MELAS Sundrome - Pipeline Insight, 2024

This “MELAS Syndrome - Pipeline Insight, 2024,” report provides comprehensive insights about 4+ companies and 4+ pipeline drugs in MELAS 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

MELAS Syndrome Understanding

MELAS Syndrome: Overview

MELAS (Mitochondrial Encephalopathy, Lactic Acidosis, and Stroke-like episodes) syndrome is a rare disorder that begins in childhood, usually between two and fifteen years of age, and mostly affects the nervous system and muscles. The most common early symptoms are seizures, recurrent headaches, loss of appetite and recurrent vomiting. Symptoms of MELAS syndrome usually begin between the ages of two and fifteen years, but delayed onset cases have also been reported between fifteen and forty years and late onset cases after forty years. MELAS is caused by mutations in mitochondrial DNA. Some cases of MELAS syndrome appear to occur as the result of a new spontaneous mutation in a mitochondrial gene and are not inherited. It affects males and females in equal numbers.

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

MELAS Syndrome Emerging Drugs Chapters

This segment of the MELAS Syndrome 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.

MELAS Syndrome Emerging Drugs

CY-6463: Cyclerion Therapeutics, Inc.

CY 6463 (previously known as IW 6463) is an orally administered small molecule soluble guanylate cyclase (s GC) stimulant, being developed by Cyclerion. IW-6463 is an sGC stimulator, a positive allosteric modulator of sGC that amplifies endogenous NO signaling. Cyclerion Therapeutics has planned the phase IIa MELAS trial forCY-6463.

KL133: Abliva ABThe KL1333 project is in Phase I stage of development, intended to document the use for chronic oral treatment of primary mitochondrial disorders, in particular MELAS-MIDD spectrum disorders. KL1333 is a potent modulator of the cellular levels of NAD+, a central co-enzyme in the cell’s energy metabolism. KL1333 is currently being evaluated in patient studies and has been granted orphan drug designation in both the United States and Europe.

MELAS Syndrome: Therapeutic Assessment

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

Major Players in MELAS Syndrome

There are approx. 4+ key companies which are developing the therapies for MELAS Syndrome. The companies which have their MELAS Syndrome drug candidates in the most advanced stage, i.e. phase II include, Cyclerion Therapeutics Inc.

Phases

This report covers around 4+ 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

MELAS 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

• Oral
• Intravenous
• Molecule Type

Products have been categorized under various Molecule types such as

• Modulator
• Peptides
• Small molecule
• Product Type

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

MELAS Syndrome: Pipeline Development Activities

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

MELAS Syndrome Report Insights

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

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

Key Players

• Cyclerion Therapeutics Inc.
• Khondrion BV
• Abliva AB

Key Products

• CY-6463
• KH176
• Kl133

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