Alexion Pharmaceuticals to File for FDA Approval of Soliris Successor in Mid-2018

Alexion Pharmaceuticals closed out its first quarter plans to seek regulatory approval for its paroxysmal nocturnal hemoglobinuria (PNH) treatment, ALXN1210, which is considered the successor to the company’s blockbuster blood-disorder drug Soliris.

On Thursday the company announced topline data from a second Phase III switch trial for ALXN1210, the company’s investigational long-acting C5 complement inhibitor. Results of the switch trial showed patients with paroxysmal nocturnal hemoglobinuria (PNH) can be effectively and safely switched from treatment with Alexion’s own Soliris.  ALXN1210 is an improved version of its blockbuster blood-disorder drug Soliris. Trial results show that patients could safely be dosed once every eight weeks with ALXN1210 compared to every two weeks with Soliris. ALXN1210 showed non-inferiority against Soliris in patients with PNH who had been stable on Soliris based on the primary endpoint of change in lactate dehydrogenase (LDH) levels. Those levels are a direct marker of complement-mediated hemolysis in PNH.

PNH is a rare blood disease where the red blood cells are destroyed by a part of the body’s immune system known as the complement system.

That’s the second time in just a few weeks that ALXN1210 has hit the mark in a late-stage trial. In March the company announced positive results from the Phase III trial that pitted the investigational treatment against Soliris. ALXN1210 showed non-inferiority to the approved treatment for its co-primary endpoints of avoidance of transfusions and normalization of lactate dehydrogenase (LDH) levels, as well as all four key secondary endpoints.

On Thursday Alexion Chief Executive Officer Ludwig Hantson told investors and reporters during a quarterly earnings call that the company will seek regulatory approval in the United States and Europe for ALXN1210 in the mid-portion of 2018. Hantson said Alexion will also seek approval for ALXN1210 in Japan later this year.

John Orloff, Alexion’s head of research and development, said the company is pleased with the totality of the Phase III data for PNH patients.

"We believe that the differentiated profile of ALXN1210 could be a meaningful improvement for patients and clinicians," Orloff said.

That positive Phase III data wasn’t the only good news to come out of Alexion’s quarterly call on Thursday. The company reported total revenues for the first quarter of $930.9 million, which is a 7 percent increase compared to the same period in 2017. The bulk of those revenues were due to sales of Soliris, which were 800.1 million, a 2 percent increase over the same period from the previous year. Sales of Strensiq (asfotase alfa) were $110.7 million. Strensiq, which was approved in 2015, is used to treat hypophosphatasia, a rare genetic disorder characterized by the abnormal development of bones and teeth. Sales of Kanuma, which was also approved in 2015 for the treatment of people with lysosomal acid lipase deficiency, were $19.6 million.

During the call, the company also pointed to its recent acquisition of Sweden-based Wilson Therapeutics, which is developing therapies for patients with rare copper-mediated disorders. Hantson said the $855 million deal, announced April 11, marks an important first step in rebuilding the company’s clinical pipeline. Wilson Therapeutics’ product candidate WTX101, a first-in-class oral copper-binding agent, is in Phase III for development as a treatment for Wilson disease, a rare genetic disorder with hepatic and neurological consequences.

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