Elamipretide A Novel Therapeutic Approach

Preclinical and clinical studies conducted by laboratories and clinicians worldwide have supported the development of elamipretide.6, 8, 39, 42 Stealth BioTherapeutics is focused on the opportunity to treat both rare mitochondrial diseases and diseases in major therapeutic categories involving mitochondrial dysfunction. Inherited, rare mitochondrial diseases are characterized by known genetic defects, and there are more than 200 of these types of diseases. Common diseases such as heart failure, renal disease and age-related macular degeneration are also well-understood to involve mitochondrial dysfunction.4, 11, 37

Our investigational compound, elamipretide, is formulated for systemic (subcutaneous injection and intravenous infusion) and topical ophthalmic delivery. Elamipretide is uniquely able to target the inner mitochondrial membrane, restoring electron transport to improve cellular energy production and reduce oxidative stress created by the over-production of ROS.8

mitochondrial gene therapy

Clinical Milestones

The clinical development for elamipretide has been structured to assess whether there is evidence of potentially favorable clinical effects across multiple therapeutic areas and to advance toward later-stage trials and registration in both rare and common diseases.

Mitochondrial Platform

Clinical Studies and Pipeline Development

Clinical-Pipeline-final 2016 1H 2H 2017 1H 2H Elampretide Development MMPOWERPrimary Mito Disease MMPOWER-2Primary Mito Disease MOTIONMuscle Energy Improvement SkeletalMuscle DATA* 1H 2016 DATA* 1H 2017 DATA* 2H 2016 ReSIGHTLHON ReVEALFuchs’ ReCLAIMDry AMD DATA* 2H 2017 DATA* 2H 2016 DATA* 1H 2017 Barth Syndrome PROGRESS-HFHFrEF RESTORE-HFHFpEF DATA* 2H 2017 DATA* 2H 2017 IDDEA-HFAcute HF DATA* 2H 2017 Preclinical enabling studies Rare Disease Indication Common Disease Indication *Topline data Ocular Cardiorenal Ph. II Ph. II Ph. II Ph. II Ph. II Ph. II Ph. II Ph. II Ph. II Ph. I