Alexion Pharmaceuticals, Inc. (ALXN - Free Report) announced that encouraging interim data from an ongoing phase II study on its pipeline candidate asfotase alfa was presented by researchers. The study evaluated the candidate in infants and young children suffering from HPP-an ultra-rare metabolic deficiency.
Results from the study revealed that treatment with asfotase alfa for 24 weeks caused a significant improvement in skeletal mineralization of the disease over time. Alexion stated in its press release that the favorable response was detected following only 12 weeks of asfotase alfa therapy and the patients continued to show improvements at 48 weeks. Data from the open-label study also revealed that majority of the patients treated with asfotase alfa improved or preserved respiratory function at 48 weeks with over survival rate of 93%. Moreover, the candidate was found to be safe and well tolerated in the interim analysis.
We note that asfotase alfa enjoys Breakthrough Therapy designation from the U.S. Food and Drug Administration for treating patients suffering from hypophosphatasia. The designation is part of the FDA Safety and Innovation Act of 2012. The symptoms were witnessed in patients prior to attaining adulthood. Alexion Pharma stated in its press release that the status is assigned to speed up the development of the drug for treating a serious or life-threatening disorder. Successful development and commercialization of asfotase alfa would broaden Alexion’s product portfolio and bring in additional revenues to the company.
Alexion, which currently is a one-drug company with Soliris being its sole marketed product, is working on developing its pipeline to reduce its dependence on Soliris. Alexion’s pipeline includes ALXN1102/ALXN1103 (hematology) and ALXN1007 (inflammatory disorders) apart from asfotase alfa.
Alexion currently carries Zacks Rank #3 (Hold). Stocks which appear to be more attractive, include Gilead Sciences Inc. (GILD - Free Report) , Actelion Ltd. and Biogen Idec Inc. (BIIB - Free Report) , each with a Zacks Rank #1 (Strong Buy).