AbbVie and REGENXBIO Announce Updates on the ABBV-RGX-314 Clinical Program
AbbVie (ABBV) and REGENXBIO (RGNX) have announced key updates for their ABBV-RGX-314 clinical program. The companies expect pivotal data on the safety and efficacy of subretinal delivery for wet age-related macular degeneration (wet AMD) patients in 2026 through the ATMOSPHERE® and ASCENT™ trials.
Additionally, both companies will plan a Phase 3 clinical program for diabetic retinopathy (DR) treatment using suprachoroidal delivery via the SCS Microinjector®. This gene therapy aims to address the treatment burden of chronic, frequent dosing in retinal diseases, which are among the leading causes of blindness. ABBV-RGX-314 is being developed as the first potential one-time gene therapy for both wet AMD and DR patients.
AbbVie (ABBV) e REGENXBIO (RGNX) hanno annunciato aggiornamenti fondamentali per il loro programma clinico ABBV-RGX-314. Le aziende si aspettano dati cruciali sulla sicurezza e l'efficacia della somministrazione subretinica per i pazienti con degenerazione maculare senile umida (AMD umida) nel 2026 attraverso i trial ATMOSPHERE® e ASCENT™.
Inoltre, entrambe le aziende pianificheranno un programma clinico di Fase 3 per il trattamento della retinopatia diabetica (DR) utilizzando la somministrazione suprachoroidale tramite il SCS Microinjector®. Questa terapia genica mira a affrontare il carico terapeutico della somministrazione cronica e frequente nelle malattie retiniche, che sono tra le principali cause di cecità. L'ABBV-RGX-314 è in fase di sviluppo come la prima potenziale terapia genica da somministrare una sola volta per i pazienti con AMD umida e DR.
AbbVie (ABBV) y REGENXBIO (RGNX) han anunciado actualizaciones clave para su programa clínico ABBV-RGX-314. Las compañías esperan datos fundamentales sobre la seguridad y eficacia de la administración subretiniana para los pacientes con degeneración macular relacionada con la edad húmeda (AMD húmeda) en 2026 a través de los ensayos ATMOSPHERE® y ASCENT™.
Además, ambas compañías planean un programa clínico de Fase 3 para el tratamiento de la retinopatía diabética (DR) utilizando la administración suprachoroidal mediante el SCS Microinjector®. Esta terapia génica tiene como objetivo abordar la carga del tratamiento de la dosificación crónica y frecuente en enfermedades retinianas, que son una de las principales causas de ceguera. El ABBV-RGX-314 se está desarrollando como la primera terapia génica potencial de una sola vez para pacientes con AMD húmeda y DR.
AbbVie (ABBV)와 REGENXBIO (RGNX)는 ABBV-RGX-314 임상 프로그램에 대한 주요 업데이트를 발표했습니다. 이들 회사는 2026년까지 습성 연령 관련 황반 변성 (습성 AMD) 환자에 대한 망막하 약물 전달의 안전성과 유효성에 대한 중요한 데이터를 예상하고 있습니다. 이는 ATMOSPHERE® 및 ASCENT™ 임상 시험을 통해 진행됩니다.
추가로, 두 회사는 SCS Microinjector®를 통한 상풍지 전달을 이용한 당뇨망막병증(DR) 치료를 위한 3상 임상 프로그램을 계획할 것입니다. 이 유전자 치료는 만성적이고 자주 약물을 투여해야 하는 문제를 해결하여 망막 질환에서의 치료 부담을 줄이는 것을 목표로 하고 있으며, 이는 실명 원인 중 하나입니다. ABBV-RGX-314는 습성 AMD와 DR 환자를 위한 잠재적인 단회 투여 유전자 치료로 개발되고 있습니다.
AbbVie (ABBV) et REGENXBIO (RGNX) ont annoncé des mises à jour clés concernant leur programme clinique ABBV-RGX-314. Les entreprises prévoient des données pivots sur la sécurité et l'efficacité de l'administration subrétinienne pour les patients atteints de dégénérescence maculaire liée à l'âge humide (AMD humide) en 2026 grâce aux essais ATMOSPHERE® et ASCENT™.
De plus, les deux entreprises vont planifier un programme clinique de phase 3 pour le traitement de la rétinopathie diabétique (DR) utilisant l'administration suprachoroïdienne via le SCS Microinjector®. Cette thérapie génique vise à alléger la charge de traitement de la posologie chronique et fréquente dans les maladies rétiniennes, qui sont parmi les principales causes de cécité. L'ABBV-RGX-314 est en cours de développement comme la première thérapie génique potentielle à administrer en une seule fois pour les patients atteints d'AMD humide et de DR.
AbbVie (ABBV) und REGENXBIO (RGNX) haben wichtige Updates zu ihrem klinischen Programm ABBV-RGX-314 bekannt gegeben. Die Unternehmen erwarten im 2026 Jahr entscheidende Daten zur Sicherheit und Wirksamkeit der subretinalen Verabreichung bei Patienten mit feuchter altersbedingter Makuladegeneration (feuchte AMD) durch die Studien ATMOSPHERE® und ASCENT™.
Darüber hinaus planen beide Unternehmen ein Phase-3-Klinikprogramm zur Behandlung der diabetischen Retinopathie (DR), das eine suprakoreale Verabreichung über den SCS Microinjector® nutzt. Diese Gentherapie zielt darauf ab, die Behandlungsbelastung durch chronische, häufige Dosierung bei Netzhauterkrankungen, die zu den häufigsten Ursachen von Erblindung gehören, zu verringern. ABBV-RGX-314 wird als die erste potenzielle genbasierte Therapie entwickelt, die nur einmalig für Patienten mit feuchter AMD und DR angewendet werden kann.
- Development of first potential one-time gene therapy for wet AMD and DR
- Advancement to pivotal trials for wet AMD indication
- Planned expansion into Phase 3 trials for diabetic retinopathy
- Pivotal trial data not expected until 2026, indicating a lengthy timeline to potential commercialization
Insights
The ABBV-RGX-314 clinical program represents a significant advancement in gene therapy for ophthalmology. The dual-delivery approach (subretinal and suprachoroidal) demonstrates sophisticated strategic planning to address different retinal conditions. The ATMOSPHERE® and ASCENT™ pivotal trials for wet AMD using subretinal delivery are particularly noteworthy, as this method allows for precise targeting of retinal cells, potentially enabling sustained therapeutic protein expression.
The expansion into diabetic retinopathy with suprachoroidal delivery using the SCS Microinjector® is strategically important. This in-office procedure could significantly reduce the barrier to treatment adoption, as it's less invasive than subretinal delivery. The market potential is substantial - wet AMD affects approximately 1.1 million Americans, while diabetic retinopathy impacts over 7.7 million. Current standard treatments like anti-VEGF injections require frequent administration, creating a significant burden on patients and healthcare systems.
For investors, the 2026 timeline for pivotal data suggests a potential market entry in 2027-2028, assuming positive results and regulatory approval. The program could establish AbbVie as a leader in ophthalmologic gene therapy, a rapidly growing market segment projected to reach
This clinical program update reveals AbbVie's strategic positioning in the high-value ophthalmology market. The collaboration with REGENXBIO combines AbbVie's commercial strength with REGENXBIO's gene therapy expertise. The addressable market is substantial - the global wet AMD therapeutics market alone is valued at
The program's potential to disrupt the current treatment paradigm is significant. If successful, ABBV-RGX-314 could challenge established players like Regeneron's Eylea® and Roche's Vabysmo®. A one-time gene therapy solution would be revolutionary in a market dominated by recurring treatments. This represents a major revenue opportunity for AbbVie, potentially helping offset future biosimilar competition to Humira.
The dual-indication strategy (wet AMD and DR) maximizes the commercial potential and diversifies clinical risk. Success in either indication would establish AbbVie as a pioneer in ophthalmic gene therapy, potentially leading to premium pricing power and significant market share capture.
- Pivotal data evaluating the safety and efficacy of the subretinal delivery of ABBV-RGX-314 in patients with wet age-related macular degeneration (wet AMD) are expected in 2026
- AbbVie and REGENXBIO will plan the Phase 3 clinical program of investigational ABBV-RGX-314 in diabetic retinopathy (DR)
ABBV-RGX-314 in Wet Age-Related Macular Degeneration (wet AMD), Subretinal Delivery
Data from the ATMOSPHERE® and ASCENT™ pivotal trials evaluating the safety and efficacy of the subretinal delivery of ABBV-RGX-314 in patients with wet AMD are expected in 2026.
ABBV-RGX-314 in Diabetic Retinopathy (DR), Suprachoroidal Delivery
AbbVie and REGENXBIO will plan a Phase 3 clinical program. The clinical program will utilize the in-office SCS Microinjector® to deliver gene therapy to the suprachoroidal space of the eye.
"Retinal diseases are progressive, with wet AMD and DR among the leading causes of blindness," said Michael Robinson, M.D., vice president, global head of ophthalmology clinical development, AbbVie. "More treatment options are needed to help relieve the current treatment burden of chronic, frequent dosing. We are excited to continue moving closer to our goal of delivering an additional treatment option to patients with wet AMD and DR in hopes of addressing their significant unmet needs."
"ABBV-RGX-314 has the potential to help millions of people living with wet AMD and DR, globally, who are facing these debilitating diseases," said Curran Simpson, president and chief executive officer, REGENXBIO. "Together with AbbVie, we are excited to continue developing ABBV-RGX-314 as the first potential one-time gene therapy for wet AMD and DR."
About ABBV-RGX-314
ABBV-RGX-314 is being investigated as a potential one-time treatment for wet AMD, diabetic retinopathy and potentially other chronic retinal conditions. ABBV-RGX-314 consists of the NAV® AAV8 vector, which encodes an antibody fragment designed to inhibit vascular endothelial growth factor (VEGF). ABBV-RGX-314 is believed to inhibit the VEGF pathway by which new, leaky blood vessels grow and contribute to the accumulation of fluid in the retina.1
AbbVie and REGENXBIO are advancing the development of two separate routes of administration of ABBV-RGX-314 to the eye, through a standardized subretinal delivery procedure as well as delivery to the suprachoroidal space. REGENXBIO has licensed certain exclusive rights to the SCS Microinjector® from Clearside Biomedical, Inc. to deliver gene therapy treatments to the suprachoroidal space of the eye.
About Wet AMD
Wet AMD is characterized by loss of vision due to new, leaky blood vessel formation in the retina.2 Wet AMD is a significant cause of vision loss in the United States, Europe and Japan, with up to two million people living with wet AMD in these geographies alone.3 Current anti-VEGF therapies have significantly changed the landscape for treatment of wet AMD, becoming the standard of care due to their ability to prevent progression of vision loss in the majority of patients.4 These therapies, however, require life-long repeated intraocular injections, to maintain efficacy.5,6 Due to the burden of treatment, patients often experience a decline in vision with reduced frequency of treatment over time.7
About Diabetic Retinopathy
Diabetic retinopathy (DR) is the leading cause of vision loss in adults between 24 and 75 years of age worldwide.8 DR affects nearly 10 million people in
About AbbVie
AbbVie's mission is to discover and deliver innovative medicines and solutions that solve serious health issues today and address the medical challenges of tomorrow. AbbVie strives to have a remarkable impact on people's lives across several key therapeutic areas – immunology, oncology, neuroscience, and eye care – and products and services in AbbVie's Allergan Aesthetics portfolio. For more information about AbbVie, please visit us at www.abbvie.com. Follow @abbvie on LinkedIn, Facebook, Instagram, X (formerly Twitter), and YouTube.
ABOUT REGENXBIO Inc.
REGENXBIO is a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy. Since its founding in 2009, REGENXBIO has pioneered the development of AAV Therapeutics, an innovative class of gene therapy medicines. REGENXBIO is advancing a pipeline of AAV Therapeutics for rare and retinal diseases, including RGX-202 for the treatment of Duchenne, ABBV-RGX-314 for the treatment of wet AMD and diabetic retinopathy, being developed in collaboration with AbbVie, and RGX 121 for the treatment of MPS II. Thousands of patients have been treated with REGENXBIO's AAV Therapeutic platform, including Novartis' ZOLGENSMA® for children with spinal muscular atrophy. Designed to be one-time treatments, AAV Therapeutics have the potential to change the way healthcare is delivered for millions of people. For more information, please visit www.regenxbio.com.
Forward-Looking Statements
Some statements in this news release are, or may be considered, forward-looking statements for purposes of the Private Securities Litigation Reform Act of 1995. The words "believe," "expect," "anticipate," "project" and similar expressions and uses of future or conditional verbs, generally identify forward-looking statements. AbbVie cautions that these forward-looking statements are subject to risks and uncertainties that may cause actual results to differ materially from those expressed or implied in the forward-looking statements. Such risks and uncertainties include, but are not limited to, challenges to intellectual property, competition from other products, difficulties inherent in the research and development process, adverse litigation or government action, and changes to laws and regulations applicable to our industry. Additional information about the economic, competitive, governmental, technological and other factors that may affect AbbVie's operations is set forth in Item 1A, "Risk Factors," of AbbVie's 2023 Annual Report on Form 10-K, which has been filed with the Securities and Exchange Commission, as updated by its subsequent Quarterly Reports on Form 10-Q. AbbVie undertakes no obligation, and specifically declines, to release publicly any revisions to forward-looking statements as a result of subsequent events or developments, except as required by law.
1 Penn JS, Madan A, Caldwell RB, et al. Vascular endothelial growth factor in eye disease. Prog Retin Eye Res. 2008;27(4):331-71.
2 Carmeliet P. Angiogenesis in life, disease and medicine. Nature. 2005;438:932-6.
3 Decision Resources Group, 2019
4 Alexandru MR, Alexandra NM. Wet age related macular degeneration management and follow-up. Rom J Ophthalmol. 2016;60:9–13.
5 AAO PPP. Preferred Practice Patterns: Age related macular degeneration. American Academy of Ophthalmology. 2019.
6 Dugel PU, Koh A, Ogura Y, et al. HAWK and HARRIER: phase 3, multicenter, randomized, double-masked trials of brolucizumab for neovascular age-related macular degeneration. Ophthalmology. 2020;127(1):72-84.
7 Holz FG et al. Br J Ophthalmol. 2015;99:220.
8 Cheung N, Mitchell P, Wong TY. Diabetic retinopathy. Lancet. 2010;376(9735):124–36.
9 Lundeen EA, Burke-Conte Z, Rein DB, Wittenborn JS, Saaddine J, Lee AY, Flaxman AD. Prevalence of Diabetic Retinopathy in the US in 2021. JAMA Ophthalmology. 2023;141(8):747-754.
10 Berrocal MD, Alexandra Acabá. Current Management of Diabetic Retinopathy, 2018
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SOURCE AbbVie
FAQ
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