Understanding Alexion’s Robust Pipeline



Alexion’s advanced pipeline

Alexion Pharmaceuticals (ALXN) is expanding the label for its key drug Soliris. Along with Soliris—also known as eculizumab—Alexion’s other key pipeline molecules in the advanced development stage include ALXN 1210, ALXN 5500, ALXN 1007, ALXN 1101, and SBC-103.

We’ll discuss ALXN 1210 in the next article. First, let’s have a look at Alexion’s other advanced molecules.

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ALXN 1007

ALXN 1007, a Phase II development stage molecule, is being evaluated for inflammatory disorders such as gastrointestinal graft versus host disease (or GI-GVHD). In the most severe form of the disease, the mortality rate is ~30%–40% within the first six months post-transplant.


SBC-103 is being investigated in Phase I and Phase II studies for the indication of Mucopolysaccharidosis IIIB (or MPS IIIB). The devastating and rare disease causes severe cognitive decline, speech loss, behavioral problems, increased loss of mobility, and premature death. It occurs in children during the first few years of life.

MPS IIIB is caused by genetic mutations that result in decreased alpha-N-acetyl-glucosaminidase (or NAGLU) enzyme activity. This leads to a “buildup of abnormal amounts of heparan sulfate (or HS) in the brain and throughout the body,” according to the company.

SBC-103 is designed to replace the missing or deficient NAGLU enzyme. It won orphan drug designation by the FDA and the EMA (European Medicines Agency) in April 2013 and June 2013, respectively.

Other companies conducting research on rare diseases include BioMarin (BMRN), Shire (SHPG), and Biogen (BIIB).

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ALXN 1101

ALXN 1101 is in the Phase II and III stages for the indication of Molybdenum Cofactor Deficiency (or MoCD) Disease Type A (MoCD Type A) in neonates. The ultra-rare disorder causes rapidly progressive neurologic damage leading to death in newborns.

ALXN 5500

ALXN 5500 is a next-generation complement inhibitor. It’s in Phase I, an early clinical development stage.

It’s often risky to directly invest in biotechnology companies, as any news releases regarding the success or failure of their drugs can result in volatility in their stock prices.

To remain comparatively safe, investors can choose to invest in ETFs such as the First Trust NYSE Arca Biotechnology Index ETF (FBT), which holds 2.8% in Alexion Pharmaceuticals’ stock.


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