Alexion expands rare disease portfolio with $930m acquisition

By Vassia Barba

- Last updated on GMT

(Image: Getty/Kritchanut)
(Image: Getty/Kritchanut)

Related tags Alexion Rare disease Achillion acquisition

Alexion acquires Achillion to develop the latter’s investigational treatments for complement alternative pathway-mediated rare diseases.

Alexion Pharmaceuticals announced the acquisition of Achillion Pharmaceuticals, a clinical-stage biopharmaceutical company developing oral small molecule Factor D inhibitors for diseases including paroxysmal nocturnal hemoglobinuria and C3 glomerulopathy.

Under the agreement, Alexion will pay Achillion $930m (€839.58m) and will also be acquiring the cash currently on Achillion’s balance sheet, which, as of September 30, 2019, was approximately $230m (€207.64m).

The transaction is expected to close in the first half of 2020.

This deal comes only a few days after Alexion signed a $30m deal with Stealth BioTherapeutics​ for the co-development and commercialization of a primary mitochondrial myopathy treatment.

Achillion’s pipeline

Achillion’s development platform is focused on advancing oral small molecules that inhibit Factor D, a serine protease that functions as a ‘critical control point’ in the alternative pathway (AP) of the complement system, a part of the innate immune system.

The small molecules can potentially be used in the treatment of immune-related diseases in which complement AP plays a critical role.

Achillion is currently working on the development of two candidates, danicopan (ACH-4471), which is in Phase II clinical studies, and developed under breakthrough therapy designation from the US Food and Drug Administration, and ACH-5228, which is currently in Phase I studies.

Alexion’s CEO, Ludwig Hantson stated that Achillion’s approach has the potential to help patients with diseases not currently addressed through C5 inhibition, which Alexion’s lead-selling product, Soliris (eculizumab), already targets.

“Targeting a different part of the complement system [...] by inhibiting Factor D production addresses uncontrolled complement activation further upstream in the complement cascade, and importantly, leaves the rest of the complement system intact, which is critical in maintaining the body’s ability to fight infection,” ​Hantson explained.

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