Alexion stalks rare disease drug from Stealth with $30m opt-in deal

Alexion has taken an option on a rare disease drug from Stealth Therapeutics in late-stage development, bolstering its pipeline as it faces a patent challenge for cash-cow product Soliris.

The US biopharma company is paying a $30 million opt-in fee for rights to elamipretide, currently in a phase 3 trial for primary mitochondrial myopathy (PMM), a genetic disease affecting mitochondria that has no approved treatments.

Mitochondria regulate energy in cells, and in PMM this process breaks down, leading to debilitating skeletal muscle weakness, chronic fatigue and exercise intolerance. The disease is estimated to affect about 40,000 people in the US.

Alexion is angling for shared development and promotion rights to elamipretide in the US, and excusive rights elsewhere, for PMM and follow-up indications Barth syndrome and Leber’s hereditary optic neuropathy (LHON) which are in mid-stage clinical testing but so far have generated mixed results.

The licensing deal comes two years into a change in leadership and a major restructuring – prompted by a probe into financial irregularities – intended to prepare Alexion for life after it loses patent protection for blockbuster C5 inhibitor Soliris (eculizumab).

Alexion’s biggest product had sales of $3.5 billion last year, accounting for 86% of the company’s total product revenues, and is predicted to break the $4 billion threshold in 2019.

It’s facing a challenge in the US from Amgen however that is trying to allow biosimilars to enter the market in 2021, six years before Alexion’s claimed patent protection on the product runs out.

The Stealth deal is one of several signed by Alexion to reduce its reliance on Soliris, coming after acquisitions of Wilson Therapeutics and Syntimmune for $855 million and $1.2 billion, respectively, as well as partnerships with Complement Pharma, Dicerna, Caelium, Affibody and Zealand Pharma.

The company also has its first approval for a follow-up C5 inhibitor, Ultomiris (ravulizumab), which has improved dosing but still has a long way to go before it can compensate for the expected impact on Soliris of biosimilar competition.

Meanwhile, Alexion is in the middle of another round of leadership changes after chief financial officer Paul Clancy unexpectedly announced he was moving on last month, to be replaced by head of business development Aradhana Sarin.

Investors were spooked by the departure as Clancy was part of the turnaround team under CEO  Ludwig Hantson, and shares in the company dipped after the announcement.

This week, Hantson added another C-suite executive to the management team by appointing Tanisha Carino to the newly-created role of chief corporate affairs officer, responsible for government affairs, public policy and communications.

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