Alexion Pharmaceuticals Inc. and Stealth BioTherapeutics Corp. agreed to co-develop and commercialize Stealth's genetic disorder drug elamipretide.
Newton, Mass.-based Stealth's elamipretide is being evaluated in late-stage studies for the treatment of genetic disorders including primary mitochondrial myopathy, or PMM, and Barth syndrome. It is also being investigated for the treatment of Leber's hereditary optic neuropathy, or LHON, and geographic atrophy associated with dry age-related macular degeneration, or GA.
Under the agreement, Boston-based Alexion will have the option to partner with Stealth in the development of subcutaneous elamipretide based on the final results of the ongoing PMM study. If Alexion takes the option, the companies will co-develop the drug in the U.S. for PMM, Barth syndrome and LHON.
The agreement gives Alexion exclusive rights to develop and commercialize subcutaneous elamipretide outside the U.S. However, the companies will co-promote the drug to the same degree in the U.S.
Alexion will make initial payments of $30 million, including an option fee, an equity investment and development funding. Upon exercising the option, additional payments will be made, including an option exercise fee, an additional equity investment, development funding and potential regulatory and commercial milestone payments.
Elamipretide has received fast-track and orphan drug designations for PMM, Barth syndrome and LHON, as well as a fast track designation for GA.
