The results of the international MMPOWER-3 trial evaluating the safety and efficacy of double-blind, placebo-controlled subcutaneous elamipretide in 218 adults with primary mitochondrial myopathy have been published:
- the majority of participants (74%) had a mitochondrial DNA mutation, the others a nuclear DNA mutation;
- elamipretide subcutaneous for 6 months was well tolerated;
- neither the gain in distance covered in the 6-minute walk test, nor the improvement in the PMMSA (Primary Mitochondrial Myopathy Symptom Assessment) total fatigue score at the end of 6 months, the two main endpoints of the trial, were significantly different with elamipretide or placebo;
- however, a post-hoc analysis showed a significant improvement in the 6-minute walk test in participants with a nuclear DNA mutation;
- the NuPower trial, currently underway, is evaluating the efficacy of 48 weeks of subcutaneous elamipretide in 130 participants with primary mitochondrial myopathy associated with a nuclear DNA mutation.
Since May 2022, elamipretide has had orphan drug status for myopathic mitochondrial DNA depletion syndromes in Europe.
