First Mitochondria Drug, Year One: $17.5 Million and a Pipeline

Jeff's Daily Pick. The first drug aimed directly at mitochondria turned one this year. It treats an ultra rare disease, earned $17.5 million, and is now being tested against eye disease and aging.

In the United States, Mighty Therapeutics, formerly Stealth BioTherapeutics, reported on October 14, 2026 on the first year of Forzinity (elamipretide). The FDA granted the drug accelerated approval on September 19, 2025 to improve muscle strength in patients with Barth syndrome weighing at least 30 kg. It was the first approved therapy aimed directly at mitochondria, the energy producing structures in cells.

The launch in numbers

From the soft launch in December 2025 through September 2026, the company reports gross revenue of $17.5 million, including $8.1 million in the third quarter of 2026, 60 patient enrollment forms and 48 unique prescribers. It says more than 90% of lives across major national health plans have coverage. A global Phase 4 study, 4TAZPower, is underway to confirm efficacy in people at least 5 years old, a requirement of accelerated approval.

The bigger bets

The Phase 3 ReNEW trial of elamipretide in dry age related macular degeneration is fully enrolled, with topline data expected in the second half of 2027. The company plans to start a Phase 2 study of bevemipretide eye drops, ReBUILD, by year end. It is requesting an FDA meeting on a pivotal trial in mitochondrial disease due to POLG mutations, with a start expected in 2027. Earlier stage work covers Parkinson's disease, Leigh syndrome, Friedreich's ataxia and fatty acid oxidation disorders. A team from Mighty and the University of Washington was named a Milestone 2 awardee in the XPRIZE Healthspan competition and is preparing a placebo controlled trial of elamipretide in older adults running through 2029.

The money comes partly from debt. In July, Mighty secured up to $150 million: a credit facility of up to $125 million from K2 HealthVentures, with an initial $30 million tranche, and $25 million in a Series B from founding investor Morningside. The company says this secures runway to projected positive cash flow in early 2029.

The 2027 dry AMD readout is the test that matters commercially: macular degeneration affects millions, Barth syndrome a few hundred people in the US. Until then, the mitochondria thesis rests on one accelerated approval.