Stargardt Disease (STGD) - Pipeline Insight, 2024

This “Stargardt Disease (STGD)- Pipeline Insight, 2024” report provides comprehensive insights about 20+ companies and 25+ pipeline drugs in Stargardt Disease (STGD) 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 Disease (STGD): Understanding

Stargardt Disease (STGD): Overview

Stargardt disease (STGD), also known as Stargardt macular dystrophy or fundus flavimaculatus, is the most common inherited form of juvenile macular degeneration. It primarily affects the central vision due to the degeneration of the macula, the part of the retina responsible for sharp, detailed vision. The disease is usually diagnosed in children, teenagers, or young adults, though symptoms can emerge later in life as well. Stargardt disease is predominantly caused by mutations in the ABCA4 gene, which disrupts the normal function of the photoreceptor cells in the retina. This progressive condition leads to a gradual decline in central vision but typically spares peripheral vision.

The hallmark symptom of Stargardt disease is a progressive loss of central vision, which can affect activities like reading, recognizing faces, and driving. Early symptoms often include difficulty seeing in low-light conditions (night blindness) and a gradual reduction in visual acuity. Some individuals may also experience color vision deficits. The vision loss is generally bilateral (affects both eyes) and tends to worsen over time, although the rate of progression can vary. Central scotomas, or blind spots in the center of vision, may develop, further impairing detailed visual tasks.

The ABCA4 gene encodes a protein involved in the visual cycle, responsible for clearing toxic byproducts formed during the process of phototransduction in the photoreceptor cells. In individuals with Stargardt disease, mutations in the ABCA4 gene impair this function, leading to the accumulation of toxic substances such as lipofuscin in the retinal pigment epithelium (RPE). This accumulation damages the RPE and photoreceptor cells, resulting in their gradual degeneration. As the macula degenerates, the ability to process fine detail deteriorates, leading to central vision loss. In some cases, Stargardt disease may also involve other retinal structures, leading to more widespread visual impairment.

Diagnosis of Stargardt disease is typically made through a combination of clinical examination, imaging techniques, and genetic testing. Fundus autofluorescence (FAF) imaging is often used to detect lipofuscin accumulation, while optical coherence tomography (OCT) can assess the structural damage to the retina. Electroretinography (ERG) may also be used to evaluate retinal function. Genetic testing can confirm the presence of ABCA4 mutations.

Currently, there is no cure for Stargardt disease, but treatments focus on managing symptoms and slowing progression. Patients are advised to protect their eyes from excessive light exposure, as UV light may exacerbate retinal damage. Emerging therapies, including gene therapy, stem cell therapy, and pharmacological interventions targeting the visual cycle, are under investigation in clinical trials, offering hope for future treatment options.

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

Stargardt Disease (STGD) Emerging Drugs Chapters

This segment of the Stargardt Disease (STGD) 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.

Stargardt Disease (STGD) Emerging Drugs

Emixustat: Kubota Vision

Emixustat hydrochloride (emixustat, formerly ACU-4429) is an orally administered small molecule that modulates the visual cycle by inhibiting a critical enzyme of this pathway, retinal pigment epithelium protein 65 (RPE65). The visual cycle is the process by which vitamin A is recycled in the eye; vitamin A is crucial to the visual process. Slowing the visual cycle reduces the availability of vitamin A derivatives (11-cis- and all-trans-retinal) to form precursors of toxic A2E and related compounds.

In addition, reducing the availability of 11-cis-retinal decreases retinal metabolic demands under dark conditions. Kubota Vision is exploring emixustat’s potential to stop or slow the progression of vision loss in patients diagnosed with Stargardt disease and other disorders in the clinical studies. Currently, the drug is in the Phase III stage of its development for the treatment of Stargardt Disease (STGD).

MCO-010: Nanoscope Therapeutics

MCO-010 (sonpiretigene isteparvovec, suspension for intravitreal injection) is the only broadband, fast, and highly light-sensitive opsin currently in clinical trials. With bipolar cell targeting via mGluR6 promoter-enhancer, the MCO-010 expression cassette is designed for restoring high-quality vision in real-world environments. The proprietary AAV2 vector allows robust transduction of MCO-010 in bipolar cells upon intravitreal injection. The Phase I/IIa study of MCO-010 in advanced RP patients demonstrated favorable safety and dose-dependent improvement in visual acuity. Currently, the drug is in the Phase II stage of its development for the treatment of Stargardt Disease (STGD).

OCU410ST: Ocugen

OCU410ST utilizes an AAV delivery platform for the retinal delivery of the RORA (RAR Related Orphan Receptor A) gene. It represents Ocugen’s modifier gene therapy approach, which is based on Nuclear Hormone Receptor (NHR) RORA that regulates pathway links to Stargardt disease such as lipofuscin formation, oxidative stress, compliment formation, inflammation, and cell survival networks. Currently, the drug is in the Phase I stage of its development for the treatment of Stargardt Disease (STGD).

Stargardt Disease (STGD): Therapeutic Assessment

This segment of the report provides insights about the different Stargardt Disease (STGD) drugs segregated based on following parameters that define the scope of the report, such as:

Major Players in Stargardt Disease (STGD)

There are approx. 20+ key companies which are developing the therapies for Stargardt Disease (STGD). The companies which have their Stargardt Disease (STGD) drug candidates in the most advanced stage, i.e. Phase III include, Kubota Vision.

Phases

The report covers around 25+ 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 Disease (STGD) 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.

Stargardt Disease (STGD): Pipeline Development Activities

The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Stargardt Disease (STGD) 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 Disease (STGD) drugs.

Stargardt Disease (STGD) Report Insights

• Stargardt Disease (STGD) Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

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

Key Players

• Kubota Vision
• Nanoscope Therapeutics
• Ocugen
• Belite Bio
• Alkeus Pharmaceuticals
• Biophytis
• reVision Therapeutics, Inc
• IIVERIC bio
• Ascidian Therapeutics
• Gene Vector Technologies
• ReVision Therapeutics

Key Products

• Emixustat
• MCO-010
• OCU410ST
• Tinlarebant
• Gildeuretinol
• BIO-201
• REV-0100
• Avacincaptad pegol
• ACDN 01
• JWK 006
• REV 0100

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