Mitochondria, which are sometimes referred to as the “powerhouse of the cell,” are accountable for the creation of around 90 percent of the energy that is used by human cells. These “powerhouses” are present in all human cells other than adult red blood cells, but they are absent from mature red blood cells. The transformation of food into adenosine triphosphate, sometimes known as ATP, is the process through which mitochondria generate energy. This occurs as a result of a cascade of processes that take place within the inner folds of the mitochondria. These reactions are regulated by the electron transport chain.The inner mitochondrial membrane of typical mitochondria is severely folded, resulting in the formation of curves known as cristae. The electron transport chain, which is made up of five different protein complexes and is responsible for the synthesis of ATP in mitochondria, is housed within the cristae. Cardiolipin is a phospholipid that may be found in the inner membrane of mitochondria. It is crucial for constructing the cristae architecture and enhancing the performance of the machinery that generates ATP, including the electron transport chain.

When mitochondria are dysfunctional, their capacity to create ATP may be reduced, and they may also produce higher quantities of reactive oxygen species, also known as ROS. ROS is one of the primary factors that contribute to oxidative stress.

At high quantities, reactive oxygen species (ROS) can cause damage to the cell’s proteins and membrane lipids, despite the fact that ROS at low levels can serve as key signalling molecules in the cell. In particular, cardiolipin is very prone to oxidative damage, which can lead to a disruption in the structure of the mitochondria and a cycle of increasing levels of reactive oxygen species (ROS) generation. This cycle can result in inflammation, fibrosis, senescence, and cell death, all of which are implicated in a wide variety of human diseases.

The malfunction of mitochondrial DNA is a widespread finding across a wide range of disorders, from the most common to the rarest. Mutations in a person’s genes, the natural process of ageing, environmental variables, or any combination of these can all be factors that contribute to mitochondrial dysfunction. These deficits have the potential to damage a variety of organ systems, particularly those with high energy needs, such as the heart, eye, brain, kidneys, and skeletal muscle.Stealth is centred on trying to mitigate mitochondrial dysfunction in a wide range of common age-related diseases, such as dry age-related macular degeneration, as well as in a number of rare diseases linked with cardiotoxicity, Barth syndrome, and Leber’s hereditary optic neuropathy7-8. These diseases affect a small percentage of the population.

Both Healthy and Unhealthy Options

Different Types of Unhealthy and Healthy Mitochondria
Elamipretide is a peptide molecule that quickly penetrates cell walls and targets the mitochondrion membrane, where it binds to cardiolipin in a reversible manner. It is the primary investigational product candidate that we are now working on.
Elamipretide has been shown to boost mitochondrial respiration, improve electron transport chain performance and ATP generation, and minimise the creation of harmful ROS levels in either preclinical or clinical research settings. These are the observations that we have made. It has been demonstrated that the connection between elamipretide and cardioolipin can normalise the structure of the inner mitochondrial membrane, which in turn improves the function of the mitochondria. The functional advantage is accomplished by increasing the amount of ATP that is produced, as well as by preventing and maybe reversing the detrimental effects of oxidative stress. In addition to conducting a clinical study on dry age-related macular degeneration, we are conducting late stage clinical studies on elamipretide for the treatment of three primary mitochondrial diseases. These include rare diseases characterised by cardiomyopathy, Barth syndrome, and Leber’s hereditary optic neuropathy.

Programs

TAZPOWER (Barth Syndrome) SEE MORE DETAIL
Barth syndrome, or Barth, is characterised by muscle atrophy, delayed growth, fatigue, various degrees of severe impairment, heart muscle weakness, or cardiomyopathy, a low white blood cell count, also known as neutropenia (which can put a person at an elevated risk for bacterial infections), and methylglutaconic aciduria (which is an inherited condition that causes the body to produce excessive amounts of methylglutaconic acid) uptick in an organic acid that leads to abnormal cellular function).
22-24 The frequency of Barth syndrome is thought to range from one in 300,000 to one in 400,000 newborns, according to estimates. 22 There are no treatments that have been authorised for the treatment of Barth by either the FDA or the EMA. 24 In relation to the research and development of elamipretide for use in this indication, the FDA has granted us both the Fast Track and Orphan Drug designations.

TAZPOWER is a randomised, double-blind, placebo-controlled phase 2/3 crossover study designed to evaluate the safety, tolerability, and efficacy of 12 weeks’ diagnosis with daily subcutaneous transfusions Using elamipretide in patients who have genetic evidence indicating they have Barth syndrome, followed by an open-label treatment extension. The study will be conducted in individuals who have Barth syndrome.

The ReCLAIM-2 (Age-Related Macular Degeneration) SEE MORE DETAIL
Symptoms of dry age-related macular degeneration, also known as dry AMD, include blurred vision, distorted vision, a loss in low luminance visual acuity, and a decline in overall visual acuity.
30-31 There are no therapies that have been authorised by the FDA or the EMA for age-related macular degeneration (AMD), which is a condition that is believed to affect more than 10 million people in the United States alone and is the primary cause of blindness among older persons in the industrialised world. 30-33 It is considered that mitochondrial dysfunction is a crucial factor in the pathogenesis of dry age-related macular degeneration (AMD). 38, This makes the mitochondrial network an interesting target for improving retinal function and reducing disease loads in this patient population.

The purpose of the ReCLAIM-2 clinical investigation, which is a phase 2 randomised, double-masked, placebo-controlled study, is to investigate the effectiveness, safety, and pharmacokinetics of subcutaneous injections of elamipretide in patients who have dry AMD with geographic atrophy.

Leber’s Hereditary Optic Neuropathy, also known as ReSIGHT SEE MORE DETAIL
Loss of central vision is the primary symptom of Leber’s hereditary optic neuropathy, often known as LHON. This condition is caused by mitochondrial dysfunction and damages the eyes.
26 Our best guess is that there are roughly 10,000 people in the United States who have been identified as having LHON. 27 There are currently no treatments that have been authorised for the treatment of LHON by the FDA. 28 In relation to the research and development of elamipretide for use in this indication, the FDA has granted us both the Fast Track and Orphan Drug designations.

ReSIGHT is a randomised, double-masked, vehicle-controlled study that aims to evaluate the safety, tolerability, and efficacy of a 52-week treatment with a half topical eye drop composition of elamipretide for the purpose of treating patients with LHON, the most common form of mitochondrial optic neuropathy.

Phase 1 of the SBT-272 SEE MORE DETAIL
SBT-272 is an innovative peptidomimetic that is now in the process of being researched as a potential therapy for neurodegenerative illnesses that are associated with mitochondrial dysfunction. In preclinical experiments, the administration of SBT-272 to defective mitochondria led to an increase in the generation of adenosine triphosphate (ATP) and a decrease in the levels of reactive oxygen species (ROS). In comparison to elamipretide, Stealth’s first-in-class lead chemical, SBT-272 exhibits much higher mitochondrial uptake, significantly higher concentrations in the brain, and significantly enhanced oral bioavailability. In a mouse model of amyotrophic lateral sclerosis (ALS), a progressive neurodegenerative disease characterised by the deterioration of motor neurons and the atrophy of muscle tissue, treatment with SBT-272 was associated with a dose-dependent delay in the onset of neurological disease, a reduction in systemic markers of neurodegeneration, and a prolonged lifespan. ALS is one of the most common forms of neurodegenerative disease. The compound is presently being investigated in a further preclinical model of amyotrophic lateral sclerosis (ALS), as well as in a preclinical model indicative of activity in multiple system atrophy (MSA). MSA is a neurological condition that can result in parkinsonism, cerebellar ataxia, dysautonomia, and other motor and non-motor symptoms. It is thought that mitochondrial dysfunction has a role in the course of neurodegenerative disorders such as ALS and MSA, as well as other conditions such as Parkinson’s disease, Huntington’s disease, and Alzheimer’s disease.

Pipeline

Stealth BioTherapeutics is continually broadening its knowledge of mitochondrial biology and innovative chemistries. This gives us the ability to enhance our mitochondrial platform, which consists of late-stage clinical projects and pipeline candidates.

We are now engaged in a research and development programme, the primary objective of which is to create novel medicinal compounds through the use of innovative, proprietary methods with the goal of optimising their absorption, distribution, metabolism, and excretion characteristics. We have a developing compound library consisting of small compounds and new peptides, both of which are now being subjected to active screening in order to enhance our existing portfolio of mitochondrial product candidates.

Certain drugs in the pipeline are now being investigated for potential neurodegenerative applications including mitochondrial dysfunction. Rare cellular diseases such as Leigh’s symptoms and Friedreich’s ataxia, as well as other rare diseases such as amyotrophic lateral sclerosis (ALS), have been linked to mitochondrial dysfunction as a possible contributing factor. Mitochondrial dysfunction has also been linked to age-related neurodegenerative diseases such as Alzheimer’s disease and Parkinson’s disease.

In addition to this, we are working on the development of a platform known as Mitochondrial Carrier Technology (MCT) to take use of the extraordinary capability of our patented chemicals to transport bioactive cargo to cells.. The preliminary findings indicate that our carrier molecules are able to successfully target mitochondria as the destination for the delivery of biologically active cargo. This method looks promising for mitochondrial isolation of small molecules, and it may also have had the ability to transport peptides, proteins, and oligonucleotides. Specifically, the mitochondrial localization of small molecules shows promise.

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