Business, Results

Alexion preps an FDA pitch on Soliris successor while signaling more deals ahead for rare disease drugs

Alexion used its Q1 update Thursday to unveil another set of Phase III non-inferior stats for its Soliris successor, adding to its regulatory package for new approvals that should ship later this year.

Following earlier proof of non-inferiority for treatment-naive patients, the new Phase III highlighted their successful switching of Soliris patients to ALXN1210, a shift from dosing every two weeks to eight weeks.

Ludwig Hantson

To be sure, the investigators underscored the same kind of improvements they found over Soliris on key measures, but there was nothing statistically significant about it. That will be good for shoring up their defense of the franchise drug for years to come — company execs talked of patents stretching out to 2035 — as rivals press along with new drugs they hope can beat the standard therapy for paroxysmal nocturnal hemoglobinuria (PNH).

Now the biotech is prepping regulatory pitches that should arrive in a matter of months.

Earlier this month Alexion announced a deal to buy a Stockholm-based biotech for $855 million in cash, bagging a new drug for rare cases of Wilson disease. The company is using that as a model for more deals just like it.

Paul Clancy

CFO Paul Clancy noted:

I would characterize the bias towards products, not towards platforms. Never say never, but this is rare disease. Wilson represents a good example of our bias. Rare disease, devastating diseases, a potential product to transform the disease, and we’re building up our ability to do that inside the company.

CEO Ludwig Hantson sought to reassure analysts that they were satisfied that they could convert a large number of patients to the new drug, which some analysts believe will have to come with a discount for payers. During the call with analysts, the CEO said:

We have a differentiated profile with 1210. I don’t think anybody will dispute that. And, for sure, we’re going to try to get the best label. And we believe that the data that we have will be reflected in the clinical trial section. The way I look at it, we don’t need the superiority claim to be successful for a fast conversion. We have a strong, differentiated clinical profile, a robust data package.

It’s not ideal, but analysts like Geoffrey Porges saw reasons for applause.

Alexion noted that ‘1210 was numerically superior on both the primary and all key secondary endpoints. We are also encouraged by the disclosure that zero subjects treated with ‘1210 experienced breakthrough hemolysis versus 5 subjects treated with Soliris. In terms of safety, there were no discontinuations due to adverse events, neutralizing antibodies, or cases of meningococcal infection.

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Sr. Manager, Regulatory Affairs, CMC
CytomX Therapeutics San Francisco, CA
Marketing Associate - Demand Generation
Catalytic Data Science Charleston, SC
Associate Principal, Life Sciences Partnerships
Flatiron Health New York City or San Francisco

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