DelveInsight’s, “Stargardt – Pipeline Insights, 2021,” report provides comprehensive insights about 8+ companies and 8+ pipeline drugs in Stargardt 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.
Stargardt: Overview
Stargardt disease is an inherited eye disease that causes vision loss in children and young adults and has a prevalence of 1 in 8000 - 10000. It is a form of macular degeneration, in which macula, a part of the retina that provides sharp, straight-ahead vision breaks down. Stargardt disease is also known as Stargardt macular dystrophy, juvenile macular degeneration, or fundus flavimaculatus. The most common form of the disease is caused by a genetic mutation of the ABCA4 gene leading to the accumulation of toxic vitamin A byproducts (primarily A2E) in the retina, which results in the gradual deterioration of photoreceptors and vision.
The most common symptom of Stargardt disease is variable, often slow loss of central vision in both eyes. People with the disease might notice gray, black, or hazy spots in the center of their vision, or that it takes longer than usual for their eyes to adjust when moving from light to dark environments.
Eyes may be more sensitive to bright light. Some people also develop color blindness later in the disease.The progression of symptoms in Stargardt disease is different for each person. People with an earlier onset of disease tend to have more rapid vision loss. Vision loss may decrease slowly at first, then worsen rapidly until it levels off. Most people with Stargardt disease will end up with 20/200 vision or worse. People with Stargardt disease may also begin to lose some of their peripheral (side) vision as they get older.
A positive diagnosis of Stargardt disease by examining the retina. Lipofuscin deposits can be seen as yellowish flecks in and under the macula. The flecks are irregular in shape and usually extend outward from the macula in a ring-like pattern. Lipofuscin are fat deposits from normal cell activity and build up is more in people with Stargardt disease. The number, size, color, and appearance of these flecks are widely variable. A fluorescein angiography test may be used to diagnose Stargardt disease. Genetic testing is also available to precisely diagnose what type of macular degeneration a patient has.
Currently, there is no proven treatments, there are three main avenues of intervention being explored, with human clinical trials of stem cell therapy, gene replacement therapy and pharmacological approaches. Gene therapy which involves repairing or replacing the defective ABCA4 gene also holds promise for treating Stargardt disease. A number of services and devices can help people with Stargardt disease carry out daily activities and maintain their independence. Wearing sunglasses may help with the bright light sensitivity of Stargardt disease and also prevent further retina damage from the sun’s harmful ultraviolet (UV) rays. Stargardt disease pateints should not smoke cigarettes or be around cigarette smoke. Also, some studies suggest that taking a lot of vitamin A could may make the disease worse.
This segment of the Stargardt 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.
Emixustat hydrochloride (emixustat, formerly ACU-4429) is an experimental drug that is a Retinoid isomerohydrolase inhibitor. It is an orally administered small molecule that inhibits the visual cycle isomerohydrolase, RPE65. The FDA (U.S. Food and Drug Administration) and European Medicines Agency (EMA) granted orphan drug designation to emixustat for the treatment of Stargardt disease.
ALK-001 is once-a-day oral drug candidate currently in Phase 2 for the treatment of Stargardt disease. Due to a genetic defect in Stargardt, toxic vitamin A aggregates called "dimers" are known to rapidly form and cause the degeneration of the retina. ALK-001 is form of vitamin A that has been chemically-modified to prevent vitamin A aggregation into dimers.
Further product details are provided in the report……..
This segment of the report provides insights about the different Stargardt drugs segregated based on following parameters that define the scope of the report, such as:
There are approx. 8+ key companies which are developing the therapies for Stargardt. The companies which have their Stargardt drug candidates in the most advanced stage, i.e. phase III include, Kubota Vision.
DelveInsight’s report covers around 30+ products under different phases of clinical development like
Stargardt pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as
Products have been categorized under various Molecule types such as
Drugs have been categorized under various product types like Mono, Combination and Mono/Combination.
Stargardt: Pipeline Development Activities
The report provides insights into different therapeutic candidates in phase III, II, I, preclinical and discovery stage. It also analyses Stargardt 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 Stargardt drug
Stargardt Report Insights
Stargardt Report Assessment
Current Treatment Scenario and Emerging Therapies:
Introduction
Executive Summary
Stargardt: Overview
• Causes
• Mechanism of Action
• Signs and Symptoms
• Diagnosis
• Disease Management
Pipeline Therapeutics
• Comparative Analysis
Therapeutic Assessment
• Assessment by Product Type
• Assessment by Stage and Product Type
• Assessment by Route of Administration
• Assessment by Stage and Route of Administration
• Assessment by Molecule Type
• Assessment by Stage and Molecule Type
Stargardt – DelveInsight’s Analytical Perspective
In-depth Commercial Assessment
• Stargardt companies’ collaborations, Licensing, Acquisition -Deal Value Trends
Stargardt Collaboration Deals
• Company-Company Collaborations (Licensing / Partnering) Analysis
• Company-University Collaborations (Licensing / Partnering) Analysis
Late Stage Products (Phase III)
• Comparative Analysis
Emixustat: Kubota Vision
• Product Description
• Research and Development
• Product Development Activities
Mid Stage Products (Phase II)
• Comparative Analysis
ALK-001: Alkeus Pharmaceuticals
• Product Description
• Research and Development
• Product Development Activities
Drug profiles in the detailed report…..
Early Stage Products (Phase I)
• Comparative Analysis
LBS-008: Belite Bio
• Product Description
• Research and Development
• Product Development Activities
Drug profiles in the detailed report…..
Pre-clinical and Discovery Stage Products
• Comparative Analysis
Zeaxanthin - Cardax Pharmaceuticals
• Product Description
• Research and Development
• Product Development Activities
Drug profiles in the detailed report…..
Inactive Products
• Comparative Analysis
Stargardt Key Companies
Stargardt Key Products
Stargardt- Unmet Needs
Stargardt- Market Drivers and Barriers
Stargardt- Future Perspectives and Conclusion
Stargardt Analyst Views
Stargardt Key Companies
Appendix
List of Tables
Table 1 Total Products for Stargardt
Table 2 Late Stage Products
Table 3 Mid Stage Products
Table 4 Early Stage Products
Table 5 Pre-clinical & Discovery Stage Products
Table 6 Assessment by Product Type
Table 7 Assessment by Stage and Product Type
Table 8 Assessment by Route of Administration
Table 9 Assessment by Stage and Route of Administration
Table 10 Assessment by Molecule Type
Table 11 Assessment by Stage and Molecule Type
Table 12 Inactive Products
List of Figures
Figure 1 Total Products for Stargardt
Figure 2 Late Stage Products
Figure 3 Mid Stage Products
Figure 4 Early Stage Products
Figure 5 Preclinical and Discovery Stage Products
Figure 6 Assessment by Product Type
Figure 7 Assessment by Stage and Product Type
Figure 8 Assessment by Route of Administration
Figure 9 Assessment by Stage and Route of Administration
Figure 10 Assessment by Molecule Type
Figure 11 Assessment by Stage and Molecule Type
Figure 12 Inactive Products
• Kubota Vision Inc.
• Alkeus Pharmaceuticals
• Stargazer Pharmaceuticals, Inc.
• IVERIC bio, Inc.
• Astellas Pharma, Inc.
• Cardax Pharmaceuticals
• Beam Therapeutics
• Belite Bio
