Mitochondrial Myopathies - Pipeline Insight, 2024

This “Mitochondrial Myopathies- Pipeline Insight, 2024” report provides comprehensive insights about 10+ companies and 10+ pipeline drugs in Mitochondrial Myopathies 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

Mitochondrial Myopathies: Understanding

Mitochondrial Myopathies: Overview

Mitochondrial disorders impair the function of mitochondria, the tiny compartments in every cell of the body that produce the energy needed by cells. Depending on which cells have fewer or lower-functioning mitochondria, different symptoms may occur. Organs and other parts of the body that require more energy, such as the heart, muscles and brain, are often affected. Mitochondrial disease is the name for a large number of mitochondrial disorders, with different genetic causes and presentations. Mitochondrial disorders are named and classified by their genetic causes or by their symptoms and the results of non-genetic clinical tests. Because mitochondrial disorders are grouped in two ways - by genetic cause and by symptoms - particular disorders can fall into more than one category. Mitochondrial myopathies are forms of mitochondrial disease that cause prominent muscle problems, a grouping based on both symptoms and non-genetic clinical tests. Three types of muscles can be affected by the myopathy: Skeletal - the muscles around the bones and eyes, Cardiac - the heart muscles, Smooth - the muscles lining the intestines, gastrointestinal tract, bladder and blood vessels. There are nine main forms of mitochondrial myopathy: Kearns-Sayre syndrome (KSS), Leigh syndrome, Mitochondrial DNA (mt DNA) depletion syndrome, Mitochondrial encephalopathy, lactic acidosis and stroke-like episodes (MELAS) syndrome, Mitochondrial neurogastrointestinal encephalopathy (MNGIE), Myoclonic epilepsy with ragged red fibers (MERFF) Neuropathy, ataxia and retinitis pigmentosa (NARP) syndrome, Pearson syndrome and Progressive external ophthalmoplegia (PEO). Mitochondrial myopathies may be caused by mutations in the body’s nuclear DNA (the DNA found in the nucleus of cells) or by mutations or deletions in the body’s mitochondrial DNA (mt DNA, the DNA found in cells’ mitochondria). Symptoms of mitochondrial myopathies vary widely by type and from case to case. There is also a great deal of overlap in symptoms among different mitochondrial conditions. Two patients may have similar symptoms with different genetic causes, and will therefore be diagnosed with different mitochondrial disorders. Further, two patients with the same genetic disorder may have very different symptoms. If mitochondrial myopathy is suspected based on the patient’s physical symptoms and history, additional tests are performed to make a diagnosis. These clinical diagnostic tests may include: Blood tests, including complete blood count and tests to look for high concentrations of lactic acid and other abnormalities, Urine test to look for abnormal levels of amino acids, glucose and other metabolites, Stool sample analysis to look for high levels of fat suggestive of deficient pancreatic enzymes, Cerebrospinal fluid (CSF) analysis to look for elevated protein levels, MRI, CT scan or other imaging of the brain, muscle tissue or other organs andmanyothers.

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

Mitochondrial Myopathies Emerging Drugs Chapters

This segment of the Mitochondrial Myopathies 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.

Mitochondrial Myopathies Emerging Drugs

Elamipretide: Stealth Bio TherapeuticsElamipretide, is a peptide compound that readily penetrates cell membranes, and targets the inner mitochondrial membrane where it binds reversibly to cardiolipin. In preclinical or clinical studies, it was observed that elamipretide increases mitochondrial respiration, improves electron transport chain function and ATP production and reduces formation of pathogenic ROS levels. This elamipretide-cardiolipin association has been shown to normalize the structure of the inner mitochondrial membrane, thereby improving mitochondrial function. Functional benefit is achieved through improvement of ATP production and interruption and potential reversal of damaging oxidative stress. Currently, the drug is in Phase III stage of Clinical trial evaluation for the treatment of Primary MitochondrialMyopathy.

Sonlicromanol: Khondrion Khondrion’s wholly-owned investigational lead asset, sonlicromanol (formerly known as KH176), is a potential first-in-class medicine and one of the most advanced disease-modifying drug candidates for primary mitochondrial disease in clinical development. Sonlicromanol targets the key underlying mechanisms of mitochondrial disease based on its scientifically validated and unique triple mode of action. Sonlicromanol is a reductive and oxidative distress modulator with anti-inflammatory properties. Currently, the drug is in Phase II stage of Clinical trial evaluation for the treatment of MitochondrialMyopathy.

REN 001: Reneo Pharmaceuticals REN001, is a potent and selective agonist of the peroxisome proliferator-activated receptor delta (PPARd) that has been shown to increase transcription of genes involved in mitochondrial function and increase fatty acid oxidation, and promote formation of new mitochondria.REN001 is being developed in two rare diseases, primary mitochondrial myopathies (PMM) and long-chain fatty acid oxidation disorders (LC-FAOD). . Currently, the drug is in Phase II/III stage of Clinical trial evaluation for the treatment of Primary Mitochondrial Myopathy.

Mitochondrial Myopathies: Therapeutic Assessment

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

Major Players in Mitochondrial Myopathies

There are approx. 10+ key companies which are developing the therapies for Mitochondrial Myopathies. The companies which have their Mitochondrial Myopathies drug candidates in the most advanced stage, i.e. phase III include, Stealth BioTherapeutics.

Phases

This report covers around 10+ 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

Mitochondrial Myopathies pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as

• Intra-articular
• Intraocular
• Intrathecal
• Intravenous
• Ophthalmic
• Oral
• Parenteral
• Subcutaneous
• Topical
• Transdermal

Molecule Type

Products have been categorized under various Molecule types such as

• Oligonucleotide
• Peptide
• Small molecule

Product Type

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

Mitochondrial Myopathies: Pipeline Development Activities

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

Mitochondrial Myopathies Report Insights

• Mitochondrial Myopathies Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

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

Key Players

• Stealth BioTherapeutics
• Mitobridge
• Reneo Pharmaceuticals
• Abliva AB
• Khondrion
• Reata Pharmaceuticals
• Modis Therapeutics

Key Products

• Elamipretide
• Bocidelpar
• REN 001
• KL 1333
• Sonlicromanol
• Omaveloxolone
• MT1621

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