officer as it Prepares to Initiate Pivotal Program (GlobeNewswire) - "Ixo-vec is currently being evaluated in the Phase 2 LUNA clinical trial. The company plans to announce LUNA 52-week data and pivotal program details in the 4th quarter of 2024. In addition, the company is on track to initiate the Ixo-vec pivotal program in the 1st half of 2025."

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

Exploring new horizons in neovascular age-related macular degeneration: novel mechanisms of action and future therapeutic avenues. (PubMed, Eye (Lond)) - "Gene therapies, including RGX-314 and Ixo-vec, express an anti-VEGF protein, and 4D-150, expresses an anti-VEGF protein and a VEGF-C inhibitory miRNA. Anti-VEGF associated therapeutics include OPT-302, targeting VEGF-C and VEGF-D, BI 836880, which inhibits VEGF-A and Ang-2 activity, and Tarcocimab tedromer, inhibiting all VEGF-A isoforms. Agents with novel mechanisms of action include UBX1325, which inhibits an anti-apoptotic protein, Restoret (EYE103), a Wnt agonist, and the tyrosine kinase inhibitors, EYP-1901, OTX-TKI, CLS-AX, and KHK4951."

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

Adverum Biotechnologies Reports Second Quarter 2024 Financial Results and Provides Program and Corporate Highlights (GlobeNewswire) - "Upcoming Anticipated Milestones...4Q 2024: Clinical data from the landmark LUNA 9-month analysis; 4Q 2024: Phase 3 pivotal trial design update; 1Q 2025: Clinical data from the landmark LUNA 52-week analysis; H1 2025: Planned initiation of Phase 3 trial."

Clinical protocol • New P3 trial • P2 data • Age-related Macular Degeneration • Diabetic Macular Edema • Diabetic Retinopathy • Ophthalmology • Wet Age-related Macular Degeneration

Adverum Biotechnologies Announces FDA Regenerative Medicine Advanced Therapy (RMAT) Designation Granted for Ixo-vec for the Treatment of Wet AMD (GlobeNewswire) - "Adverum Biotechnologies, Inc...today announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation for Ixo-vec, its clinical-stage gene therapy product candidate, for the treatment of wet AMD...'The RMAT designation is based on the clinical data from Ixo-vec to date and recognizes the transformative potential of our gene therapy for patients with wet AMD...we look forward to continuing our close collaborations with both the FDA and EMA on our pivotal program and to sharing updates from regulatory discussions in the fourth quarter of this year."

FDA event • Macular Degeneration • Ophthalmology • Wet Age-related Macular Degeneration

Adverum Biotechnologies Presents Positive Ixo-vec Clinical Data from the 26-Week Interim Analysis of the LUNA Phase 2 Trial at the 2024 ASRS Annual Meeting (GlobeNewswire) - "Upcoming Anticipated Milestones: 2H 2024: Continued FDA and EMA formal and informal regulatory interactions; 4Q 2024: Presentation of landmark LUNA 9-month analysis; 4Q 2024: Phase 3 pivotal trial design update; 1Q 2025: Presentation of landmark LUNA 52-week analysis; H1 2025: Planned initiation of Phase 3 trial."

Clinical protocol • European regulatory • FDA event • New P3 trial • P2 data • Age-related Macular Degeneration • Macular Degeneration • Ophthalmology • Wet Age-related Macular Degeneration

Adverum Biotechnologies Presents Positive Ixo-vec Clinical Data from the 26-Week Interim Analysis of the LUNA Phase 2 Trial at the 2024 ASRS Annual Meeting (GlobeNewswire) - P2 | N=69 | LUNA (NCT05536973) | Sponsor: Adverum Biotechnologies, Inc. | "Adverum Biotechnologies, Inc...today announced results from the landmark 26-week interim analysis of the ongoing LUNA Phase 2 trial of Ixo-vec in patients with wet age-related macular degeneration (AMD)...Ixo-vec demonstrated a significant proportion of patients injection free at both doses at week 26. 6E10: 76% of patients injection free; (n=29)2E11: 83% of patients injection free (n=29)...Visual acuity was maintained at both dose levels – least squares mean BCVA change from baseline at week 26 (95% CI): 6E10: -1.1 (-3.5, 1.2); 2E11: -2.2 (-4.5, 0.2)...Ixo-vec was well tolerated at both doses. No Ixo-vec-related serious adverse events...All Ixo-vec-related adverse events were either mild or moderate."

P2 data • Age-related Macular Degeneration • Macular Degeneration • Ophthalmology • Wet Age-related Macular Degeneration

Adverum Biotechnologies to Host Webcast to Review Clinical Data from the 26-Week Interim Analysis of the Ongoing LUNA Phase 2 Trial in Wet AMD Being Presented at ASRS Annual Meeting (GlobeNewswire) - "Adverum Biotechnologies, Inc...announced that the company will host a webcast to review the interim 26-week landmark data from its ongoing LUNA Phase 2 study of ixoberogene soroparvovec (Ixo-vec) for the treatment of wet age-related macular degeneration (wet AMD) being presented at the American Society of Retina Specialists (ASRS) Annual Scientific Meeting. The webcast will be held on Wednesday, July 17 at 8:00 a.m. ET and will include a presentation from Dr. Charles C. Wykoff, Director of Research, Retina Consultants of Texas, Professor of Clinical Ophthalmology, Blanton Eye Institute, Houston Methodist Hospital, and principal investigator in the LUNA study."

P2 data • Age-related Macular Degeneration • Ophthalmology • Wet Age-related Macular Degeneration

Gene Therapy for Non-Hereditary Retinal Disease: Age-Related Macular Degeneration, Diabetic Retinopathy, and Beyond. (PubMed, Genes (Basel)) - "This manuscript reviews ongoing gene therapy clinical trials for these disorders, including ABBV-RGX-314, ixoberogene soroparvovec (ixo-vec), and 4D-150. ABBV-RGX-314 utilizes an adeno-associated virus (AAV) vector to deliver a transgene encoding a ranibizumab-like anti-VEGF antibody fragment, demonstrating promising results in Phase 1/2a and ongoing Phase 2b/3 trials...Other therapies reviewed include EXG102-031, FT-003, KH631, OLX10212, JNJ-1887, 4D-175, and OCU410. These therapies offer potential advantages of reduced treatment frequency and enhanced safety profiles, representing a paradigm shift in management towards durable and efficacious cellular-based biofactories. These advancements in gene therapy hold promise for improving outcomes in AMD and addressing the complex challenges of DME and DR, providing new avenues for the treatment of diabetic eye diseases."

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