Age-Related Macular Degeneration (AMD) - Pipeline Insight, 2025

This “Age-Related Macular Degeneration (AMD) - Pipeline Insight, 2025” report provides comprehensive insights about 70+ companies and 75+ pipeline drugs in Age-Related Macular Degeneration (AMD) pipeline landscape. It covers the pipeline drug profiles, including clinical and nonclinical stage products. It also covers the therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.

Age-Related Macular Degeneration (AMD): Understanding

Age-Related Macular Degeneration (AMD): Overview

Age-Related Macular Degeneration (AMD) is a chronic, progressive eye disease that primarily affects individuals over the age of 50. It damages the macula, the central part of the retina, leading to blurred or complete loss of central vision, while peripheral vision usually remains intact. AMD is classified into two main types: dry (atrophic) and wet (neovascular), with dry AMD being more common but less severe. Risk factors include aging, genetics, smoking, and high blood pressure. Currently, there is no cure, but treatments aim to slow disease progression and manage symptoms. Age-Related Macular Degeneration (AMD) typically presents with a gradual loss of central vision, making it difficult to read, recognize faces, or perform tasks requiring fine visual detail. Early symptoms may include blurred or distorted vision, where straight lines appear wavy (known as metamorphopsia). As the disease progresses, a dark or empty area may appear in the center of vision. Colors may seem less vivid, and increased sensitivity to glare or low-light conditions can occur. In the wet form of AMD, vision loss can be more sudden and severe due to abnormal blood vessel growth.

Age-Related Macular Degeneration (AMD) is primarily caused by the aging of the macula, the part of the retina responsible for sharp central vision. Over time, accumulation of waste material called drusen beneath the retina can damage retinal cells and impair vision, particularly in dry AMD. In wet AMD, abnormal blood vessels grow under the retina and leak fluid or blood, leading to rapid vision loss. Genetic factors, such as mutations in the CFH and ARMS2 genes, significantly increase risk. Environmental factors like smoking, poor diet, obesity, and excessive sun exposure also contribute to disease development.

Age-Related Macular Degeneration (AMD) is broadly classified into two main types:

Dry AMD (Atrophic AMD): This is the more common form, accounting for about 85-90% of cases. It involves the gradual thinning of the macula and accumulation of drusen, leading to slow, progressive vision loss.

Wet AMD (Neovascular or Exudative AMD): Less common but more severe, this type is characterized by abnormal blood vessel growth under the retina that leaks fluid or blood, causing rapid and significant central vision loss.

Age-Related Macular Degeneration (AMD) is diagnosed through a comprehensive eye exam that includes a visual acuity test and a dilated retinal examination. Amsler grid testing is used to detect visual distortions characteristic of AMD. Imaging techniques like optical coherence tomography (OCT) help visualize retinal layers and detect fluid or atrophy. Fluorescein angiography may be used to identify abnormal blood vessels in wet AMD. Early detection is key to slowing disease progression.

Treatment for Age-Related Macular Degeneration (AMD) depends on the type and stage of the disease. For dry AMD, there is no cure, but progression may be slowed with lifestyle changes and nutritional supplements, particularly those in the AREDS2 formulation containing antioxidants and zinc. For wet AMD, anti-VEGF injections (e.g., ranibizumab, aflibercept) are the mainstay treatment, targeting abnormal blood vessel growth and leakage. Other options include photodynamic therapy and laser therapy in select cases. Ongoing research is exploring gene therapy, complement inhibitors, and regenerative treatments.

"Age-Related Macular Degeneration (AMD) - Pipeline Insight, 2025" report outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Age-Related Macular Degeneration (AMD) pipeline landscape is provided which includes the disease overview and Age-Related Macular Degeneration (AMD) treatment guidelines. The assessment part of the report embraces, in depth Age-Related Macular Degeneration (AMD) commercial assessment and clinical assessment of the pipeline products under development. In the report, detailed description of the drug is given which includes mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, Age-Related Macular Degeneration (AMD) collaborations, licensing, mergers and acquisition, funding, designations and other product related details.

Report Highlights
The companies and academics are working to assess challenges and seek opportunities that could influence Age-Related Macular Degeneration (AMD) R&D. The therapies under development are focused on novel approaches to treat/improve Age-Related Macular Degeneration (AMD).

Age-Related Macular Degeneration (AMD) Emerging Drugs Chapters

This segment of the Age-Related Macular Degeneration (AMD) report encloses its detailed analysis of various drugs in different stages of clinical development, including phase III, II, II/III I, preclinical and Discovery. It also helps to understand clinical trial details, expressive pharmacological action, agreements and collaborations, and the latest news and press releases.

Age-Related Macular Degeneration (AMD) Emerging Drugs

Surabgene Lomparvovec: AbbVie

Surabgene Lomparvovec (ABBV-RGX-314) is an investigational one-time gene therapy being developed by AbbVie in collaboration with REGENXBIO. It utilizes an AAV8 viral vector to deliver a gene encoding an anti-VEGF monoclonal antibody fragment, aiming to inhibit abnormal blood vessel growth in the retina. The therapy is being evaluated in both subretinal and suprachoroidal delivery formats for chronic retinal conditions such as wet Age-Related Macular Degeneration (wet AMD) and diabetic retinopathy. Interim Phase 2 data have shown promising results, including a significant reduction (up to 97%) in the need for supplemental anti-VEGF injections and stable visual acuity outcomes. Two pivotal Phase 3 trials - ATMOSPHERE (subretinal delivery) and ASCENT (suprachoroidal delivery) - are currently ongoing, with topline data expected in 2026. Currently, the drug is in the Phase III stage of its development for the treatment of Age Related Macular Degeneration.

Elamipretide Stealth BioTherapeutics

Elamipretide, developed by Stealth BioTherapeutics, is a mitochondria-targeting tetrapeptide designed to bind cardiolipin in the inner mitochondrial membrane, thereby enhancing ATP production and reducing oxidative stress  . Currently in late-stage development across several indications, it is the subject of the global Phase III ReNEW trial for dry Age-Related Macular Degeneration (AMD), evaluating daily subcutaneous dosing to slow photoreceptor loss, with topline data expected in 2026. Earlier Phase II results supported the FDA’s recognition of ellipsoid zone attenuation as a valid endpoint and elamipretide holds Fast Track designation for dry AMD, its application for Barth syndrome is under FDA review after a recent resubmission request, with the agency showing openness to accelerated approval based on muscle-strength endpoints.

Iptacopan: Novartis Pharmaceuticals

Iptacopan (LNP023) is an oral complement factor B inhibitor being developed by Novartis as a potential treatment for early to intermediate Age-Related Macular Degeneration (AMD), aimed at reducing progression to late-stage disease. It is currently in a Phase II, randomized, placebo-controlled proof-of-concept trial, enrolling patients aged ≥50 who have early/intermediate AMD in one eye and neovascular AMD in the other; the study’s primary goal is to prevent progression to atrophy or late-stage AMD over two years. As an oral therapy, it offers a major advantage over current intravitreal treatments by potentially improving patient compliance, convenience, and safety in elderly populations eliminating injection-related risks. Currently, the drug is in the Phase II stage of its development for the treatment of Age Related Macular Degeneration.

BBRP 11001: BenoBio

BBRP 11001, also known as BBC-1501, is an investigational small-molecule inhibitor developed by BenoBio that targets BET (bromodomain and extra-terminal) proteins. It is designed to modulate epigenetic mechanisms underlying inflammation and angiogenesis, making it a promising candidate for the treatment of neovascular (wet) Age-Related Macular Degeneration (AMD). The therapy is currently undergoing a Phase I dose-escalation clinical trial (NCT05803785) in Australia, where it is being evaluated for safety, tolerability, and preliminary efficacy in patients who have shown inadequate response to standard anti-VEGF treatments. Administered via intravitreal injection, BBRP 11001 is being tested in ascending doses ranging from 1.25 µg to 5 µg. Following the initiation of this first-in-human trial in mid-2024, a Phase 2 study is anticipated in 2025. If successful, BBRP 11001 could offer a novel, epigenetically driven therapeutic option for managing chronic retinal diseases. Currently, the drug is in the Phase I stage of its development for the treatment of Age Related Macular Degeneration.

SZN 8143: Surrozen

SZN 8143 is an innovative trispecific antibody candidate from Surrozen, leveraging their Wnt pathway-modulating SWAP™ platform to address serious retinal diseases. It uniquely combines Fzd4 agonism, VEGF antagonism, and IL 6 antagonism, aiming to not only suppress pathological angiogenesis but also promote healthy vessel regrowth and reduce inflammation - offering potential therapeutic advantages over standard anti VEGF monotherapy in diabetic macular edema, wet AMD, and uveitic macular edema. Currently in advanced preclinical development, Surrozen is preparing to advance SZN 8143 toward IND-enabling studies alongside its sister candidate SZN 8141, supported by a focused $175 million funding round aimed at accelerating these ophthalmology programs. Given its trispecific mechanism and strategic development plan, SZN 8143 represents a hopeful new approach to treating retinopathies with multifaceted pathological components. Currently, the drug is in the Preclinical stage of its development for the treatment of Age Related Macular Degeneration.

Age-Related Macular Degeneration (AMD): Therapeutic Assessment

This segment of the report provides insights about the different Age-Related Macular Degeneration (AMD) drugs segregated based on following parameters that define the scope of the report.

Major Players in Age-Related Macular Degeneration (AMD)
There are approx. 70+ key companies which are developing the therapies for Age-Related Macular Degeneration (AMD). The companies which have their Age-Related Macular Degeneration (AMD) drug candidates in the most advanced stage, i.e. Phase III include, AbbVie.

Phases
The report covers around 75+ products under different phases of clinical development, like:

• Late stage products (Phase III)
• Mid-stage products (Phase II)
• Early-stage product (Phase I) along with the details of:
• Pre-clinical and Discovery stage candidates
• Discontinued & Inactive candidates

Route of Administration
Age-Related Macular Degeneration (AMD) pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs, such as:

• Intravenous
• Subcutaneous
• Oral
• Intramuscular

Molecule Type
Products have been categorized under various Molecule types, such as:

• Monoclonal antibody
• Small molecule
• Peptide

Product Type
Drugs have been categorized under various product types like Mono, Combination and Mono/Combination.

Age-Related Macular Degeneration (AMD): Pipeline Activities

The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Age-Related Macular Degeneration (AMD) therapeutic drugs key players involved in developing key drugs.

Development Activities

The report covers the detailed information of collaborations, acquisition and merger, licensing along with a thorough therapeutic assessment of emerging Age-Related Macular Degeneration (AMD) drugs.

Age-Related Macular Degeneration (AMD) Report Insights

• Age-Related Macular Degeneration (AMD) Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

Age-Related Macular Degeneration (AMD) Report Assessment

• Pipeline Product Profiles
• Therapeutic Assessment
• Pipeline Assessment
• Inactive drugs assessment
• Unmet Needs

Key Questions

Current Treatment Scenario and Emerging Therapies:

• How many companies are developing Age-Related Macular Degeneration (AMD) drugs?
• How many Age-Related Macular Degeneration (AMD) drugs are developed by each company?
• How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Age-Related Macular Degeneration (AMD)?
• What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the Age-Related Macular Degeneration (AMD) therapeutics?
• What are the recent trends, drug types and novel technologies developed to overcome the limitation of existing therapies?
• What are the clinical studies going on for Age-Related Macular Degeneration (AMD) and their status?
• What are the key designations that have been granted to the emerging drugs?

Key Players

• AbbVie
• RemeGen
• Stealth BioTherapeutics
• Theratocular Biotek
• Novartis Pharmaceuticals
• Clearside Biomedical
• BenoBio
• Chengdu Origen Biotechnology Co., Ltd
• Surrozen
• Anida Pharma
• Belite Bio, Inc
• Luxa Biotechnology LLC

Key Products

• Surabgene Lomparvovec
• RC 28 E
• Elamipretide
• TO O 1002
• Iptacopan (LNP023)
• CLS-AX
• BBRP 11001
• KH658
• SZN 8143
• AP 001
• Tinlarebant
• RPESC-RPE-4W

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