Company

Stealth BioTherapeutics

Last updated Sat May 30 2026 00:00:00 GMT+0000 (Coordinated Universal Time)

What it is

Stealth BioTherapeutics develops drugs targeting mitochondrial dysfunction. Lead asset: elamipretide (SS-31), a tetrapeptide that binds cardiolipin in the mitochondrial inner membrane, stabilising cristae structure and improving electron-transport-chain efficiency.

Programmes

Primary mitochondrial myopathy (Barth syndrome): granted approval pathway in 2024 after positive results.
Dry AMD: Phase 3 ReNEW and ReGAIN trials read out 2024 with mixed results.
Heart failure with preserved ejection fraction (HFpEF): pre-clinical and early clinical work.
Friedreich’s ataxia: investigated.
Geographic atrophy (AMD subtype).

Why interesting for aging biology

Mitochondrial dysfunction is a hallmark of aging. Drugs that improve cristae structure could in principle address underlying mitochondrial decline across multiple age-related diseases. Elamipretide is the most-advanced clinical-stage cristae-targeted drug.

State

Mixed Phase 3 results in AMD have created uncertainty about elamipretide’s broader utility, but the Barth syndrome path provides a beachhead. Future indications depend on hit-or-miss across the mitochondrial-disease landscape.