Alexion drops kidney disease program for drug that was part of $930M Achillion buyout last year

The company said in its 2Q earnings that it would halt development of ALXN2040 for C3 glomerulopathy, or C3G, citing the drug’s lack of potency. However, it may develop a second, more potent drug from the Achillion acquisition, ALXN2050, in the same disease.

Last fall, Alexion Pharmaceuticals spent more than $900 million to acquire a company with a drug in development for a rare kidney disease. On Thursday, it dropped the program.

The Boston-based biotech company said in its second quarter earnings Thursday that following a review of Phase II data, it would drop the development of ALXN2040 in C3 glomerulopathy, or C3G, a disease with an incidence of about 1-2 million per year that is diagnosed in 1-2% of kidney biopsies.

Shares of Alexion rose 2.6% on the Nasdaq Thursday morning following the release of the company’s earnings.

The company said that the Phase II study data showed that the clinical response to ALXN2040, a Factor D inhibitor whose chemical name is danicopan, was suboptimal due to insufficient pharmacokinetic and pharmacodynamic response and incomplete inhibition of the alternative pathway. However, the company said a more potent Factor D inhibitor, ALXN2050, could also be assessed. ALXN2050 is currently undergoing a Phase II study in paroxysmal nocturnal hemoglobinuria. Alexion is also planning to start a Phase III study of ALXN2040 as an add-on therapy to C5 inhibitor therapy – meaning Soliris (eculizumab) or Ultomiris (ravulizumab-cwvz) – for PNH patients by the end of this year. The trial page is already posted on and listed as not yet recruiting.

Both drugs were part of Alexion’s $930 million acquisition of Achillion Pharmaceuticals, which was announced in October 2019. At the time of the acquisition, executives from Alexion said the deal provided the opportunity to diversify into complement-mediated diseases beyond ones it had focused on, like PNH and atypical hemolytic uremic syndrome (aHUS), using oral therapies. At the same time, they downplayed the suggestion that it was an attempt to protect the company from biosimilar competition to Soliris, its lead product, which stands to lose patent protection in the coming years.

Soliris had sales of $975.5 million in the second quarter, according to the earnings reports. Its longer-acting successor, Ultomiris, had sales of $251.1 million. Net product sales for the quarter were more than $1.4 billion, compared with $1.2 billion in second quarter 2019.

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