Dry Age-related Macular Degeneration (Dry-AMD) - Pipeline Insight, 2024

This “Dry Age-related Macular Degeneration (Dry-AMD)- Pipeline Insight, 2024” report provides comprehensive insights about 70+ companies and 80+ pipeline drugs in Dry Age-related Macular Degeneration (Dry-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.

Geography Covered

• Global coverage

Dry Age-related Macular Degeneration (Dry-AMD): Understanding

Dry Age-related Macular Degeneration (Dry-AMD): Overview

Age-related macular degeneration (AMD) is a degenerative disease of the center area of the ocular-posterior segment (the macula lutea) that causes slow degeneration in central vision and severe disability in affected persons. “Age-related” means that it mainly occurs with elderly adults, while “Macular” means that the macula part of the eye is damaged. AMD mostly affects people aged above 50, and it was first reported more than 80 years ago. One of the top four causes of blindness in the elderly is AMD. It is also the leading cause of irreversible blindness in the developed countries. A total population of about 170 million individuals is affected with AMD globally, thus being the third leading cause of vision loss in the world. Usually, dry AMD progresses very slowly whereas, some people experience severe visual impairment within days or weeks of the onset of wet AMD. No symptoms appear during the early stage of the disease. During this initial stage, an ophthalmologist can detect drusen long before symptoms occur. Symptoms that typically occur during the intermediate stage include minor alterations in eyesight; however, many people do not have any symptoms at this time. Some people see black or grey areas in the middle of their visual field or have difficulty adapting from a brightly illuminated environment to a dark environment.

Currently, clinicians monitor morphological changes in the retina/RPE/choroid by fundus exam, color fundus photography, FAF, OCT, and infrared (IR) reflectance. In the past two decades, OCT140 imaging technologies have had a profound impact on early detection, monitoring of progression, and treatment-efficacy evaluation of dry AMD. The current generation of commercialized OCT systems, SD-OCT, provides volumetric and cross-sectional views of the retina facilitating the visualization, measurement, monitoring, and phenotyping of the retinal layers and RPE,141-143 hyperreflective foci,144 GA,145,146 and drusen147 in eyes with dry AMD. While SD-OCT imaging is currently widely used in clinics as the standard of care for dry AMD diagnosis and prognosis, some emerging developments in OCT technology may lead to paradigm shifts in the way that dry AMD imaging is performed for research and clinical applications. One such technology is the polarization-sensitive OCT (PS-OCT) with tissue-selective imaging capabilities.

In contrast, there are not any approved treatments for dry AMD. The management of dry AMD consists of observation, regular follow-up evaluations, and documentation for timely recognition of visual function deterioration with appropriate rehabilitation and early CNV detection. Several therapeutic avenues are being investigated, aiming at (1) disease prevention, (2) halting disease progression, and/or (3) restoration of vision. Treatment options includes behavioral and lifestyle modification, age-related eye disease study (AREDS) supplements, reduction of toxic by-products, visual cycle modulators, and anti-inflammatory agents and complement inhibitors, gene therapy and cell based therapies.

"Dry Age-related Macular Degeneration (Dry-AMD)- Pipeline Insight, 2024" report outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Dry Age-related Macular Degeneration (Dry-AMD) pipeline landscape is provided which includes the disease overview and Dry Age-related Macular Degeneration (Dry-AMD) treatment guidelines. The assessment part of the report embraces, in depth Dry Age-related Macular Degeneration (Dry-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, Dry Age-related Macular Degeneration (Dry-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 Dry Age-related Macular Degeneration (Dry-AMD) R&D. The therapies under development are focused on novel approaches to treat/improve Dry Age-related Macular Degeneration (Dry-AMD).

Dry Age-related Macular Degeneration (Dry-AMD) Emerging Drugs Chapters

This segment of the Dry Age-related Macular Degeneration (Dry-AMD) report encloses its detailed analysis of various drugs in different stages of clinical development, including phase II, 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.

Dry Age-related Macular Degeneration (Dry-AMD) Emerging Drugs

Tinlarebant (LBS-008): Belite Bio

LBS-008 is a first-in-class oral therapy that prevents the buildup of toxins (A2E) in the eye that cause Stargardt disease and contribute to atrophic Age-related Macular Degeneration (dry AMD). The toxins are by-products of the eye’s visual cycle which are produced from vitamin A. LBS-008 works by reducing and modulating a carrier protein, Retinol-Binding Protein 4 (RBP4) that is to transport vitamin A to the eye. LBS-008 does not directly interfere with the visual cycle, and therefore is unlikely to affect the visual cycle rate. LBS-008 received US and EU orphan drug designation (ODD) in 2017 and 2018, and rare pediatric disease designation (RPD) from the FDA in 2018 for the treatment of Stargardt disease. Currently, the drug is in Phase III stage of its development for the treatment of Dry AMD.

XIFLAM: Inflammx Therapeutics

Xiflam, an orally administered NLRP3 inflammasome inhibitor with broad application that is formulated as a tablet. InflammX will initially focus on the ophthalmic indications of Diabetic Macular Edema (DME), the intermediate stage of Aged-Related Macular Degeneration (iAMD) and the Geographic Atrophy (GA) form of AMD. Xiflam’s unique mechanism of action and its ability to cross the blood-brain and blood-retinal barriers provide a novel therapeutic approach for treating retinal diseases with an orally administered tablet capable of treating both eyes simultaneously. This disruptive technology can provide a much needed clinical alternative for treating the millions of patients with diabetic retinopathy and age-related macular degeneration. Currently, the drug is in Phase II stage of Clinical trial evaluation for the treatment of Geographic Atrophy.

OCU410: Ocugen

OCU410 (AAV-RORA) is a modifier gene therapy product candidate being developed for the treatment of dry age-related macular degeneration (Dry AMD). OCU410 utilizes an AAV delivery platform for the retinal delivery of the RORA (RAR Related Orphan Receptor A) gene. Various genes associated with AMD are regulated by RORA. The RORA protein plays an important role in lipid metabolism and demonstrates an anti-inflammatory role, which we believe could be a potential therapeutic candidate for dry AMD based on in-vitro and in-vivo (animal model) studies. Currently, the drug is in Phase I/II stage of Clinical trial evaluation for the treatment of Dry AMD.

OLX301A: OliX Pharmaceuticals

OLX301A aims to be a first-in-class therapeutics for treating both dry and wet age-related macular degeneration (AMD). The program targets a gene that plays a major role in retinal pigment epithelium (RPE) cell damage. Currently, there are no approved therapies to treat patients with both geographic atrophy (GA) and wet AMD, and one drug approved for GA is with concern regarding its efficacy. OLX301A has the potential to be a novel drug for wet AMD patients who is not responsive to conventional VEGF therapies. Currently, the drug is in Phase I stage of Clinical trial evaluation for the treatment of Dry AMD.

GS030: Gensight Biologics

GS030 is an innovative combination of two complementary components: A gene therapy product encoding a photo activatable channel rhodopsin protein, delivered via a modified AAV2 vector known as AAV2 7m8 and biomimetic goggles that stimulate the engineered retinal cells. GS030 uses optogenetics, a biologic technique that involves the transfer of a gene that encodes for a light-sensitive protein, which in turn causes neuronal cells to respond to light stimulation. GS030 includes a bio-engineered AAV2 gene therapy vector that introduces the gene of a photosensitive protein (to which we have exclusive rights in ontogenetic) into the nucleus of the target cells, in case the retinal ganglion cells, or RGCs. Currently the drug is currently in the preclinical stage of development for the treatment of Dry AMD.

Dry Age-related Macular Degeneration (Dry-AMD): Therapeutic Assessment

This segment of the report provides insights about the different Dry Age-related Macular Degeneration (Dry-AMD) drugs segregated based on following parameters that define the scope of the report, such as:

Major Players in Dry Age-related Macular Degeneration (Dry-AMD)

• There are approx. 70+ key companies which are developing the therapies for Dry Age-related Macular Degeneration (Dry-AMD). The companies which have their Dry Age-related Macular Degeneration (Dry-AMD) drug candidates in the most advanced stage, i.e. Phase III include, Belite Bio

Phases

This report covers around 80+ 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

Dry Age-related Macular Degeneration (Dry-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

• Oral
• Intravenous
• Subcutaneous
• Parenteral
• Topical
• Molecule Type

Products have been categorized under various Molecule types such as

• Recombinant fusion proteins
• Small molecule
• Monoclonal antibody
• Peptide
• Polymer
• Gene therapy
• Product Type

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

Dry Age-related Macular Degeneration (Dry-AMD): Pipeline Development Activities

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

Pipeline Development Activities

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

Dry Age-related Macular Degeneration (Dry-AMD) Report Insights

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

Dry Age-related Macular Degeneration (Dry-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 Dry Age-related Macular Degeneration (Dry-AMD) drugs?
• How many Dry Age-related Macular Degeneration (Dry-AMD) drugs are developed by each company?
• How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Dry Age-related Macular Degeneration (Dry-AMD)?
• What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the Dry Age-related Macular Degeneration (Dry-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 Dry Age-related Macular Degeneration (Dry-AMD) and their status?
• What are the key designations that have been granted to the emerging drugs?

Key Players

• Belite Bio
• Inflammx Therapeutics
• Ocugen
• OliX Pharmaceuticals
• Gensight Biologics
• Alkeus Pharmaceuticals
• Dobecure
• Cognition Therapeutics
• Aviceda Therapeutics
• Galimedix Therapeutics
• Mitotech
• Annexon, Inc.
• NGM Biopharmaceuticals
• Johnson & Johnson
• Lineage Cell Therapeutics
• Astellas Pharma
• Regenerative Patch Technologies
• Hoffmann-La Roche
• Boehringer Ingelheim
• ONL Therapeutics

Key Products

• Tinlarebant (LBS-008)
• XIFLAM
• OCU410
• OLX301A
• GS030
• ALK-001
• Etamsylate
• CT1812
• AVD-104
• GAL-101(Topical)
• Visomitin
• ANX 007
• NGM621
• JNJ 81201887
• OpRegen
• MA09-hRPE
• CPCB-RPE1
• RO7303359
• BI 771716
• ONL1204

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