Stealth BioTherapeutics is advancing a new pipeline candidate into investigational new drug (IND) enabling studies in collaboration with long-term partner Evotec AG.

Stealth’s nomination of this novel pipeline candidate marks the culmination of a development initiative resulting in a diverse proprietary pipeline of compounds for the treatment of mitochondrial dysfunction arising in the context of both genetic mitochondrial diseases as well as age-related diseases. Stealth’s lead candidate, elamipretide, has received Orphan Drug and Fast Track designation with respect to late stage clinical development efforts in primary mitochondrial myopathy (PMM), Barth syndrome and Leber’s hereditary optic neuropathy (LHON). Stealth is also developing elamipretide for dry age-related macular degeneration, the leading cause of blindness in the elderly. 

“We believe that our first-in-class mitochondrial medicines offer a novel approach to the treatment of human disease,” said Reenie McCarthy, chief executive officer of Stealth. “We hope to utilize our deep knowledge of mitochondrial therapeutics to improve the treatment paradigm for patients suffering from genetic diseases involving mitochondrial dysfunction, as well as to potentially extend health span in the context of age-related diseases to which mitochondrial dysfunction is a known contributor.”

Stealth expects to advance its new pipeline candidate to clinical-stage studies by the end of 2019 utilizing Evotec’s INDiGO platform. INDiGO is a market-leading integrated drug development solution that accelerates early drug candidates into the clinic by reducing time from nomination to IND submission, typically in less than 52 weeks. Under the terms of the collaboration, Evotec will be responsible for all preclinical and toxicology/safety studies ahead of clinical trials in 2019.

“Our INDiGO platform reduces time and cost while consistently producing a quality data package for clinical trial agreements and investigational new drug filings,” said Dr. Mario Polywka, chief operating officer of Evotec. “We have a great scientific and personal relationship with Stealth and are excited for the opportunity to continue to help advance their programs.”