Leigh Syndrome - Pipeline Insight, 2024

This “Leigh Syndrome - Pipeline Insight, 2024” report provides comprehensive insights about 3+ companies and 3+ pipeline drugs in Leigh Syndrome 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

Leigh Syndrome: Understanding

Leigh Syndrome: Overview

Leigh Syndrome, is a clinically and genetically heterogeneous disorder resulting from defective mitochondrial energy generation. It most commonly presents as a progressive and severe neurodegenerative disorder with onset within the first months or years of life, and may result in early death. Affected individuals usually show global developmental delay or developmental regression, hypotonia, ataxia, dystonia, and ophthalmologic abnormalities, such as nystagmus or optic atrophy. The neurologic features are associated with the classic findings of T2-weighted hyperintensities in the basal ganglia and/or brainstem on brain imaging. Leigh syndrome can also have detrimental multisystemic effects on the cardiac, hepatic, gastrointestinal, and renal organs.

Leigh syndrome is characterized by the development of bilateral symmetrical lesions in the brainstem and basal ganglia, showing gliosis, vacuolation, capillary proliferation, and relative neuronal preservation. These lesions are accompanied by hyperlacticacidemia in the blood and/or cerebrospinal fluid. The underlying molecular mechanisms driving this unique neuropathology are not fully understood, but likely involve severe ATP depletion, gliosis, reactive oxygen species, and potentially excitotoxicity.

Leigh syndrome manifests with various signs and symptoms, including poor sucking ability and failure to thrive in infancy, delayed development, weakness, muscle tone issues like hypotonia, and movement problems such as ataxia and dystonia. Respiratory issues like difficulty breathing and acute respiratory failure, along with eye muscle weakness, nystagmus, and optic atrophy, are common.

The diagnosis of Leigh syndrome is typically based on clinical features confirmed by imaging tests like MRI to detect damaged areas of brain tissue (lesions). Additionally, blood tests are conducted to check for lactic acidosis and enzyme markers indicating Leigh syndrome. The disease is characterized by specific neuropathological features seen in brain imaging, often showing lesions in the brainstem and basal ganglia, which are crucial for diagnosis.

The treatment and management of Leigh syndrome involves a multi-faceted approach. Supplementation with vitamins like thiamine, coenzyme Q10, L-carnitine, and a vitamin cocktail can help manage symptoms and improve energy production. Dietary interventions, such as implementing a high-fat, low-carbohydrate ketogenic diet, may benefit individuals with Leigh syndrome, especially those with metabolic issues. Additionally, ongoing clinical trials with experimental therapies like EPI-743, cysteamine bitartrate, and KH176 show promise in managing Leigh syndrome symptoms. Supportive care from a multidisciplinary medical team, including neurologists and cardiologists, is essential for addressing the various complications and symptoms to enhance the quality of life for individuals with Leigh syndrome.

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

Leigh Syndrome Emerging Drugs Chapters

This segment of the Leigh Syndrome report encloses its detailed analysis of various drugs in different stages of clinical development, including phase III, II/III, 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.

Leigh Syndrome Emerging Drugs

Vatiquinone: PTC Therapeutics

Vatiquinone (EPI-743) is an orally bioavailable small molecule being developed by the company for inherited mitochondrial diseases. EPI-743 is a member of the para-benzoquinone class of drugs. The mechanism of action of EPI-743 involves augmenting the synthesis of glutathione, optimizing metabolic control, enhancing the expression of genetic elements critical for cellular management of oxidative stress, and acting at the mitochondria to regulate electron transport. The drug is currently being evaluated under Phase III clinical trial for the treatment of patients with Leigh Syndrome.

Leigh Syndrome: Therapeutic Assessment

This segment of the report provides insights about the different Leigh Syndrome drugs segregated based on following parameters that define the scope of the report, such as:

Major Players in Leigh Syndrome

There are approx. 3+ key companies which are developing the therapies for Leigh Syndrome. The companies which have their Leigh Syndrome drug candidates in the most advanced stage, i.e. Phase III include, PTC Therapeutics.

Phases

DelveInsight’s report covers around 3+ 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

Leigh Syndrome 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.

Leigh Syndrome: Pipeline Development Activities

The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Leigh Syndrome 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 Leigh Syndrome drugs.

Leigh Syndrome Report Insights

• Leigh Syndrome Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

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

Key Players

• PTC Therapeutics
• CAMP Therapeutics

Key Products

• Vatiquinone
• CMP-3013

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