#United States #Needham #Stealth BioTherapeutics #FORZINITY #mitochondrial diseases

Stealth BioTherapeutics: Pioneering Solutions for Mitochondrial Dysfunction

Stealth BioTherapeutics Inc., a biotechnology company at the forefront of innovation in therapies for mitochondrial dysfunction, has made significant strides since its receipt of accelerated approval for its flagship product, FORZINITY (elamipretide). On January 6, 2026, the firm outlined its developmental and commercial progress, setting the stage for ambitious strategic goals heading into 2026.

Commercial Landscape

Since FORZINITY's FDA approval in September 2025, Stealth has established a robust commercial foundation. This drug has transcended previous market expectations, becoming available for eligible patients diagnosed with Barth syndrome (BTHS), an ultra-rare but critical condition affecting muscle strength and overall health. The response from healthcare providers has been overwhelmingly positive, contributing to a rapid uptake of the drug since its launch through AnovoRx Specialty Pharmacy, the exclusive provider for FORZINITY.

Reenie McCarthy, CEO of Stealth BioTherapeutics, commented, "Our collaboration with prescribers and payers remains integral as we strive to ensure consistent access for all eligible patients suffering from this debilitating condition." This commitment to patient accessibility is crucial, especially given the challenges faced by those diagnosed with BTHS, which affects approximately 150 individuals in the U.S.

To further support this initiative, Stealth launched a patient support program named Mito Assist™, aimed at providing comprehensive assistance throughout each patient's treatment journey. Efforts are also in place to engage with pharmacy benefit providers to streamline the reimbursement process for patients, ensuring financial accessibility for those who need it most.

Research and Development Progress

In addition to its commercial achievements, Stealth is maintaining momentum in its research and development sector. The company is poised to meet with the FDA in early 2026 to discuss regulatory pathways for patients with BTHS who do not meet the current weight criteria for FORZINITY, which applies to individuals weighing at least 30 kg (66 lbs). Until new regulations are finalized, continued access to elamipretide is being provided through an expanded access program for younger patients.

The company is also making headway in research regarding primary mitochondrial disease (PMD) and dry age-related macular degeneration (AMD). Alongside advancing current treatments, Stealth is preparing to launch additional studies to evaluate the efficacy of elamipretide in this new indication while progressing with its fully-enrolled Phase 3 ReNEW clinical trial targeting dry AMD, with results expected by late 2027.

Moreover, their innovative compound, bevemipretide, is showing promise in both ophthalmic and neurological applications, with early studies suggesting neuroprotective benefits in Parkinson's disease.

The Road Ahead

As Stealth BioTherapeutics looks towards 2026, maintaining a laser focus on innovation is at the core of its mission. The company's roadmap emphasizes addressing urgent unmet needs across mitochondrial diseases, ranging from BTHS to other forms of PMD and even conditions like dry AMD. As the medical landscape for mitochondrial therapy expands, Stealth is committed to remaining a pioneering force, shaping outcomes that not only improve muscle strength but potentially life expectancy for individuals impacted by these rare diseases.

In conclusion, Stealth BioTherapeutics is not only advancing in commercial success but is also paving the way for a transformative future within the realm of mitochondrial research and treatment. With their ongoing commitment to innovation and patient care, they are well-positioned to make lasting impacts in the treatment of mitochondrial dysfunction.

For further information on FORZINITY and ongoing clinical trials, interested parties can visit Stealth BioTherapeutics' official website or contact their investor relations team directly.

Contact

For media inquiries, reach out to Anna Stallmann at Ascent Strategic Communications. For investor-related questions, contact Austin Murtagh at Precision AQ.

Stealth BioTherapeutics Inc. continues to stand at the forefront of the biotechnology sector, advocating for patients affected by rare mitochondrial diseases, and ensuring that they receive the care and innovation they deserve.