Dry Age-related Macular Degeneration- Pipeline Insight, 2023

DelveInsight's , "Dry Age-related Macular Degeneration- Pipeline Insight, 2023" report provides comprehensive insights about 58+ companies and 64+ pipeline drugs in Dry Age-related Macular Degeneration 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: Understanding

Dry Age-related Macular Degeneration: 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. AMD is a complex multifactorial disease whose pathogenesis is incompletely understood and evolving, with a detailed evaluation behind the scope of this review. Several factors are believed to contribute to its pathogenesis, including genetic, oxidative stress, environmental, inflammatory and ischaemic. The presence of drusen is considered the hallmark of earlier stages of AMD, which may enlarge, become confluent and evolve to drusenoid retinal pigmented epithelium (RPE) detachments. This causes a disruption in the interaction between Bruch's membrane and RPE, inhibiting RPE function, including the crucial ability to transport photoreceptor debris across Bruch's membrane to the choriocapillaris, further facilitaing accumulation of lipofuscin and other related products including A2E. Drusen contain several proinflammatory factors including complement pathway components (and lipofuscin-related products), which have been identified as one of the main contributors to AMD development. 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.

"Dry Age-related Macular Degeneration- Pipeline Insight, 2023" report by DelveInsight outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Dry Age-related Macular Degeneration pipeline landscape is provided which includes the disease overview and Dry Age-related Macular Degeneration treatment guidelines. The assessment part of the report embraces, in depth Dry Age-related Macular Degeneration 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 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 R&D. The therapies under development are focused on novel approaches to treat/improve Dry Age-related Macular Degeneration.

Dry Age-related Macular Degeneration Emerging Drugs Chapters

This segment of the Dry Age-related Macular Degeneration 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 Emerging Drugs

ALK-001: Alkeus Pharmaceuticals

ALK-001 is once-a-day oral drug candidate currently being tested for the treatment of geographic atrophy (advanced form of dry AMD) and Stargardt disease. Toxic vitamin A aggregates called "dimers" are known to rapidly form due to a genetic defect in Stargardt, or to accumulate with age in the case of dry AMD. These toxic dimers have been implicated in the degeneration of the retina seen in Stargardt and AMD. Dimers form when two molecules of vitamin A are chemically combined. ALK-001 is form of vitamin A that has been chemically-modified to prevent its aggregation into dimers.

Elamipretide: Stealth BioTherapeutics

Elamipretide (Stealth BioTherapeutics) is a novel tetrapeptide drug currently in development for both common and orphan diseases that involve dysfunction of the mitochondria, the cellular organelles that generates chemical energy in the form of adenosine triphosphate (ATP). At the cellular level, mitochondrial dysfunction causes loss of ATP production, increased levels of reactive oxygen species (eg, superoxide), calcium dysregulation, and, if prolonged, cell death.

Elamipretide, given as systemic injection, penetrates tissues and cells and enters the mitochondria, where it reverses mitochondrial dysfunction (improved ATP production, restored mitochondrial membrane potential, normal calcium flux, and reduced superoxide generation).

CPCB-RPE1: Regenerative Patch Technologies

Regenerative Patch Technologies has developed composite subretinal implant, termed the California Project to Cure Blindness-Retinal Pigment Epithelium 1 (CPCB-RPE1), consisting of a polarized monolayer of human embryonic stem cell-derived RPE (hESC-RPE) on an ultrathin, synthetic parylene substrate designed to mimic Bruch's membrane.

AAV.sFH: Aevitas Therapeutics

AAV.sFH, is an investigational drug being developed by Aevitas Therapeutics. Aevitas advances AAV gene therapies of short-form human factor H (sFH) to restore homeostasis in disorders of complement dysregulation: dry age-related macular degeneration (Dry AMD), atypical hemolytic uremic syndrome (aHUS) and other disorders. sFH restored complement regulation in eyes of FH knockout mice with subretinal administration.

Supraphysiological expression of sFH with subretinal administration was shown in non-human primate eye. Good vector genome transfer to ocular target tissues with intravitreal administration was demonstrated in Yorkshire pigs. Transgene product sFH was present in all eye compartments with potentially clinically relevant FH levels in the porcine eye. Currently, the drug is in Preclinical stage of development for the treatment of Dry AMD.

Further product details are provided in the report……..

Dry Age-related Macular Degeneration: Therapeutic Assessment

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

Major Players in Dry Age-related Macular Degeneration

There are approx. 55+ key companies which are developing the therapies for Dry Age-related Macular Degeneration. The companies which have their Dry Age-related Macular Degeneration drug candidates in the most advanced stage, i.e. Phase III include, Alkeus Pharmaceuticals.

Phases

DelveInsight's report covers around 60+ 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 pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as:

• Intra-articular
• Intraocular
• Intrathecal
• Intravenous
• Ophthalmic
• Oral
• Parenteral
• Subcutaneous
• Topical
• Transdermal
• Molecule Type

Products have been categorized under various Molecule types such as:

• Oligonucleotide
• Peptide
• Small molecule
• Product Type

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

Dry Age-related Macular Degeneration: 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 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 drugs.

Dry Age-related Macular Degeneration Report Insights

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

Dry Age-related Macular Degeneration 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 drugs?
• How many Dry Age-related Macular Degeneration 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?
• What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the Dry Age-related Macular Degeneration 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 and their status?
• What are the key designations that have been granted to the emerging drugs?

Key Players

• Molecular Partners
• Stealth BioTherapeutics
• Regenerative Patch Technologies
• Aevitas Therapeutics
• NGM Biopharmaceuticals
• InflammX Therapeutics
• Lineage Cell Therapeutics
• Alexion AstraZeneca Rare Disease
• Belite Bio
• Katairo
• Cognition Therapeutics
• Apellis Pharmaceuticals
• Galimedix Therapeutics
• Amarna Therapeutics
• 4D Molecular Therapeutics
• Aviceda Therapeutics
• Isarna Therapeutics

Key Products

• ALK-001
• Elamipretide
• CPCB-RPE1
• AAV.Sfh
• NGM621
• Xiflam
• OpRegen
• ALXN2040
• Tinlarebant (LBS-008)
• Remofuscin
• CT1812
• Gene therapies
• GAL-101
• AMA004
• 4D-175
• AVD-104
• TGF-beta antisense oligonucleotides