Alexion Pharmaceuticals’ (ALXN) Kanuma is a recombinant form of the human LAL (lysosomal acid lipase) enzyme and is the only enzyme-replacement therapy approved by the FDA for the treatment of patients suffering from LAL deficiency (or LAL-D).
Total sales of Kanuma increased from $12 million in the first quarter of 2017 to $19.6 million in the first quarter of 2018. Kanuma’s US sales increased from $8.7 million in the first quarter of 2017 to $11.9 million in the first quarter of 2018. Its European sales increased from $1.8 million in the first quarter of 2017 to $5.9 million in the first quarter of 2018.
Acquisition of Wilson Therapeutics
In April, Alexion announced a tender offer to acquire Sweden-based Wilson Therapeutics, a biopharmaceutical company. Wilson Therapeutics develops novel therapies for patients suffering from rare copper-mediated disorders.
This acquisition will help Alexion add Wilson Therapeutics’ drug candidate WTX101 to its clinical pipeline. WTX101 is currently in a Phase 3 clinical trial for the treatment of patients with Wilson’s disease. The transaction value is expected to be ~$855 million.
Alexion Pharmaceuticals’ research pipeline includes ALXN1210, Soliris, ALXN1101, and ALXN6000. ALXN1210 is an innovative, long-acting C5 inhibitor. It’s being developed in intravenous and subcutaneous forms targeted at PNH (paroxysmal nocturnal hemoglobinuria) and aHUS (atypical hemolytic uremic syndrome). The drug was granted orphan drug designation by the FDA in January 2017.
ALXN1210 for PNH
The company has completed enrolment in two Phase 3 clinical studies of ALXN1210 for patients with PNH. Alexion also began enrolling for a Phase 3 open-label study for evaluating the safety and efficacy of ALXN1210 administered by intravenous infusion to pediatric patients with PNH in February.
Notably, in March, Alexion announced that in the pivotal Phase 3 study of ALXN1210 for evaluating the safety and efficacy of ALXN1210 versus Soliris, intravenous infusion every eight weeks showed ALXN1210 to be non-inferior to Soliris in complement inhibitor treatment-naive patients.
In the next part of the series, we’ll take a look at Alexion Pharmaceuticals’ other clinical programs.