Biopharmaceutical company Alexion Pharmaceuticals has entered a deal with biotechnology firm Stealth BioTherapeutics to develop and commercialise elamipretide to treat mitochondrial diseases.

Elamipretide acts on the inner mitochondrial membrane, where it links to cardiolipin, a phospholipid involved in various mitochondrial processes such as respiration and energy conversion.

The association between elamipretide and cardiolipin was found to restore the structure of the inner mitochondrial membrane and in turn, improve mitochondrial function.

The drug is undergoing Phase III clinical trial in patients with primary mitochondrial myopathy (PMM), a genetic mitochondrial disease currently lacking approved therapies.

The agreement will provide Alexion with an exclusive option to work with Stealth for the development of subcutaneous elamipretide after the Phase III PMM study results are delivered.

If Alexion exercises the option, the partners will co-develop the subcutaneous formulation in the US for PMM and Barth syndrome and Leber’s hereditary optic neuropathy (LHON).

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According to the deal, upon commercialisation, the companies will be able to equally promote in the US, while Alexion will gain exclusive rights for subcutaneous elamipretide outside the US.

Alexion Pharmaceuticals executive vice-president and Research & Development head John Orloff said: “Given our strong existing relationships with neuromuscular specialists, who play a critical role in treating PMM, we believe this is an exciting potential opportunity to further expand our rare neurology portfolio and look forward to the possibility of working with Stealth to realise the promise of elamipretide for patients.”

The company will make initial payments of $30m to Stealth, including an option fee, equity investment and development funding.

Furthermore, Stealth could also receive an option exercise fee, additional equity investment, development funding and potential regulatory and commercial milestones.