Stargardt Disease (STGD) - Pipeline Insight, 2020

“Stargardt – Pipeline Insight, 2020,” 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.

Geography Covered
• Global coverage

Stargardt Understanding

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.

Symptoms
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.

Diagnosis
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.

Treatment
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.

Stargardt Emerging Drugs Chapters
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.

Stargardt Emerging Drugs
• Emixustat: Kubota Vision
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: Alkeus Pharmaceuticals
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……..

Stargardt: Therapeutic Assessment
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:
• Major Players in Stargardt
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.
• Phases
report covers around 30+ 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
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
• Oral
• Parenteral
• intravitreal
• Subretinal
• Topical.
• Molecule Type
Products have been categorized under various Molecule types such as
• Monoclonal Antibody
• Peptides
• Polymer
• Small molecule
• Gene therapy

• Product Type
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 drugs.

Report Highlights
• The companies and academics are working to assess challenges and seek opportunities that could influence Stargardt R&D. The therapies under development are focused on novel approaches to treat/improve Stargardt.
• On October 28, 2020, reVision Therapeutics, Inc developing candidate REV-0100 received Orphan-Drug and a Rare Pediatric Disease Drug designation for the treatment of Stargardt disease.

Stargardt Report Insights
• Stargardt Pipeline Analysis

• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

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

Key Players
• Kubota Vision Inc.
• Alkeus Pharmaceuticals
• Stargazer Pharmaceuticals, Inc.
• IVERIC bio, Inc.
• Astellas Pharma, Inc.
• Cardax Pharmaceuticals
• Beam Therapeutics
• Belite Bio

Key Products
• Emixustat
• ALK-001
• Zeaxanthin
• STG 001
• Avacincaptad pegol
• ASP7317
• LBS-008