Science & Pipeline

Mighty is pioneering mitochondrial medicine and developed the first approved mitochondria-targeted therapeutic. In September 2025, the FDA granted accelerated approval to elamipretide, which is being marketed in the U.S. under the trade name FORZINITY^TM (elamipretide) injection, a mitochondrial cardiolipin binder indicated to improve muscle strength in adult and pediatric patients with Barth syndrome weighing at least 30 kg. FORZINITY is the first-ever treatment option for Barth syndrome. We are also investigating elamipretide in late-stage clinical studies in ophthalmic diseases entailing mitochondrial dysfunction, such as dry AMD, rare neuromuscular disorders, such as primary mitochondrial myopathy.

Elamipretide is a peptide compound that readily penetrates cell membranes, and targets the inner mitochondrial membrane where it binds reversibly to cardiolipin.¹³ In preclinical or clinical studies, we have observed that elamipretide increases mitochondrial respiration, improves electron transport chain function and ATP production and reduces formation of pathogenic ROS levels.^13-19 This elamipretide-cardiolipin association has been shown to normalize the structure of the inner mitochondrial membrane, thereby improving mitochondrial function.¹³ Functional benefit is achieved through improvement of ATP production and interruption and potential reversal of damaging oxidative stress.¹³

We are evaluating our second-generation clinical-stage candidate, bevemipretide (SBT-272), an eye drop formula, in dry AMD, and have a deep pipeline of novel compounds under evaluation for rare neurological and cardiac disease indications.

Healthy & Unhealthy

Programs

TAZPOWER (Barth syndrome)

Barth syndrome is an ultra-rare genetic condition characterized by mitochondrial abnormalities leading to exercise intolerance, muscle weakness, debilitating fatigue, heart failure, recurrent infections, and delayed growth. The disease is associated with reduced life expectancy, with 85% of early deaths occurring by age 5. Barth syndrome occurs primarily in males and is estimated to affect one in 300,000-400,000 male births or around 150 known individuals in the United States. Development efforts for commercial availability for patients with Barth syndrome outside of the US are currently ongoing with trusted partners.

NuPOWER (nuclear DNA mutations nPMD)

The NuPOWER is a completed Phase 3 clinical trial designed from the clinical experience on the prespecified subgroup of patients with nPMD who appeared to respond to elamipretide therapy in the Company’s MMPOWER-3 trial. The nPMD subgroup of patients demonstrated a meaningful efficacy signal of improvement in the 6-minute walk test (6MWT), while the mtDNA subgroup did not show the same degree of benefit.

ReNEW (Dry Age-Related Macular Degeneration)

ReNEW is an ongoing Phase 3 global clinical trial evaluating the efficacy and safety of once-daily subcutaneous injections of elamipretide in participants with dry AMD. The primary endpoint for the trial is the rate of change in the macular area of photoreceptor loss assessed by spectral domain-optical coherence tomography and ellipsoid zone mapping. In the ReNEW trial, 360 patients will be randomized 2:1 to either elamipretide or placebo for 96 weeks. To learn more about the ReNEW trial, please visit https://dry-amdclinicaltrials.com/.

Bevemipretide (SBT-272)

Bevemipretide is a novel investigational small molecule that targets cardiolipin, a phospholipid within the inner mitochondrial membrane that is essential for mitochondrial structure and function. Bevemipretide has demonstrated mitochondria- and neuro-protective effects across preclinical models of alpha-synucleinopathy, amyotrophic lateral sclerosis, fronto temporal dementia, Huntington’s disease, and ischemic stroke. Bevemipretide-mediated improvements in functional assessments, lifespan, inflammation, and reduction of protein aggregates have been observed in these preclinical models. Data from a Phase 1 study evaluating subcutaneous Bevemipretide in healthy volunteers supports further clinical development. Bevemipretide is also being investigated in dry AMD. A Phase 1 study with bevemipretide topical eyedrops is currently underway with additional clinical studies planned in the near future.

Pipeline (SBT-255 and 580 series)

SBT-255 is a novel follow-on compound to our lead product candidate elamipretide. SBT-255 has a similar cardiolipin binding mechanism of action but has been clearly differentiated from other compounds across pre-clinical studies. It is currently being evaluated in several different models of cardiac and muscle myopathy including hypertrophic cardiomyopathy (HCM), idiopathic cardiomyopathy, and aging cardiac and skeletal muscle.

The SBT-580 series of compounds are promising novel molecules, with a unique mechanism of action, that acts on mitochondrial pathways essential for cellular health and energy production. The lead candidate in the series, SBT-589, has been studied in Leigh syndrome and Friedreich’s ataxia (FA) patient-derived cells, isolated heart mitochondria, and an aggressive mouse model of FA cardiomyopathy. SBT-589 has been shown to improve bioenergetics in FA patient-derived cells and mitochondria and display significantly reduced cardiac hypertrophy. It has also been shown to delay the onset of mortality in mouse models.

Pipeline

Mighty Therapeutics continues to expand our broad knowledge of mitochondrial biology and novel chemistries, enabling us to advance our mitochondrial platform of proprietary late-stage clinical programs and pipeline candidates.

We have an active discovery and development program focused on the development of novel therapeutic compounds using proprietary new approaches to optimize absorption, distribution, metabolism and excretion properties. We have a growing compound library of small molecules and novel peptides that we are actively screening to broaden our existing mitochondrial product candidate portfolio.

For more information on Mighty Therapeutics’ Research and Discovery, please contact us discovery@mightytx.com.

Resources

For more information about clinical trials, please visit clinicaltrials.gov.

To read our latest announcements, please visit https://mightytx.com/latest-news